Gene therapy injection aims to slow vision loss in stargardt disease
NCT ID NCT06467344
First seen Jan 11, 2026 · Last updated May 15, 2026 · Updated 18 times
Summary
This early-stage study tests a single injection of ACDN-01 into the eye for people with Stargardt disease or cone-rod dystrophy caused by ABCA4 gene mutations. The main goals are to check safety and find the best dose. About 15 adults with advanced vision loss will take part. The therapy is designed to target the underlying genetic problem, but it is not expected to be a cure.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Bascom Palmer Eye Institute
RECRUITINGMiami, Florida, 33136, United States
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Casey Eye Institute OHSU
RECRUITINGPortland, Oregon, 97239, United States
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Cincinnati Eye Institute
RECRUITINGCincinnati, Ohio, 45245, United States
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Massachusetts Eye and Ear
RECRUITINGBoston, Massachusetts, 02114, United States
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Retina Consultants of Texas
RECRUITINGHouston, Texas, 77401, United States
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Retina Foundation of the Southwest
RECRUITINGDallas, Texas, 75382, United States
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University of Michigan Kellogg Eye Center
RECRUITINGAnn Arbor, Michigan, 48105, United States
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University of San Francisco
RECRUITINGSan Francisco, California, 94158, United States
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Vitreo Retinal Associates
RECRUITINGGainesville, Florida, 32607, United States
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Wilmer Eye Institute at John Hopkins
RECRUITINGBaltimore, Maryland, 21218, United States
Conditions
Explore the condition pages connected to this study.