X-Linked Retinitis PigmentosaPhase 2NCT06646289
A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
A Phase 2 study testing AAV5-hRKp.RPGR in people with X-Linked Retinitis Pigmentosa, run by MeiraGTx Ocular UK Ltd at 3 sites in 2 countries.
Trial summary
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa. Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- X-Linked Retinitis Pigmentosa
- What is being tested?
- AAV5-hRKp.RPGR
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 10 Oct 2024to 24 Oct 2030
- Phase
- Phase 2
- 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 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or is currently enrolled in Study MGT010
- Must sign an informed consent form indicating that they understand the purpose and procedures of the study and is willing to participate in the study
- Willing to adhere to the protocol and long-term follow-up
Who cannot join
- There are no specific exclusion criteria to enroll in this study
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114
About this study
Official title: An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With 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)
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
- Conditions
- X-Linked Retinitis Pigmentosa
- Being tested
- AAV5-hRKp.RPGR, No intervention (Follow-Up assessment)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV5-hRKp.RPGRBiological
Also called JNJ-74765340, botaretigene sparoparvovec
AAV5-hRKp.RPGR will be administered sub-retinally.
No intervention (Follow-Up assessment)Other
Participants will not receive any intervention and will undergo follow-up assessment.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Participants will receive a dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 depending on the dosage administered in study MGT009 (NCT03252847) in the past. After receiving the treatment, participants will be assessed for safety. Receives: Biological: AAV5-hRKp.RPGR |
| Cohort 2 | Experimental | Participants will receive the treatment dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 in the second eye once the safety will be determined in Cohort 1. Receives: Biological: AAV5-hRKp.RPGR |
| Cohort 3 | Other | The participants who are not willing to undergo surgery or are not eligible for surgery will be assessed in this cohort. They will be assessed yearly until 5 years after their initial eye surgery in previous study MGT009. Receives: Other: No intervention (Follow-Up assessment) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AE)
An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non investigational) product, whether or not related to that medicinal (investigational or non investigational) product.
Measured: From baseline up to 5.5 years
Change from Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Scores
Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
Measured: Baseline, Months 12, 24, 36, 48 and 60
Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score
Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.
Measured: Baseline, Months 12, 24, 36, 48 and 60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2024
- Study start10 Oct 2024
- Primary completion24 Oct 2030 (estimated)
- Study completion24 Oct 2030 (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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 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
- NCT06646289
- Sponsor's protocol ID
- 74765340RPG2001
- Status last verified
- Sep 2026
- First submitted
- 16 Oct 2024
- 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
- An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With 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)
- ClinicalTrials.gov ID
- NCT06646289
- Acronym / sponsor ID
- 74765340RPG2001
- Conditions
- X-Linked Retinitis Pigmentosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (actual)
- Lead sponsor
- MeiraGTx Ocular UK LtdIndustry (pharma, biotech, devices)
- Study start
- 10 Oct 2024 (actual)
- Primary completion
- 24 Oct 2030 (estimated)
- Study completion
- 24 Oct 2030 (estimated)
- First posted
- 17 Oct 2024 (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.
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: AAV5-hRKp.RPGRParticipants will receive a dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 depending on the dosage administered in study MGT009 (NCT03252847) in the past. After receiving the treatment, participants will be assessed for safety. |
| Cohort 2 | Experimental | Biological: AAV5-hRKp.RPGRParticipants will receive the treatment dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 in the second eye once the safety will be determined in Cohort 1. |
| Cohort 3 | Other | Other: No intervention (Follow-Up assessment)The participants who are not willing to undergo surgery or are not eligible for surgery will be assessed in this cohort. They will be assessed yearly until 5 years after their initial eye surgery in previous study MGT009. |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or is currently enrolled in Study MGT010
- Must sign an informed consent form indicating that they understand the purpose and procedures of the study and is willing to participate in the study
- Willing to adhere to the protocol and long-term follow-up
Key exclusion criteria (1 listed)
- There are no specific exclusion criteria to enroll in this study
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 |
|---|---|---|
| AAV5-hRKp.RPGRAlso: JNJ-74765340, botaretigene sparoparvovecAAV5-hRKp.RPGR will be administered sub-retinally. | Biological | Cohort 1, Cohort 2 |
| No intervention (Follow-Up assessment)Participants will not receive any intervention and will undergo follow-up assessment. | Other | Cohort 3 |
Primary outcome
Number of Participants With Adverse Events (AE)
Time frame: From baseline up to 5.5 years
An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non investigational) product, whether or not related to that medicinal (investigational or non investigational) product.
Change from Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Scores
Time frame: Baseline, Months 12, 24, 36, 48 and 60
Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score
Time frame: Baseline, Months 12, 24, 36, 48 and 60
Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.
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
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AAV5-hRKp.RPGR in people with X-Linked Retinitis Pigmentosa, run by MeiraGTx Ocular UK Ltd at 3 sites in 2 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 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 3 sites in United States, United Kingdom. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Adverse Events (AE) (time frame: From baseline up to 5.5 years). The study lists 3 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.