MedTrace Logo

RESCUE and REVERSE Long-term Follow-up

NCT03406104 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 62

Last updated 2026-03-02

Study results available
· View outcomes & findings →

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.

Conditions

  • Leber Hereditary Optic Neuropathy

Interventions

GENETIC

GS010

Lenadogene nolparvovec Intravitreal ocular unilateral Injection

OTHER

Sham Intravitreal Injection

Lenadogene nolparvovec Intravitreal ocular unilateral Injection

Sponsors & Collaborators

  • GenSight Biologics

    lead INDUSTRY

Principal Investigators

  • Nancy Newman, MD · Emory University Hospital Atlanta, Georgia, United States, 30322

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-01-09
Primary Completion
2022-07-04
Completion
2022-07-04
FDA Drug
Yes

Countries

  • United States
  • France
  • Germany
  • Italy
  • United Kingdom

Study Locations

More Related Trials

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 NCT03406104 on ClinicalTrials.gov