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

Phase 3

Active, not recruiting

• 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

Study Timeline

• Study Start: 2020-12-04
• Study First Submitted: 2020-12-15
• Study First Submitted QC: 2021-03-08
• Study First Posted: 2021-03-11
• Status Verified: 2025-10
• Last Update Submitted: 2025-10-09
• Last Update Posted: 2025-10-10
• Primary Completion: 2029-09-20
• Study Completion: 2029-12-19

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 97

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

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.

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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