Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (NCT04794101)

🧪 Interventions in This Study

• Genetic: AAV5-hRKp.RPGR Intermediate Dose (BIOLOGICAL)
• Genetic: AAV5-hRKp.RPGR Low Dose (BIOLOGICAL)

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (28 Sites)

Showing the first 25 of 28 sites.

• Shiley Eye Institute Jacobs Retina Center — La Jolla, California
• Childrens Hospital Los Angeles — Los Angeles, California
• Stanford Health Care — Palo Alto, California
• VitreoRetinal Associates, PA — Gainesville, Florida
• Emory University — Atlanta, Georgia
• Massachusetts Eye and Ear Infirmary — Boston, Massachusetts
• Univ of Michigan Medical Center — Ann Arbor, Michigan
• Duke Eye Center — Durham, North Carolina
• University of Pittsburgh Medical Center (UPMC) — Pittsburgh, Pennsylvania
• Retina Consultants of Houston — Bellaire, Texas
• Ghent University Hospital — Ghent,
• Hospital For Sick Children — Toronto, Ontario
• Rigshospitalet Glostrup — Glostrup Municipality,
• Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts — Paris,
• Hadassah Medical Center — Jerusalem,
• Azienda Ospedaliero Universitaria Careggi — Florence,
• Ospedale San Paolo — Milan,
• Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli — Naples,
• IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS — Rome,
• VUMC Amsterdam — Amsterdam,
• Radboudumc — Nijmegen,
• Hosp Univ Fund Jimenez Diaz — Madrid,
• University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical — Basel,
• Universite de Lausanne, Hopital ophtalmique Jules-Gonin — Lausanne,
• NHS Lothian — Edinburgh,

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in X-Linked Retinitis Pigmentosa

• Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations — Durham, North Carolina
• A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP — Scottsdale, Arizona
• A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) — Boston, Massachusetts
• A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) — Jacksonville, Florida
• 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) — Aurora, Colorado

Explore by condition: All X-Linked Retinitis Pigmentosa clinical trials

💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Review the official NIH record below to find the coordinating site.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.