Gene therapy trial offers hope for boys and men with rare blinding eye condition
NCT ID NCT05878860
First seen May 01, 2026 · Last updated May 23, 2026 · Updated 3 times
Summary
This study tests a gene therapy called ATSN-201 for people with X-linked retinoschisis, a rare inherited eye disease that splits the retina and can lead to vision loss. The trial includes males aged 6 and older, and aims to see if the treatment is safe and can improve vision. The study has three parts, including a phase 3 randomized controlled portion to measure changes in how well the retina senses light.
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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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Children's Hospital of Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Oregon Health Sciences University
RECRUITINGPortland, Oregon, 97239, United States
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Conditions
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