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 range5 Years

SexMALE

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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

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