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Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE) | Clinical Trials | Clareo Health

COMPLETEDPHASE3

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)

Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for More Than 6 Months and To 12 Months by LHON Due to the G11778A Mutation in the ND4 Gene

NCT02652780•GenSight Biologics

View on ClinicalTrials.gov

Summary

The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for more than six months and up to one year.

Eligibility

Age

15 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Selection Criteria:
•Participants must meet all the following criteria at the Screening Visit (Visit 1) in order to be included into the study.
•1. Age 15 years or older.
•2. Onset of vision loss based on medically documented history or participants testimony, in both eyes for 181 and ≤365 days in duration.
•3. Each eye of the participant maintaining visual ability to allow at least for counting of the examiner's fingers at any distance.
•4. Female participants (if of childbearing potential) must agree to use effective methods of birth control up to 6 months after IVT injection and male participants must agree to use condoms for up to 6 months after IVT injection.
•5. Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing.
•6. Signed written informed consent.
•Participants included in the study must satisfy all the following criteria at the Inclusion Visit (Visit 2).
•1. 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 participant's mitochondrial DNA.
+3 more criteria

Exclusion Criteria

•Non-Selection Criteria:
•Participants who meet at least one of the following criteria at the Screening Visit (Visit 1) will not be included into the study.
•1. Any known allergy or hypersensitivity to GS010 or its constituents.
•2. Contraindication to IVT injection.
•3. IVT drug delivery to either eye within 30 days prior to the Screening Visit (Visit 1).
•4. Previous vitrectomy in either eye.
•5. Narrow angle in either eye contra-indicating pupillary dilation.
•6. Presence of disorders of the ocular media, such as the cornea and lens, which may interfere with visual acuity and other ocular assessments during the study period.
•7. Vision disorders, other than LHON, involving visual disability or with the potential to cause further vision loss during the trial period.
•8. Causes of optic neuropathy other than LHON and glaucoma.
+18 more criteria

Locations (7)

Doheny Eye Center, University of California, Los Angeles

Los Angeles, California, United States

Department of Ophthalmology, Emory University School of Medicine

Atlanta, Georgia, United States

Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Centre National Hospitalier d'Ophtalmologie des Quinze-Vingt

Paris, Paris, France

Department of Neurology, University of Munich, Friedrich-Baur-Institute

Munich, Germany

IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria

Bologna, Bologna, Italy

Moorfields Eye Hospital NHS Foundation Trust

London, United Kingdom

Key Dates

Start Date

January 1, 2016

Primary Completion Date

January 1, 2018

Completion Date

December 1, 2018

Last Updated

March 2, 2026

Enrollment

ACTUAL participants

Conditions

Optic, Atrophy, Hereditary, Leber

Interventions

GS010

BIOLOGICAL

Sham Intravitreal Injection

DEVICE

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

NCT02652767

Optic, Atrophy, Hereditary, Leber

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 2, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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