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.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for USHER'S SYNDROME are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Investigational Site Number : 250001
Paris, 75012, France
-
Oregon Health and Science University Site Number : 840001
Portland, Oregon, 97239-3098, United States