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

NCT07303296 | Recruiting Phase 2 DMC

• 1 other identifier: GS-LHON-CLIN-08
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief 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

Keywords

Heredity Optic atrophy; leber hereditary optic atrophy; LHON; Eye diseases; gene therapy; AAV2 vectors; Inborn genetic disease; Mitochondrial disease; nervous system disease; Intravitreal injection; neurodegenerative disease; leber disease

Outcome Measures

Primary Outcomes (1)

• The primary endpoint will be the BCVA change from baseline to 1.5 years post-treatment in the study eyes.

  from baseline to 1.5 years post-treatment

Secondary Outcomes (3)

• 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.

  from baseline to 1.5 years post-treatment

• BCVA change from baseline to 1.5 years post-treatment in both eyes.

  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.

  from baseline to 1.5 years post-treatment

Study Arms (2)

GS010 High dose

ACTIVE COMPARATOR

GS010, at a dose of 3.9E11 VG/eye (High Dose) in ARM H

Genetic: GS010 High dose

GS010 Low dose

ACTIVE COMPARATOR

GS010, at a dose of 1.3E11 VG/eye (Low Dose) in ARM L.

Genetic: GS010 Low dose

Interventions

GS010 High dose GENETIC

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).

Also known as: Lumevoq, GS010, lenadogene nolparvovec

GS010 High dose

GS010 Low dose GENETIC

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).

Also known as: GS010, Lenadogene nolparvovec, Lumevoq

GS010 Low dose

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 15 years or older at the time of vision loss onset
• Clinically manifested vision loss due to ND4 LHON in both eyes
• BCVA of at least LogMAR +2.39, measured at the FrACT scale, in both eyes
• 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.
• 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
• Clear ocular media and adequate pupillary dilation to permit thorough ocular examination, as assessed by the Investigator
• Human immunodeficiency virus (HIV) negative serology
• 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).
• Willing and able to comply with the protocol, follow study instructions, attend study visits as required and complete all study assessments
• Patient - and parent/legal guardian if the patient is under 18 years of age - has provided signed, written informed consent

You may not qualify if:

• 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;

Sponsors & Collaborators

• GenSight Biologics: lead

Study Sites (1)

Hopital national des quinze-vingts

Paris, 75012, France

RECRUITING

Related Links

• GenSight Biologics website

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber; Eye Diseases; Genetic Diseases, Inborn; Mitochondrial Diseases; Nervous System Diseases; Neurodegenerative Diseases

Condition Hierarchy (Ancestors)

Optic Atrophies, Hereditary; Optic Atrophy; Optic Nerve Diseases; Cranial Nerve Diseases; Heredodegenerative Disorders, Nervous System; Eye Diseases, Hereditary; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

GenSight Biologics

CONTACT

clinics@gensight-biologics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The objective of the Phase 2 study REVISE is to assess the efficacy, retinal mitochondrial activity and safety of two dose levels (high and low doses) of bilateral GS010 IVT measured by ddPCR in ND4 LHON patients, in order to determine the optimal dose for future development.

Study Record Dates

• First Submitted: December 11, 2025
• First Posted: December 24, 2025
• Study Start: January 22, 2026
• Primary Completion (Estimated): May 15, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: March 2, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)