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A gene therapy injected into the eye aims to restore vision lost to a rare inherited retinal disease

NCT ID NCT01496040

Cure Sponsor: Nantes University Hospital 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 14, 2026 · Last updated Sep 15, 2026 · Updated 1 time

Summary

Researchers are testing a gene therapy called rAAV2/4.hRPE65 in people with Leber congenital amaurosis or severe early-onset retinal degeneration caused by mutations in the RPE65 gene. The treatment delivers a working copy of the gene directly into one eye through a single injection. The trial enrolls nine participants, including children, in three groups that receive different doses. The main goal is to check whether the therapy is safe, with vision tests and immune measurements used to look for signs of benefit.

What this could mean

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

Active substance
a gene therapy that delivers a working copy of the RPE65 gene into the eye
What this could lead to
If it works, this approach could restore some vision for people born with RPE65-related retinal dystrophy, a condition that currently has no cure.
What could go wrong
This is a very small, early-stage trial with only nine participants, so safety and benefit are far from proven. Eye surgery and gene therapy carry risks of inflammation, infection, or damage to the retina, and the treatment may not help or may stop working over time.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

9 people

The number who actually took part.

Start date

Sep 2011

Finished

Aug 2014

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

6 to 50 years

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: * Mutations that code for abnormal RPE65 protein * Presence of characteristic abnormalities in fundus * Dramatic reduction of both rods ans cones ERG responses * Low visual acuity \<0.32 * inform consent signed Exclusion Criteria: * Patients with chronic conditions such a haematological, cardiac, renal diseases * Patients with, within the past 6 months, a clinically significant cardiac disease or known congestive heart failure, cardiac rhytm and conduction abnormalities * Patients with pulmonaty dysfunction * Patients with suspected rheumatoid arthritis * Patients with current systemic infection........

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Conditions

The condition(s) this trial relates to.

Leber congenital amaurosis Leber congenital amaurosis 2

As listed by the trial registrant

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

Leber congenital amaurosis

Contacts and locations

Locations

  • CHU Nantes

    Nantes, 44000, France

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