Active — Not RecruitingPhase 3

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

United States97 participantsStarted 2020-12-04

Plain-language summary

A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Who can participate

Age range3 Years

SexALL

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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: * 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. Exclusion Criteria: * None

What they're measuring

Number of Participants with Ocular and Non-ocular Adverse Events

Timeframe: Day 1 - Month 60

Number of Participants With Abnormalities in Laboratory Assessments

Timeframe: 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

Timeframe: From Baseline - Month 60

Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment

Timeframe: From Baseline- Month 60

Trial details

NCT IDNCT04794101

SponsorJanssen Research & Development, LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-09-20

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