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Can a One-Time gene injection slow inherited blindness?

NCT ID NCT01736592

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 26, 2026 · Last updated Aug 27, 2026 · Updated 1 time

Summary

This study follows patients with Stargardt's macular degeneration, an inherited eye disease that causes progressive vision loss, who previously received an experimental gene therapy called SAR422459. The goal is to check the long-term safety and tolerability of the treatment and to see if it continues to show biological activity. Participants will have regular eye exams and lab tests over an extended period to monitor for side effects and any signs that the therapy is working.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
SAR422459, a gene therapy given as a subretinal injection
What this could lead to
If successful, this could support the long-term safety and potential benefit of gene therapy for Stargardt disease, a form of inherited vision loss.
What could go wrong
This is a small, open-label follow-up study without a control group, so results may not prove effectiveness. Risks include potential side effects from the injection or gene therapy.

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

27 people

The number who actually took part.

Started

Dec 2012

Expected to finish

Aug 2033

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

6 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: Patients must meet ALL of the following criteria: 1. Provide signed and dated written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act \[HIPAA\]) 2. Must have been enrolled in protocol TDU13583 (SG1/001/10) 3. Must have received a subretinal injection of SAR422459 4. Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit. Exclusion Criteria: The following would exclude Patients from participation in the study: 1\. Did not receive SAR422459 as part of the TDU13583 protocol.

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Conditions

The condition(s) this trial relates to.

Stargardt disease

As listed by the trial registrant

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

Stargardt's disease

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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