Actively Recruiting

Phase 2
Age: 15Years +
All Genders
NCT07303296

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

Led by GenSight Biologics · Updated on 2026-03-02

14

Participants Needed

1

Research Sites

123 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 15Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Aged 15 years or older at the time of vision loss onset
  • Vision loss due to ND4 LHON in both eyes
  • Best corrected visual acuity (BCVA) of at least LogMAR +2.39 in both eyes
  • Genetic testing confirming pathogenic ND4 LHON mutation(s) without other optic nerve or retinal pathogenic mutations
  • Vision loss duration between 6 months and 1.5 years in the first affected eye at inclusion
  • No limitations preventing quality OCT imaging in both eyes
  • Clear ocular media and adequate pupil dilation for thorough eye examination
  • HIV negative serology
  • Female patients of childbearing potential agree to use effective birth control for 6 months after treatment; male patients agree to use condoms for 6 months after treatment
  • Willing and able to comply with protocol, attend visits, and complete assessments
  • Signed informed consent obtained from patient or legal guardian if under 18 years old
Not Eligible

You will not qualify if you...

  • Allergy or hypersensitivity to GS010 or its components
  • Contraindication to intravitreal injection in any eye
  • Prior relevant intraocular surgery or injection within 6 months before inclusion, or planned surgery
  • Optic neuropathy from causes other than LHON
  • History of recurrent or active intraocular inflammation or uveitis
  • Indication for unilateral GS010 treatment due to asymmetric disease, amblyopia, or patient preference
  • Use of idebenone within 7 days before inclusion
  • Inability to tolerate anti-inflammatory treatment
  • Ocular or systemic diseases or conditions that affect safety or study assessments
  • Use of investigational drugs or devices within 90 days or 5 half-lives before inclusion, or plans for other investigational studies
  • Previous ocular gene therapy in either eye
  • Pregnancy or breastfeeding

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Hopital national des quinze-vingts

Paris, France, 75012

Actively Recruiting

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Research Team

G

GenSight Biologics

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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