Gene therapy trial offers hope for inherited blindness
NCT ID NCT04850118
First seen Apr 22, 2026 · Last updated Apr 23, 2026 · Updated 1 time
Summary
This study tests a gene therapy called AGTC-501 in males aged 12 to 50 with X-linked retinitis pigmentosa, an inherited eye disease that causes vision loss. The treatment aims to improve vision in low light by replacing a faulty gene. Participants receive either a low or high dose of the therapy or no treatment, and their vision is tracked over time.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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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
Conditions
Explore the condition pages connected to this study.