NCT07303296

Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients

A Dose-ranging Randomized, Open-label Study Evaluating the Effect of Bilateral Intravitreal Injection of GS010 at Two Dose Levels on Visual Acuity and Retinal Mitochondrial Activity in Patients Affected With ND4 Leber Hereditary Optic Neuropathy - The REVISE Study

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (estimated)
• Sponsor: GenSight Biologics · Industry
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

Summary

The goal of this Clinical trial is to assess the safety and efficacy of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy (LHON)

Conditions

• Leber Hereditary Optic Disease

Interventions

Type	Name	Description
GENETIC	GS010 High dose	For patients allocated dose H GS010 will be administered at a dose of 3.9E11 VG/eye in a final volume of 90 μL (ARM H).
GENETIC	GS010 Low dose	For patients allocated dose L, GS010 will be administered at a dose of 1.3E11 VGVG/eye in a final volume of 30 μL (ARM L).

Timeline

• Start date: 2026-01-22
• Primary completion: 2028-05-15
• Completion: 2028-06-01
• First posted: 2025-12-24
• Last updated: 2026-03-02

Locations

1 site across 1 country: France