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

Running, not enrolling · Phase 3

Conditions studied: X-Linked Retinitis Pigmentosa

In brief

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

Key facts

Study ID

NCT04794101

Run by

Janssen Research & Development, LLC

People needed

97

Starts

2020-12-04

Expected to finish

2029-12-07

Last updated by the study team

2026-07-06

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…

• None

Where it is running

• 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
• Ghent University Hospital — 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 — Rome, 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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