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    Clinical Trial Results:
    Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene

    Summary
    EudraCT number
    		 2020-002873-88
    Trial protocol
    		 IE   NL   BE   ES   FR   DK   DE   IT   Outside EU/EEA  
    Global end of trial date
    30 Sep 2024

    Results information
    Results version number
    		 v2(current)
    This version publication date
    04 Jul 2025
    First version publication date
    13 Apr 2025
    Other versions
    		 v1
    Version creation reason
    Summary report(s)
    		 Rationale_extension request_

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    MGT-RPGR-021
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04671433
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Janssen Research & Development, LLC
    Sponsor organisation address
    920 Route 202 South, Raritan, New Jersey, United States, 08869
    Public contact
    Clinical Registry Group, Janssen Research & Development, LLC, ClinicalTrialsEU@its.jnj.com
    Scientific contact
    Clinical Registry Group, Janssen Research & Development, LLC, ClinicalTrialsEU@its.jnj.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002827-PIP01-20
    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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    30 Sep 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Sep 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the effect of bilateral treatment with an adeno-associated virus vector with a serotype 5 capsid, a human rhodopsin kinase promoter, and the retinitis pigmentosa guanosine triphosphatase regulator gene (AAV5-hRKp.RPGR) on functional vision as measured by vision-guided mobility assessment (VMA).
    Protection of trial subjects
    This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practices and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Mar 2021
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 48 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 7
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    Switzerland: 10
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    Spain: 7
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    United Kingdom: 13
    Country: Number of subjects enrolled
    Israel: 6
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    Netherlands: 9
    Country: Number of subjects enrolled
    United States: 35
    Worldwide total number of subjects
    97
    EEA total number of subjects
    32
    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)
    2
    Adolescents (12-17 years)
    4
    Adults (18-64 years)
    91
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Total 97 adult and pediatric subjects were enrolled, out of which 94 subjects completed. Study consisted of Cohort (C) 1, 2 and pediatric cohort. C 1 subjects were randomised to assess safety after treatment up to Week 10. Randomisation for C 2 began after the IDMC completed their review of C 1 safety data and recommended to continue study.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Deferred Group (Control Group)
    Arm description
    		 Adults (cohort [C] 1 and 2) and pediatric subjects with X-linked retinitis pigmentosa (XLRP) caused by mutations in the human retinitis pigmentosa guanosine triphosphatase regulator (RPGR) gene (RPGR-XLRP) were randomised in the Deferred Group and did not receive any treatment in the study. Subjects were followed up for safety from Day 1 up to Week 52.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Arm title
    		 Immediate Treatment Group: RPGR2e11
    Arm description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive an adeno-associated virus vector with a serotype 5 capsid, a human rhodopsin kinase promoter, and the retinitis pigmentosa guanosine triphosphatase regulator gene (AAV5-hRKp.RPGR) therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 2.0*10^11 viral genome per millilitre (vg/mL) (RPGR2e11) dose (300 microlitres [mcL] to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug on Day 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    RPGR2e11
    Investigational medicinal product code
    Other name
    Botaretigene sparoparvovec
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subretinal use
    Dosage and administration details
    Subjects received AAV5-hRKp.RPGR gene therapy with (RPGR2e11) at a dose of 300 mcL to 800 mcL in each eye on the day of first eye surgery on Day 1 and on the day of second eye surgery between Day 8 to Day 22.

    Arm title
    		 Immediate Treatment Group: RPGR4e11
    Arm description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive AAV5-hRKp.RPGR gene therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 4.0*10^11 vg/mL (RPGR4e11) dose (300 mcL to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug on Day 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    RPGR4e11
    Investigational medicinal product code
    Other name
    Botaretigene sparoparvovec
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subretinal use
    Dosage and administration details
    Subjects received AAV5-hRKp.RPGR gene therapy with (RPGR4e11) at a dose of 300 mcL to 800 mcL in each eye on the day of first eye surgery on Day 1 and on the day of second eye surgery between Day 8 to Day 22.

    Number of subjects in period 1
    		Deferred Group (Control Group) Immediate Treatment Group: RPGR2e11 Immediate Treatment Group: RPGR4e11
    Started
    33
    33
    31
    Treated
    0 [1]
    32
    30
    Completed
    33
    32
    29
    Not completed
    0
    1
    2
         Consent withdrawn by subject
    -
    1
    -
         Subject did not meet inclusion criteria
    -
    -
    1
         Lost to follow-up
    -
    -
    1
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: Only reported subjects were planned to be included in this milestone.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Deferred Group (Control Group)
    Reporting group description
    		 Adults (cohort [C] 1 and 2) and pediatric subjects with X-linked retinitis pigmentosa (XLRP) caused by mutations in the human retinitis pigmentosa guanosine triphosphatase regulator (RPGR) gene (RPGR-XLRP) were randomised in the Deferred Group and did not receive any treatment in the study. Subjects were followed up for safety from Day 1 up to Week 52.

    Reporting group title
    Immediate Treatment Group: RPGR2e11
    Reporting group description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive an adeno-associated virus vector with a serotype 5 capsid, a human rhodopsin kinase promoter, and the retinitis pigmentosa guanosine triphosphatase regulator gene (AAV5-hRKp.RPGR) therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 2.0*10^11 viral genome per millilitre (vg/mL) (RPGR2e11) dose (300 microlitres [mcL] to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug on Day 1.

    Reporting group title
    Immediate Treatment Group: RPGR4e11
    Reporting group description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive AAV5-hRKp.RPGR gene therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 4.0*10^11 vg/mL (RPGR4e11) dose (300 mcL to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug on Day 1.

    Reporting group values
    		 Deferred Group (Control Group) Immediate Treatment Group: RPGR2e11 Immediate Treatment Group: RPGR4e11 Total
    Number of subjects
    		 33 33 31 97
    Age categorical
    Units: Subjects
        In Utero
    		 0 0 0 0
        Preterm newborn infants (gestional age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days - 23 months)
    		 0 0 0 0
        Children (2 - 11 years)
    		 1 0 1 2
        12 - 17 years
    		 1 2 1 4
        Adults (18 - 64 years)
    		 31 31 29 91
        From 65 - 84 years
    		 0 0 0 0
        85 years and over
    		 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 24.9 ( 7.47 ) 27.0 ( 9.36 ) 28.0 ( 7.54 ) -
    Gender categorical
    Units: Subjects
        Male
    		 32 31 31 94
        Female
    		 1 2 0 3

    End points

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    End points reporting groups
    Reporting group title
    Deferred Group (Control Group)
    Reporting group description
    		 Adults (cohort [C] 1 and 2) and pediatric subjects with X-linked retinitis pigmentosa (XLRP) caused by mutations in the human retinitis pigmentosa guanosine triphosphatase regulator (RPGR) gene (RPGR-XLRP) were randomised in the Deferred Group and did not receive any treatment in the study. Subjects were followed up for safety from Day 1 up to Week 52.

    Reporting group title
    Immediate Treatment Group: RPGR2e11
    Reporting group description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive an adeno-associated virus vector with a serotype 5 capsid, a human rhodopsin kinase promoter, and the retinitis pigmentosa guanosine triphosphatase regulator gene (AAV5-hRKp.RPGR) therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 2.0*10^11 viral genome per millilitre (vg/mL) (RPGR2e11) dose (300 microlitres [mcL] to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug on Day 1.

    Reporting group title
    Immediate Treatment Group: RPGR4e11
    Reporting group description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive AAV5-hRKp.RPGR gene therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 4.0*10^11 vg/mL (RPGR4e11) dose (300 mcL to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug on Day 1.

    Subject analysis set title
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The pooled immediate treatment group included cohort 2 subjects of RPGR2e11 and RPGR4e11 dose groups combined.

    Subject analysis set title
    C 2: Control Group
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Cohort 2 subjects with RPGR-XLRP were randomised and did not receive any treatment in the study. Subjects were followed up for safety from Day 1 up to Week 52.

    Primary: Cohort 2: Percentage of Subjects With Vision-guided Mobility Assessment (VMA) Response in Binocular Assessment at Week 52

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    End point title
    		 Cohort 2: Percentage of Subjects With Vision-guided Mobility Assessment (VMA) Response in Binocular Assessment at Week 52
    End point description
    Percentage of subjects with VMA response in binocular assessment at Week 52 was reported. The VMA tests the ability of a subject to navigate through a maze at various illumination (lux) levels and presents an environment with obstacles that was meant to simulate real-life conditions at varying light levels. VMA responder was defined as a subject who passes a lux level at least 2 levels lower at Week 52 compared to the lowest lux level the subject passed at baseline. Intent-to-treat (ITT) analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Primary
    End point timeframe
    At Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Percentage of responders
        number (not applicable)
    27.3
    13.3
    Statistical analysis title
    		 Statistical Analysis-1
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.247
    Method
    		 Fisher exact
    Parameter type
    		 Difference in percentage of responders
    Point estimate
    13.4
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.6
         upper limit
    		 30.5

    Secondary: Cohort 2: Change From Baseline in Mean Retinal Sensitivity (MRS) Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52

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    End point title
    		 Cohort 2: Change From Baseline in Mean Retinal Sensitivity (MRS) Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
    End point description
    Change from baseline in MRS10 at Week 52 were reported. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Decibels (dB)
        least squares mean (standard error)
    0.88 ( 0.22 )
    -0.36 ( 0.29 )
    Statistical analysis title
    		 Statistical Analysis-2
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Least square (LS) mean difference
    Point estimate
    1.24
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.54
         upper limit
    		 1.94

    Secondary: Cohort 2: Percentage of Subjects With VMA Response in Binocular Assessment at Week 52 Comparing RPGR2e11 Group with Control Group

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    End point title
    		 Cohort 2: Percentage of Subjects With VMA Response in Binocular Assessment at Week 52 Comparing RPGR2e11 Group with Control Group [1]
    End point description
    Percentage of subjects with VMA response in binocular assessment at Week 52 comparing RPGR2e11 group with control group was reported. The VMA tests the ability of a subject to navigate through a maze at various illumination (lux) levels and presents an environment with obstacles that was meant to simulate real-life conditions at varying light levels. VMA responder was defined as a subject who passes a lux level at least 2 levels lower at Week 52 compared to the lowest lux level the subject passed at baseline. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint data for immediate treatment groups RPGR2e11 and deferred treatment was planned to be reported.
    End point type
    Secondary
    End point timeframe
    At Week 52
    Notes
    [1] - 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 planned to be reported for specified arms only.
    End point values
    		 Deferred Group (Control Group) Immediate Treatment Group: RPGR2e11
    Number of subjects analysed
    30
    29
    Units: Percentage of responders
        number (not applicable)
    13.3
    24.1
    Statistical analysis title
    		 Statistical Analysis-3
    Comparison groups
    Deferred Group (Control Group) v Immediate Treatment Group: RPGR2e11
    Number of subjects included in analysis
    59
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Difference in percentage of responders
    Point estimate
    10
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -9.7
         upper limit
    		 29.7

    Secondary: Cohort 2: Percentage of Subjects With VMA Response in Binocular Assessment at Week 52 Comparing RPGR4e11 Group with Control Group

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    End point title
    		 Cohort 2: Percentage of Subjects With VMA Response in Binocular Assessment at Week 52 Comparing RPGR4e11 Group with Control Group [2]
    End point description
    Percentage of subjects with VMA response in binocular assessment at Week 52 comparing RPGR4e11 group with control group was reported. The VMA tests the ability of a subject to navigate through a maze at various illumination (lux) levels and presents an environment with obstacles that was meant to simulate real-life conditions at varying light levels. VMA responder was defined as a subject who passes a lux level at least 2 levels lower at Week 52 compared to the lowest lux level the subject passed at baseline. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint data for immediate treatment groups RPGR4e11 and deferred treatment was planned to be reported.
    End point type
    Secondary
    End point timeframe
    At Week 52
    Notes
    [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 planned to be reported for specified arms only.
    End point values
    		 Deferred Group (Control Group) Immediate Treatment Group: RPGR4e11
    Number of subjects analysed
    30
    26
    Units: Percentage of responders
        number (not applicable)
    13.3
    30.8
    Statistical analysis title
    		 Statistical Analysis-4
    Comparison groups
    Deferred Group (Control Group) v Immediate Treatment Group: RPGR4e11
    Number of subjects included in analysis
    56
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Difference in percentage of responders
    Point estimate
    16.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -5
         upper limit
    		 38.7

    Secondary: Cohort 2: Percentage of Pointwise Responders in Full Visual Field at Week 52

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    End point title
    		 Cohort 2: Percentage of Pointwise Responders in Full Visual Field at Week 52
    End point description
    Percentage of pointwise responders in full visual field at Week 52 were reported. A responder at Week 52 was defined as a subject with at least 1 treated eye with greater than or equal to (>=)7 dB increase from baseline in >=5 loci repeated at Week 52 and 1 or more other time points prior to Week 52. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    At Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Percentage of responders
        number (not applicable)
    58.2
    23.3
    Statistical analysis title
    		 Statistical Analysis-5
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Difference in percentage of responders
    Point estimate
    36.6
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 18.2
         upper limit
    		 54.9

    Secondary: Cohort 2: Percentage of Pointwise Responders in the Central 30 Degrees Visual Field at Week 52

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    End point title
    		 Cohort 2: Percentage of Pointwise Responders in the Central 30 Degrees Visual Field at Week 52
    End point description
    Percentage of pointwise responders in the central 30 degrees visual field at Week 52 were reported. A responder at Week 52 was defined as a subject with at least 1 treated eye with a >=7 dB increase from baseline in >=5 loci repeated at Week 52 and 1 or more other time points prior to Week 52. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    At Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Percentage of responders
        number (not applicable)
    47.3
    10.0
    Statistical analysis title
    		 Statistical Analysis-6
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Difference in percentage of responders
    Point estimate
    38.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 21.4
         upper limit
    		 54.7

    Secondary: Cohort 2: Percentage of VMA Responders in the “Worse-seeing Eye” at Week 52

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    End point title
    		 Cohort 2: Percentage of VMA Responders in the “Worse-seeing Eye” at Week 52
    End point description
    The VMA tests the ability of a subject to navigate through a maze at various illumination (lux) levels and presents an environment with obstacles that was meant to simulate real-life conditions at varying light levels. VMA responder was defined as a subject who passes a lux level at least 2 levels lower at Week 52 compared to the lowest lux level the subject passed at baseline. Worse-seeing Eye was determined by best corrected visual acuity (BCVA). If BCVA was identical in both eyes, static perimetry mean retinal sensitivity (MRS) was used to determine the worse-seeing eye. If both BCVA and MRS were equal in both eyes, the right eye was the first eye receiving treatment (and considered as the worse-seeing eye). ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    At Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Percentage of responders
        number (not applicable)
    29.1
    23.3
    Statistical analysis title
    		 Statistical Analysis-7
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Difference in percentage of responders
    Point estimate
    5.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -13.4
         upper limit
    		 25.3

    Secondary: Cohort 2: Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ)- Extreme Lighting Domain Score at Week 52

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    End point title
    		 Cohort 2: Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ)- Extreme Lighting Domain Score at Week 52
    End point description
    The mLLQ was 30-item disease-specific questionnaire for use in eye diseases to assess self reported visual problems under low luminance and at night. The instrument consisted of 6 domains and was scored by each of its 6 domains (extreme lighting, mobility, general dim lighting, peripheral vision, driving, emotional distress). The extreme lighting domain comprised of 7 items (Items 1 to 7) and was used to assess patient-reported functional vision under low light/at night and in extreme (ie, bright light) conditions. Response options included 5- point Likert Scales ranging from 'No difficulty at all' to 'Not able to' and 'None of the time' to 'All of the time'. The mLLQ uses scale from 0 to 100, higher scores reflect a higher level of functioning. A positive change from baseline reflects improvement; negative reflects worsening. ITT analysis set. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Score on a scale
        least squares mean (standard error)
    1.80 ( 1.50 )
    -5.49 ( 2.05 )
    Statistical analysis title
    		 Statistical Analysis-8
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 LS mean difference
    Point estimate
    7.29
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.2
         upper limit
    		 12.38

    Secondary: Cohort 2: Change From Baseline in Low Luminance Visual Acuity (LLVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52

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    End point title
    		 Cohort 2: Change From Baseline in Low Luminance Visual Acuity (LLVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52
    End point description
    Change from baseline in LLVA by ETDRS chart letter score in monocular assessment at Week 52 were reported. The LLVA was determined using the ETDRS chart by counting the number of ETDRS letters read under low light conditions. ETDRS chart letter score ranged from 0 to 100 letters. 20/20 snellen was equivalent to 85 letters on the ETDRS chart or 0 logMAR. The higher ETDRS score indicated the better vision. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Number of ETDRS letters
        least squares mean (standard error)
    6.86 ( 0.94 )
    2.11 ( 1.26 )
    Statistical analysis title
    		 Statistical Analysis-9
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 LS mean difference
    Point estimate
    4.75
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.64
         upper limit
    		 7.86

    Secondary: Cohort 2: Change From Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Monocular Assessment at Week 52

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    End point title
    		 Cohort 2: Change From Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Monocular Assessment at Week 52
    End point description
    Change from baseline in BCVA by ETDRS chart letter score in monocular assessment at Week 52 were reported. The BCVA was determined using the ETDRS chart by counting the number of ETDRS letters read under normal lighting conditions. ETDRS chart letter score ranged from 0 to 100 letters. 20/20 snellen was equivalent to 85 letters on the ETDRS chart or 0 logMAR. The higher ETDRS score indicated the better vision. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Number of ETDRS letters
        least squares mean (standard error)
    1.79 ( 0.54 )
    0.83 ( 0.73 )
    Statistical analysis title
    		 Statistical Analysis-10
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 LS mean difference
    Point estimate
    0.96
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.84
         upper limit
    		 2.76

    Secondary: Cohort 2: Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma in Static Perimetry (MRS90) at Week 52

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    End point title
    		 Cohort 2: Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma in Static Perimetry (MRS90) at Week 52
    End point description
    Change from baseline in MRS90 at Week 52 were reported. ITT analysis set included all adult subjects who were randomly assigned to a treatment group (treated or control group) in Cohort 2. In this endpoint pooled data for immediate treatment groups RPGR2e11 and RPGR4e11 was planned to be reported.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 C 2: Control Group
    Number of subjects analysed
    55
    30
    Units: Decibels (dB)
        least squares mean (standard error)
    0.00 ( 0.19 )
    -0.93 ( 0.26 )
    Statistical analysis title
    		 Statistical Analysis-11
    Comparison groups
    C 2: Pooled Immediate Treatment Group: RPGR2e11 & RPGR4e11 v C 2: Control Group
    Number of subjects included in analysis
    85
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 LS mean difference
    Point estimate
    0.92
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.29
         upper limit
    		 1.55

    Secondary: Number of Subjects With Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Subjects With Treatment-Emergent Adverse Events (TEAEs)
    End point description
    Adverse event (AE) was any untoward medical occurrence in a clinical study subject administered a medicinal (investigational or non-investigational) product. AE does not necessarily have a causal relationship with intervention. Any AE occurring on or after the initial administration of study drug and on or before the last visit of the study was considered treatment-emergent for immediate treatment group. Any AE occurring on or after the last baseline visit (Day 1) and on or before the last visit of the study was considered treatment-emergent for deferred group. TEAEs included serious and non-serious events. Analysis population included all subjects randomised to immediate treatment and had received AAV5-hRKp.RPGR administration in at least 1 eye, and all subjects randomised to deferred treatment and had completed at least 1 baseline assessment during deferred period.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Week 52
    End point values
    		 Deferred Group (Control Group) Immediate Treatment Group: RPGR2e11 Immediate Treatment Group: RPGR4e11
    Number of subjects analysed
    33
    32
    30
    Units: Subjects
    19
    32
    30
    No statistical analyses for this end point

    Secondary: Number of Subjects With Clinically Significant Change in Laboratory Parameters

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    End point title
    		 Number of Subjects With Clinically Significant Change in Laboratory Parameters
    End point description
    Number of subjects with clinically significant change in laboratory parameters (clinical chemistry and hematology) were reported. Analysis population included all subjects randomised to immediate treatment and had received AAV5-hRKp.RPGR administration in at least 1 eye, and all subjects randomised to deferred treatment and had completed at least 1 baseline assessment during deferred period.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Week 52
    End point values
    		 Deferred Group (Control Group) Immediate Treatment Group: RPGR2e11 Immediate Treatment Group: RPGR4e11
    Number of subjects analysed
    33
    32
    30
    Units: Subjects
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to Week 52
    Adverse event reporting additional description
    Analysis population included all subjects randomised to immediate treatment and had received AAV5-hRKp.RPGR administration in at least 1 eye, and all subjects randomised to deferred treatment and had completed at least 1 baseline assessment during deferred period.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    Deferred Group (Control Group)
    Reporting group description
    		 Adults (cohort 1 and 2) and pediatric subjects with X-linked retinitis pigmentosa (XLRP) caused by mutations in the human retinitis pigmentosa guanosine triphosphatase regulator (RPGR) gene (RPGR-XLRP) were randomised in the Deferred Group and did not receive any treatment in the study. Subjects were followed up for safety from Day 1 up to Week 52.

    Reporting group title
    Immediate Treatment Group: RPGR2e11
    Reporting group description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive adeno-associated virus vector with a serotype 5 capsid-human rhodopsin kinase promoter retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR) gene therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 2.0*10^11 viral genome per millilitre (vg/mL) (RPGR2e11) dose (300 microlitres [mcL] to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug (Day 1).

    Reporting group title
    Immediate Treatment Group: RPGR4e11
    Reporting group description
    		 Adults (cohort 1 and 2) and pediatric subjects with RPGR-XLRP were randomised to receive AAV5-hRKp.RPGR gene therapy as a subretinal injection on the day of first eye surgery (Day 1) and second eye surgery (any time between Day 8 to Day 22) with 4.0*10^11 vg/mL (RPGR4e11) dose (300 mcL to 800 mcL in each eye). Subjects were followed up for safety up to Week 52 after first dose of study drug (Day 1).

    Serious adverse events
    		 Deferred Group (Control Group) Immediate Treatment Group: RPGR2e11 Immediate Treatment Group: RPGR4e11
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 33 (0.00%)
    5 / 32 (15.63%)
    5 / 30 (16.67%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Intraocular Pressure Increased
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    3 / 30 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Urinary Retention Postoperative
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Choroidal Haemorrhage
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    0 / 30 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hypotony of Eye
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Rhegmatogenous Retinal Detachment
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    0 / 30 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Anal Abscess
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Deferred Group (Control Group) Immediate Treatment Group: RPGR2e11 Immediate Treatment Group: RPGR4e11
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    13 / 33 (39.39%)
    32 / 32 (100.00%)
    30 / 30 (100.00%)
    Investigations
    Blood Bicarbonate Decreased
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    2 / 30 (6.67%)
         occurrences all number
    0
    1
    2
    Intraocular Pressure Decreased
         subjects affected / exposed
    1 / 33 (3.03%)
    6 / 32 (18.75%)
    8 / 30 (26.67%)
         occurrences all number
    1
    7
    11
    Intraocular Pressure Increased
         subjects affected / exposed
    0 / 33 (0.00%)
    11 / 32 (34.38%)
    14 / 30 (46.67%)
         occurrences all number
    0
    30
    35
    Injury, poisoning and procedural complications
    Hyphaema
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    3 / 30 (10.00%)
         occurrences all number
    0
    0
    4
    Lens Feathering
         subjects affected / exposed
    0 / 33 (0.00%)
    6 / 32 (18.75%)
    1 / 30 (3.33%)
         occurrences all number
    0
    10
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    2 / 30 (6.67%)
         occurrences all number
    0
    2
    9
    Eye disorders
    Anterior Chamber Flare
         subjects affected / exposed
    0 / 33 (0.00%)
    4 / 32 (12.50%)
    7 / 30 (23.33%)
         occurrences all number
    0
    5
    9
    Anterior Chamber Cell
         subjects affected / exposed
    0 / 33 (0.00%)
    14 / 32 (43.75%)
    15 / 30 (50.00%)
         occurrences all number
    0
    28
    31
    Conjunctival Haemorrhage
         subjects affected / exposed
    0 / 33 (0.00%)
    20 / 32 (62.50%)
    21 / 30 (70.00%)
         occurrences all number
    0
    32
    40
    Cataract Subcapsular
         subjects affected / exposed
    4 / 33 (12.12%)
    11 / 32 (34.38%)
    8 / 30 (26.67%)
         occurrences all number
    4
    19
    17
    Cataract
         subjects affected / exposed
    1 / 33 (3.03%)
    3 / 32 (9.38%)
    5 / 30 (16.67%)
         occurrences all number
    2
    8
    11
    Anterior Chamber Inflammation
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    2 / 30 (6.67%)
         occurrences all number
    0
    3
    2
    Conjunctival Oedema
         subjects affected / exposed
    0 / 33 (0.00%)
    8 / 32 (25.00%)
    6 / 30 (20.00%)
         occurrences all number
    0
    10
    10
    Conjunctival Hyperaemia
         subjects affected / exposed
    0 / 33 (0.00%)
    16 / 32 (50.00%)
    15 / 30 (50.00%)
         occurrences all number
    0
    28
    25
    Epiretinal Membrane
         subjects affected / exposed
    2 / 33 (6.06%)
    3 / 32 (9.38%)
    4 / 30 (13.33%)
         occurrences all number
    3
    5
    5
    Diplopia
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    4
    Cystoid Macular Oedema
         subjects affected / exposed
    0 / 33 (0.00%)
    3 / 32 (9.38%)
    3 / 30 (10.00%)
         occurrences all number
    0
    4
    4
    Corneal Infiltrates
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    0 / 30 (0.00%)
         occurrences all number
    0
    3
    0
    Eye Inflammation
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    1 / 30 (3.33%)
         occurrences all number
    0
    3
    2
    Eye Irritation
         subjects affected / exposed
    0 / 33 (0.00%)
    3 / 32 (9.38%)
    1 / 30 (3.33%)
         occurrences all number
    0
    3
    2
    Eye Movement Disorder
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    1 / 30 (3.33%)
         occurrences all number
    0
    2
    1
    Iridocyclitis
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    0 / 30 (0.00%)
         occurrences all number
    0
    2
    0
    Hypotony of Eye
         subjects affected / exposed
    0 / 33 (0.00%)
    3 / 32 (9.38%)
    1 / 30 (3.33%)
         occurrences all number
    0
    4
    1
    Flat Anterior Chamber of Eye
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    2 / 30 (6.67%)
         occurrences all number
    0
    1
    2
    Eye Pain
         subjects affected / exposed
    0 / 33 (0.00%)
    3 / 32 (9.38%)
    7 / 30 (23.33%)
         occurrences all number
    0
    3
    11
    Lenticular Opacities
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    2 / 30 (6.67%)
         occurrences all number
    0
    2
    3
    Posterior Capsule Opacification
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    4
    Photophobia
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    2 / 30 (6.67%)
         occurrences all number
    0
    4
    3
    Papilloedema
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    4
    Metamorphopsia
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    3 / 30 (10.00%)
         occurrences all number
    0
    2
    4
    Macular Oedema
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    2 / 30 (6.67%)
         occurrences all number
    0
    10
    3
    Macular Degeneration
         subjects affected / exposed
    0 / 33 (0.00%)
    3 / 32 (9.38%)
    0 / 30 (0.00%)
         occurrences all number
    0
    4
    0
    Low Luminance Best-Corrected Visual Acuity Decreased
         subjects affected / exposed
    4 / 33 (12.12%)
    2 / 32 (6.25%)
    1 / 30 (3.33%)
         occurrences all number
    5
    2
    1
    Loss of Visual Contrast Sensitivity
         subjects affected / exposed
    1 / 33 (3.03%)
    3 / 32 (9.38%)
    5 / 30 (16.67%)
         occurrences all number
    1
    7
    9
    Punctate Keratitis
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    3 / 30 (10.00%)
         occurrences all number
    0
    2
    5
    Retinal Degeneration
         subjects affected / exposed
    0 / 33 (0.00%)
    4 / 32 (12.50%)
    0 / 30 (0.00%)
         occurrences all number
    0
    8
    0
    Visual Acuity Reduced
         subjects affected / exposed
    1 / 33 (3.03%)
    4 / 32 (12.50%)
    5 / 30 (16.67%)
         occurrences all number
    1
    4
    6
    Vision Blurred
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    0 / 30 (0.00%)
         occurrences all number
    0
    4
    0
    Subretinal Fluid
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    2 / 30 (6.67%)
         occurrences all number
    0
    1
    4
    Retinal Oedema
         subjects affected / exposed
    1 / 33 (3.03%)
    0 / 32 (0.00%)
    6 / 30 (20.00%)
         occurrences all number
    1
    0
    12
    Retinal Haemorrhage
         subjects affected / exposed
    0 / 33 (0.00%)
    3 / 32 (9.38%)
    4 / 30 (13.33%)
         occurrences all number
    0
    4
    4
    Visual Impairment
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    2
    Vitreal Cells
         subjects affected / exposed
    1 / 33 (3.03%)
    8 / 32 (25.00%)
    10 / 30 (33.33%)
         occurrences all number
    2
    18
    23
    Vitreous Disorder
         subjects affected / exposed
    0 / 33 (0.00%)
    3 / 32 (9.38%)
    1 / 30 (3.33%)
         occurrences all number
    0
    3
    1
    Vitritis
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    3
    Vitreous Opacities
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    3 / 30 (10.00%)
         occurrences all number
    0
    3
    5
    Vitreous Haemorrhage
         subjects affected / exposed
    0 / 33 (0.00%)
    5 / 32 (15.63%)
    2 / 30 (6.67%)
         occurrences all number
    0
    5
    2
    Vitreous Floaters
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    3
    Gastrointestinal disorders
    Abdominal Pain Upper
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    2 / 30 (6.67%)
         occurrences all number
    0
    1
    2
    Vomiting
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    2
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    2
    Dermatitis
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    0 / 30 (0.00%)
         occurrences all number
    0
    2
    0
    Acne
         subjects affected / exposed
    0 / 33 (0.00%)
    1 / 32 (3.13%)
    3 / 30 (10.00%)
         occurrences all number
    0
    1
    3
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 33 (0.00%)
    2 / 32 (6.25%)
    1 / 30 (3.33%)
         occurrences all number
    0
    2
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 33 (3.03%)
    5 / 32 (15.63%)
    5 / 30 (16.67%)
         occurrences all number
    1
    6
    8
    Localised Infection
         subjects affected / exposed
    0 / 33 (0.00%)
    0 / 32 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    0
    2
    Covid-19
         subjects affected / exposed
    1 / 33 (3.03%)
    2 / 32 (6.25%)
    4 / 30 (13.33%)
         occurrences all number
    1
    2
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Jul 2020
    The overall reason for this amendment was to add research sample in Schedule of Activities for Deferred Treatment Group, correct the inclusion and exclusion criteria, and correct route of drug administration in Appendix 6.
    26 Aug 2020
    The overall reason for this amendment was to update the inclusion criteria to a retinal function assessment instead of a functional vision assessment, address health authority feedback, and to make administrative and operational changes.
    04 Feb 2021
    The overall reason for this amendment was to update the screening timeline, respond to regulatory input requiring subjects to consent to the long-term follow up study at the time of consent to MGT-RPGR-021, address safety in the second eye by gating the first 3 treated patients, and make administrative and operational changes.
    31 Mar 2021
    The overall reason for this amendment was to update numerous sections of the protocol with new or revised details, to note changes in procedures/methods, and to clarify content.
    15 Jul 2021
    The overall reason for this amendment was to update numerous sections of the protocol with new or revised details, to note changes in procedures/methods, and to make and/or clarify administrative and operational changes.
    10 Nov 2021
    The overall reason for this amendment was to revise time frames in relation to randomization and Non-Ocular Exclusion Criteria, to update sections of the protocol with new or revised details, and to make and/or clarify administrative and operational changes.
    24 Dec 2021
    The overall reason for this amendment was to update sections of the protocol with additional or revised specifics to enable clarity and implement administrative and operational changes. The update also contains changes to the population for efficacy analysis to allow for maze refinement prior to starting of Cohort 2.
    06 Apr 2022
    The overall reason for this amendment was to revise the definition of Cohort 1 and add that subjects who discontinue from Cohort 1 prior to Week 10 may be replaced. Additional changes have been made to align the protocol with requests made by Health Authorities following their review and also to provide further clarification to investigators on some aspects of the study.
    14 Jul 2022
    The overall reason for this amendment was to revise the objectives and endpoints of the study to align with data from other studies and utilize a more sensitive and appropriate primary endpoint of functional vision. The section on statistical power has been updated based on the change in primary endpoint. Additional time points for assessing viral shedding have been added and the total blood volume to be collected has been updated.
    16 Oct 2023
    The overall reason for this amendment was to update sections of the protocol with additional or revised specifics to enable clarity and implement administrative and operational changes including sponsorship transfer from MeiraGTx UK II Ltd to Janssen Research & Development.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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