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Clinical Trial Results:
A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Summary
EudraCT number	2016-003852-60
Trial protocol	GB
Global end of trial date	18 Nov 2020

Results information
Results version number	v2(current)
This version publication date	20 Jun 2021
First version publication date	02 Jun 2021
Other versions	v1
Version creation reason	Correction of full data set Update in justification to the outcome measure.

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

274RP101 (NSR-RPGR-01)

ISRCTN number

US NCT number

NCT03116113

WHO universal trial number (UTN)

Sponsor organisation name

Biogen

Sponsor organisation address

225 Binney Street, Cambridge, United States, 02142

Public contact

Biogen Study Medical Director, Biogen, clinicaltrials@biogen.com

Scientific contact

Biogen Study Medical Director, Biogen, clinicaltrials@biogen.com

Is trial part of an agreed paediatric investigation plan (PIP)

Yes

EMA paediatric investigation plan number(s)

EMEA-002601-PIP01-19

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Analysis stage

Final

Date of interim/final analysis

24 Mar 2021

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

18 Nov 2020

Was the trial ended prematurely?

Main objective of the trial

The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in subjects with X-linked retinitis pigmentosa (XLRP).

Protection of trial subjects

Written informed consent was obtained from each subject or subject’s legally authorized representative (e.g., legal guardian), as applicable, prior to evaluations performed for eligibility. Subjects or the subject’s legally authorized representative were given adequate time to review the information in the informed consent/assent and were allowed to ask, and have answered, questions concerning all portions of the conduct of the study.

Background therapy

In Part I, all subjects were prescribed to take 1 milligram per kilogram per day (mg/kg/day) prednisone/prednisolone for a total of 10 days (beginning 2 days before the vector injection, on the day of injection, and then for 7 days); followed by 0.5 mg/kg/day for 7 days; 0.25 mg/kg/day for 2 days; and 0.125 mg/kg/day for 2 days (21 days in total). In Part II subjects in treated groups were given a 9-week course of oral prednisone/prednisolone and instructed to start taking the drug 3 days before Visit 2.

Evidence for comparator

Actual start date of recruitment

16 Mar 2017

Long term follow-up planned

Yes

Long term follow-up rationale

Efficacy, Safety

Long term follow-up duration

5 Years

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

United Kingdom: 25

Country: Number of subjects enrolled

United States: 25

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Subjects were enrolled at investigational sites in the United Kingdom (UK) and the United States (US) from 16 March, 2017 to 18 November, 2020.

Screening details

A total of 50 subjects with X-Linked Retinitis Pigmentosa (XLRP) were randomised in the study and received treatment (18 subjects in Part 1 and 32 subjects in Part 2). Of these, 47 subjects completed the study (18 subjects in Part 1 and 29 in Part 2).

Period 1 title

Overall Period

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Blinding implementation details

Part 1 of the study was open label and Part II was double-masked to the assigned dose, and open-label with respect to the treatment procedure. Primary efficacy endpoint data for Part 2 was also masked.

Are arms mutually exclusive

Yes

Part 1: BIIB112 Dose 1

Arm description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 1 (5 × 10^9 vector genomes {vg}) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Part 1: BIIB112 Dose 2

Arm description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 2 (1 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Part 1: BIIB112 Dose 3

Arm description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 3 (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Part 1: BIIB112 Dose 4

Arm description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 4 (1 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Part 1: BIIB112 Dose 5

Arm description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 5 (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Part 1: BIIB112 Dose 6

Arm description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 6 (5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Part 2: Untreated Group

Arm description

Subjects received no intervention to allow for a controlled comparison.

Arm type

No intervention

Investigational medicinal product name

No investigational medicinal product assigned in this arm

Part 2: BIIB112 Low Dose

Arm description

Followed by vitrectomy and retinal detachment in study eye, subjects received a single low dose (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Part 2: BIIB112 High Dose

Arm description

Followed by vitrectomy and retinal detachment in study eye, subjects received a single high dose (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Arm type

Experimental

Investigational medicinal product name

BIIB112

Investigational medicinal product code

Other name

AAV8-RPGR

Pharmaceutical forms

Suspension for injection

Routes of administration

Intraocular use

Dosage and administration details

Single, subretinal injection, Day 0.

Number of subjects in period 1	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Started	3	3	3	3	3	3	9	11	12
Completed	3	3	3	3	3	3	9	10	10
Not completed	0	0	0	0	0	0	0	1	2
Withdrew due to COVID-19	-	-	-	-	-	-	-	1	2

Baseline characteristics

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Reporting group title

Part 1: BIIB112 Dose 1

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 1 (5 × 10^9 vector genomes {vg}) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 2

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 2 (1 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 3

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 3 (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 4

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 4 (1 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 5

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 5 (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 6

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 6 (5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 2: Untreated Group

Reporting group description

Subjects received no intervention to allow for a controlled comparison.

Reporting group title

Part 2: BIIB112 Low Dose

Reporting group description

Followed by vitrectomy and retinal detachment in study eye, subjects received a single low dose (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 2: BIIB112 High Dose

Reporting group description

Followed by vitrectomy and retinal detachment in study eye, subjects received a single high dose (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose	Total
Number of subjects	3	3	3	3	3	3	9	11	12	50
Age Categorical
Units: subjects
Age Continuous
Units: years
arithmetic mean (standard deviation)	36.3 ( 6.35 )	34.3 ( 10.69 )	30.0 ( 2.65 )	33.3 ( 14.57 )	25.0 ( 5.00 )	32.3 ( 15.31 )	33.7 ( 13.83 )	30.6 ( 8.83 )	26.8 ( 7.11 )	-
Gender Categorical
Units: subjects
Female	0	0	0	0	0	0	0	0	0	0
Male	3	3	3	3	3	3	9	11	12	50
Ethnicity
Units: Subjects
Hispanic or Latino	0	0	0	0	0	1	2	3	5	11
Not Hispanic or Latino	3	3	3	3	3	2	7	8	7	39
Race
Units: Subjects
White	3	3	3	3	3	3	6	9	12	45
Black, of African Heritage	0	0	0	0	0	0	0	1	0	1
Asian	0	0	0	0	0	0	0	1	0	1
Multiple	0	0	0	0	0	0	1	0	0	1
Other	0	0	0	0	0	0	2	0	0	2

End points

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Reporting group title

Part 1: BIIB112 Dose 1

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 1 (5 × 10^9 vector genomes {vg}) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 2

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 2 (1 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 3

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 3 (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 4

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 4 (1 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 5

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 5 (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 6

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 6 (5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 2: Untreated Group

Reporting group description

Subjects received no intervention to allow for a controlled comparison.

Reporting group title

Part 2: BIIB112 Low Dose

Reporting group description

Followed by vitrectomy and retinal detachment in study eye, subjects received a single low dose (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 2: BIIB112 High Dose

Reporting group description

Followed by vitrectomy and retinal detachment in study eye, subjects received a single high dose (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Primary: Part 1: Number of Subjects with Dose-Limiting Toxicities (DLTs)

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End point title

Part 1: Number of Subjects with Dose-Limiting Toxicities (DLTs) ^[1] ^[2]

End point description

DLTs are defined as any of the following events considered to be related to AAV8-RPGR: Sustained decrease in best-corrected visual acuity (BCVA) of ≥30 letters on the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart compared to baseline (sustained is defined as lasting 48 hours or more until recovery, with recovery defined as visual acuity (VA) returning to within 10 letters of baseline VA. An exception is made for surgery-related events occurring in close temporal association {within <24 hours} of the surgery); Vitreous inflammation, vitritis (>Grade 3 using standardised Nussenblatt vitreous inflammation scale grading); Any clinically significant retinal damage observed that is not directly attributed to complications of surgery; Any clinically relevant suspected unexpected serious adverse reaction, with the exception of vision loss or vision threatening. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Primary

End point timeframe

Up to Month 24

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Only descriptive analysis was planned for this endpoint.
[2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: subjects	0	0	0	0	0	0

No statistical analyses for this end point

Primary: Part 1: Number of Subjects with Treatment-Emergent Adverse Events (TEAEs)

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End point title

Part 1: Number of Subjects with Treatment-Emergent Adverse Events (TEAEs) ^[3] ^[4]

End point description

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Primary

End point timeframe

Up to Month 24

Notes
[3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Only descriptive analysis was planned for this endpoint.
[4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: subjects	3	3	3	3	3	3

No statistical analyses for this end point

Primary: Part 2: Percentage of Study Eyes with ≥7 Decibels (dB) Improvement from Baseline at ≥5 of the 16 Central Loci of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry

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End point title

Part 2: Percentage of Study Eyes with ≥7 Decibels (dB) Improvement from Baseline at ≥5 of the 16 Central Loci of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry ^[5]

End point description

MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’, or a positive integer. The point labelled as ‘< 0’ was assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points out of the 16 central points. Intent-to-treat (ITT) analysis set included all subjects that were randomised under the 3-arm randomisation schedules.

End point type

Primary

End point timeframe

Month 12

Notes
[5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9 ^[6]	8 ^[7]	8 ^[8]
Units: percentage of study eyes
number (confidence interval 80%)	22.2 (6.1 to 49.0)	37.5 (14.7 to 65.5)	25.0 (6.9 to 53.8)

Notes
[6] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[7] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[8] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

Untreated Group Vs BIIB112 Low Dose

Comparison groups

Part 2: Untreated Group v Part 2: BIIB112 Low Dose

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3181

Method

Fisher’s Exact-Boschloo test

Parameter type

Difference from Untreated Group

Confidence interval

level

80%

sides

2-sided

lower limit

-13.9

upper limit

Untreated Group Vs BIIB112 High Dose

Comparison groups

Part 2: Untreated Group v Part 2: BIIB112 High Dose

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5177

Method

Fisher’s Exact-Boschloo test

Parameter type

Difference from Untreated Group

Confidence interval

level

80%

sides

2-sided

lower limit

-24.4

upper limit

30.5

Primary: Part 2: Number of Subjects with TEAEs

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End point title

Part 2: Number of Subjects with TEAEs ^[9] ^[10]

End point description

End point type

Primary

End point timeframe

Up to Month 12

Notes
[9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Only descriptive analysis was planned for this endpoint.
[10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	11	12
Units: subjects	5	11	12

No statistical analyses for this end point

Secondary: Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry

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End point title

Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry ^[11]

End point description

MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ was assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points out of the 16 central points. Safety analysis set consists of all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3 ^[12]	2 ^[13]	3 ^[14]	3 ^[15]	3 ^[16]	3 ^[17]
Units: percentage of study eyes
number (confidence interval 95%)
Month 1 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	100.0 (29.2 to 100.0)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 3 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 6 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 9 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 12 (n=3,2,3,3,2,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)
Month 18 (n=3,2,3,3,3,2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)
Month 24 (n=3,1,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 97.5)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)

Notes
[12] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[13] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[14] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[15] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[16] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[17] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry

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End point title

Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry ^[18]

End point description

MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ was assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in whole grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points of the grid as a whole (68 points). Safety analysis set consists of all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[18] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3 ^[19]	2 ^[20]	3 ^[21]	3 ^[22]	3 ^[23]	3 ^[24]
Units: percentage of study eyes
number (confidence interval 95%)
Month 1 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	100.0 (29.2 to 100.0)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 3 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 6 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	100.0 (29.2 to 100.0)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)
Month 9 (n=3,2,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Month 12 (n=3,2,3,3,2,3)	0.0 (0.0 to 70.8)	50.0 (1.3 to 98.7)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	50.0 (1.3 to 98.7)	66.7 (9.4 to 99.2)
Month 18 (n=3,2,3,3,3,2)	0.0 (0.0 to 70.8)	50.0 (1.3 to 98.7)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)	50.0 (1.3 to 98.7)
Month 24 (n=3,1,3,3,3,3)	0.0 (0.0 to 70.8)	0.0 (0.0 to 97.5)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)

Notes
[19] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[20] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[21] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[22] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[23] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[24] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Mean Sensitivity of the 16 Central Loci

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End point title

Part 1: Change from Baseline in Mean Sensitivity of the 16 Central Loci ^[25]

End point description

MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ is assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points out of the 16 central points. Here negative values indicate a decline in retinal sensitivity. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). 99999 indicates that standard deviation was not evaluable as there was only 1 subject. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9, 12, 18 and 24

Notes
[25] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3 ^[26]	2 ^[27]	3 ^[28]	3 ^[29]	3 ^[30]	3 ^[31]
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=3,2,3,3,3,3)	0.33 ( 1.748 )	1.78 ( 1.282 )	3.35 ( 0.911 )	6.52 ( 3.398 )	13.46 ( 6.316 )	7.75 ( 7.742 )
Baseline: Non-Study Eye (n=3,2,3,3,3,3)	0.23 ( 1.358 )	3.31 ( 2.563 )	6.92 ( 2.221 )	8.21 ( 5.570 )	13.71 ( 8.447 )	7.54 ( 7.542 )
Change at Month 1: Study Eye (n=3,2,3,3,3,3)	0.52 ( 0.477 )	-0.09 ( 0.928 )	6.10 ( 0.844 )	1.19 ( 6.094 )	-0.63 ( 4.154 )	-1.90 ( 7.686 )
Change at Month 1: Non-Study Eye (n=3,2,3,3,3,3)	0.42 ( 1.122 )	-0.81 ( 1.768 )	-0.98 ( 1.531 )	-0.19 ( 0.272 )	1.40 ( 2.690 )	0.19 ( 2.063 )
Change at Month 3: Study Eye (n=3,2,3,3,3,3)	0.40 ( 0.407 )	0.47 ( 1.282 )	5.63 ( 2.394 )	1.73 ( 8.714 )	-3.58 ( 7.476 )	-3.35 ( 2.894 )
Change at Month 3: Non-Study Eye (n=3,2,3,3,3,3)	-0.06 ( 0.534 )	-0.91 ( 0.575 )	-1.15 ( 2.094 )	0.33 ( 1.134 )	2.06 ( 1.137 )	-0.42 ( 0.289 )
Change at Month 6: Study Eye (n=3,2,3,3,3,3)	0.54 ( 0.485 )	-0.06 ( 0.265 )	5.10 ( 1.890 )	3.25 ( 8.199 )	-2.38 ( 5.177 )	0.85 ( 5.105 )
Change at Month 6: Non-Study Eye (n=3,2,3,3,3,3)	-0.17 ( 0.344 )	-0.34 ( 2.607 )	-1.19 ( 1.535 )	2.10 ( 3.437 )	2.19 ( 0.933 )	-0.65 ( 1.156 )
Change at Month 9: Study Eye (n=3,2,3,3,3,3)	0.21 ( 0.219 )	0.09 ( 1.282 )	4.65 ( 3.851 )	3.69 ( 6.308 )	-2.50 ( 5.314 )	0.77 ( 7.096 )
Change at Month 9: Non-Study Eye (n=3,2,3,3,3,3)	-0.35 ( 0.509 )	0.19 ( 2.210 )	0.20 ( 2.751 )	-0.06 ( 1.789 )	2.52 ( 1.714 )	0.40 ( 2.032 )
Change at Month 12: Study Eye (n=3,2,3,3,2,3)	0.19 ( 0.272 )	0.13 ( 1.945 )	4.10 ( 2.908 )	3.98 ( 5.090 )	2.25 ( 2.121 )	0.56 ( 5.590 )
Change at Month 12: Non-Study Eye (n=3,2,3,3,2,3)	-0.10 ( 0.355 )	-1.09 ( 1.370 )	-1.02 ( 0.581 )	0.42 ( 1.951 )	2.31 ( 2.990 )	-0.29 ( 1.835 )
Change at Month 18: Study Eye (n=3,2,3,3,3,2)	0.21 ( 0.473 )	-1.13 ( 0.177 )	2.69 ( 2.507 )	2.92 ( 5.550 )	-2.79 ( 6.733 )	-1.09 ( 5.436 )
Change at Month 18: Non-Study Eye (n=3,2,3,3,3,2)	-0.33 ( 0.577 )	-1.09 ( 1.282 )	0.02 ( 0.940 )	-0.67 ( 0.806 )	1.92 ( 3.085 )	-0.59 ( 2.607 )
Change at Month 24: Study Eye (n=3,1,3,3,3,3)	0.04 ( 0.253 )	-1.88 ( 99999 )	2.75 ( 2.683 )	1.48 ( 5.236 )	-4.90 ( 5.250 )	-1.88 ( 5.862 )
Month 24: Non-Study Eye (n=3,1,3,3,3,3)	-0.46 ( 0.469 )	-1.63 ( 99999 )	-0.33 ( 1.347 )	-0.56 ( 1.207 )	0.71 ( 3.263 )	-2.19 ( 2.189 )

Notes
[26] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[27] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[28] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[29] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[30] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[31] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Mean Sensitivity of the 68 Central Loci

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End point title

Part 1: Change from Baseline in Mean Sensitivity of the 68 Central Loci ^[32]

End point description

MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ is assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in whole grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points of the grid as a whole (68 points). Here negative values indicate a decline in retinal sensitivity. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). 99999 indicates that standard deviation was not evaluable as there was only 1 subject. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9, 12, 18 and 24

Notes
[32] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3 ^[33]	2 ^[34]	3 ^[35]	3 ^[36]	3 ^[37]	3 ^[38]
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=3,2,3,3,3,3)	-0.19 ( 0.690 )	0.20 ( 0.281 )	0.21 ( 0.435 )	2.22 ( 1.536 )	8.87 ( 10.784 )	4.26 ( 6.732 )
Baseline: Non-Study Eye (n=3,2,3,3,3,3)	-0.38 ( 0.540 )	1.47 ( 1.061 )	1.50 ( 1.419 )	2.30 ( 1.559 )	9.11 ( 12.466 )	5.35 ( 8.579 )
Change at Month 1: Study Eye (n=3,2,3,3,3,3)	0.12 ( 0.309 )	0.24 ( 1.185 )	2.42 ( 0.283 )	0.08 ( 2.436 )	0.56 ( 3.243 )	-1.69 ( 4.341 )
Change at Month 1: Non-Study Eye (n=3,2,3,3,3,3)	0.18 ( 0.481 )	-0.76 ( 1.165 )	-0.08 ( 0.938 )	-0.11 ( 0.111 )	1.24 ( 1.639 )	-0.66 ( 1.474 )
Change at Month 3: Study Eye (n=3,2,3,3,3,3)	0.14 ( 0.137 )	0.74 ( 1.820 )	2.44 ( 1.564 )	0.58 ( 5.061 )	-1.87 ( 4.073 )	-0.60 ( 1.383 )
Change at Month 3: Non-Study Eye (n=3,2,3,3,3,3)	-0.06 ( 0.292 )	-0.29 ( 1.549 )	-0.29 ( 1.351 )	0.68 ( 1.223 )	1.38 ( 1.005 )	-0.65 ( 0.407 )
Change at Month 6: Study Eye (n=3,2,3,3,3,3)	0.04 ( 0.334 )	0.19 ( 0.957 )	2.28 ( 1.534 )	0.90 ( 5.011 )	-0.60 ( 1.596 )	0.51 ( 2.154 )
Change at Month 6: Non-Study Eye (n=3,2,3,3,3,3)	-0.24 ( 0.331 )	-0.32 ( 1.872 )	-0.51 ( 0.773 )	3.86 ( 5.648 )	1.76 ( 0.470 )	-0.84 ( 1.101 )
Change at Month 9: Study Eye (n=3,2,3,3,3,3)	-0.18 ( 0.145 )	0.31 ( 1.456 )	2.02 ( 2.177 )	0.90 ( 4.233 )	-0.87 ( 2.229 )	-0.13 ( 4.044 )
Change at Month 9: Non-Study Eye (n=3,2,3,3,3,3)	-0.37 ( 0.467 )	-0.12 ( 1.830 )	1.66 ( 1.790 )	0.77 ( 1.405 )	2.05 ( 0.816 )	-0.41 ( 1.157 )
Change at Month 12: Study Eye (n=3,2,3,3,2,3)	-0.16 ( 0.165 )	0.30 ( 1.612 )	1.72 ( 1.892 )	1.28 ( 3.541 )	-0.22 ( 2.600 )	-0.41 ( 4.208 )
Change at Month 12: Non-Study Eye (n=3,2,3,3,2,3)	-0.30 ( 0.417 )	-0.46 ( 1.633 )	-0.24 ( 0.153 )	0.76 ( 1.345 )	1.72 ( 1.144 )	-0.82 ( 1.248 )
Change at Month 18: Study Eye (n=3,2,3,3,3,2)	-0.23 ( 0.213 )	-0.50 ( 0.458 )	1.16 ( 1.144 )	1.37 ( 4.233 )	-0.72 ( 1.253 )	-1.32 ( 4.721 )
Change at Month 18: Non-Study Eye (n=3,2,3,3,3,2)	-0.33 ( 0.412 )	-0.57 ( 1.435 )	0.07 ( 0.509 )	0.18 ( 0.191 )	1.24 ( 1.265 )	-1.19 ( 2.267 )
Change at Month 24: Study Eye (n=3,1,3,3,3,3)	-0.40 ( 0.382 )	-0.96 ( 99999 )	1.17 ( 1.190 )	0.98 ( 3.510 )	-2.37 ( 1.225 )	-2.26 ( 6.009 )
Change at Month 24: Non-Study Eye (n=3,1,3,3,3,3)	-0.37 ( 0.427 )	-1.44 ( 99999 )	0.14 ( 0.221 )	0.24 ( 0.153 )	0.75 ( 1.427 )	-1.73 ( 2.481 )

Notes
[33] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[34] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[35] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[36] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[37] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[38] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Best Corrected Visual Acuity (BCVA) Score

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End point title

Part 1: Change from Baseline in Best Corrected Visual Acuity (BCVA) Score ^[39]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Here negative values indicate decline in BCVA. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9, 12, 18 and 24

Notes
[39] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: letters
arithmetic mean (standard deviation)
Baseline: Study Eye	27.67 ( 8.145 )	49.67 ( 9.452 )	64.33 ( 7.095 )	63.67 ( 20.551 )	70.67 ( 4.163 )	67.33 ( 15.044 )
Baseline: Non-Study Eye	60.67 ( 2.082 )	57.33 ( 5.686 )	66.33 ( 2.517 )	62.67 ( 22.279 )	73.33 ( 4.163 )	75.00 ( 4.583 )
Change at Month 1: Study Eye	3.67 ( 3.055 )	0.33 ( 2.517 )	-1.00 ( 1.732 )	-1.33 ( 6.506 )	-0.67 ( 4.041 )	-0.67 ( 10.693 )
Change at Month 1: Non-Study Eye	3.00 ( 3.000 )	1.00 ( 4.359 )	1.00 ( 1.000 )	0.33 ( 0.577 )	2.00 ( 4.583 )	-2.67 ( 1.155 )
Change at Month 3: Study Eye	6.33 ( 2.309 )	0.00 ( 3.606 )	0.67 ( 2.082 )	1.67 ( 4.726 )	0.33 ( 6.028 )	0.67 ( 6.506 )
Change at Month 3: Non-Study Eye	3.00 ( 1.000 )	1.67 ( 1.528 )	-1.67 ( 1.528 )	2.33 ( 2.309 )	4.67 ( 4.509 )	-0.33 ( 0.577 )
Change at Month 6: Study Eye	5.00 ( 1.000 )	-3.67 ( 3.786 )	0.33 ( 0.577 )	3.67 ( 5.686 )	-3.67 ( 8.737 )	-0.67 ( 5.033 )
Change at Month 6: Non-Study Eye	-1.00 ( 7.211 )	-0.67 ( 4.726 )	-0.33 ( 3.215 )	3.67 ( 3.055 )	4.00 ( 4.359 )	-0.67 ( 1.528 )
Change at Month 9: Study Eye	1.33 ( 4.619 )	-0.67 ( 0.577 )	1.33 ( 4.509 )	4.00 ( 5.292 )	-3.67 ( 16.073 )	-2.33 ( 9.074 )
Change at Month 9: Non-Study Eye	-5.00 ( 5.292 )	-0.67 ( 5.033 )	1.00 ( 1.000 )	3.00 ( 3.606 )	5.67 ( 8.622 )	2.00 ( 4.359 )
Change at Month 12: Study Eye	4.67 ( 4.509 )	-2.00 ( 3.464 )	0.67 ( 5.132 )	4.33 ( 4.933 )	-15.00 ( 40.286 )	-2.33 ( 8.327 )
Change at Month 12: Non-Study Eye	-1.67 ( 2.309 )	-0.33 ( 3.215 )	-0.33 ( 0.577 )	2.00 ( 4.359 )	6.67 ( 6.429 )	1.00 ( 2.000 )
Change at Month 18: Study Eye	3.00 ( 8.718 )	-2.00 ( 3.606 )	-3.33 ( 5.132 )	3.33 ( 3.786 )	-9.33 ( 28.919 )	-5.00 ( 8.544 )
Change at Month 18: Non-Study Eye	-4.00 ( 6.245 )	0.00 ( 5.000 )	-0.33 ( 2.517 )	1.33 ( 2.517 )	5.00 ( 7.810 )	-0.33 ( 5.508 )
Change at Month 24: Study Eye	-2.00 ( 15.588 )	-2.00 ( 5.292 )	-3.33 ( 3.215 )	5.00 ( 7.000 )	-13.33 ( 32.747 )	-5.67 ( 6.110 )
Change at Month 24: Non-Study Eye	-3.67 ( 5.508 )	0.67 ( 0.577 )	-2.33 ( 1.528 )	2.33 ( 4.509 )	2.00 ( 7.810 )	-2.00 ( 3.000 )

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Low Luminance Visual Acuity (LLVA) Score

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End point title

Part 1: Change from Baseline in Low Luminance Visual Acuity (LLVA) Score ^[40]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Here negative values indicate decline in LLVA. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9. 12. 18 and 24

Notes
[40] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[41]	0 ^[42]	2 ^[43]	3 ^[44]	3 ^[45]	3 ^[46]
Units: letters
arithmetic mean (standard deviation)
Baseline: Study Eye	( )	( )	41.00 ( 18.385 )	34.33 ( 29.939 )	58.00 ( 3.000 )	41.67 ( 34.646 )
Baseline: Non-Study Eye	( )	( )	37.50 ( 24.749 )	37.67 ( 32.808 )	61.00 ( 2.646 )	57.33 ( 14.844 )
Change at Month 1: Study Eye	( )	( )	10.00 ( 9.899 )	0.33 ( 0.577 )	3.00 ( 5.292 )	4.67 ( 26.502 )
Change at Month 1: Non-Study Eye	( )	( )	10.00 ( 18.385 )	-0.33 ( 7.506 )	3.00 ( 5.196 )	-4.33 ( 7.572 )
Change at Month 3: Study Eye	( )	( )	14.00 ( 11.314 )	7.33 ( 6.658 )	-14.33 ( 38.214 )	-7.67 ( 10.263 )
Change at Month 3: Non-Study Eye	( )	( )	10.00 ( 16.971 )	1.00 ( 1.000 )	3.00 ( 4.583 )	2.00 ( 3.464 )
Change at Month 6: Study Eye	( )	( )	11.50 ( 7.778 )	8.33 ( 7.234 )	-16.67 ( 35.810 )	7.33 ( 9.713 )
Change at Month 6: Non-Study Eye	( )	( )	8.00 ( 15.556 )	1.67 ( 2.887 )	5.33 ( 1.155 )	0.67 ( 3.215 )
Change at Month 9: Study Eye	( )	( )	0.50 ( 4.950 )	10.00 ( 6.557 )	-14.33 ( 37.820 )	4.33 ( 8.083 )
Change at Month 9: Non-Study Eye	( )	( )	0.50 ( 4.950 )	2.00 ( 4.359 )	2.00 ( 3.464 )	3.33 ( 2.309 )
Change at Month 12: Study Eye	( )	( )	3.50 ( 0.707 )	16.00 ( 13.454 )	-11.33 ( 40.612 )	8.33 ( 11.590 )
Change at Month 12: Non-Study Eye	( )	( )	7.00 ( 9.899 )	6.67 ( 8.327 )	9.00 ( 4.359 )	4.00 ( 6.083 )
Change at Month 18: Study Eye	( )	( )	6.50 ( 7.778 )	10.67 ( 8.622 )	-12.33 ( 40.079 )	8.33 ( 5.508 )
Change at Month 18: Non-Study Eye	( )	( )	9.00 ( 21.213 )	2.33 ( 2.517 )	5.67 ( 4.726 )	-4.33 ( 7.234 )
Change at Month 24: Study Eye	( )	( )	3.00 ( 8.485 )	9.33 ( 14.295 )	-14.67 ( 38.371 )	-1.67 ( 6.506 )
Change at Month 24: Non-Study Eye	( )	( )	5.50 ( 24.749 )	0.67 ( 4.041 )	3.33 ( 3.512 )	-12.67 ( 11.547 )

Notes
[41] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[42] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[43] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[44] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[45] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[46] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for BCVA

Top of page

End point title

Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for BCVA ^[47]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[47] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 1: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 12: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 24: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for LLVA

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End point title

Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for LLVA ^[48]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[48] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[49]	0 ^[50]	2 ^[51]	3 ^[52]	3 ^[53]	3 ^[54]
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 1: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 6: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 12: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 24: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)

Notes
[49] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[50] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[51] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[52] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[53] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[54] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for BCVA

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End point title

Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for BCVA ^[55]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[55] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 1: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 12: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 12: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for LLVA

Top of page

End point title

Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for LLVA ^[56]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[56] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[57]	0 ^[58]	2 ^[59]	3 ^[60]	3 ^[61]	3 ^[62]
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	66.7 (9.4 to 99.2)
Month 1: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 3: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	66.7 (9.4 to 99.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 6: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 9: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 12: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 18: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 18: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)

Notes
[57] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[58] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[59] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[60] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[61] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[62] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for BCVA

Top of page

End point title

Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for BCVA ^[63]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18, and 24

Notes
[63] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	66.7 (9.4 to 99.2)
Month 1: Non-Study Eye	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 3: Study Eye	100.0 (29.2 to 100.0)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 3: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)	0.0 (0.0 to 70.8)
Month 6: Study Eye	66.7 (9.4 to 99.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 9: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 9: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 12: Study Eye	66.7 (9.4 to 99.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 12: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Study Eye	66.7 (9.4 to 99.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 24: Study Eye	66.7 (9.4 to 99.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for LLVA

Top of page

End point title

Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for LLVA ^[64]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[64] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[65]	0 ^[66]	2 ^[67]	3 ^[68]	3 ^[69]	3 ^[70]
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Month 1: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 3: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 3: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 6: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 6: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)	0.0 (0.0 to 70.8)
Month 9: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)
Month 9: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 12: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Month 12: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	100.0 (29.2 to 100.0)	33.3 (0.8 to 90.6)
Month 18: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 18: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 24: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)

Notes
[65] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[66] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[67] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[68] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[69] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[70] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for BCVA

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End point title

Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for BCVA ^[71]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[71] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 1: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 9: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 12: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 24: Study Eye	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for LLVA

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End point title

Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for LLVA ^[72]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[72] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[73]	0 ^[74]	2 ^[75]	3 ^[76]	3 ^[77]	3 ^[78]
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 1: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 3: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 9: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 12: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 24: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)

Notes
[73] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[74] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[75] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[76] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[77] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[78] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for BCVA

Top of page

End point title

Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for BCVA ^[79]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[79] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 1: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 9: Non-Study Eye	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 12: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 18: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 24: Study Eye	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 24: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for LLVA

Top of page

End point title

Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for LLVA ^[80]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[80] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[81]	0 ^[82]	2 ^[83]	3 ^[84]	3 ^[85]	3 ^[86]
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 1: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 3: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 3: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 9: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 12: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 24: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 24: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)

Notes
[81] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[82] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[83] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[84] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[85] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[86] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for BCVA

Top of page

End point title

Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for BCVA ^[87]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[87] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 1: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 3: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 3: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)
Month 6: Non-Study Eye	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 9: Non-Study Eye	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 12: Non-Study Eye	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 18: Non-Study Eye	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 24: Study Eye	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 24: Non-Study Eye	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for LLVA

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End point title

Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for LLVA ^[88]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[88] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[89]	0 ^[90]	2 ^[91]	3 ^[92]	3 ^[93]	3 ^[94]
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 1: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)
Month 3: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 3: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 6: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 6: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 9: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 9: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 12: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 12: Non-Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)
Month 18: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	0.0 (0.0 to 70.8)
Month 18: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	66.7 (9.4 to 99.2)
Month 24: Study Eye	( to )	( to )	0.0 (0.0 to 84.2)	0.0 (0.0 to 70.8)	33.3 (0.8 to 90.6)	33.3 (0.8 to 90.6)
Month 24: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	0.0 (0.0 to 70.8)	0.0 (0.0 to 70.8)	100.0 (29.2 to 100.0)

Notes
[89] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[90] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[91] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[92] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[93] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[94] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for BCVA

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End point title

Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for BCVA ^[95]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[95] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: percentage of eyes
number (confidence interval 95%)
Month 1: Study Eye	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Month 1: Non-Study Eye	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Month 3: Study Eye	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Month 3: Non-Study Eye	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Month 6: Study Eye	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)
Month 6: Non-Study Eye	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Month 9: Study Eye	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Month 9: Non-Study Eye	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Month 12: Study Eye	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)
Month 12: Non-Study Eye	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Month 18: Study Eye	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)
Month 18: Non-Study Eye	33.3 (0.8 to 90.6)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)
Month 24: Study Eye	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)
Month 24: Non-Study Eye	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)

No statistical analyses for this end point

Secondary: Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for LLVA

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End point title

Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for LLVA ^[96]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR).

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, 12, 18 and 24

Notes
[96] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	0 ^[97]	0 ^[98]	2 ^[99]	3 ^[100]	3 ^[101]	3 ^[102]
Units: percentage of eyes
number (confidence interval 95%)
Change at Month 1: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)
Change at Month 1: Non-Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)
Change at Month 3: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	33.3 (0.8 to 90.6)
Change at Month 3: Non-Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Change at Month 6: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)
Change at Month 6: Non-Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Change at Month 9: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)
Change at Month 9: Non-Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Change at Month 12: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Change at Month 12: Non-Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)
Change at Month 18: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	100.0 (29.2 to 100.0)
Change at Month 18: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	33.3 (0.8 to 90.6)
Change at Month 24: Study Eye	( to )	( to )	100.0 (15.8 to 100.0)	100.0 (29.2 to 100.0)	66.7 (9.4 to 99.2)	66.7 (9.4 to 99.2)
Change at Month 24: Non-Study Eye	( to )	( to )	50.0 (1.3 to 98.7)	100.0 (29.2 to 100.0)	100.0 (29.2 to 100.0)	0.0 (0.0 to 70.8)

Notes
[97] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[98] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[99] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[100] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[101] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.
[102] - 'Number of subjects analysed' signifies number of subjects analysed in this endpoint.

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Central Ellipsoid Area

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End point title

Part 1: Change from Baseline in Central Ellipsoid Area ^[103]

End point description

Spectral Domain Optical Coherence Tomography (SD-OCT) was used to assess change in central ellipsoid area. Here negative values indicate decline in central ellipsoid area. Safety analysis included all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9, 12, 18 and 24

Notes
[103] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: millimeter square (mm^2)
arithmetic mean (standard deviation)
Baseline: Study Eye (n=3,3,3,3,3,3)	0.140 ( 0.1637 )	2.750 ( 4.7631 )	0.137 ( 0.1582 )	0.193 ( 0.1686 )	8.327 ( 13.8600 )	3.107 ( 5.2001 )
Baseline: Non-Study Eye (n=3,3,3,2,3,3)	0.130 ( 0.0985 )	2.313 ( 3.7500 )	0.093 ( 0.0814 )	0.465 ( 0.1061 )	14.307 ( 24.1914 )	5.257 ( 8.8720 )
Change at Month 1: Study Eye (n=3,3,3,3,3,3)	-0.077 ( 0.1159 )	-0.493 ( 0.8545 )	-0.103 ( 0.1050 )	-0.050 ( 0.0458 )	-1.360 ( 2.2863 )	-1.310 ( 2.2258 )
Change at Month 1: Non-Study Eye (n=3,3,3,2,3,3)	-0.060 ( 0.0265 )	-0.383 ( 0.4508 )	-0.020 ( 0.0693 )	-0.080 ( 0.0566 )	-0.410 ( 0.7645 )	0.873 ( 1.5824 )
Change at Month 3: Study Eye (n=3,3,3,3,2,3)	-0.100 ( 0.1249 )	-0.560 ( 0.9699 )	-0.110 ( 0.1153 )	-0.060 ( 0.0529 )	-2.255 ( 3.0335 )	-2.960 ( 5.0319 )
Change at Month 3: Non-Study Eye (n=3,3,3,2,3,2)	-0.093 ( 0.0404 )	-0.407 ( 0.4895 )	-0.043 ( 0.0379 )	-0.095 ( 0.0495 )	-0.407 ( 0.7778 )	-0.040 ( 0.0566 )
Change at Month 6: Study Eye (n=3,3,3,3,3,2)	-0.120 ( 0.1375 )	-0.527 ( 0.9122 )	-0.113 ( 0.1206 )	-0.070 ( 0.0624 )	-4.847 ( 8.1176 )	-0.055 ( 0.0778 )
Change at Month 6: Non-Study Eye (n=3,3,3,2,3,2)	-0.093 ( 0.0404 )	-0.410 ( 0.4951 )	-0.060 ( 0.0529 )	-0.120 ( 0.0424 )	-0.983 ( 1.7747 )	-0.050 ( 0.0707 )
Change at Month 9: Study Eye (n=3,3,3,3,3,3)	-0.127 ( 0.1419 )	-0.540 ( 0.9353 )	-0.117 ( 0.1258 )	-0.063 ( 0.0551 )	-5.640 ( 9.4830 )	-2.960 ( 5.0664 )
Change at Month 9: Non-Study Eye (n=3,3,3,2,3,2)	-0.097 ( 0.0451 )	-0.437 ( 0.5326 )	-0.063 ( 0.0569 )	-0.120 ( 0.0141 )	-1.100 ( 1.9504 )	-0.050 ( 0.0707 )
Change at Month 12: Study Eye (n=3,3,3,3,3,3)	-0.130 ( 0.1473 )	-0.560 ( 0.9699 )	-0.117 ( 0.1258 )	-0.053 ( 0.0551 )	-5.623 ( 9.4975 )	-3.007 ( 5.1386 )
Change at Month 12: Non-Study Eye (n=3,3,3,2,3,3)	-0.097 ( 0.0451 )	-0.447 ( 0.5353 )	-0.033 ( 0.1266 )	-0.120 ( 0.0424 )	-1.010 ( 2.0313 )	0.997 ( 1.8312 )
Change at Month 18: Study Eye (n=3,3,3,3,3,3)	-0.133 ( 0.1528 )	-0.567 ( 0.9815 )	-0.137 ( 0.1582 )	0.027 ( 0.1617 )	-5.633 ( 9.4975 )	-2.993 ( 5.1155 )
Change at Month 18: Non-Study Eye (n=3,3,3,2,3,3)	-0.113 ( 0.0709 )	-0.450 ( 0.5408 )	-0.027 ( 0.1201 )	-0.115 ( 0.0919 )	-1.107 ( 2.0124 )	0.590 ( 1.1364 )
Change at Month 24: Study Eye (n=3,3,3,3,3,3)	-0.133 ( 0.1528 )	-0.573 ( 0.9930 )	-0.137 ( 0.1582 )	0.010 ( 0.1552 )	-5.657 ( 9.5292 )	-3.010 ( 5.1357 )
Change at Month 24: Non-Study Eye (n=3,3,3,2,3,3)	-0.117 ( 0.0764 )	-0.443 ( 0.5297 )	-0.060 ( 0.0794 )	-0.150 ( 0.0283 )	-1.163 ( 2.0063 )	0.367 ( 0.7505 )

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Central Horizontal Ellipsoid Width

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End point title

Part 1: Change from Baseline in Central Horizontal Ellipsoid Width ^[104]

End point description

SD-OCT was used to assess change in central horizontal ellipsoid width. Here negative values indicate decline in central horizontal ellipsoid width. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9, 12, 18 and 24

Notes
[104] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: Micron (um)
arithmetic mean (standard deviation)
Baseline: Study Eye (n=3,3,3,3,3,3)	162.67 ( 175.104 )	1375.33 ( 2382.147 )	248.00 ( 275.158 )	462.33 ( 400.775 )	2411.33 ( 3048.253 )	745.67 ( 774.448 )
Baseline: Non-Study Eye (n=3,3,3,2,3,3)	355.67 ( 280.878 )	1383.67 ( 2396.581 )	250.00 ( 223.139 )	792.00 ( 100.409 )	3341.00 ( 4556.473 )	937.67 ( 1088.262 )
Change at Month 1: Study Eye (n=3,3,3,3,3,3)	-56.33 ( 73.894 )	-311.00 ( 538.668 )	-174.00 ( 162.567 )	-26.33 ( 24.007 )	-203.33 ( 248.605 )	88.67 ( 422.044 )
Change at Month 1: Non-Study Eye (n=3,3,3,2,3,3)	-62.33 ( 40.216 )	-321.67 ( 557.143 )	47.33 ( 226.509 )	-56.00 ( 63.640 )	-14.00 ( 113.331 )	977.33 ( 1814.500 )
Change at Month 3: Study Eye (n=3,3,3,3,3,3)	-73.00 ( 70.193 )	-318.00 ( 550.792 )	-174.33 ( 163.022 )	-89.33 ( 125.644 )	-369.00 ( 271.070 )	-453.33 ( 554.795 )
Change at Month 3: Non-Study Eye (n=3,3,3,2,3,3)	-149.00 ( 80.988 )	-333.33 ( 577.350 )	-60.67 ( 78.258 )	-151.00 ( 15.556 )	-19.00 ( 123.503 )	1008.33 ( 1869.924 )
Change at Month 6: Study Eye (n=3,3,3,3,3,3)	-115.67 ( 105.624 )	-318.67 ( 551.947 )	-175.67 ( 164.852 )	-146.67 ( 129.454 )	-1070.67 ( 1365.685 )	-461.33 ( 553.453 )
Change at Month 6: Non-Study Eye (n=3,3,3,2,3,2)	-158.67 ( 65.033 )	-347.00 ( 601.022 )	-76.33 ( 79.135 )	-195.50 ( 6.364 )	-20.00 ( 144.503 )	-108.50 ( 153.442 )
Change at Month 9: Study Eye (n=3,3,3,3,3,3)	-127.00 ( 121.025 )	-321.33 ( 556.566 )	-178.33 ( 168.548 )	-149.00 ( 131.936 )	-1096.33 ( 1343.289 )	-441.00 ( 505.720 )
Change at Month 9: Non-Study Eye (n=3,3,3,2,3,2)	-175.00 ( 39.585 )	-348.00 ( 602.754 )	-77.33 ( 79.053 )	-202.50 ( 4.950 )	-81.67 ( 217.721 )	-124.00 ( 175.362 )
Change at Month 12: Study Eye (n=3,3,3,3,3,3)	-130.00 ( 125.300 )	-238.33 ( 412.805 )	-217.00 ( 225.980 )	-106.33 ( 132.666 )	-1055.33 ( 1394.128 )	-463.00 ( 537.199 )
Change at Month 12: Non-Study Eye (n=3,3,3,2,3,3)	-181.67 ( 30.665 )	-349.33 ( 605.063 )	-35.67 ( 255.895 )	-113.50 ( 47.376 )	-143.00 ( 221.007 )	1108.00 ( 2145.532 )
Change at Month 18: Study Eye (n=3,3,3,3,3,3)	-131.00 ( 126.740 )	-239.33 ( 414.537 )	-248.00 ( 275.158 )	21.67 ( 332.031 )	-1059.67 ( 1438.362 )	-461.67 ( 530.381 )
Change at Month 18: Non-Study Eye (n=3,3,3,2,3,3)	-316.67 ( 213.601 )	-354.67 ( 614.301 )	-168.67 ( 161.122 )	-188.00 ( 65.054 )	-113.00 ( 242.736 )	1078.67 ( 2094.819 )
Change at Month 24: Study Eye (n=3,3,3,3,3,3)	-131.33 ( 127.222 )	-242.33 ( 419.734 )	-248.00 ( 275.158 )	-43.33 ( 243.904 )	-1162.00 ( 1352.050 )	-465.67 ( 587.699 )
Change at Month 24: Non-Study Eye (n=3,3,3,2,3,3)	-329.33 ( 235.434 )	-358.00 ( 620.074 )	-145.67 ( 162.543 )	-254.50 ( 7.778 )	-159.67 ( 265.038 )	1065.67 ( 2074.136 )

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Fundus Autofluoroscence- Total Area of Preserved Autofluoroscence

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End point title

Part 1: Change from Baseline in Fundus Autofluoroscence- Total Area of Preserved Autofluoroscence ^[105]

End point description

Fundus Autofluoroscence was used to assess change in total area of preserved autofluoroscence. Here negative values indicate decline in total area of preserved autofluoroscence. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). 99999 indicates that standard deviation was not evaluable as there was only 1 subject. 999999 indicates that data was not evaluable at given time point. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 12, 18 and 24

Notes
[105] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: mm^2
arithmetic mean (standard deviation)
Baseline: Study Eye (n=1,0,0,0,0,0)	27.050 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Baseline: Non-Study Eye (n=1,0,1,0,0,0)	22.910 ( 99999 )	999999 ( 999999 )	50.640 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 1: Study Eye (n=1,0,0,0,0,0)	-1.820 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 1: Non-Study Eye (n=1,0,1,0,0,0)	-2.420 ( 99999 )	999999 ( 999999 )	-8.620 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 3: Study Eye (n=1,0,0,0,0,0)	-2.940 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 3: Non-Study Eye (n=1,0,1,0,0,0)	-2.650 ( 99999 )	999999 ( 999999 )	-5.150 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 6: Study Eye (n=1,0,0,0,0,0)	-3.460 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 6: Non-Study Eye (n=1,0,1,0,0,0)	-3.050 ( 99999 )	999999 ( 999999 )	-6.530 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 9: Study Eye (n=1,0,0,0,0,0)	-3.470 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 9: Non-Study Eye (n=1,0,1,0,0,0)	-3.100 ( 99999 )	999999 ( 999999 )	-9.130 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 12: Study Eye (n=1,0,0,0,0,0)	-3.640 ( 99999 )	999999 ( 9999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 12: Non-Study Eye (n=1,0,1,0,0,0)	-3.760 ( 99999 )	999999 ( 999999 )	-9.220 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 18: Study Eye (n=1,0,0,0,0,0)	-3.670 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 18: Non-Study Eye (n=1,0,1,0,0,0)	-3.830 ( 99999 )	999999 ( 99999 )	-11.700 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 24: Study Eye (n=1,0,0,0,0,0)	-3.740 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )
Change at Month 24: Non-Study Eye (n=1,0,0,0,0,0)	-4.120 ( 99999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Fundus Autofluoroscence- Distance from Foveal Center (FC) to Nearest Border of Preserved Autofluoroscence

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End point title

Part 1: Change from Baseline in Fundus Autofluoroscence- Distance from Foveal Center (FC) to Nearest Border of Preserved Autofluoroscence ^[106]

End point description

Fundus Autofluoroscence was used to assess change in distance from foveal center (FC) to nearest border of preserved autofluoroscence. Here negative values indicate decline in total area of preserved autofluoroscence. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). 99999 indicates that standard deviation was not evaluable as there was only 1 subject. 999999 indicates that data was not evaluable at given time point. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9, 12, 18 and 24

Notes
[106] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: mm^2
arithmetic mean (standard deviation)
Baseline: Study Eye (n=2,1,1,1,1,0)	2394.50 ( 665.387 )	-39.00 ( 99999 )	3018.00 ( 99999 )	361.00 ( 99999 )	2853.00 ( 99999 )	999999 ( 999999 )
Baseline: Non-Study Eye (n=2,1,1,1,1,1)	2609.00 ( 386.080 )	-24.00 ( 99999 )	3052.00 ( 99999 )	369.00 ( 99999 )	2816.00 ( 99999 )	4047.00 ( 99999 )
Change at Month 1: Study Eye (n=2,1,1,1,1,0)	-33.00 ( 5.657 )	-14.00 ( 99999 )	-52.00 ( 99999 )	-14.00 ( 99999 )	-5.00 ( 99999 )	999999 ( 999999 )
Change at Month 1: Non-Study Eye (n=2,1,1,1,1,1)	-255.50 ( 297.692 )	-19.00 ( 99999 )	-281.00 ( 99999 )	-18.00 ( 99999 )	-12.00 ( 99999 )	-3.00 ( 99999 )
Change at Month 3: Study Eye (n=2,1,1,1,1,0)	-87.00 ( 24.042 )	-16.00 ( 99999 )	-138.00 ( 99999 )	-89.00 ( 99999 )	-104.00 ( 99999 )	999999 ( 999999 )
Change at Month 3: Non-Study Eye (n=2,1,1,1,1,1)	-276.50 ( 324.562 )	-30.00 ( 99999 )	-375.00 ( 99999 )	-22.00 ( 99999 )	-93.00 ( 99999 )	-21.00 ( 99999 )
Change at Month 6: Study Eye (n=2,1,1,1,1,0)	-128.50 ( 54.447 )	-24.00 ( 99999 )	-179.00 ( 99999 )	-93.00 ( 99999 )	-122.00 ( 99999 )	999999 ( 999999 )
Change at Month 6: Non-Study Eye (n=2,1,1,1,1,1)	-297.00 ( 349.311 )	-41.00 ( 99999 )	-462.00 ( 99999 )	-36.00 ( 99999 )	-102.00 ( 99999 )	999999 ( 999999 )
Change at Month 9: Study Eye (n=2,1,1,1,1,0)	-140.00 ( 41.012 )	-35.00 ( 99999 )	-281.00 ( 99999 )	-94.00 ( 99999 )	-140.00 ( 99999 )	999999 ( 999999 )
Change at Month 9: Non-Study Eye (n=2,1,1,1,1,1)	-310.50 ( 333.047 )	-58.00 ( 99999 )	-413.00 ( 99999 )	-38.00 ( 99999 )	92.00 ( 99999 )	999999 ( 999999 )
Change at Month 12: Study Eye (n=2,0,1,1,1,0)	-1489.50 ( 1829.285 )	999999 ( 999999 )	-282.00 ( 99999 )	-101.00 ( 99999 )	-708.00 ( 99999 )	999999 ( 999999 )
Change at Month 12: Non-Study Eye (n=2,0,1,1,1,1)	-353.50 ( 318.905 )	999999 ( 999999 )	-422.00 ( 99999 )	-39.00 ( 99999 )	89.00 ( 99999 )	999999 ( 999999 )
Change at Month 18: Study Eye (n=2,1,1,1,1,0)	-1493.00 ( 1829.992 )	-55.00 ( 99999 )	-310.00 ( 99999 )	-123.00 ( 99999 )	-1278.00 ( 99999 )	999999 ( 999999 )
Change at Month 18: Non-Study Eye (n=2,1,1,1,1,0)	-358.00 ( 318.198 )	-86.00 ( 99999 )	-425.00 ( 99999 )	-11.00 ( 99999 )	65.00 ( 99999 )	999999 ( 999999 )
Change at Month 24: Study Eye (n=2,1,1,1,1,0)	-1501.00 ( 1820.093 )	-69.00 ( 99999 )	-226.00 ( 99999 )	-125.00 ( 99999 )	-1293.00 ( 99999 )	999999 ( 999999 )
Change at Month 24: Non-Study Eye (n=2,1,1,1,1,0)	-391.00 ( 301.227 )	-91.00 ( 99999 )	-2646.00 ( 99999 )	-13.00 ( 99999 )	62.00 ( 99999 )	999999 ( 999999 )

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Volume of 30-Degree Hill of Vision

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End point title

Part 1: Change from Baseline in Volume of 30-Degree Hill of Vision ^[107]

End point description

Visual field testing was performed to assess change in volume of 30-degree hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this endpoint. Here negative values indicate decline in volume of 30-degree hill vision. Safety analysis set. 99999= standard deviation was not evaluable as there was only 1 subject. 999999=data was not evaluable at given time point. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 6, 12 and 24

Notes
[107] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=0,0,0,0,1,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	2.73 ( 99999 )	5.07 ( 99999 )
Baseline: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	5.17 ( 99999 )
Change at Month 6: Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-2.14 ( 99999 )
Change at Month 6: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-1.10 ( 99999 )
Change at Month 12: Study Eye (n=0,0,0,0,1,1),	999999 ( 999999 )	999999 ( 999999 )	9999999 ( 999999 )	999999 ( 999999 )	0.33 ( 999999 )	-2.80 ( 99999 )
Change at Month 12: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-1.02 ( 99999 )
Change at Month 24: Study Eye (n=0,0,0,0,1,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-1.89 ( 99999 )	-3.08 ( 99999 )
Change at Month 24: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-2.11 ( 99999 )

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Volume of Full Field Hill of Vision

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End point title

Part 1: Change from Baseline in Volume of Full Field Hill of Vision ^[108]

End point description

Visual field testing was performed to assess change in volume of full field of hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this endpoint. Here negative values indicate decline in volume of 30-degree hill vision. Safety analysis set. 99999= standard deviation was not evaluable as there was only 1 subject. 999999= data was not evaluable at given time point. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 6, 12, and 24

Notes
[108] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=0,0,0,0,1,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	15.77 ( 99999 )	18.31 ( 99999 )
Baseline: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	99999 ( 999999 )	23.41 ( 99999 )
Change at Month 6: Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-0.86 ( 99999 )
Change at Month 6: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-4.91 ( 99999 )
Change at Month 12: Study Eye (n=0,0,0,0,1,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	1.29 ( 99999 )	-2.98 ( 99999 )
Change at Month 12: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-3.70 ( 99999 )
Change at Month 24: Study Eye (n=0,0,0,0,1,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-6.28 ( 99999 )	-5.36 ( 99999 )
Change at Month 24: Non-Study Eye (n=0,0,0,0,0,1)	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	999999 ( 999999 )	-7.87 ( 99999 )

No statistical analyses for this end point

Secondary: Part 1: Change from Baseline in Contrast Sensitivity

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End point title

Part 1: Change from Baseline in Contrast Sensitivity ^[109]

End point description

Change in contrast sensitivity (CS) was assessed by Pelli-Robson chart which uses a single large letter size (20/60 optotype), with contrast varying across groups of letters. Chart uses letters (6 per line), arranged in groups whose contrast varies from high to low. Subjects read the letters, starting with the highest contrast, until they are unable to read two or three letters in a single group. Each group has three letters of the same contrast level, so there are three trials per contrast level. Subject is assigned a score based on the contrast of the last group in which two or three letters were correctly read. Score is a measure of the subject’s log contrast sensitivity ranging from 0-2.25, with 0 being no letters read, and 2.25 being all letters read. Total CS score = [(total # letters correct - 3) x 0.05]. Safety analysis set consist of all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Part 1: Month 3, 6, 12 and 24

Notes
[109] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 1, hence data was reported for Part 1 reporting arm groups only.

End point values	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6
Number of subjects analysed	3	3	3	3	3	3
Units: Score on scale
arithmetic mean (standard deviation)
Baseline: Study Eye (n=3,3,3,3,3,3)	0.300 ( 0.1732 )	0.533 ( 0.2309 )	0.900 ( 0.1732 )	0.883 ( 0.3884 )	1.267 ( 0.0289 )	1.067 ( 0.6110 )
Baseline: Non-Study (n=3,3,3,3,3,3)	0.433 ( 0.2082 )	0.583 ( 0.2255 )	1.050 ( 0.2598 )	0.933 ( 0.4646 )	1.350 ( 0.1803 )	1.100 ( 0.3279 )
Change at Month 3: Study Eye (n=3,3,3,3,3,3)	0.167 ( 0.1528 )	0.183 ( 0.2566 )	0.150 ( 0.0866 )	0.133 ( 0.1155 )	-0.033 ( 0.4163 )	-0.167 ( 0.3014 )
Change at Month 3: Non-Study Eye (n=3,3,3,3,3,3)	0.000 ( 0.0000 )	0.017 ( 0.4010 )	0.050 ( 0.0500 )	0.050 ( 0.0500 )	0.100 ( 0.0000 )	0.000 ( 0.1000 )
Change at Month 6: Study Eye (n=3,3,3,3,3,3)	0.133 ( 0.2082 )	0.067 ( 0.2363 )	0.100 ( 0.1323 )	0.133 ( 0.0577 )	-0.183 ( 0.4072 )	0.117 ( 0.2021 )
Change at Month 6: Non-Study Eye (n=3,3,3,3,3,3)	0.000 ( 0.1732 )	0.033 ( 0.2255 )	0.083 ( 0.0764 )	0.050 ( 0.1000 )	-0.100 ( 0.0500 )	0.000 ( 0.0500 )
Change at Month 12: Study Eye (n=2,3,3,3,3,3)	0.100 ( 0.1414 )	0.133 ( 0.3512 )	-0.017 ( 0.1041 )	0.183 ( 0.0577 )	-0.167 ( 0.6788 )	-0.017 ( 0.3329 )
Change at Month 12: Non-Study Eye (n=2,3,3,3,3,3)	-0.100 ( 0.2828 )	0.067 ( 0.3512 )	0.000 ( 0.0000 )	0.000 ( 0.0500 )	-0.100 ( 0.1323 )	-0.050 ( 0.1323 )
Change at Month 24: Study Eye (n=3,3,3,3,3,3)	0.000 ( 0.2291 )	0.017 ( 0.5965 )	-0.100 ( 0.0000 )	0.217 ( 0.1893 )	-0.217 ( 0.5346 )	-0.117 ( 0.2466 )
Change at Month 24: Non-Study Eye (n=3,3,3,3,3,3)	-0.183 ( 0.1258 )	0.117 ( 0.3884 )	-0.100 ( 0.1732 )	0.017 ( 0.2021 )	-0.050 ( 0.1323 )	-0.050 ( 0.0500 )

No statistical analyses for this end point

Secondary: Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry

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End point title

Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry ^[110]

End point description

MAIA microperimetry assessment was measured in decibel (dB) using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ is assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points out of the 16 central points. ITT analysis set included all subjects who were randomized, under the 3-arm randomization schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6 and 9

Notes
[110] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of study eyes
number (confidence interval 80%)
Month 1 (n=9,10,10)	22.2 (6.1 to 49.0)	30.0 (11.6 to 55.2)	40.0 (18.8 to 64.6)
Month 2 (n=9,10,10)	22.2 (6.1 to 49.0)	30.0 (11.6 to 55.2)	50.0 (26.7 to 73.3)
Month 3 (n=8,10,10)	12.5 (1.3 to 40.6)	30.0 (11.6 to 55.2)	60.0 (35.4 to 81.2)
Month 6 (n=9,9,8)	22.2 (6.1 to 49.0)	33.3 (12.9 to 59.9)	62.5 (34.5 to 85.3)
Month 9 (n=9,7,7)	22.2 (6.1 to 49.0)	28.6 (7.9 to 59.6)	42.9 (17.0 to 72.1)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry

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End point title

Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry ^[111]

End point description

MAIA microperimetry assessment was measured in decibel (dB) using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ was assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in whole grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points of the grid as a whole (68 points). ITT analysis set included all subjects who were randomized, under the 3-arm randomization schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9 and 12

Notes
[111] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of study eyes
number (confidence interval 80%)
Month 1 (n=9,10,10)	33.3 (12.9 to 59.9)	60.0 (35.4 to 81.2)	40.0 (18.8 to 64.6)
Month 2 (n=9,10,10)	22.2 (6.1 to 49.0)	80.0 (55.0 to 94.5)	80.0 (55.0 to 94.5)
Month 3 (n=8,10,10)	25.0 (6.9 to 53.8)	80.0 (55.0 to 94.5)	70.0 (44.8 to 88.4)
Month 6 (n=9,9,8)	33.3 (12.9 to 59.9)	77.8 (51.0 to 93.9)	100.0 (75.0 to 100.0)
Month 9 (n=9,7,7)	33.3 (12.9 to 59.9)	85.7 (54.7 to 98.5)	71.4 (40.4 to 92.1)
Month 12 (n=9,8,8)	33.3 (12.9 to 59.9)	62.5 (34.5 to 85.3)	62.5 (34.5 to 85.3)

No statistical analyses for this end point

Secondary: Part 2: Change from Baseline in Mean Sensitivity of the 16 Central Loci

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End point title

Part 2: Change from Baseline in Mean Sensitivity of the 16 Central Loci ^[112]

End point description

MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ was assigned a value of ‘-1’ by MAIA in the calculation. Mean Sensitivity in center grid was defined as the mean in dB of the 16 points located in the center of the grid. Here negative values indicate a decline in retinal sensitivity. ITT analysis set included all subjects who were randomized, under the 3-arm randomization schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 2, 3, 6, 9 and 12

Notes
[112] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=9,10,10)	6.78 ( 3.488 )	7.39 ( 5.207 )	7.38 ( 3.095 )
Baseline: Non-Study Eye (n=9,10,10)	9.56 ( 3.321 )	9.09 ( 5.003 )	8.98 ( 3.583 )
Change at Month 1: Study Eye (n=9,10,10)	1.53 ( 3.086 )	2.70 ( 3.731 )	0.10 ( 5.723 )
Change at Month 1: Non-Study Eye (n=9,10,10)	-0.26 ( 1.692 )	-0.76 ( 1.793 )	-0.08 ( 0.834 )
Change at Month 2: Study Eye (n=9,10,10)	1.62 ( 2.846 )	2.73 ( 4.360 )	1.55 ( 5.886 )
Change at Month 2: Non-Study Eye (n=9,10,10)	-0.34 ( 2.027 )	-0.44 ( 1.723 )	0.26 ( 1.118 )
Change at Month 3: Study Eye (n=8,10,10)	1.17 ( 2.760 )	2.77 ( 4.598 )	1.00 ( 6.862 )
Change at Month 3: Non-Study Eye (n=8,10,10)	-1.24 ( 1.253 )	0.07 ( 1.393 )	-0.24 ( 0.500 )
Change at Month 6: Study Eye (n=9,9,8)	0.72 ( 3.208 )	3.38 ( 4.770 )	3.74 ( 3.931 )
Change at Month 6: Non-Study Eye (n=9,9,8)	-0.69 ( 2.199 )	-0.35 ( 1.125 )	-0.66 ( 0.702 )
Change at Month 9: Study Eye (n=9,7,7)	0.65 ( 3.272 )	2.52 ( 4.619 )	2.54 ( 6.317 )
Change at Month 9: Non-Study Eye (n=9,8,7)	-1.01 ( 1.943 )	0.30 ( 1.654 )	-0.54 ( 0.636 )
Change at Month 12: Study Eye (n=9,8,8)	-0.11 ( 4.446 )	2.79 ( 4.663 )	1.16 ( 5.660 )
Change at Month 12: Non-Study Eye (n=9,9,8)	-2.23 ( 4.018 )	-0.44 ( 1.078 )	-1.04 ( 1.292 )

No statistical analyses for this end point

Secondary: Part 2: Change from Baseline in Mean Sensitivity of the 68 Central Loci

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End point title

Part 2: Change from Baseline in Mean Sensitivity of the 68 Central Loci ^[113]

End point description

MAIA microperimetry assessment was measured in decibel (dB) using a 10-2 grid of 68 points. Each point was labelled as ‘< 0’, ‘0’ or a positive integer. The point labelled as ‘< 0’ was assigned a value of ‘-1’ by MAIA in the calculation. Improvement in Retinal Sensitivity in whole grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points of the grid as a whole (68 points). Here negative values indicate a decline in retinal sensitivity. ITT analysis set included all subjects who were randomized, under the 3-arm randomization schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 2, 3, 6, 9, and 12

Notes
[113] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=9,10,10)	2.15 ( 2.182 )	2.46 ( 1.911 )	3.84 ( 2.138 )
Baseline: Non-Study Eye (n=9,10,10)	3.32 ( 2.539 )	3.34 ( 2.092 )	4.47 ( 2.331 )
Change at Month 1: Study Eye (n=9,10,10)	0.91 ( 1.389 )	1.60 ( 2.225 )	-0.07 ( 3.898 )
Change at Month 1: Non-Study Eye (n=9,10,10)	0.12 ( 1.214 )	-0.46 ( 1.044 )	-0.03 ( 0.850 )
Change at Month 2: Study Eye (n=9,10,10)	0.90 ( 0.972 )	2.34 ( 2.353 )	1.18 ( 3.367 )
Change at Month 2: Non-Study Eye (n=9,10,10)	0.30 ( 1.111 )	-0.23 ( 0.859 )	0.13 ( 0.668 )
Change at Month 3: Study Eye (n=8,10,10)	0.46 ( 1.064 )	2.64 ( 2.962 )	0.24 ( 3.962 )
Change at Month 3: Non-Study Eye (n=8,10,10)	-0.16 ( 0.743 )	-0.03 ( 0.516 )	-0.11 ( 0.619 )
Change at Month 6: Study Eye (n=9,9,8)	0.32 ( 1.301 )	3.15 ( 3.283 )	2.05 ( 3.007 )
Change at Month 6: Non-Study Eye (n=9,9,8)	-0.13 ( 1.283 )	-0.27 ( 0.527 )	-0.50 ( 0.632 )
Change at Month 9: Study Eye (n=9,7,7)	0.40 ( 1.456 )	2.80 ( 3.116 )	1.71 ( 3.605 )
Change at Month 9: Non- Study Eye (n=9,8,7)	-0.41 ( 1.150 )	-0.05 ( 0.790 )	0.09 ( 0.544 )
Change at Month 12: Study Eye (n=9,8,8)	0.11 ( 1.592 )	2.79 ( 3.045 )	0.89 ( 3.132 )
Change at Month 12: Non-Study Eye (n=9,9,8)	-0.84 ( 1.749 )	-0.28 ( 0.476 )	-0.35 ( 1.004 )

No statistical analyses for this end point

Secondary: Part 2: Change From Baseline in BCVA

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End point title

Part 2: Change From Baseline in BCVA ^[114]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Here negative values indicate a decline in BCVA. ITT analysis set consist of all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 2, 3, 6, 9, and 12

Notes
[114] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: letters
arithmetic mean (standard deviation)
Baseline: Study Eye (n=9,10,10)	68.78 ( 5.954 )	65.90 ( 10.104 )	68.20 ( 8.991 )
Baseline: Non-Study Eye (n=9,10,10)	72.89 ( 6.846 )	68.50 ( 9.675 )	68.80 ( 6.989 )
Change at Month 1: Study Eye (n=9,10,10)	0.44 ( 2.833 )	1.60 ( 6.450 )	0.60 ( 7.545 )
Change at Month 1: Non-Study Eye (n=9,10,10)	-0.89 ( 2.028 )	0.00 ( 4.110 )	3.80 ( 3.360 )
Change at Month 2: Study Eye (n=9,10,10)	0.22 ( 2.108 )	-1.00 ( 12.392 )	0.00 ( 8.957 )
Change at Month 2: Non-Study Eye (n=9,10,10)	-0.11 ( 1.269 )	0.60 ( 2.836 )	3.10 ( 2.644 )
Change at Month 3: Study Eye (n=8,10,10)	1.38 ( 2.615 )	0.10 ( 6.540 )	-0.30 ( 11.036 )
Change at Month 3: Non-Study Eye (n=8,10,10)	0.13 ( 2.642 )	1.10 ( 2.470 )	3.20 ( 2.860 )
Change at Month 6: Study Eye (n=9,9,8)	1.00 ( 2.000 )	2.44 ( 5.003 )	-0.38 ( 5.630 )
Change at Month 6: Non-Study Eye (n=9,9,8)	-1.56 ( 2.833 )	0.67 ( 1.581 )	1.00 ( 4.629 )
Change at Month 9: Study Eye (n=9,8,7)	0.44 ( 1.944 )	-2.50 ( 14.263 )	-0.57 ( 1.397 )
Change at Month 9: Non-Study Eye (n=9,8,7)	-1.78 ( 2.949 )	1.00 ( 2.000 )	1.71 ( 3.147 )
Change at Month 12: Study Eye (n=9,9,8)	0.22 ( 2.949 )	-4.00 ( 17.436 )	-0.75 ( 3.370 )
Change at Month 12: Non-Study Eye (n=9,9,7)	-4.11 ( 3.983 )	0.89 ( 3.100 )	2.14 ( 3.976 )

No statistical analyses for this end point

Secondary: Part 2: Change From Baseline in LLVA

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End point title

Part 2: Change From Baseline in LLVA ^[115]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Here negative values indicate decline in LLVA. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 1, 3, 6, 9, and 12

Notes
[115] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9 ^[116]	10 ^[117]	10 ^[118]
Units: letters
arithmetic mean (standard deviation)
Baseline: Study Eye (n=9,10,10)	50.22 ( 13.349 )	39.30 ( 22.246 )	47.90 ( 14.970 )
Baseline: Non-Study Eye (n=9,10,10)	54.78 ( 11.872 )	49.50 ( 19.127 )	51.60 ( 9.524 )
Change at Month 1: Study Eye (n=9,10,10)	0.56 ( 7.618 )	11.30 ( 16.918 )	-1.30 ( 18.397 )
Change at Month 1: Non-Study Eye (n=9,10,10)	0.22 ( 5.215 )	-3.00 ( 9.165 )	3.50 ( 4.882 )
Change at Month 3: Study Eye (n=8,10,10)	3.63 ( 7.230 )	8.10 ( 16.251 )	-3.50 ( 22.609 )
Change at Month 3: Non-Study Eye (n=8,10,10)	-0.63 ( 8.158 )	0.50 ( 3.749 )	1.20 ( 3.553 )
Change at Month 6: Study Eye (n=9,9,8)	1.22 ( 7.032 )	8.00 ( 18.581 )	3.25 ( 14.636 )
Change at Month 6: Non-Study Eye (n=9,9,8)	0.11 ( 6.274 )	-3.56 ( 8.472 )	2.13 ( 2.850 )
Change at Month 9: Study Eye (n=9,8,7)	-1.78 ( 7.225 )	8.38 ( 16.088 )	6.00 ( 12.356 )
Change at Month 9: Non-Study Eye (n=9,8,7)	-3.11 ( 7.491 )	-1.13 ( 6.749 )	4.00 ( 4.082 )
Change at Month 12: Study Eye (n=9,9,8)	-2.22 ( 7.412 )	5.11 ( 14.426 )	-0.63 ( 11.575 )
Change at Month 12: Non-Study Eye (n=9,9,8)	-4.44 ( 7.002 )	-3.56 ( 11.652 )	-1.50 ( 4.309 )

Notes
[116] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.
[117] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.
[118] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥15 Letter Increase From Baseline for BCVA

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End point title

Part 2: Percentage of Eyes with a ≥15 Letter Increase From Baseline for BCVA ^[119]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9, and 12

Notes
[119] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 1: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 2: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 2: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 3: Study Eye (n=8,10,10)	0.0 (0.0 to 25.0)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 3: Non-Study Eye (n=8,10,10)	0.0 (0.0 to 25.0)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 6: Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)
Month 6: Non-Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)
Month 9: Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)	0.0 (0.0 to 28.0)
Month 9: Non-Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)	0.0 (0.0 to 28.0)
Month 12: Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)
Month 12: Non-Study Eye (n=9,9,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 28.0)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥15 Letter Increase From Baseline for LLVA

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End point title

Part 2: Percentage of Eyes with a ≥15 Letter Increase From Baseline for LLVA ^[120]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, and 12

Notes
[120] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	40.0 (18.8 to 64.6)	20.0 (5.5 to 45.0)
Month 1: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 3: Study Eye (n=8,10,10)	0.0 (0.0 to 25.0)	40.0 (18.8 to 64.6)	30.0 (11.6 to 55.2)
Month 3: Non-Study Eye (n=8,10,10)	0.0 (0.0 to 25.0)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 6: Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	33.3 (12.9 to 59.9)	12.5 (1.3 to 40.6)
Month 6: Non-Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)
Month 9: Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	25.0 (6.9 to 53.8)	28.6 (7.9 to 59.6)
Month 9: Non-Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)	0.0 (0.0 to 28.0)
Month 12: Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	33.3 (12.9 to 59.9)	12.5 (1.3 to 40.6)
Month 12: Non-Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥10 Letter Increase From Baseline for BCVA

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End point title

Part 2: Percentage of Eyes with a ≥10 Letter Increase From Baseline for BCVA ^[121]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9, and 12

Notes
[121] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 1: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 2: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	20.0 (5.5 to 45.0)
Month 2: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 3: Study Eye (n=8,10,10)	0.0 (0.0 to 25.0)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 3: Non-Study Eye (n=8,10,10)	0.0 (0.0 to 25.0)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 6: Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	12.5 (1.3 to 40.6)
Month 6: Non-Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)
Month 9: Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)	0.0 (0.0 to 28.0)
Month 9: Non-Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)	0.0 (0.0 to 28.0)
Month 12: Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	11.1 (1.2 to 36.8)	0.0 (0.0 to 25.0)
Month 12: Non-Study Eye (n=9,9,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 28.0)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥10 Letter Increase From Baseline for LLVA

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End point title

Part 2: Percentage of Eyes with a ≥10 Letter Increase From Baseline for LLVA ^[122]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, and 12

Notes
[122] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	11.1 (1.2 to 36.8)	40.0 (18.8 to 64.6)	30.0 (11.6 to 55.2)
Month 1: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 3: Study Eye (n=8,10,10)	25.0 (6.9 to 53.8)	40.0 (18.8 to 64.6)	30.0 (11.6 to 55.2)
Month 3: Non-Study Eye (n=8,10,10)	12.5 (1.3 to 40.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 6: Study Eye (n=9,9,8)	11.1 (1.2 to 36.8)	44.4 (21.0 to 69.9)	37.5 (14.7 to 65.5)
Month 6: Non-Study Eye (n=9,9,8)	11.1 (1.2 to 36.8)	11.1 (1.2 to 36.8)	0.0 (0.0 to 25.0)
Month 9: Study Eye (n=9,8,7)	11.1 (1.2 to 36.8)	37.5 (14.7 to 65.5)	28.6 (7.9 to 59.6)
Month 9: Non-Study Eye (n=9,8,7)	11.1 (1.2 to 36.8)	12.5 (1.3 to 40.6)	14.3 (1.5 to 45.3)
Month 12: Study Eye (n=9,9,8)	11.1 (1.2 to 36.8)	33.3 (12.9 to 59.9)	12.5 (1.3 to 40.6)
Month 12: Non-Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	11.1 (1.2 to 36.8)	0.0 (0.0 to 25.0)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥5 Letter Increase From Baseline for BCVA

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End point title

Part 2: Percentage of Eyes with a ≥5 Letter Increase From Baseline for BCVA ^[123]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9, and 12

Notes
[123] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	60.0 (35.4 to 81.2)	20.0 (5.5 to 45.0)
Month 1: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	40.0 (18.8 to 64.6)
Month 2: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	60.0 (35.4 to 81.2)	30.0 (11.6 to 55.2)
Month 2: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	30.0 (11.6 to 55.2)
Month 3: Study Eye (n=8,10,10)	12.5 (1.3 to 40.6)	20.0 (5.5 to 45.0)	30.0 (11.6 to 55.2)
Month 3: Non-Study Eye (n=8,10,10)	0.0 (0.0 to 25.0)	0.0 (0.0 to 20.6)	20.0 (5.5 to 45.0)
Month 6: Study Eye (n=9,9,8)	11.1 (1.2 to 36.8)	44.4 (21.0 to 69.9)	12.5 (1.3 to 40.6)
Month 6: Non-Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	25.0 (6.9 to 53.8)
Month 9: Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	37.5 (14.7 to 65.5)	0.0 (0.0 to 28.0)
Month 9: Non-Study Eye (n=9,8,7)	0.0 (0.0 to 22.6)	0.0 (0.0 to 25.0)	42.9 (17.0 to 72.1)
Month 12: Study Eye (n=9,9,8)	0.0 (0.0 to 22.6)	22.2 (6.1 to 49.0)	0.0 (0.0 to 25.0)
Month 12: Non-Study Eye (n=9,9,7)	0.0 (0.0 to 22.6)	11.1 (1.2 to 36.8)	28.6 (7.9 to 59.6)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥5 Letter Increase From Baseline for LLVA

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End point title

Part 2: Percentage of Eyes with a ≥5 Letter Increase From Baseline for LLVA ^[124]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, and 12

Notes
[124] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9 ^[125]	10 ^[126]	10 ^[127]
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	22.2 (6.1 to 49.0)	60.0 (35.4 to 81.2)	40.0 (18.8 to 64.6)
Month 1: Non-Study Eye (n=9,10,10)	22.2 (6.1 to 49.0)	30.0 (11.6 to 55.2)	30.0 (11.6 to 55.2)
Month 3: Study Eye (n=8,10,10)	37.5 (14.7 to 65.5)	50.0 (26.7 to 73.3)	30.0 (11.6 to 55.2)
Month 3: Non-Study Eye (n=8,10,10)	25.0 (6.9 to 53.8)	20.0 (5.5 to 45.0)	10.0 (1.0 to 33.7)
Month 6: Study Eye (n=9,9,8)	44.4 (21.0 to 69.9)	66.7 (40.1 to 87.1)	50.0 (24.0 to 76.0)
Month 6: Non-Study Eye (n=9,9,8)	22.2 (6.1 to 49.0)	11.1 (1.2 to 36.8)	25.0 (6.9 to 53.8)
Month 9: Study Eye (n=9,8,7)	22.2 (6.1 to 49.0)	62.5 (34.5 to 85.3)	57.1 (27.9 to 83.0)
Month 9: Non-Study Eye (n=9,8,7)	11.1 (1.2 to 36.8)	12.5 (1.3 to 40.6)	42.9 (17.0 to 72.1)
Month 12: Study Eye (n=9,9,8)	22.2 (6.1 to 49.0)	44.4 (21.0 to 69.9)	12.5 (1.3 to 40.6)
Month 12: Non-Study Eye (n=9,9,8)	11.1 (1.2 to 36.8)	22.2 (6.1 to 49.0)	0.0 (0.0 to 25.0)

Notes
[125] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.
[126] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.
[127] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥15 Letters Loss From Baseline for BCVA

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End point title

Part 2: Percentage of Eyes with a ≥15 Letters Loss From Baseline for BCVA ^[128]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set consist of all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9, and 12

Notes
[128] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	11	12
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,11,12)	0.0 (0.0 to 22.6)	0.0 (0.0 to 18.9)	8.3 (0.9 to 28.7)
Month 1: Non-Study Eye (n=9,11,12)	0.0 (0.0 to 22.6)	0.0 (0.0 to 18.9)	0.0 (0.0 to 17.5)
Month 2: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	0.0 (0.0 to 20.6)
Month 2: Non- Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 3: Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	0.0 (0.0 to 18.9)	8.3 (0.9 to 28.7)
Month 3: Non- Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	0.0 (0.0 to 18.9)	0.0 (0.0 to 17.5)
Month 6: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 6: Non- Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 9: Study Eye (n=9,9,9)	0.0 (0.0 to 22.6)	11.1 (1.2 to 36.8)	11.1 (1.2 to 36.8)
Month 9: Non- Study Eye (n=9,9,9)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)
Month 12: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	0.0 (0.0 to 20.6)
Month 12: Non- Study Eye (n=9,10,9)	0.0 (0.0 to 222.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 22.6)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥15 Letters Loss From Baseline for LLVA

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End point title

Part 2: Percentage of Eyes with a ≥15 Letters Loss From Baseline for LLVA ^[129]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, and 12

Notes
[129] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	11	12
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,11,12)	0.0 (0.0 to 22.6)	0.0 (0.0 to 18.9)	33.3 (15.4 to 55.9)
Month 1: Non-Study Eye (n=9,11,12)	0.0 (0.0 to 22.6)	18.2 (4.9 to 41.5)	0.0 (0.0 to 17.5)
Month 3: Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	9.1 (1.0 to 31.0)	33.3 (15.4 to 55.9)
Month 3: Non-Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	0.0 (0.0 to 18.9)	0.0 (0.0 to 17.5)
Month 6: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	20.0 (5.5 to 45.0)	20.0 (5.5 to 45.0)
Month 6: Non-Study Eye(n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	0.0 (0.0 to 20.6)
Month 9: Study Eye (n=9,9,9)	0.0 (0.0 to 22.6)	11.1 (1.2 to 36.8)	11.1 (1.2 to 36.8)
Month 9: Non-Study Eye (n=9,9,9)	11.1 (1.2 to 36.8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)
Month 12: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	20.0 (5.5 to 45.0)
Month 12: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	0.0 (0.0 to 20.6)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥10 Letters Loss From Baseline for BCVA

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End point title

Part 2: Percentage of Eyes with a ≥10 Letters Loss From Baseline for BCVA ^[130]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set consist of all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9, and 12

Notes
[130] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	11	12
Units: percentage of eyes
arithmetic mean (confidence interval 80%)
Month 1: Study Eye (n=9,11,12)	0.0 (0.0 to 22.6)	9.1 (1.0 to 31.0)	8.3 (0.9 to 28.7)
Month 1: Non-Study Eye (n=9,11,12)	0.0 (0.0 to 22.6)	0.0 (0.0 to 18.9)	0.0 (0.0 to 17.5)
Month 2: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	20.0 (5.5 to 45.0)	10.0 (1.0 to 33.7)
Month 2: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 3: Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	9.1 (1.0 to 31.0)	8.3 (0.9 to 28.7)
Month 3: Non-Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	0.0 (0.0 to 18.9)	0.0 (0.0 to 17.5)
Month 6: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 6: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	0.0 (0.0 to 20.6)	0.0 (0.0 to 20.6)
Month 9: Study Eye (n=9,9,9)	0.0 (0.0 to 22.6)	11.1 (1.2 to 36.8)	11.1 (1.2 to 36.8)
Month 9: Non-Study Eye (n=9,9,9)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)
Month 12: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	20.0 (5.5 to 45.0)	10.0 (1.0 to 33.7)
Month 12: Non-Study Eye (n=9,10,9)	11.1 (1.2 to 36.8)	0.0 (0.0 to 20.6)	0.0 (0.0 to 22.6)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥10 Letters Loss From Baseline for LLVA

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End point title

Part 2: Percentage of Eyes with a ≥10 Letters Loss From Baseline for LLVA ^[131]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, and 12

Notes
[131] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	11	12
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,11,12)	11.1 (1.2 to 36.8)	0.0 (0.0 to 18.9)	33.3 (15.4 to 55.9)
Month 1: Non-Study Eye (n=9,11,12)	0.0 (0.0 to 22.6)	18.2 (4.9 to 41.5)	0.0 (0.0 to 17.5)
Month 3: Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	18.2 (4.9 to 41.5)	33.3 (15.4 to 55.9)
Month 3: Non-Study Eye (n=8,11,12)	12.5 (1.3 to 40.6)	0.0 (0.0 to 18.9)	0.0 (0.0 to 17.5)
Month 6: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	20.0 (5.5 to 45.0)	20.0 (5.5 to 45.0)
Month 6: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	20.0 (5.5 to 45.0)	0.0 (0.0 to 20.6)
Month 9: Study Eye (n=9,9,9)	11.1 (1.2 to 36.8)	11.1 (1.2 to 36.8)	11.1 (1.2 to 36.8)
Month 9: Non-Study Eye (n=9,9,9)	11.1 (1.2 to 36.8)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)
Month 12: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	30.0 (11.6 to 55.2)
Month 12: Non-Study Eye (n=9,10,10)	33.3 (12.9 to 59.9)	10.0 (1.0 to 33.7)	0.0 (0.0 to 20.6)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥5 Letters Loss From Baseline for BCVA

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End point title

Part 2: Percentage of Eyes with a ≥5 Letters Loss From Baseline for BCVA ^[132]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. Safety analysis set consist of all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9, and 12

Notes
[132] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	11	12
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,11,12)	11.1 (1.2 to 36.8)	9.1 (1.0 to 31.0)	33.3 (15.4 to 55.9)
Month 1: Non-Study Eye (n=9,11,12)	11.1 (1.2 to 36.8)	9.1 (1.0 to 31.0)	0.0 (0.0 to 17.5)
Month 2: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	20.0 (5.5 to 45.0)	40.0 (18.8 to 64.6)
Month 2: Non-Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	0.0 (0.0 to 20.6)
Month 3: Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	18.2 (4.9 to 41.5)	33.3 (15.4 to 55.9)
Month 3: Non-Study Eye (n=8,11,12)	0.0 (0.0 to 25.0)	18.2 (4.9 to 41.5)	0.0 (0.0 to 17.5)
Month 6: Study Eye (n=9,10,10)	0.0 (0.0 to 22.6)	10.0 (1.0 to 33.7)	30.0 (11.6 to 55.2)
Month 6: Non-Study Eye (n=9,10,10)	22.2 (6.1 to 49.0)	0.0 (0.0 to 20.6)	10.0 (1.0 to 33.7)
Month 9: Study Eye (n=9,9,9)	22.2 (6.1 to 49.0)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)
Month 9: Non-Study Eye (n=9,9,9)	22.2 (6.1 to 49.0)	0.0 (0.0 to 22.6)	0.0 (0.0 to 22.6)
Month 12: Study Eye (n=9,10,10)	11.1 (1.2 to 36.8)	20.0 (5.5 to 45.0)	20.0 (5.5 to 45.0)
Month 12: Non-Study Eye (n=9,10,9)	33.3 (12.9 to 59.9)	10.0 (1.0 to 33.7)	0.0 (0.0 to 22.6)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with a ≥5 Letters Loss From Baseline for LLVA

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End point title

Part 2: Percentage of Eyes with a ≥5 Letters Loss From Baseline for LLVA ^[133]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. Safety analysis set included all subjects who received study treatment (vitrectomy/AAV8-RPGR). n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, and 12

Notes
[133] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	11	12
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,11,12)	33.3 (12.9 to 59.9)	18.2 (4.9 to 41.5)	41.7 (21.9 to 63.8)
Month 1: Non-Study Eye (n=9,11,12)	33.3 (12.9 to 59.9)	36.4 (16.9 to 59.9)	0.0 (0.0 to 17.5)
Month 3: Study Eye (n=8,11,12)	12.5 (1.3 to 40.6)	18.2 (4.9 to 41.5)	41.7 (21.9 to 63.8)
Month 3: Non-Study Eye (n=8,11,12)	37.5 (14.7 to 65.5)	9.1 (1.0 to 31.0)	0.0 (0.0 to 17.5)
Month 6: Study Eye (n=9,10,10)	22.2 (6.1 to 49.0)	20.0 (5.5 to 45.0)	30.0 (11.6 to 55.2)
Month 6: Non-Study Eye (n=9,10,10)	33.3 (12.9 to 59.9)	50.0 (26.7 to 73.3)	0.0 (0.0 to 20.6)
Month 9: Study Eye (n=9,9,9)	33.3 (12.9 to 59.9)	22.2 (6.1 to 49.0)	33.3 (12.9 to 59.9)
Month 9: Non-Study Eye (n=9,9,9)	55.6 (30.1 to 79.0)	33.3 (12.9 to 59.9)	0.0 (0.0 to 22.6)
Month 12: Study Eye (n=9,10,10)	55.6 (30.1 to 79.0)	30.0 (11.6 to 55.2)	40.0 (18.8 to 64.6)
Month 12: Non-Study Eye (n=9,10,10)	44.4 (21.0 to 69.9)	40.0 (18.8 to 64.6)	30.0 (11.6 to 55.2)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with Change From Baseline >-5 Letters for BCVA

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End point title

Part 2: Percentage of Eyes with Change From Baseline >-5 Letters for BCVA ^[134]

End point description

BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 2, 3, 6, 9, and 12

Notes
[134] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	88.9 (63.2 to 98.8)	90.0 (66.3 to 99.0)	70.0 (44.8 to 88.4)
Month 1: Non-Study Eye (n=9,10,10)	88.9 (63.2 to 98.8)	90.0 (66.3 to 99.0)	100.0 (79.4 to 100.0)
Month 2: Study Eye (n=9,10,10)	100.0 (77.4 to 100.0)	80.0 (55.0 to 94.5)	60.0 (35.4 to 81.2)
Month 2: Non-Study Eye (n=9,10,10)	100.0 (77.4 to 100.0)	90.0 (66.3 to 99.0)	100.0 (79.4 to 100.0)
Month 3: Study Eye (n=8,10,10)	100.0 (75.0 to 100.0)	80.0 (55.0 to 94.5)	70.0 (44.8 to 88.4)
Month 3: Non-Study Eye (n=8,10,10)	100.0 (75.0 to 100.0)	90.0 (66.3 to 99.0)	100.0 (79.4 to 100.0)
Month 6: Study Eye (n=9,9,8)	100.0 (77.4 to 100.0)	88.9 (63.2 to 98.8)	75.0 (46.2 to 93.1)
Month 6: Non-Study Eye (n=9,9,8)	77.8 (51.0 to 93.9)	100.0 (77.4 to 100.0)	87.5 (59.4 to 98.7)
Month 9: Study Eye (n=9,8,7)	100.0 (77.4 to 100.0)	75.0 (46.2 to 93.1)	100.0 (72.0 to 100.0)
Month 9: Non-Study Eye (n=9,8,7)	77.8 (51.0 to 93.9)	100.0 (75.0 to 100.0)	100.0 (72.0 to 100.0)
Month 12: Study Eye (n=9,9,8)	88.9 (63.2 to 98.8)	77.8 (51.0 to 93.9)	87.5 (59.4 to 98.7)
Month 12: Non-Study Eye (n=9,9,7)	66.7 (40.1 to 87.1)	88.9 (63.2 to 98.8)	100.0 (72.0 to 100.0)

No statistical analyses for this end point

Secondary: Part 2: Percentage of Eyes with Change From Baseline >-5 Letters for LLVA

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End point title

Part 2: Percentage of Eyes with Change From Baseline >-5 Letters for LLVA ^[135]

End point description

LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Month 1, 3, 6, 9, and 12

Notes
[135] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9 ^[136]	10 ^[137]	10 ^[138]
Units: percentage of eyes
number (confidence interval 80%)
Month 1: Study Eye (n=9,10,10)	66.7 (40.1 to 87.1)	80.0 (55.0 to 94.5)	70.0 (44.8 to 88.4)
Month 1: Non-Study Eye (n=9,10,10)	66.7 (40.1 to 87.1)	60.0 (35.4 to 81.2)	100.0 (79.4 to 100.0)
Month 3: Study Eye (n=8,10,10)	87.5 (59.4 to 98.7)	80.0 (55.0 to 94.5)	60.0 (35.4 to 81.2)
Month 3: Non-Study Eye (n=8,10,10)	62.5 (34.5 to 85.3)	100.0 (79.4 to 100.0)	100.0 (79.4 to 100.0)
Month 6: Study Eye (n=9,9,8)	77.8 (51.0 to 93.9)	77.8 (51.0 to 93.9)	75.0 (46.2 to 93.1)
Month 6: Non-Study Eye (n=9,9,8)	66.7 (40.1 to 87.1)	44.4 (21.0 to 69.9)	100.0 (75.0 to 100.0)
Month 9: Study Eye (n=9,8,7)	66.7 (40.1 to 87.1)	75.0 (46.2 to 93.1)	71.4 (40.4 to 92.1)
Month 9: Non-Study Eye (n=9,8,7)	44.4 (21.0 to 69.9)	62.5 (34.5 to 85.3)	100.0 (72.0 to 100.0)
Month 12: Study Eye (n=9,9,8)	44.4 (21.0 to 69.9)	66.7 (40.1 to 87.1)	75.0 (46.2 to 93.1)
Month 12: Non-Study Eye (n=9,9,8)	55.6 (30.1 to 79.0)	55.6 (30.1 to 79.0)	62.5 (34.5 to 85.3)

Notes
[136] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.
[137] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.
[138] - 'Number of Subjects Analyzed' signifies number of subjects analyzed in this endpoint.

No statistical analyses for this end point

Secondary: Part 2: Change from Baseline in Volume of 30-Degree Hill of Vision Assessed by Octopus 900

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End point title

Part 2: Change from Baseline in Volume of 30-Degree Hill of Vision Assessed by Octopus 900 ^[139]

End point description

Visual field testing was performed to assess change in volume of 30-degree hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this endpoint. Here negative values indicate decline in the volume of 30-degree hill vision. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 3, 6 and 12

Notes
[139] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=8,10,10)	3.61 ( 3.436 )	3.58 ( 2.549 )	5.70 ( 3.161 )
Baseline: Non-Study Eye (n=8,10,10)	3.05 ( 3.223 )	3.71 ( 2.668 )	6.23 ( 2.956 )
Change at Month 3: Study Eye (n=7,10,8)	0.80 ( 1.236 )	0.63 ( 1.162 )	-0.01 ( 5.288 )
Change at Month 3: Non-Study Eye (n=7,10,10)	0.52 ( 1.315 )	0.45 ( 0.759 )	-0.02 ( 1.110 )
Change at Month 6: Study Eye (n=8,8,7)	0.13 ( 0.499 )	0.57 ( 1.354 )	-1.22 ( 1.566 )
Change at Month 6: Non-Study Eye (n=7,9,8)	0.20 ( 0.516 )	0.30 ( 0.665 )	0.19 ( 0.576 )
Change at Month 12: Study Eye (n=7,8,8)	-0.36 ( 0.615 )	0.26 ( 1.164 )	-1.91 ( 1.498 )
Change at Month 12: Non-Study Eye (n=7,8,8)	0.29 ( 0.487 )	-0.10 ( 0.781 )	-0.56 ( 0.337 )

No statistical analyses for this end point

Secondary: Part 2: Change from Baseline in Volume of Full Field Hill of Vision Assessed by Octopus 900

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End point title

Part 2: Change from Baseline in Volume of Full Field Hill of Vision Assessed by Octopus 900 ^[140]

End point description

Visual field testing was performed to assess change in volume of full field of hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Only reliable measurements were included for analysis of this endpoint. Here negative values indicate decline in volume of full field hill vision. ITT analysis set included all subjects that were randomised under the 3-arm randomisation schedules. n=number of subjects analysed at specific timepoint.

End point type

Secondary

End point timeframe

Baseline, Month 3, 6 and 12

Notes
[140] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This endpoint was for Part 2, hence data was reported for Part 2 reporting arm groups only.

End point values	Part 2: Untreated Group	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Number of subjects analysed	9	10	10
Units: decibel
arithmetic mean (standard deviation)
Baseline: Study Eye (n=8,10,10)	7.26 ( 8.624 )	17.51 ( 17.302 )	19.48 ( 16.248 )
Baseline: Non-Study Eye (n=8,10,10)	7.05 ( 8.821 )	16.33 ( 16.803 )	21.08 ( 17.560 )
Change at Month 3: Study Eye (n=7,10,8)	1.84 ( 3.168 )	1.26 ( 3.327 )	-0.10 ( 4.059 )
Change at Month 3: Non-Study Eye (n=7,10,10)	1.36 ( 2.460 )	2.01 ( 2.832 )	0.55 ( 3.097 )
Change at Month 6: Study Eye (n=8,8,7)	-0.35 ( 1.461 )	0.98 ( 2.197 )	1.52 ( 3.897 )
Change at Month 6: Non-Study Eye (n=7,9,8)	0.46 ( 1.124 )	0.99 ( 2.324 )	1.38 ( 2.526 )
Change at Month 12: Study Eye (n=7,8,8)	-1.18 ( 1.302 )	0.81 ( 3.709 )	-0.95 ( 3.992 )
Change at Month 12: Non-Study Eye (n=7,8,8)	0.66 ( 1.193 )	0.28 ( 2.736 )	-0.91 ( 2.001 )

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Up to Day 1344 (end of study)

Adverse event reporting additional description

Safety analysis set consists of all subjects who received study treatment (vitrectomy/AAV8-RPGR).

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

23.1

Reporting group title

Part 1: BIIB112 Dose 1

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 1 (5 × 10^9 vector genomes {vg}) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 2

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 2 (1 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 3

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 3 (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 4

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 4 (1 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 5

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 5 (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 1: BIIB112 Dose 6

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single Dose 6 (5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 2: Untreated

Reporting group description

Subjects received no intervention to allow for a controlled comparison.

Reporting group title

Part 2: BIIB112 Low Dose

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single low dose (5 × 10^10 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Reporting group title

Part 2: BIIB112 High Dose

Reporting group description

Followed by vitrectomy and retinal detachment in the study eye, subjects received a single high dose (2.5 × 10^11 vg) of BIIB112 by sub-retinal injection on Day 0 (surgery day).

Serious adverse events	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6	Part 2: Untreated	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Total subjects affected by serious adverse events
subjects affected / exposed	1 / 3 (33.33%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	2 / 3 (66.67%)	1 / 9 (11.11%)	3 / 11 (27.27%)	5 / 12 (41.67%)
number of deaths (all causes)	0	0	0	0	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0	0	0	0	0
Immune system disorders
Anaphylactic reaction
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Eye disorders
Noninfective retinitis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	1 / 1	0 / 0	0 / 0	1 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Retinal detachment
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 0	1 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Subretinal fluid
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	3 / 12 (25.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	3 / 3
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Visual acuity reduced
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	0 / 9 (0.00%)	1 / 11 (9.09%)	2 / 12 (16.67%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	1 / 1	1 / 1	0 / 0	2 / 2	3 / 3
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Visual impairment
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Food poisoning
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Renal and urinary disorders
Nephrolithiasis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Infections and infestations
Appendicitis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 9 (11.11%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Urinary tract infection
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Part 1: BIIB112 Dose 1	Part 1: BIIB112 Dose 2	Part 1: BIIB112 Dose 3	Part 1: BIIB112 Dose 4	Part 1: BIIB112 Dose 5	Part 1: BIIB112 Dose 6	Part 2: Untreated	Part 2: BIIB112 Low Dose	Part 2: BIIB112 High Dose
Total subjects affected by non serious adverse events
subjects affected / exposed	3 / 3 (100.00%)	3 / 3 (100.00%)	3 / 3 (100.00%)	3 / 3 (100.00%)	3 / 3 (100.00%)	3 / 3 (100.00%)	5 / 9 (55.56%)	11 / 11 (100.00%)	12 / 12 (100.00%)
Vascular disorders
Hot flush
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Hypertension
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Surgical and medical procedures
Tooth extraction
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Immune system disorders
Multiple allergies
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Seasonal allergy
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Respiratory, thoracic and mediastinal disorders
Bronchiectasis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Cough
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	2 / 9 (22.22%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	2	0	0
Dyspnoea
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Epistaxis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	1	1	0	0	0
Psychiatric disorders
Depression
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Investigations
Blood bilirubin increased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Blood creatinine increased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	2	0
Blood glucose increased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	1	0
Blood triglycerides increased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Intraocular pressure decreased
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	1	0	0	0	0	0	1	0
Intraocular pressure increased
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	1 / 3 (33.33%)	1 / 3 (33.33%)	2 / 3 (66.67%)	1 / 9 (11.11%)	6 / 11 (54.55%)	5 / 12 (41.67%)
occurrences all number	0	1	1	3	1	4	1	7	7
Lymphocyte count decreased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Neutrophil count increased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	2 / 11 (18.18%)	4 / 12 (33.33%)
occurrences all number	0	0	0	0	0	1	0	2	4
Visual field tests abnormal
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Vitamin d decreased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
White blood cell count increased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	1	2
Injury, poisoning and procedural complications
Corneal abrasion
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
Dysphotopsia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Eye contusion
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
Muscle rupture
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Ocular procedural complication
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	0	2
Post procedural discomfort
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0	0	0	0	0	0	0
Post procedural inflammation
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	1	0	0	0	0	0	0	0
Post-traumatic neck syndrome
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Post-traumatic pain
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	1	0	0	0	0
Procedural pain
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	1	2
Skin laceration
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 9 (11.11%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	1	0	0	1	0	0
Suture related complication
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	5 / 12 (41.67%)
occurrences all number	0	0	0	0	0	1	0	0	5
Cardiac disorders
Atrial fibrillation
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 9 (11.11%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	1	0	0
Palpitations
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 9 (11.11%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0	0	0	0	1	0	0
Nervous system disorders
Dizziness
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Headache
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	2 / 11 (18.18%)	1 / 12 (8.33%)
occurrences all number	0	0	1	0	0	1	0	3	2
Paraesthesia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Tunnel vision
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Visual field defect
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	1	1	0	0	1
Ear and labyrinth disorders
Eustachian tube dysfunction
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0	0	0	0	0	0	0
Eye disorders
Aniseikonia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	1	1
Anterior chamber cell
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	5 / 11 (45.45%)	6 / 12 (50.00%)
occurrences all number	0	0	0	0	0	2	0	5	8
Anterior chamber flare
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	3 / 12 (25.00%)
occurrences all number	0	0	0	0	0	0	0	0	3
Anterior chamber inflammation
subjects affected / exposed	0 / 3 (0.00%)	2 / 3 (66.67%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	2 / 11 (18.18%)	0 / 12 (0.00%)
occurrences all number	0	2	0	1	0	0	0	2	0
Asthenopia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Blepharitis
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	1	0	0	0	0	0	0	0	1
Blindness transient
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Cataract
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 9 (11.11%)	3 / 11 (27.27%)	3 / 12 (25.00%)
occurrences all number	0	1	0	0	0	1	1	4	3
Cataract subcapsular
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	1 / 12 (8.33%)
occurrences all number	0	0	0	1	1	0	0	1	1
Choroidal detachment
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Chromatopsia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Conjunctival haemorrhage
subjects affected / exposed	3 / 3 (100.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	6 / 11 (54.55%)	9 / 12 (75.00%)
occurrences all number	3	0	0	0	0	0	0	7	11
Conjunctival hyperaemia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	1 / 12 (8.33%)
occurrences all number	0	0	0	1	0	0	0	1	2
Conjunctival irritation
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
Conjunctival oedema
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	2	0
Corneal deposits
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	1	1	0	0	0	0
Cystoid macular oedema
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	2 / 11 (18.18%)	4 / 12 (33.33%)
occurrences all number	0	0	0	0	0	0	0	2	4
Delayed light adaptation
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Detachment of macular retinal pigment epithelium
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	1	0	0	0	0
Diplopia
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	1	0	0	0	0	0	0	0	1
Dry eye
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	1	0	1	0	0	0	0	0	1
Dyschromatopsia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	1	0	0	0	0	1	0
Eye inflammation
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	2 / 3 (66.67%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	2 / 12 (16.67%)
occurrences all number	0	0	1	1	3	0	0	1	3
Eye pain
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	2 / 11 (18.18%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	1	0	2	6
Eye pruritus
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Eyelid oedema
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Eyelid ptosis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Foreign body sensation in eyes
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	1 / 11 (9.09%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	1	0	1	2
Hypotony of eye
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Iridocyclitis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	1	1	0	0	0	0	0
Iritis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	2 / 11 (18.18%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	2	3
Lacrimation increased
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	0	2
Lenticular pigmentation
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Macular fibrosis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Maculopathy
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	1	0	0	0	0
Metamorphopsia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	4 / 12 (33.33%)
occurrences all number	0	0	0	0	0	0	0	1	4
Noninfective retinitis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	3 / 3 (100.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	3 / 12 (25.00%)
occurrences all number	0	0	0	1	2	5	0	1	3
Ocular discomfort
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	2 / 11 (18.18%)	2 / 12 (16.67%)
occurrences all number	0	0	1	0	0	0	0	2	2
Ocular hypertension
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	2
Photophobia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	0	2
Posterior capsule opacification
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 9 (11.11%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	1	0	0
Retinal artery embolism
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Retinal cyst
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Retinal degeneration
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	2
Retinal deposits
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	0	2
Retinal fovea disorder
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Retinal haemorrhage
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	1	2
Retinal infiltrates
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Retinal oedema
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0	0	0	0	0	0	0
Retinal tear
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	0	2
Retinal thickening
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Retinoschisis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Scleritis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Subretinal fluid
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	2 / 11 (18.18%)	3 / 12 (25.00%)
occurrences all number	0	0	1	0	0	0	0	2	3
Vision blurred
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	1 / 3 (33.33%)	1 / 3 (33.33%)	1 / 9 (11.11%)	1 / 11 (9.09%)	4 / 12 (33.33%)
occurrences all number	0	0	0	1	1	1	1	1	7
Visual acuity reduced
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	3 / 12 (25.00%)
occurrences all number	0	0	0	0	1	0	0	1	3
Visual impairment
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	3 / 12 (25.00%)
occurrences all number	0	0	0	0	0	0	0	1	4
Vitreal cells
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	4 / 12 (33.33%)
occurrences all number	0	0	0	0	0	0	0	1	4
Vitreous detachment
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Vitritis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	5 / 12 (41.67%)
occurrences all number	0	0	0	0	0	0	0	0	6
Gastrointestinal disorders
Dyspepsia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Nausea
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	2 / 12 (16.67%)
occurrences all number	0	0	0	0	0	0	0	1	2
Stomatitis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Vomiting
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	1	1
Skin and subcutaneous tissue disorders
Acne
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	3 / 12 (25.00%)
occurrences all number	0	0	0	0	0	1	0	0	4
Erythema
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Night sweats
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	0	0	0	0	0	0	1
Rash
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	1	0	0	0	0
Urticaria
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	1	0	0	0	0	0	0	0
Musculoskeletal and connective tissue disorders
Chondrocalcinosis pyrophosphate
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Muscle spasms
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	1	0	0	0	0	0	0	0
Pain in extremity
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Infections and infestations
Bursitis infective
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 9 (11.11%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	1	0	0
Conjunctivitis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	1	0	0	0	0	1	0
Ear infection
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	1	0	0	0	0	0	0	1	0
Gastroenteritis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	1	0	0	0	0
Hypopyon
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Infected dermal cyst
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Influenza
subjects affected / exposed	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0	0	0	0	0	0	0
Lower respiratory tract infection
subjects affected / exposed	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 9 (0.00%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	1	0	0	0	0	0	2	0
Nasopharyngitis
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 3 (0.00%)	0 / 9 (0.00%)	2 / 11 (18.18%)	0 / 12 (0.00%)
occurrences all number	0	0	1	0	1	0	0	3	0
Upper respiratory tract infection
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 3 (33.33%)	0 / 9 (0.00%)	2 / 11 (18.18%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	4	0	2	0
Urinary tract infection
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 9 (11.11%)	0 / 11 (0.00%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	1	0	0
Metabolism and nutrition disorders
Hyperglycaemia
subjects affected / exposed	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 9 (11.11%)	1 / 11 (9.09%)	0 / 12 (0.00%)
occurrences all number	0	0	0	0	0	0	1	1	0

More information

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Were there any global substantial amendments to the protocol? Yes

01 Dec 2016

Updated exclusion criteria to include time frame (3 months) within which subjects were to be compliant with the use of barrier contraception.

26 May 2017

Updated inclusion criteria for BCVA in maximum tolerated dose (MTD) cohort to allow inclusion of a wider study population in this cohort. LLVA and full field stimulus threshold added as additional assessments, in order to provide additional data to measure changes in disease progression over time. Removed microperimetry assessment at visit 4 because utility of the assessment at this timepoint was expected to be low. Updated schedule of procedures to include triplicate testing at screening/baseline, year 1, and early termination (ET) visit for EDTRS BCVA, LLVA, microperimetry, and visual fields. Triplicates were added to improve data quality by mitigating potential learning effects and providing a measure of test-retest variability. Added a corticosteroid compliance review to clarify that corticosteroid compliance would be monitored. Screening/baseline visit timing changed from within 4 weeks of visit 2 (± 2 weeks) to within 8 weeks of visit 2 (± 2 weeks) in order to provide more flexibility in scheduling subsequent visits.

15 Dec 2017

Study design updated to confirm assessment of Cohort 4 (1 × 10^11 vg) as standard, with potential Cohorts 5 (2.5 × 10^11 vg) and 6 (5 × 10^11 vg) to allow wider dose exploration, if appropriate. Study design updated to include additional visits 10 (Month 18) and 11 (Year 2) to allow longer duration of post-treatment follow-up (24 months in total), in line with feedback from regulatory agencies. Updated MTD cohort eligibility criteria to include subjects ≥ 10 years of age, to allow inclusion of a wider study population in this cohort. Updated eligibility criteria to specify that ellipsoid zone (EZ) at screening must be within the borders of the spectral domain optical coherence tomography (SD-OCT) scan, to ensure that subjects have active disease within the macular region (without which the central reading center was unable to adequately grade the SD-OCT images which would negatively impact secondary study endpoints). DLT definition updated to exclude events related to the surgical procedure, to ensure only genuine cases of DLTs were identified and reported. Removed several study assessments from specific visits, e.g., blood pressure and pulse (visits 5, 9, and ET), viral shedding (visit 6), fundus autofluorescence (Visit 4), triplicate microperimetry and visual field assessments (visits 9 and ET). Following feedback from the central reading center and several study sites, the utility of the assessments at these specific timepoints was considered low.

16 Jan 2018

Updated inclusion criteria to clarify and allow the consent of male subjects ≥ 10 years who would require parental permission and subject assent (if applicable). Exclusion criteria (use of contraception method) was clarified for male subjects ≥ 10 years to include “if applicable”. Removal of the following study procedures at visits 5, 11, and ET: blood pressure and pulse; collection of safety blood samples.

18 May 2018

Added the expansion of an active-control cohort of subjects in Part II to occur concurrently with the expansion of the MTD cohort in a randomized, double-masked fashion, in order to provide greater efficacy information through a comparison of low- and high-dose outcomes. Clarified the consent procedures for subjects under 10 years of age. Removed assessments of LLVA, blood pressure, pulse, and safety blood sampling at various visits as there was no rationale for the assessments and it lessened the burden on the site. Clarified the primary endpoint in Part I regarding the incidence of DLTs and AEs. Specified timepoints for the secondary endpoints for precision of analysis. Cohorts 5 and 6 in part I altered from optional to protocol-defined due to a lack of DLTs observed in the ongoing study cohorts 1 to 4. Specified that if DLTs were observed in cohort 5, no escalation to cohort 6 would occur.

16 Nov 2018

Clarified that dose 5 and 3 from part I were now defined as high (2.5 × 10^11 vg) and low (5 × 10^10 vg) dose arms in Part II, based on rationale that these were most likely to define safety and efficacy. Added a third untreated arm to better establish efficacy and safety of gene therapy. Specified that subjects were to be randomized in a 1:1:1 allocation, with double masking to dose in treated arm, and masked assessments of efficacy. Study duration shortened to 12 months of follow-up for part II dose expansion because a long-term follow up study was initiated to continue follow-up of AAV8 RPGR-treated subjects from 12 months out to 5 years. Phase 1 subjects were still followed for 24 months and then also invited to participate in long-term study. Endpoints and inclusion criteria separated for parts I and II to improve clarity. Endpoints were also updated for both parts I and II. Added microperimetry at 3 months as an efficacy endpoint in part II. Added an inclusion range for microperimetry to part II to assure inclusion of subjects with modifiable disease and avoid inclusion of those with microperimetry values subject to ceiling effects. Removed reading test, color vision, and full field stimulus threshold assessments due to the excessive burden of study visits on subjects, and limited value of these assessments in understanding both disease and effects of the treatment. Modified steroid regimen to improve safety and to prevent potential inflammatory response to therapy. A standardized adult dose was selected, the taper lengthened, and a 2-month visit added to assess participants at the end of the steroid therapy. A standardized adult dose was selected, taper lengthened, and a 2-month visit added to assess subjects at the end of the steroid therapy. Added information of the procedures related to unmasking in compliance with good clinical practices (GCP). Serious adverse events (SAE) reporting changed from within 7 days to immediately, per regional recommendations.

10 Dec 2018

Clarified the procedures and requirements for assessments performed at each visit compared with the schedule of study procedures: Added footnotes for BCVA assessment, immunogenicity sampling, and corticosteroid review to the in-text footnotes and schedule of study procedures; Removed fundus photography from visits 6 and 7; Removed viral shedding assessment from visit 6; Added autofluorescence assessment to the unscheduled visit.

18 Dec 2018

Modified the unmasking information to adhere more closely to good clinical practice (GCP). Added mobility test to the early termination (ET) visit list of procedures, to be conducted if feasible.

08 Mar 2019

Changed the date from which subjective ophthalmic assessments were made by a masked assessor from Month 1 (visit 5) to Month 3 (visit 6), to ensure that any residual signs or symptoms related to the procedure had completely resolved and did not unmask the assessor. Corrected a typographical error in the doses during the steroid tapering regimen.

14 Aug 2019

Added an administrative interim analysis after all part II subjects completed 3 months of post treatment follow-up, to enable continued alignment with regulatory agencies on the acceptability of the approach taken in evaluating the safety and efficacy of AAV8 RPGR. Modified the following inclusion and exclusion criteria. Removed the requirement that participants have a measurable EZ on optical coherence imaging, to allow the broader study of XLRP participants without a measurable EZ. Documentation of a positive genetic screen updated to specify that a pathogenic mutation must have been present for clarification of test results from genetic screening. Specified that participants must achieve a BCVA ≥ 34 letters in both eyes and not just the study eye, to exclude monocular participants or participants with very poor vision in the fellow eye. Updated criteria to exclude participants who were unsuitable candidates for surgery for any reason. Improved the study masking language and descriptions of the specific assessments that must be conducted in a masked fashion. Added a description of the maintenance of masking of study teams during the interim analysis (IA) proceedings. Removed adaptive optics-optical coherence tomography (AO-OCT) assessments due to lack of operational feasibility. Changed some statistical analyses and endpoints. Updated the protocol template language regarding safety assessments to include better definitions of SAEs and reporting of SAEs, consistent with other protocols. Better defined the SAE of vision loss post treatment, particularly regarding the timing. Added a description of the DMC for Part II.

10 Oct 2019

Increased the maximum time period between the baseline/screening visit and the day of surgery (visit 2) from 8 weeks to 12 weeks, to provide operational flexibility for the sites and investigators and to minimize subject burden.

01 Oct 2020

Reduced the sample size to 29 subjects; study drug expired at the end of September 2019, and it was not possible to extend the shelf-life or use a new drug supply without causing significant delay to the study. This change resulted in multiple updates throughout the study design and statistical analyses sections of the protocol. Removed the requirement for an IA because it was no longer deemed necessary. Removed month 9 from the timepoints at which the endpoint of fundus autofluorescence was assessed (in both parts I), because it was added in error. Updated Part II secondary endpoints to separate the central 16-loci microperimetry mean change from baseline from the 68-loci microperimetry mean change from baseline. More granularity was needed for this endpoint as multiple analysis were to be conducted on this measure. Removed months 1, 2, and 9 timepoints from the part II endpoints of visual field since this is in error given that they are not collected at these timepoints. Added the timepoints for microperimetry and safety and secondary efficacy assessments in the description of study design with the schedule of assessments table. Added text providing instructions in case of missed final visits. Corrected the no observed adverse effect level (NOAEL) from greater than to equal to 3.54 × 10^9 vg/eye. Updated text to clarify that efficacy assessments were only masked at certain timepoints, not throughout the study. Clarified the definition of the end of study.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

http://www.ncbi.nlm.nih.gov/pubmed/32094925

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Clinical trials

Clinical Trial Results: A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Part 1: Number of Subjects with Dose-Limiting Toxicities (DLTs)

Primary: Part 1: Number of Subjects with Dose-Limiting Toxicities (DLTs)

Primary: Part 1: Number of Subjects with Treatment-Emergent Adverse Events (TEAEs)

Primary: Part 1: Number of Subjects with Treatment-Emergent Adverse Events (TEAEs)

Primary: Part 2: Percentage of Study Eyes with ≥7 Decibels (dB) Improvement from Baseline at ≥5 of the 16 Central Loci of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry

Primary: Part 2: Percentage of Study Eyes with ≥7 Decibels (dB) Improvement from Baseline at ≥5 of the 16 Central Loci of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry

Primary: Part 2: Number of Subjects with TEAEs

Primary: Part 2: Number of Subjects with TEAEs

Secondary: Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry

Secondary: Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry

Secondary: Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry

Secondary: Part 1: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry

Secondary: Part 1: Change from Baseline in Mean Sensitivity of the 16 Central Loci

Secondary: Part 1: Change from Baseline in Mean Sensitivity of the 16 Central Loci

Secondary: Part 1: Change from Baseline in Mean Sensitivity of the 68 Central Loci

Secondary: Part 1: Change from Baseline in Mean Sensitivity of the 68 Central Loci

Secondary: Part 1: Change from Baseline in Best Corrected Visual Acuity (BCVA) Score

Secondary: Part 1: Change from Baseline in Best Corrected Visual Acuity (BCVA) Score

Secondary: Part 1: Change from Baseline in Low Luminance Visual Acuity (LLVA) Score

Secondary: Part 1: Change from Baseline in Low Luminance Visual Acuity (LLVA) Score

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Increase from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Increase from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Increase from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥15 Letters Loss from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥10 Letters Loss from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for BCVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with a ≥5 Letters Loss from Baseline for LLVA

Secondary: Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for BCVA

Secondary: Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for BCVA

Secondary: Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for LLVA

Secondary: Part 1: Percentage of Eyes with Change from Baseline > -5 Letters for LLVA

Secondary: Part 1: Change from Baseline in Central Ellipsoid Area

Secondary: Part 1: Change from Baseline in Central Ellipsoid Area

Secondary: Part 1: Change from Baseline in Central Horizontal Ellipsoid Width

Secondary: Part 1: Change from Baseline in Central Horizontal Ellipsoid Width

Secondary: Part 1: Change from Baseline in Fundus Autofluoroscence- Total Area of Preserved Autofluoroscence

Secondary: Part 1: Change from Baseline in Fundus Autofluoroscence- Total Area of Preserved Autofluoroscence

Secondary: Part 1: Change from Baseline in Fundus Autofluoroscence- Distance from Foveal Center (FC) to Nearest Border of Preserved Autofluoroscence

Secondary: Part 1: Change from Baseline in Fundus Autofluoroscence- Distance from Foveal Center (FC) to Nearest Border of Preserved Autofluoroscence

Secondary: Part 1: Change from Baseline in Volume of 30-Degree Hill of Vision

Secondary: Part 1: Change from Baseline in Volume of 30-Degree Hill of Vision

Secondary: Part 1: Change from Baseline in Volume of Full Field Hill of Vision

Secondary: Part 1: Change from Baseline in Volume of Full Field Hill of Vision

Secondary: Part 1: Change from Baseline in Contrast Sensitivity

Secondary: Part 1: Change from Baseline in Contrast Sensitivity

Secondary: Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry

Secondary: Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry

Secondary: Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry

Secondary: Part 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry

Secondary: Part 2: Change from Baseline in Mean Sensitivity of the 16 Central Loci

Secondary: Part 2: Change from Baseline in Mean Sensitivity of the 16 Central Loci

Secondary: Part 2: Change from Baseline in Mean Sensitivity of the 68 Central Loci

Secondary: Part 2: Change from Baseline in Mean Sensitivity of the 68 Central Loci

Clinical Trial Results:
A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)