Active — Not RecruitingPhase 2

A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)

United States, United Kingdom24 participantsStarted 2024-10-10

Plain-language 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.

Who can participate

Age range

5 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 Exclusion Criteria: \- There are no specific exclusion criteria to enroll in this study

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants With Adverse Events (AE)

Timeframe: 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

Timeframe: Baseline, Months 12, 24, 36, 48 and 60

Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score

Timeframe: Baseline, Months 12, 24, 36, 48 and 60

Trial details

NCT IDNCT06646289

SponsorJanssen Research & Development, LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-10-24

View on ClinicalTrials.gov More X-Linked Retinitis Pigmentosa trials

Who can participate

Age range

5 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Number of Participants With Adverse Events (AE)

Timeframe: 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

Timeframe: Baseline, Months 12, 24, 36, 48 and 60

Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score

Timeframe: Baseline, Months 12, 24, 36, 48 and 60