Gene therapy offers hope for rare Blindness-Causing eye disease
NCT ID NCT04850118
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called AGTC-501 in males aged 12 to 50 with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. Participants receive one of two doses of the therapy or no treatment, and researchers measure changes in vision under low light over time. The goal is to see if the treatment can slow or improve vision loss safely.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Bascom Palmer Eye Institute- University of Miami
Miami, Florida, 33136, United States
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Baylor Eye Institute
Houston, Texas, 77030, United States
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Casey Eye Institute, OHSU
Portland, Oregon, 97239, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Cincinnati Eye Institute
Cincinnati, Ohio, 45242, United States
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Cole Eye Institute - Cleveland Clinic
Cleveland, Ohio, 44195, United States
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Duke Eye Center
Durham, North Carolina, 27710, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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McGill University Health Centre - Glen Site
Montreal, Quebec, H4A3J1, Canada
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Mid Atlantic Retina
Philadelphia, Pennsylvania, 19107, United States
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Midwest Eye Institute (Retina Partners Midwest)
Carmel, Indiana, 46290, United States
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Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
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Ophthalmic Consultants of Boston
Boston, Massachusetts, 02114, United States
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Oxford Eye Hospital
Oxford, OX39DU, United Kingdom
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Retina Consultants of San Antonio Texas
San Antonio, Texas, 78240, United States
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Retina Consultants of Texas
Bellaire, Texas, 77401, United States
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Retina Foundation of the Southwest
Dallas, Texas, 75231, United States
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Retina Macula Institute of Arizona
Scottsdale, Arizona, 85255, United States
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Royal Victorian Eye & Ear Hospital
East Melbourne, Victoria, 3002, Australia
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Sydney Eye Hospital
Sydney, New South Wales, 2000, Australia
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The Center for Advanced Retinal & Ocular Therapeutics University of Pennsylvania Perelman School of Medicine
Philadelphia, Pennsylvania, 19104, United States
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The Retina Clinic London, Institute of Ophthalmology, University College London
London, W1G7LB, United Kingdom
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University of Florida Health Jacksonville, Department of Ophthalmology
Jacksonville, Florida, 32209, United States
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University of Pittsburgh
Pittsburgh, Pennsylvania, 15219, United States
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Wilmer Eye Institute at Johns Hopkins
Baltimore, Maryland, 21287, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene Therapy's lasting effects on vision tracked in Long-Term study
- Gene therapy targets second eye in rare blindness disorder
- Gene therapy offers hope for rare blindness
- Experimental gene therapy aims to halt vision loss in rare eye disease
- Gene therapy shows promise for rare blindness disease
- Gene therapy watch: 5-Year safety check for inherited blindness treatment