Can a single injection restore sight in a rare childhood blindness?
NCT ID NCT03920007
First seen Aug 03, 2026 · Last updated Aug 04, 2026 · Updated 1 time
Summary
This trial tests a gene therapy called ATSN-101, given as a one-time injection under the retina, for people with Leber congenital amaurosis caused by GUCY2D gene mutations. The goal is to see if the treatment is safe and can improve vision. Participants receive the therapy in one eye, with the other eye serving as a comparison. The study will monitor for side effects and measure changes in visual acuity over a year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ATSN-101, a gene therapy injected under the retina
- What this could lead to
- If successful, this one-time gene therapy could restore meaningful vision in people with a rare inherited blindness, potentially offering a long-term cure.
- What could go wrong
- This is an early-stage trial with only 15 participants, so results may not apply broadly. Risks include side effects from the injection or the therapy not working as hoped.
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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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Casey Eye Institute - Oregon Health & Science University
Portland, Oregon, 97239, United States
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Scheie Eye Institute, University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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