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Gene therapy shows promise for rare eye disease that causes blindness

NCT ID NCT03293524

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 trial tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that leads to rapid vision loss. Participants received injections of the therapy or a placebo into both eyes. The study measured changes in vision over 1.5 years, with follow-up up to 5 years. The goal was to see if GS010 could improve or stabilize eyesight in those with vision loss for less than a year.

What this could mean

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

Active substance
GS010 (lenadogene nolparvovec) gene therapy
What this could lead to
If successful, this gene therapy could slow or partially reverse vision loss in people with a specific form of Leber hereditary optic neuropathy.
What could go wrong
This is a Phase 3 trial, but results may not apply to all patients. Gene therapy carries risks like injection-related complications, and long-term effects are still being studied.

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

98 people

The number who actually took part.

Started

Mar 2018

Finished

Jul 2024

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.

Main Selection Criteria: * Age 15 years or older on the date of signed informed consent. * Clinically manifested vision loss due to ND4 LHON, to any extent, in at least one eye. * Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2). Main Non-Selection Criteria: * Contraindication to intravitreal injection in any eye. * Subjects refusing to discontinue idebenone. * Previous vitrectomy in either eye. * Narrow angle in any eye contra-indicating pupillary dilation. * Presence of known/documented mutations, other than the G11778A ND4 LHON-causing mutation, which are known to cause pathology of the optic nerve, retina or afferent visual system. * History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation. Main Inclusion Criteria: * Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2). * Each eye of the subject must maintain at least Hand Motion (HM) visual acuity, as defined by the study's SOP for visual acuity testing. * Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject's mitochondrial DNA. Main Exclusion Criteria: * Light Perception (LP) or No Light Perception (NLP) visual acuity in any eye, as defined by the study's standard operating procedure (SOP) for visual acuity testing. * Presence of active infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye. * Presence of alcoholism, alcohol dependence, or alcohol or drug abuse (excluding nicotine).

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Conditions

The condition(s) this trial relates to.

Eye Diseases, Hereditary eye disorder hereditary optic atrophy inborn mitochondrial metabolism disorder Leber hereditary optic neuropathy

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, Paris, 75012, France

  • Department of Ophthalmology, Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107, United States

  • Doheny Eye Center UCLA Pasadena

    Pasadena, California, 91105, United States

  • Emory Healthcare - The Emory Clinic

    Atlanta, Georgia, 30322, United States

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034, Spain

  • IRCCS Istituto delle Scienze Neurologiche di Bologna (ISNB) Clinica Neurologica

    Bologna, 40139, Italy

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    London, Greater London, EC1V 2PD, United Kingdom

  • Taipei Veterans General Hospital

    Taipei, 11217, Taiwan

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • University of Colorado Health Eye Center

    Aurora, Colorado, 80045, United States

  • Vanderbilt Eye Institute

    Nashville, Tennessee, 37232, United States

More trials for these conditions

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