Gene therapy injection aims to halt blinding eye disease
NCT ID NCT06467344
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early study tests a new gene therapy called ACDN-01 for people with Stargardt disease, a genetic condition that leads to progressive vision loss. The therapy is given as a one-time injection under the retina. The main goals are to check safety and find the right dose, with a first look at whether it might help preserve sight.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ACDN-01 (a gene therapy given as a one-time injection under the retina)
- What this could lead to
- If it works, this could slow or stop vision loss in people with Stargardt disease and related retinal conditions.
- What could go wrong
- This is a very early, first-in-human trial with only 15 participants. It primarily tests safety, not effectiveness. Gene therapies can cause inflammation or other side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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Study contacts
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Contact
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can stem cells restore vision in stargardt disease? a First-in-Human trial aims to find out
- Could stem cells restore sight in damaged eyes?
- New study tracks stargardt disease to pave way for future treatments
- New eye camera could unlock secrets of inherited blindness
- Tiny genetic clues may unlock eye disease mysteries
- New cell therapy aims to slow vision loss in retinal disease