CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 62 enrolled
Drug / intervention
GS010 +1 moregenetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03406104
NCT03406104Phase 3Completed

Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials (RESTORE)

GenSight Biologics·interventional·Posted Jan 23, 2018·Updated Mar 2, 2026

In Brief

A Phase 3 clinical trial evaluating GS010 and Sham Intravitreal Injection for Leber Hereditary Optic Neuropathy. Completed, enrolled 62 participants across 7 sites in 5 countries.

Detailed Summary

The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesFrance, Germany, Italy, United Kingdom, United States
Collaborators--

Timeline

Phase 3CompletedFinished
201820192020202120222023202420252026
First PostedJan 23, 2018
Enrollment StartJan 9, 2018
Primary CompletionJul 4, 2022
TodayJul 2, 2026
Enrollment to primary: 4.5 yearsPosted 8.4 years ago

Interventions

GS010genetic

Lenadogene nolparvovec Intravitreal ocular unilateral Injection

Sham Intravitreal Injectionother

Lenadogene nolparvovec Intravitreal ocular unilateral Injection