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Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year | Clinical Trials | Clareo Health

COMPLETEDPHASE3

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected With G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year

NCT03293524•GenSight Biologics

View on ClinicalTrials.gov

Summary

The goal of this clinical trial is to assess the safety and efficacy of GS010, a gene therapy, in improving the retina functional \& structural outcomes in subjects with LHON due to the G11778A ND4 mitochondrial mutation when vision loss duration is present up to one year.

Detailed Description

GS-LHON-CLIN-05 is a Phase III, global, multi-center randomized, double-masked for the primary analysis, placebo-controlled, clinical study. As LHON is a neurodegenerative disease, the goal is to administer GS010 as soon as possible upon confirmation of the LHON diagnosis and the causative mutation.

Eligibility

Age

15 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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.
+4 more criteria

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).

Locations (13)

Doheny Eye Center UCLA Pasadena

Pasadena, California, United States

University of Colorado Health Eye Center

Aurora, Colorado, United States

Emory Healthcare - The Emory Clinic

Atlanta, Georgia, United States

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Wills Eye Institute - Ocular Oncology Service

Philadelphia, Pennsylvania, United States

Vanderbilt Eye Institute

Nashville, Tennessee, United States

Universitair Ziekenhuis Gent

Ghent, Belgium

CHNO Les Quinze Vingts

Paris, France

IRCCS Istituto delle Scienze Neurologiche di Bologna UOC Clinica Neurologica

Bologna, Italy

Key Dates

Start Date

March 12, 2018

Primary Completion Date

July 23, 2024

Completion Date

July 23, 2024

Last Updated

August 26, 2025

Enrollment

ACTUAL participants

Conditions

Leber Hereditary Optic Neuropathy

Interventions

GS010

GENETIC

Placebo

DRUG

REALITY LHON Registry

NCT03295071

Leber Hereditary Optic Neuropathy

View study

TERMINATEDPHASE1/PHASE2

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

NCT05820152

Leber Hereditary Optic Neuropathy (LHON)

View study

UNKNOWNPHASE2/PHASE3

A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy

NCT03153293

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: August 26, 2025Data 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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