X-Linked Retinitis PigmentosaPhase 3NCT04671433
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
A Phase 3 study testing Genetic: AAV5-hRKp.RPGR in people with X-Linked Retinitis Pigmentosa, run by MeiraGTx Ocular UK Ltd at 28 sites in 11 countries.
Trial summary
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP) Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- X-Linked Retinitis Pigmentosa
- What is being tested?
- Genetic: AAV5-hRKp.RPGR
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 105 participants
- Where
- 28 sitesin 11 countries
- Study dates
- 4 Dec 2020to 30 Sep 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female
- 3 years of age or older
- Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory
Who cannot join
- Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
- Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
- Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
Trial locations and contacts
28 sites in 11 countries.
28 sites in 11 countries
United States10 sites
Univ of Michigan Medical Center
Ann Arbor, Michigan, 48105
Emory University
Atlanta, Georgia, 30322
Retina Consultants of Houston
Bellaire, Texas, 77401
Massachusetts General Hospital - Center for Celiac Research and Treatment
Boston, Massachusetts, 02114
Duke Eye Center
Durham, North Carolina, 27705
VitreoRetinal Associates, PA
Gainesville, Florida, 32607
Shiley Eye Institute Jacobs Retina Center
La Jolla, California, 92093 0946
Childrens Hospital
Los Angeles, California, 90027
Stanford Health Care
Palo Alto, California, 94303
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213
Italy4 sites
Azienda Ospedaliero Universitaria Careggi
Florence, 50134
Ospedale San Paolo
Milan, 20142
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
Naples, 80131
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
Roma, 00198
United Kingdom4 sites
NHS Lothian
Edinburgh, EH3 9HA
Gartnavel General Hospital
Glasgow, G12 0YN
St James University Hospital
Leeds, LS9 7TF
Moorfields Eye Hospital
London, EC1V 2PD
Netherlands2 sites
VUMC Amsterdam
Amsterdam, 1105AZ
Radboudumc
Nijmegen, 6525EX
Switzerland2 sites
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
Basel, 4031
Universite de Lausanne, Hopital ophtalmique Jules-Gonin
Lausanne, 1004
France1 site
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Paris, 75012
About this study
Official title: Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Conditions
- X-Linked Retinitis Pigmentosa
- Being tested
- Genetic: AAV5-hRKp.RPGR
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genetic: AAV5-hRKp.RPGRBiological
Also called botaretigene sparoparvovec
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Genetic: AAV5-hRKp.RPGRBiological
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)
| Study group | Role | What they receive |
|---|---|---|
| Experimental - Immediate Treatment | Experimental | Intermediate dose. Receives: Biological: Genetic: AAV5-hRKp.RPGR |
| Deferred Treatment | Other | Deferred Treatment Receives: Biological: Genetic: AAV5-hRKp.RPGR |
| Experimental Immediate Treatment | Experimental | Low dose. Receives: Biological: Genetic: AAV5-hRKp.RPGR |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.
Measured: From Baseline to 52 Weeks
Secondary outcomes (14)
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52
Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed.
Measured: From Baseline to Week 52
Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed.
Measured: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
Pointwise response in full visual field at Week 52 will be assessed.
Measured: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52
Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed.
Measured: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
Pointwise response in the central 30 degrees visual field at Week 52 will be assessed.
Measured: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed.
Measured: From Baseline to Week 52
Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52
Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed.
Measured: From Baseline to Week 52
Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52
Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52.
Measured: From Baseline to Week 52
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52.
Measured: From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52
Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52.
Measured: From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52
Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52.
Measured: From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52
Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52.
Measured: From Baseline to Week 52
Number of Participants with Ocular and Non-ocular Adverse Events
Number of participants with ocular and non-ocular adverse events will be assessed.
Measured: Day 1 - Week 52
Number of Participants With Abnormalities in Laboratory Assessments
Number of participants with abnormalities in laboratory assessments will be assessed.
Measured: Day 1 - 52 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2020
- Study start4 Dec 2020
- Primary completion30 Sep 2024
- Study completion30 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 105 participants (actual)
Sponsor and investigators
- Lead sponsor
- MeiraGTx Ocular UK Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, MeiraGTx Ocular UK Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04671433
- Sponsor's protocol ID
- CR109258
- Other IDs
- MGT-RPGR-021, 2020-002873-88
- Status last verified
- Sep 2026
- First submitted
- 5 Nov 2020
- Last updated
- 24 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
- ClinicalTrials.gov ID
- NCT04671433
- Acronym / sponsor ID
- CR109258
- Conditions
- X-Linked Retinitis Pigmentosa
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 105 participants (actual)
- Lead sponsor
- MeiraGTx Ocular UK LtdIndustry (pharma, biotech, devices)
- Study start
- 4 Dec 2020 (actual)
- Primary completion
- 30 Sep 2024 (actual)
- Study completion
- 30 Sep 2024 (actual)
- First posted
- 17 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
- Model description
- Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
| Arm | Type | Receives |
|---|---|---|
| Experimental - Immediate Treatment | Experimental | Biological: Genetic: AAV5-hRKp.RPGRIntermediate dose. |
| Deferred Treatment | Other | Biological: Genetic: AAV5-hRKp.RPGRDeferred Treatment |
| Experimental Immediate Treatment | Experimental | Biological: Genetic: AAV5-hRKp.RPGRLow dose. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Male or female
- 3 years of age or older
- Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory
Key exclusion criteria (3 listed)
- Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
- Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
- Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Genetic: AAV5-hRKp.RPGRAlso: botaretigene sparoparvovecBilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group | Biological | Experimental - Immediate Treatment, Experimental Immediate Treatment |
| Genetic: AAV5-hRKp.RPGRNo intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) | Biological | Deferred Treatment |
Primary outcome
Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
Time frame: From Baseline to 52 Weeks
Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.
14 secondary outcomes
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52
Time frame: From Baseline to Week 52
Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
Time frame: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
Time frame: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52
Time frame: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
Time frame: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
Time frame: From Baseline to Week 52
Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52
Time frame: From Baseline to Week 52
Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52
Time frame: From Baseline to Week 52
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
Time frame: From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52
Time frame: From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52
Time frame: From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52
Time frame: From Baseline to Week 52
Number of Participants with Ocular and Non-ocular Adverse Events
Time frame: Day 1 - Week 52
Number of Participants With Abnormalities in Laboratory Assessments
Time frame: Day 1 - 52 Weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
MeiraGTx Ocular UK Ltd
28 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Genetic: AAV5-hRKp.RPGR in people with X-Linked Retinitis Pigmentosa, run by MeiraGTx Ocular UK Ltd at 28 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Italy, United Kingdom, Netherlands, Switzerland and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze (time frame: From Baseline to 52 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2026.