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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

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    Trial identification
    Sponsor protocol code
    274RP101 (NSR-RPGR-01)
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03116113
    WHO universal trial number (UTN)
    -
    Sponsors
    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
    Paediatric regulatory details
    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?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    24 Mar 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Nov 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    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
    Population of trial subjects
    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
    50
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    49
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    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.

    Pre-assignment
    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
    Period 1 title
    Overall Period
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Carer, Data analyst, 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.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    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.

    Arm title
    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.

    Arm title
    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.

    Arm title
    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.

    Arm title
    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.

    Arm title
    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.

    Arm title
    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
    Arm title
    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.

    Arm title
    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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    Baseline characteristics reporting groups
    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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    End points reporting groups
    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.
    Statistical analysis title
    Untreated Group Vs BIIB112 Low Dose
    Comparison groups
    Part 2: Untreated Group v Part 2: BIIB112 Low Dose
    Number of subjects included in analysis
    17
    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
    43
    Statistical analysis title
    Untreated Group Vs BIIB112 High Dose
    Comparison groups
    Part 2: Untreated Group v Part 2: BIIB112 High Dose
    Number of subjects included in analysis
    17
    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
    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 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

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    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

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    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

    Close 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

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    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

    Close Top of page
    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

    Close Top of page
    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

    Close 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

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    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

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    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)