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Gene therapy shows promise in Long-Term study for rare blindness

NCT ID NCT03406104

Disease control Sponsor: GenSight Biologics 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 62 people with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that causes vision loss, for up to 5 years after they received a single gene therapy treatment called GS010. The goal was to see if the treatment remained safe and if any vision improvements lasted over time. Researchers tracked eye-related side effects and measured changes in visual sharpness.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

62 people

The number who actually took part.

Started

Jan 2018

Finished

Jul 2022

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

15 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 * Subject was treated with GS010 IVT injection in either of the RESCUE or REVERSE Phase III clinical studies * Subject of legal consent age has provided informed consent; subjects that are not of legal consent age have undergone their country-approved clinical trial enrollment consent process Exclusion Criteria * Subject is unwilling or unable to comply with the protocol requirements * Subject has any medical or psychological condition that, in the opinion of the Investigator, may compromise his or her safe participation in the study * Subject is taking or intending to take idebenone during the long-term follow-up study period

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Conditions

The condition(s) this trial relates to.

Eye Diseases, Hereditary eye disorder hereditary disease inborn mitochondrial metabolism disorder Leber hereditary optic neuropathy nervous system disorder neurodegenerative disease

As listed by the trial registrant

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

Leber hereditary optic neuropathy

Contacts and locations

Locations

  • CHNO Les Quinze Vingts

    Paris, 75012, France

  • Doheny Eye Center UCLA

    Pasadena, California, 91105, United States

  • Emory University Hospital

    Atlanta, Georgia, 30322, United States

  • LMU Klinikum der Universität München / Friedrich-Baur-Institut

    Munich, 80336, Germany

  • Moorfields Eye Hospital

    London, Greater London, EC1V 2PD, United Kingdom

  • Ospedale Bellaria

    Bologna, 40139, Italy

  • Wills Eye Institute

    Philadelphia, Pennsylvania, 19107, United States

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