Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
- Conditions
- X-Linked Retinitis Pigmentosa
- Registration Number
- NCT04794101
- Lead Sponsor
- Janssen Research & Development, LLC
- Brief Summary
A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 97
- 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.
- None
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method Number of Participants with Ocular and Non-ocular Adverse Events 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 Day 1 - Month 60 Number of participants with abnormalities in laboratory assessments will be assessed.
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment From Baseline- Month 60 Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment 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 From Baseline - Month 60 Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (28)
Shiley Eye Institute Jacobs Retina Center
🇺🇸La Jolla, California, United States
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Stanford Health Care
🇺🇸Palo Alto, California, United States
VitreoRetinal Associates, PA
🇺🇸Gainesville, Florida, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Massachusetts Eye and Ear Infirmary
🇺🇸Boston, Massachusetts, United States
Univ of Michigan Medical Center
🇺🇸Ann Arbor, Michigan, United States
Duke Eye Center
🇺🇸Durham, North Carolina, United States
University of Pittsburgh Medical Center (UPMC)
🇺🇸Pittsburgh, Pennsylvania, United States
Retina Consultants of Houston
🇺🇸Bellaire, Texas, United States
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