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

February 27, 2026 updated by: GenSight Biologics

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

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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

14

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
      • Paris, France, 75012
        • Recruiting
        • Hopital national des quinze-vingts
        • Contact:
        • Principal Investigator:
          • catherine vignal, Dr

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Aged 15 years or older at the time of vision loss onset
  2. Clinically manifested vision loss due to ND4 LHON in both eyes
  3. BCVA of at least LogMAR +2.39, measured at the FrACT scale, in both eyes
  4. Documented results of genotyping showing the presence of pathogenic LHON-associated mutation(s) in the ND4 mitochondrial gene and the absence of other pathogenic LHON-associated mutations in the patient's mitochondrial DNA; absence of pathogenic mutations, other than the ND4 LHON-causing mutation(s), which are known to cause pathology of the optic nerve, retina or afferent visual system. Historic results of genetic analysis are acceptable upon Sponsor approval.
  5. Vision loss duration from 6 months to 1.5 years in the first affected eye at the Inclusion visit (Day 1)
  6. No limitations to OCT image collection that would prevent high quality, reliable images from being obtained in both eyes, as determined by the reading center
  7. Clear ocular media and adequate pupillary dilation to permit thorough ocular examination, as assessed by the Investigator
  8. Human immunodeficiency virus (HIV) negative serology
  9. Female patients of childbearing potential must agree to use effective methods of birth control for up to 6 months after Treatment visit (Day 0). Male patients must agree to use condoms with their female partners for up to 6 months after Treatment visit (Day 0).
  10. Willing and able to comply with the protocol, follow study instructions, attend study visits as required and complete all study assessments
  11. Patient - and parent/legal guardian if the patient is under 18 years of age - has provided signed, written informed consent

Exclusion Criteria:

  • 1. Any known allergy or hypersensitivity to GS010 or any of its constituents 2. Contraindication to IVT in any eye according to the Investigator's clinical judgment and international guidelines (Avery, 2014) 3. Prior intraocular surgery or procedure or IVT, which occurred 6 months prior to inclusion and is considered of clinical relevance per Investigator assessment, or planned intraocular surgery or procedure 4. Presence of optic neuropathy from any cause except LHON, as determined by the Investigator 5. History of recurrent uveitis (idiopathic or immune-related) or active intraocular inflammation, as determined by the Investigator 6. Indication of a unilateral treatment with GS010, as determined by the Investigator:

    • Unilateral or asymmetric ND4 LHON disease: only one eye affected with a vision loss due to ND4 LHON, and the fellow eye with preserved BCVA (LogMAR 0 or close to 0);
    • Amblyopia: unilateral treatment of the functional eye with a vision loss due to ND4 LHON;
    • Patient's desire for unilateral treatment 7. Ingestion of idebenone less than 7 days prior to the Inclusion visit (Day -1) 8. Inability to tolerate the anti-inflammatory regimen 9. Presence of ocular disease (excluding LHON), or systemic disease, or condition (including medications and laboratory test abnormalities) that could compromise patient safety or interfere with assessment of efficacy and safety, as determined by the Investigator 10. Use of any investigational drug, or device, within 90 days, or 5 half-lives, preceding the Inclusion visit (Day-1), whichever is longer, or plans to participate in another study of an investigational drug or device during the study period 11. Previous treatment with ocular gene therapy in either eye. 12. Pregnancy or breast-feeding

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: GS010 High dose
GS010, at a dose of 3.9E11 VG/eye (High Dose) in ARM H
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).
Other Names:
  • Lumevoq
  • GS010
  • lenadogene nolparvovec
Active Comparator: GS010 Low dose
GS010, at a dose of 1.3E11 VG/eye (Low Dose) in ARM L.
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).
Other Names:
  • Lenadogene nolparvovec
  • Lumevoq
  • GS010

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The primary endpoint will be the BCVA change from baseline to 1.5 years post-treatment in the study eyes.
Time Frame: from baseline to 1.5 years post-treatment
from baseline to 1.5 years post-treatment

Secondary Outcome Measures

Outcome Measure
Time Frame
BCVA responder rates from baseline to 1.5 years post-treatment in the study eyes, defined as an improvement of at least -0.2 LogMAR.
Time Frame: from baseline to 1.5 years post-treatment
from baseline to 1.5 years post-treatment
BCVA change from baseline to 1.5 years post-treatment in both eyes.
Time Frame: from baseline to 1.5 years post-treatment
from baseline to 1.5 years post-treatment
Difference between ARM H and ARM L in BCVA change from baseline to 1.5 years post-treatment in the study eyes.
Time Frame: from baseline to 1.5 years post-treatment
from baseline to 1.5 years post-treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

GenSight Biologics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 22, 2026

Primary Completion (Estimated)

May 15, 2028

Study Completion (Estimated)

June 1, 2028

Study Registration Dates

First Submitted

December 11, 2025

First Submitted That Met QC Criteria

December 11, 2025

First Posted (Actual)

December 24, 2025

Study Record Updates

Last Update Posted (Actual)

March 2, 2026

Last Update Submitted That Met QC Criteria

February 27, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GS-LHON-CLIN-08

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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