One-Time gene injection aims to rescue sight in rare childhood blindness
NCT ID NCT07681778
First seen Jul 02, 2026 · Last updated Jul 02, 2026
Summary
This study tests a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare condition that leads to severe vision loss from a young age. The treatment is given as a single injection under the retina in the worse-seeing eye. The trial includes adults and adolescents and will check safety and whether vision can be improved or stabilized.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
OPGx-RDH12 (gene therapy)
What this could lead to
If successful, this could slow or partially reverse vision loss in people with a rare genetic form of blindness.
What could go wrong
This is an early, small trial. The therapy may not improve vision, and risks include inflammation or damage from the injection.
Disclaimer
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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.
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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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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Associated Retina Consultants
Phoenix, Arizona, 85020, United States
Contact
Contact Email: •••••@•••••
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Perelman School of Medicine, University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Contact
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Retina Consultants of Texas & Retina Group Inc.
Houston, Texas, 77056, United States
Contact