X-Linked Retinitis PigmentosaPhase 3NCT04794101
Follow-up 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 Intermediate Dose and Genetic: AAV5-hRKp.RPGR Low Dose 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-hRKp.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 Intermediate Dose and Genetic: AAV5-hRKp.RPGR Low Dose
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 97 participants
- Where
- 28 sitesin 11 countries
- Study dates
- 4 Dec 2020to 7 Dec 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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
- None
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 Eye and Ear Infirmary
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
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
Rome, 00184
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 Follow-up 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-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Conditions
- X-Linked Retinitis Pigmentosa
- Being tested
- Genetic: AAV5-hRKp.RPGR Intermediate Dose, Genetic: AAV5-hRKp.RPGR Low Dose
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genetic: AAV5-hRKp.RPGR Intermediate DoseBiological
Also called botaretigene sparoparvovec
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
Genetic: AAV5-hRKp.RPGR Low DoseBiological
Also called botaretigene sparoparvovec
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
| Study group | Role | What they receive |
|---|---|---|
| Deferred Treatment From MGT-RPGR-021 of Intermediate Dose | Experimental | Deferred treatment Receives: Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose |
| Already Treated in MGT-RPGR-021 | Experimental | Already treated Receives: Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose; Biological: Genetic: AAV5-hRKp.RPGR Low Dose |
| Deferred Treatment From MGT-RPGR-021 Low Dose | Experimental | Deferred treatment Receives: Biological: Genetic: AAV5-hRKp.RPGR Low Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 - Month 60
Number of Participants With Abnormalities in Laboratory Assessments
Number of participants with abnormalities in laboratory assessments will be assessed.
Measured: Day 1 - Month 60
Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
Measured: From Baseline - Month 60
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment
Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.
Measured: From Baseline- Month 60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2021
- Study start4 Dec 2020
- Primary completion20 Sep 2029 (estimated)
- Study completion7 Dec 2029 (estimated)
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
- 97 participants randomized in MGT-RPGR-021. 64 participants randomized to Immediate treatment arm. 33 participants randomized to the deferred arm. These participants will be treated in MGT-RPGR-022. All participants to be followed for 60 months.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 97 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
- NCT04794101
- Sponsor's protocol ID
- MGT-RPGR-022
- Other IDs
- MGT-RPGR-022, 2020-002255-37, 2024-511411-25-00
- Status last verified
- Sep 2026
- First submitted
- 15 Dec 2020
- Last updated
- 25 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 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
- ClinicalTrials.gov ID
- NCT04794101
- Acronym / sponsor ID
- MGT-RPGR-022
- Conditions
- X-Linked Retinitis Pigmentosa
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 97 participants (actual)
- Lead sponsor
- MeiraGTx Ocular UK LtdIndustry (pharma, biotech, devices)
- Study start
- 4 Dec 2020 (actual)
- Primary completion
- 20 Sep 2029 (estimated)
- Study completion
- 7 Dec 2029 (estimated)
- First posted
- 11 Mar 2021 (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-hRKp.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
- 97 participants randomized in MGT-RPGR-021. 64 participants randomized to Immediate treatment arm. 33 participants randomized to the deferred arm. These participants will be treated in MGT-RPGR-022. All participants to be followed for 60 months.
| Arm | Type | Receives |
|---|---|---|
| Deferred Treatment From MGT-RPGR-021 of Intermediate Dose | Experimental | Biological: Genetic: AAV5-hRKp.RPGR Intermediate DoseDeferred treatment |
| Already Treated in MGT-RPGR-021 | Experimental | Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose, Biological: Genetic: AAV5-hRKp.RPGR Low DoseAlready treated |
| Deferred Treatment From MGT-RPGR-021 Low Dose | Experimental | Biological: Genetic: AAV5-hRKp.RPGR Low DoseDeferred treatment |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 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 (1 listed)
- None
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.RPGR Intermediate DoseAlso: botaretigene sparoparvovecBilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group | Biological | Already Treated in MGT-RPGR-021, Deferred Treatment From MGT-RPGR-021 of Intermediate Dose |
| Genetic: AAV5-hRKp.RPGR Low DoseAlso: botaretigene sparoparvovecBilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group | Biological | Already Treated in MGT-RPGR-021, Deferred Treatment From MGT-RPGR-021 Low Dose |
Primary outcome
Number of Participants with Ocular and Non-ocular Adverse Events
Time frame: Day 1 - Month 60
Number of participants with ocular and non-ocular adverse events will be assessed.
Number of Participants With Abnormalities in Laboratory Assessments
Time frame: Day 1 - Month 60
Number of participants with abnormalities in laboratory assessments will be assessed.
Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment
Time frame: From Baseline - Month 60
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment
Time frame: From Baseline- Month 60
Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 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 Intermediate Dose and Genetic: AAV5-hRKp.RPGR Low Dose 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 1 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 25 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?
This study is still under way. Results are not available in the source record yet.
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?
Number of Participants with Ocular and Non-ocular Adverse Events (time frame: Day 1 - Month 60). The study lists 4 primary outcomes in all. 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 25 Sep 2026.