Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44393   clinical trials with a EudraCT protocol, of which   7405   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    Long-term Follow-up Study of Participants Following an Open-label, Multi-center, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children with X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

    Summary
    EudraCT number
    		 2018-000425-31
    Trial protocol
    		 GB   Outside EU/EEA  
    Global end of trial date
    17 Sep 2025

    Results information
    Results version number
    		 v1(current)
    This version publication date
    29 Mar 2026
    First version publication date
    29 Mar 2026
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    MGT010
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04312672
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Janssen Research & Development
    Sponsor organisation address
    Turnhoutseweg 30, Beerse, Belgium, B-2340
    Public contact
    Clinical Registry Group, Janssen Research & Development, ClinicalTrialsEU@its.jnj.com
    Scientific contact
    Clinical Registry Group, Janssen Research & Development, 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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    23 Nov 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Sep 2025
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of the trial was to assess the longer term safety of AAV5-hRKp.RPGR administered to participants in the MGT009 trial, measured by the presence or absence of adverse events (AEs), the assessment of visual acuity, and loss of light perception.
    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 Practice and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    19 Feb 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 29
    Country: Number of subjects enrolled
    United States: 13
    Worldwide total number of subjects
    42
    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)
    1
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    39
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 42 participants (including 3 children) were enrolled.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 Low Dose AAV5-hRKp.RPGR
    Arm description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1 milliliter (mL) dose concentration 1.0*10^11 viral genomes per milliliter (vg/mL) of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the Retinitis Pigmentosa GTPase Regulator (RPGR) gene received during study MGT009.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AAV5-hRKp.RPGR
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Ocular use
    Dosage and administration details
    No intervention was administered in the current study. Participants who received AAV5-hRKp.RPGR in MGT009 study were followed up in the current study.

    Arm title
    		 Intermediate Dose AAV5-hRKp.RPGR
    Arm description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL intermediate dose concentration 2.0*10^11 vg/mL of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AAV5-hRKp.RPGR
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Ocular use
    Dosage and administration details
    No intervention was administered in the current study. Participants who received AAV5-hRKp.RPGR in MGT009 study were followed up in the current study.

    Arm title
    		 High Dose AAV5-hRKp.RPGR
    Arm description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL high dose concentration 4.0*10^11 vg/mL of RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AAV5-hRKp.RPGR
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Ocular use
    Dosage and administration details
    No intervention was administered in the current study. Participants who received AAV5-hRKp.RPGR in MGT009 study were followed up in the current study.

    Number of subjects in period 1
    		Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Started
    17
    23
    2
    Completed
    9
    14
    1
    Not completed
    8
    9
    1
         Adverse event, serious fatal
    -
    1
    -
         Consent withdrawn by subject
    1
    -
    1
         Unspecified
    7
    8
    -

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Low Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1 milliliter (mL) dose concentration 1.0*10^11 viral genomes per milliliter (vg/mL) of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the Retinitis Pigmentosa GTPase Regulator (RPGR) gene received during study MGT009.

    Reporting group title
    Intermediate Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL intermediate dose concentration 2.0*10^11 vg/mL of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Reporting group title
    High Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL high dose concentration 4.0*10^11 vg/mL of RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Reporting group values
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR Total
    Number of subjects
    		 17 23 2 42
    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)
    		 0 1 0 1
        12 - 17 years
    		 0 2 0 2
        Adults (18 - 64 years)
    		 17 20 2 39
        From 65 - 84 years
    		 0 0 0 0
        85 years and over
    		 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 30.1 ( 12.6 ) 26.8 ( 9.48 ) 21.0 ( 4.24 ) -
    Gender categorical
    Units: Subjects
        Male
    		 17 23 2 42
        Female
    		 0 0 0 0

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Low Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1 milliliter (mL) dose concentration 1.0*10^11 viral genomes per milliliter (vg/mL) of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the Retinitis Pigmentosa GTPase Regulator (RPGR) gene received during study MGT009.

    Reporting group title
    Intermediate Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL intermediate dose concentration 2.0*10^11 vg/mL of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Reporting group title
    High Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL high dose concentration 4.0*10^11 vg/mL of RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Subject analysis set title
    Low Dose AAV5-hRKp.RPGR
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1 milliliter (mL) dose concentration 1.0*10^11 viral genomes per milliliter (vg/mL) of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the Retinitis Pigmentosa GTPase Regulator (RPGR) gene received during study MGT009.

    Subject analysis set title
    Intermediate Dose AAV5-hRKp.RPGR
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL intermediate dose concentration 2.0*10^11 vg/mL of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Subject analysis set title
    High Dose AAV5-hRKp.RPGR
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL high dose concentration 4.0*10^11 vg/mL of RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    		 Close Top of page
    End point title
    		 Number of Participants With Treatment-emergent Adverse Events (TEAEs) [1]
    End point description
    An adverse event (AE) was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. AE does not necessarily have a causal relationship with intervention. Any AE occurring at or after the initial administration of AAV5-hRKp.RPGR was considered to be treatment-emergent. TEAEs included both serious and non-serious adverse events. Safety analysis set (SAS) included all enrolled participants who were administered bota-vec in Study MGT009 and consented to Study MGT010, and performed separately for each dose level and overall, unless otherwise specified.
    End point type
    Primary
    End point timeframe
    For Deferred treatment arm: From Month 6 post treatment in study MGT009 up to 60 months; For Immediate treatment arm: From Month 12 post treatment in study MGT009 up to 60 months
    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 data was planned to be reported for this endpoint.
    End point values
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Number of subjects analysed
    17
    22
    3
    Units: Subjects
    17
    20
    3
    No statistical analyses for this end point

    Secondary: Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score

    		 Close Top of page
    End point title
    		 Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score
    End point description
    Change from baseline in BCVA by ETDRS chart letter score in monocular assessment was reported. BCVA was determined using ETDRS chart by counting 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 logarithm of the minimum angle of resolution (logMAR). Higher ETDRS score indicated better vision. Full analysis set (FAS) included all enrolled adults and pediatric participants treated with AAV5-hRKp. RPGR and completed both at least one baseline visit prior to treatment in study MGT009 and at least one visit in study MGT010. 'n' (number analyzed) signifies number of participants analyzed at specified timepoints. 99999 signifies that standard deviation was not estimable for single participant.
    End point type
    Secondary
    End point timeframe
    Baseline, Month 12, Month 18, Month 24, Month 36, Month 48, and Month 60
    End point values
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Number of subjects analysed
    17
    23
    2
    Units: Number of ETDRS letters
    arithmetic mean (standard deviation)
        At Month 12 (n=15,22,2)
    1.4 ( 4.02 )
    0.2 ( 5.68 )
    -5.0 ( 0.47 )
        At Month 18 (n=15,23,2)
    -1.8 ( 7.51 )
    0.0 ( 5.09 )
    -6.5 ( 1.65 )
        At Month 24 (n=16,21,2)
    -2.6 ( 5.51 )
    -1.7 ( 7.97 )
    -7.5 ( 0.24 )
        At Month 36 (n=16,23,2)
    -4.8 ( 9.40 )
    -5.6 ( 10.10 )
    -8.0 ( 0.47 )
        At Month 48 (n=15,22,2)
    -4.3 ( 8.41 )
    -6.4 ( 13.12 )
    -16.5 ( 5.42 )
        At Month 60 (n=9,13,1)
    -8.6 ( 13.58 )
    -13.0 ( 17.74 )
    -14.7 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Letter Score

    		 Close Top of page
    End point title
    		 Change From Baseline in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Letter Score
    End point description
    Change from baseline in LLVA by ETDRS chart letter score in monocular assessment were reported. LLVA was determined using 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 ETDRS chart or 0 logMAR. Higher ETDRS score indicated better vision. FAS included all enrolled adults and pediatric participants treated with AAV5-hRKp.RPGR and completed both at least one baseline visit prior to treatment in study MGT009 and at least one visit in study MGT010. 'N' (Overall number of participants analyzed) signifies number of participants evaluable for this endpoint. 'n' (number analyzed) signifies number of participants analyzed at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Month 12, Month 18, Month 24, Month 36, Month 48, and Month 60
    End point values
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Number of subjects analysed
    9
    9
    0 [2]
    Units: Number of ETDRS letters
    arithmetic mean (standard deviation)
        At Month 12 (n=8,8,0)
    2.9 ( 4.39 )
    -1.0 ( 15.57 )
    ( )
        At Month 18 (n=8,6,0)
    -2.4 ( 8.12 )
    1.8 ( 8.90 )
    ( )
        At Month 24 (n=9,8,0)
    -3.2 ( 8.91 )
    -4.3 ( 13.74 )
    ( )
        At Month 36 (n=9,9,0)
    -4.7 ( 14.70 )
    -12.7 ( 16.66 )
    ( )
        At Month 48 (n=7,8,0)
    -5.5 ( 16.04 )
    -8.5 ( 16.65 )
    ( )
        At Month 60 (n=3,3,0)
    -17.6 ( 30.86 )
    -31.1 ( 19.17 )
    ( )
    Notes
    [2] - LLVA data not analyzable for any subject in the high‑dose group; thus, no subjects were analyzed.
    No statistical analyses for this end point

    Secondary: Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry

    		 Close Top of page
    End point title
    		 Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry
    End point description
    Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry was reported. FAS included all enrolled adults and pediatric participants treated with AAV5-hRKp.RPGR and completed both at least one baseline visit prior to treatment in study MGT009 and at least one visit in study MGT010. Here 'n' (number analyzed) signifies number of participants analyzed at specified timepoints. In 'High Dose AAV5-hRKp.RPGR' arm, at month 60, 99999 signifies that standard deviation was not estimable for single participant.
    End point type
    Secondary
    End point timeframe
    Baseline, Month 12, Month 18, Month 24, Month 36, Month 48, and Month 60
    End point values
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Number of subjects analysed
    17
    23
    2
    Units: Decibels (dB)
    arithmetic mean (standard deviation)
        At Month 12 (n=15,20,2)
    1.0 ( 2.09 )
    1.5 ( 2.62 )
    -0.9 ( 3.15 )
        At Month 18 (n=15,20,2)
    0.1 ( 2.43 )
    0.4 ( 3.07 )
    -3.8 ( 4.41 )
        At Month 24 (n=16,20,2)
    -1.0 ( 3.12 )
    0.4 ( 2.84 )
    -2.6 ( 5.41 )
        At Month 36 (n=17,23,2)
    -1.7 ( 3.97 )
    -0.8 ( 3.54 )
    -4.2 ( 4.23 )
        At Month 48 (n=15,20,2)
    -2.7 ( 4.60 )
    -1.8 ( 3.03 )
    -6.6 ( 4.68 )
        At Month 60 (n=9,13,1)
    -3.0 ( 4.24 )
    -3.0 ( 3.03 )
    -11.9 ( 99999 )
    No statistical analyses for this end point

    Secondary: Percentage of Responders in Pointwise Data in Static Perimetry Within the Full Visual Field

    		 Close Top of page
    End point title
    		 Percentage of Responders in Pointwise Data in Static Perimetry Within the Full Visual Field
    End point description
    Percentage of responders in pointwise data in static perimetry within the full visual field was reported. A responder was defined as a participant with greater than equal to (>=) 7dB improvement from baseline in at least 5 loci at the current visit and another visit prior. FAS included all enrolled adults and pediatric participants treated with AAV5-hRKp.RPGR and completed both at least one baseline visit prior to treatment in study MGT009 and at least one visit in study MGT010. Here 'n' (number analyzed) signifies number of participants analyzed at specified timepoints.
    End point type
    Secondary
    End point timeframe
    At Month 12, Month 18, Month 24, Month 36, Month 48, and Month 60
    End point values
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Number of subjects analysed
    17
    23
    2
    Units: Percentage of responders
    number (not applicable)
        At Month 12 (n=15,20,2)
    60.0
    65.0
    50.0
        At Month 18 (n=15,20,2)
    46.7
    75.0
    0
        At Month 24 (n=16,20,2)
    50.0
    70.0
    50.0
        At Month 36 (n=17,23,2)
    47.1
    52.2
    50.0
        At Month 48 (n=15,20,2)
    40.0
    35.0
    0
        At Month 60 (n=9,13,1)
    22.2
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage of Responder in Pointwise Data in Static Perimetry Within the Central 30-Degree Visual Field

    		 Close Top of page
    End point title
    		 Percentage of Responder in Pointwise Data in Static Perimetry Within the Central 30-Degree Visual Field
    End point description
    Percentage of responder in pointwise data in static perimetry within the central 30-degree visual field was reported. FAS included all enrolled adults and pediatric participants treated with AAV5-hRKp.RPGR and completed both at least one baseline visit prior to treatment in study MGT009 and at least one visit in study MGT010. Here 'n' (number analyzed) signifies number of participants analyzed at specified timepoints.
    End point type
    Secondary
    End point timeframe
    At Month 12, Month 18, Month 24, Month 36, Month 48, and Month 60
    End point values
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Number of subjects analysed
    17
    23
    2
    Units: Percentage of responders
    number (not applicable)
        At Month 12 (n=15,20,2)
    46.7
    45.0
    0
        At Month 18 (n=15,20,2)
    26.7
    45.0
    0
        At Month 24 (n=16,20,2)
    37.5
    50.0
    50.0
        At Month 36 (n=17,23,2)
    29.4
    30.4
    50.0
        At Month 48 (n=15,20,2)
    26.7
    25.0
    0
        At Month 60 (n=9, 13, 1)
    11.1
    0
    0
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    For Deferred treatment arm: From Month 6 post treatment in study MGT009 up to 60 months; For Immediate treatment arm: From Month 12 post treatment in study MGT009 up to 60 months.
    Adverse event reporting additional description
    Safety analysis set (SAS) included all enrolled participants who administered bota-vec in Study MGT009 and consented to Study MGT010, and performed separately for each dose level and overall, unless otherwise specified.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    28.1
    Reporting groups
    Reporting group title
    Low Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1 milliliter (mL) dose concentration 1.0*10^11 viral genomes per milliliter (vg/mL) of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the Retinitis Pigmentosa GTPase Regulator (RPGR) gene received during study MGT009.

    Reporting group title
    Intermediate Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL intermediate dose concentration 2.0*10^11 vg/mL of AAV5-hRKp.RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Reporting group title
    High Dose AAV5-hRKp.RPGR
    Reporting group description
    		 Participants from MGT009 study (from deferred and immediate treatment arm) were followed up for subretinal administration of a single monocular injection of up to 1mL high dose concentration 4.0*10^11 vg/mL of RPGR AAV5-hRKp.RPGR: AAV gene therapy for defects in the RPGR gene received during study MGT009.

    Serious adverse events
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         number of deaths (all causes)
    0
    1
    0
         number of deaths resulting from adverse events
    0
    1
    0
    Eye disorders
    Noninfective Chorioretinitis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 22 (4.55%)
    0 / 3 (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
    Uveitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Completed Suicide
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Low Dose AAV5-hRKp.RPGR Intermediate Dose AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    17 / 17 (100.00%)
    20 / 22 (90.91%)
    3 / 3 (100.00%)
    Surgical and medical procedures
    Intraocular Lens Implant
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    2
    2
    0
    Cataract Operation
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    2
    2
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Peripheral Swelling
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Immune system disorders
    Seasonal Allergy
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Psychiatric disorders
    Attention Deficit Hyperactivity Disorder
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    1
    2
    0
    Depression
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Mania
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Mood Altered
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Investigations
    Alanine Aminotransferase Increased
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    1
    1
    0
    Low Density Lipoprotein Increased
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Intraocular Pressure Increased
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    3
    0
    0
    Fundus Autofluorescence
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 22 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Aspartate Aminotransferase Increased
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    Skin Laceration
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Joint Dislocation
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Cardiac disorders
    Atrial Fibrillation
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Eye disorders
    Cataract Cortical
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    1
    2
    0
    Cataract Subcapsular
         subjects affected / exposed
    3 / 17 (17.65%)
    5 / 22 (22.73%)
    0 / 3 (0.00%)
         occurrences all number
    6
    11
    0
    Eye Pain
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    2
    1
    0
    Epiretinal Membrane
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    1
    2
    0
    Cataract
         subjects affected / exposed
    3 / 17 (17.65%)
    3 / 22 (13.64%)
    1 / 3 (33.33%)
         occurrences all number
    3
    3
    1
    Aniseikonia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Anterior Chamber Cell
         subjects affected / exposed
    2 / 17 (11.76%)
    4 / 22 (18.18%)
    0 / 3 (0.00%)
         occurrences all number
    3
    10
    0
    Cystoid Macular Oedema
         subjects affected / exposed
    2 / 17 (11.76%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    2
    2
    0
    Retinal Deposits
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Retinal Haemorrhage
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Retinal Degeneration
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    0
    3
    0
    Posterior Capsule Opacification
         subjects affected / exposed
    1 / 17 (5.88%)
    3 / 22 (13.64%)
    1 / 3 (33.33%)
         occurrences all number
    2
    3
    1
    Photophobia
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    2
    1
    0
    Optic Atrophy
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Metamorphopsia
         subjects affected / exposed
    2 / 17 (11.76%)
    3 / 22 (13.64%)
    0 / 3 (0.00%)
         occurrences all number
    2
    4
    0
    Macular Pigmentary Changes
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Macular Cyst
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Uveitis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 22 (4.55%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    Iridocyclitis
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Low Luminance Best-Corrected Visual Acuity Decreased
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 22 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Vitreous Haemorrhage
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Vitreous Floaters
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Vitreal Cells
         subjects affected / exposed
    0 / 17 (0.00%)
    3 / 22 (13.64%)
    1 / 3 (33.33%)
         occurrences all number
    0
    5
    1
    Visual Impairment
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    1
    3
    0
    Visual Field Defect
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 22 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Visual Acuity Reduced
         subjects affected / exposed
    4 / 17 (23.53%)
    9 / 22 (40.91%)
    1 / 3 (33.33%)
         occurrences all number
    6
    18
    1
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Vomiting
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Abdominal Pain Upper
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    1
    1
    0
    Skin and subcutaneous tissue disorders
    Dermatitis Acneiform
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 22 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Dry Skin
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 22 (4.55%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    Pain of Skin
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Rash
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Bone Loss
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 22 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Musculoskeletal Stiffness
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 22 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Osteopenia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Pain in Extremity
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Ankylosing Spondylitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    Chorioretinitis
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Upper Respiratory Tract Infection
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    2
    1
    0
    Sinusitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Rhinitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Lower Respiratory Tract Infection
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 22 (4.55%)
    0 / 3 (0.00%)
         occurrences all number
    1
    3
    0
    Influenza
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Ear Infection
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 22 (9.09%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Covid-19
         subjects affected / exposed
    3 / 17 (17.65%)
    5 / 22 (22.73%)
    0 / 3 (0.00%)
         occurrences all number
    3
    5
    0
    Conjunctivitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Gout
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 22 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Jun 2019
    The overall reason for this amendment was to update participant numbers and include a 12 visit to align with the updated visit schedule on the MGT009 study. Full field stimulus testing assessment was removed.
    13 Aug 2020
    The overall reason for this amendment was to include assessments that have been recently included in the RPGR gene therapy trial. Provide clarification around optional assessments.
    15 Sep 2020
    The overall reason for this amendment was correction of page numbering.
    16 Oct 2020
    The overall reason for this amendment was changing of vector name. Removal of information relating to caregiver patient reported outcomes (PROs) as not applicable to study. More detail on PRO information to align with treatment study protocol (MGT009).
    09 Jun 2021
    The overall reason for this amendment was to update the number of research sites. XLRP RPGR MGT009 trial design was updated to include randomisation phase. Remove Professor Bainbridge from the declaration of Interests section as he no longer holds share in MeiraGTx or receives payment for consultancy work.
    07 Apr 2022
    The overall reason for this amendment was to include language to allow alternative reduced follow-up options. Updated the number of participants expected to be included in this study (based on MGT009 data). Removed collection of medical history from the schedule of assessments. Indicated that all medical events that occurred between the last study visit in Study MGT009 and prior to signing informed consent for Study MGT010 are to be reported as AEs in Study MGT010. Replaced “EMAS” by “Sponsor or delegate”
    03 Apr 2023
    The overall reason for this amendment was to clarify that electroretinography testing will be discontinued after M18 if responses remain undetectable or if confirmed by the electroretinography reading centre as unnecessary. Added that an interim analysis of the data is planned and others may be undertaken. Clarified that the IDMC also considers data of other studies in the RPGR program. Added that unscheduled visits may be conducted to follow-up adverse events. Reworded text on optional assessments. Updated information related to the data management vendor. Clarified that the Sponsor will disclose the results of the clinical study as required by applicable regulations.
    18 Dec 2023
    The overall rationale for the amendment was to update sections of the protocol with additional or revised specifics such as changes to the secondary endpoints, addition of exploratory endpoints and to enable clarity and implement operational changes including sponsorship transfer from MeiraGTx UK II Ltd to Janssen Research & Development (JRD) and administrative changes (For example, updates to Janssen Research & Development protocol template format).

    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.
    Study was not powered to evaluate efficacy, as all participants received bota-vec in previous study and there was no concurrent control arm in this study. Due to revision in scope, VMA, LLQ, and MRS90 endpoints were not analyzed and reported.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sun Mar 29 08:19:36 CEST 2026 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA