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

NCT07303296 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2026-03-02

No results posted yet for this study

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

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).

Sponsors & Collaborators

  • GenSight Biologics

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-01-22
Primary Completion
2028-05-15
Completion
2028-06-30

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07303296 on ClinicalTrials.gov