Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Completed · Phase 3
Conditions studied: X-Linked Retinitis Pigmentosa
In brief
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Key facts
- Study ID
- NCT04671433
- Run by
- Janssen Research & Development, LLC
- People needed
- 105
- Starts
- 2020-12-04
- Expected to finish
- 2024-09-30
- Last updated by the study team
- 2026-03-16
Who can join
Age: 3 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
- Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
- Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
Where it is running
- Shiley Eye Institute Jacobs Retina Center — La Jolla, California, United States
- Childrens Hospital — 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 General Hospital - Center for Celiac Research and Treatment — 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
- UZ Gent — Ghent, Belgium
- Hospital For Sick Children — Toronto, Ontario, Canada
- Rigshospitalet Glostrup — Glostrup Municipality, Denmark
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts — Paris, France
- Hadassah Medical Center — Jerusalem, Israel
- Azienda Ospedaliero Universitaria Careggi — Florence, Italy
- Ospedale San Paolo — Milan, Italy
- Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli — Naples, Italy
- IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS — Roma, Italy
- VUMC Amsterdam — Amsterdam, Netherlands
- Radboudumc — Nijmegen, Netherlands
- Hosp Univ Fund Jimenez Diaz — Madrid, Spain
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical — Basel, Switzerland
- Universite de Lausanne, Hopital ophtalmique Jules-Gonin — Lausanne, Switzerland
- NHS Lothian — Edinburgh, United Kingdom
Full record on ClinicalTrials.gov
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