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Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

NCT03293524 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 98

Last updated 2026-04-16

Study results available
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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.

Conditions

  • Leber Hereditary Optic Neuropathy

Interventions

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.

Sponsors & Collaborators

  • GenSight Biologics

    lead INDUSTRY

Principal Investigators

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

  • Patrick Yu-Wai-Man, PhD · John van Geest Centre for Brain Repair, University of Cambridge, Cambridge, CB2 0PY, United Kingdom

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-03-12
Primary Completion
2024-07-23
Completion
2024-07-23
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • France
  • Italy
  • Spain
  • Taiwan
  • United Kingdom

Study Locations

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Entities

Drugs

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 NCT03293524 on ClinicalTrials.gov