Gene therapy for rare Blindness-Deafness syndrome passes early safety check
NCT ID NCT02065011
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 study follows 9 adults with Usher syndrome type 1B who previously received a single injection of the gene therapy SAR421869 under their retina. Researchers are monitoring long-term safety and whether the treatment can slow retinal degeneration. The trial is active but no longer recruiting, and results will help determine if further testing is warranted.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- SAR421869 (a gene therapy given as an injection under the retina)
- What this could lead to
- If successful, this could point toward a treatment that slows or delays vision loss in people with Usher syndrome type 1B.
- What could go wrong
- This is a very small, early-phase study with only 9 participants, so results may not apply to everyone. The main goal is safety, not yet proof of effectiveness.
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
Locations
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Investigational Site Number : 250001
Paris, 75012, France
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Oregon Health and Science University Site Number : 840001
Portland, Oregon, 97239-3098, United States
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Other studies related to the condition(s) this trial covers.