NCT03293524

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 98 (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 efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.

Detailed description

The REFLECT study is a Phase 3, international, multi-center, randomized, double-masked, placebo-controlled, clinical trial. The primary objective is to assess the efficacy of intravitreal (IVT) of GS010 compared to IVT of placebo in second-affected/not-yet-affected eyes at 1.5 years post-treatment, by analyzing the change from baseline of the visual acuity in ND4 LHON subjects with vision loss up to one year.

Conditions

• Leber Hereditary Optic Neuropathy

Interventions

Type	Name	Description
GENETIC	GS010	GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 was administrated via intravitreal injection containing 1.2/1.3E11 vg in 90 μL balanced sterile saline solution (BSSS).
DRUG	Placebo	The placebo is a balanced sterile saline solution (BSSS) used for IVT. The placebo was administered via intravitreal injection in a volume of 90 μL.

Timeline

• Start date: 2018-03-12
• Primary completion: 2024-07-23
• Completion: 2024-07-23
• First posted: 2017-09-26
• Last updated: 2026-04-16
• Results posted: 2026-04-16

Locations

13 sites across 7 countries: United States, Belgium, France, Italy, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study