NCT02652780

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 37 (actual)
• Sponsor: GenSight Biologics · Industry
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Optic, Atrophy, Hereditary, Leber

Interventions

Type	Name	Description
BIOLOGICAL	GS010	Both eyes of each participant will receive standard antiseptic preparation, administration of topical local ocular anesthetic agents and will undergo pupillary dilation. Administration of an intra-ocular pressure lowering agent will precede treatment for every participant. GS010-treated Eyes: GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). Participants will receive a single dose of GS010 in one of their randomly selected eyes, via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) plus 0.001% Pluronic F68®.
DEVICE	Sham Intravitreal Injection	Both eyes of each participant will receive standard antiseptic preparation, administration of topical local ocular anesthetic agents and will undergo pupillary dilation. Administration of an intra-ocular pressure lowering agent will precede treatment for every participant. Sham-treated Eyes: One eye of each participant will undergo sham injection. Sham Intravitreal injection will be performed by applying pressure to the eye at the location of a typical intravitreal injection procedure using the blunt end of a syringe without a needle.

Timeline

• Start date: 2016-01-01
• Primary completion: 2018-01-01
• Completion: 2018-12-01
• First posted: 2016-01-12
• Last updated: 2026-03-02
• Results posted: 2020-01-23

Locations

7 sites across 5 countries: United States, France, Germany, Italy, United Kingdom

Regulatory

• FDA-regulated drug study