Trials / NCT04850118
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
Underway, not enrolling Phase 2 / Phase 3 Gene therapy
- Sponsor
- Beacon Therapeutics
- Treatment
- rAAV2tYF-GRK1-hRPGRco; Control
- Gene
- RPGR
- Conditions
- X-Linked Retinitis Pigmentosa
- Ages
- 12 Years to 50 Years
- Enrollment
- 85 participants
- Started
- 2024-03-14
- Main results expected
- 2026-06-30
- Results posted
- Not yet
- Record last updated
- 2026-04-07
What the study is testing
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.
Where it runs
Australia (2)
- Sydney Eye Hospital, Sydney, New South Wales
- Royal Victorian Eye & Ear Hospital, East Melbourne, Victoria
Canada (1)
- McGill University Health Centre - Glen Site, Montreal, Quebec
United Kingdom (3)
- Moorfields Eye Hospital, London
- The Retina Clinic London, Institute of Ophthalmology, University College London, London
- Oxford Eye Hospital, Oxford
United States (19)
- Retina Macula Institute of Arizona, Scottsdale, Arizona
- Children's Hospital Los Angeles, Los Angeles, California
- University of Florida Health Jacksonville, Department of Ophthalmology, Jacksonville, Florida
- Bascom Palmer Eye Institute- University of Miami, Miami, Florida
- Midwest Eye Institute (Retina Partners Midwest), Carmel, Indiana
- Wilmer Eye Institute at Johns Hopkins, Baltimore, Maryland
- Ophthalmic Consultants of Boston, Boston, Massachusetts
- Mayo Clinic, Rochester, Minnesota
- Duke Eye Center, Durham, North Carolina
- Cincinnati Eye Institute, Cincinnati, Ohio
- Cole Eye Institute - Cleveland Clinic, Cleveland, Ohio
- Casey Eye Institute, OHSU, Portland, Oregon
- The Center for Advanced Retinal & Ocular Therapeutics University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania
- Mid Atlantic Retina, Philadelphia, Pennsylvania
- University of Pittsburgh, Pittsburgh, Pennsylvania
- Retina Consultants of Texas, Bellaire, Texas
- Retina Foundation of the Southwest, Dallas, Texas
- Baylor Eye Institute, Houston, Texas
- Retina Consultants of San Antonio Texas, San Antonio, Texas