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A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
Sponsor: Beacon Therapeutics
Summary
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.
Official title: A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa
Key Details
Gender
MALE
Age Range
12 Years - 50 Years
Study Type
INTERVENTIONAL
Enrollment
85
Start Date
2024-03-14
Completion Date
2031-10-31
Last Updated
2026-04-07
Healthy Volunteers
No
Conditions
Interventions
rAAV2tYF-GRK1-hRPGRco
Adeno-associated virus vector expressing a human RPGR gene
Control
Untreated Control Group 3
Locations (25)
Retina Macula Institute of Arizona
Scottsdale, Arizona, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of Florida Health Jacksonville, Department of Ophthalmology
Jacksonville, Florida, United States
Bascom Palmer Eye Institute- University of Miami
Miami, Florida, United States
Midwest Eye Institute (Retina Partners Midwest)
Carmel, Indiana, United States
Wilmer Eye Institute at Johns Hopkins
Baltimore, Maryland, United States
Ophthalmic Consultants of Boston
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Duke Eye Center
Durham, North Carolina, United States
Cincinnati Eye Institute
Cincinnati, Ohio, United States
Cole Eye Institute - Cleveland Clinic
Cleveland, Ohio, United States
Casey Eye Institute, OHSU
Portland, Oregon, United States
The Center for Advanced Retinal & Ocular Therapeutics University of Pennsylvania Perelman School of Medicine
Philadelphia, Pennsylvania, United States
Mid Atlantic Retina
Philadelphia, Pennsylvania, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Retina Consultants of Texas
Bellaire, Texas, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
Baylor Eye Institute
Houston, Texas, United States
Retina Consultants of San Antonio Texas
San Antonio, Texas, United States
Sydney Eye Hospital
Sydney, New South Wales, Australia
Royal Victorian Eye & Ear Hospital
East Melbourne, Victoria, Australia
McGill University Health Centre - Glen Site
Montreal, Quebec, Canada
Moorfields Eye Hospital
London, United Kingdom
The Retina Clinic London, Institute of Ophthalmology, University College London
London, United Kingdom
Oxford Eye Hospital
Oxford, United Kingdom