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Gene therapy for rare Blindness-Deafness syndrome passes early safety check

NCT ID NCT02065011

Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

9 people

The number who actually took part.

Started

Sep 2013

Expected to finish

Jun 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria : Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA). Must have been enrolled in protocol TDU13600. Must have received a subretinal injection of SAR421869 Exclusion criteria: Did not receive SAR421869 as part of the TDU13600 protocol.

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Conditions

The condition(s) this trial relates to.

Usher syndrome Usher syndrome type 1B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Usher's syndrome

Contacts and locations

Locations

  • Investigational Site Number : 250001

    Paris, 75012, France

  • Oregon Health and Science University Site Number : 840001

    Portland, Oregon, 97239-3098, United States

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