What is GS010 studying for Leber Hereditary Optic Neuropathy?

GS010 is being studied to assess its safety and effects on vision and retinal cell activity in patients with ND4 Leber Hereditary Optic Neuropathy. The trial evaluates two different doses to understand how the treatment may impact visual acuity. This research is sponsored by GenSight Biologics.

Who may be eligible for this Leber Hereditary Optic Neuropathy clinical trial?

People aged 15 or older with vision loss in both eyes caused by ND4 LHON may qualify. Participants must have specific genetic mutations and meet vision criteria. Both females and males can participate, but safety and medical history are also considered. Please review eligibility criteria for more details.

Is there any cost to participate in this Leber Hereditary Optic Neuropathy clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel and other expenses may vary and are not guaranteed to be covered by the study.

Actively Recruiting

Phase 2

Age: 15Years +

All Genders

ID07303296

A Dose-ranging Randomized, Open-label Study Evaluating Bilateral Intravitreal Injection of GS010 at Two Dose Levels on Visual Acuity and Retinal Mitochondrial Activity in ND4 Leber Hereditary Optic Neuropathy

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

Participants Needed

Research Sites

2 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effects of GS010, a gene therapy, at two different dose levels in patients with ND4 Leber Hereditary Optic Neuropathy LHON, a condition affecting vision. This Phase 2 trial aims to measure changes in visual acuity and retinal mitochondrial activity to better understand the treatments impact on this inherited optic nerve disease. Participants receive GS010 through bilateral intravitreal injections, meaning the treatment is injected into both eyes. There are two study groups one receives a high dose 3.9E11 VGeye and the other a low dose 1.3E11 VGeye. The study is randomized and open-label, so patients are assigned to one of these doses without masking. Treatment is given once, and participants are monitored over time. During the study, participants will have their visual acuity measured at baseline and regularly up to 1.5 years post-treatment. Additional assessments include retinal imaging and genetic testing to confirm diagnosis. Researchers will monitor safety and treatment effects throughout this period. Participants are expected to comply with study visits and assessments, and the total participation time extends over 1.5 years after receiving GS010.

CONDITIONS

Brief 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

You may qualify if you...

Aged 15 years or older at vision loss onset
Clinically manifested 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 and no other optic nerve or retinal pathology mutations
Vision loss duration between 6 months and 1.5 years in the first affected eye at study start
No limitation to high-quality OCT imaging in both eyes
Clear ocular media and sufficient pupil dilation for eye exams
Negative HIV test
Female patients of childbearing potential must use effective birth control for 6 months post-treatment; males must use condoms for 6 months post-treatment
Willing and able to follow study procedures and attend visits
Signed informed consent provided by patient and guardian if under 18

You will not qualify if you...

Allergy or hypersensitivity to GS010 or its components
Contraindications to intravitreal injection in either eye
Recent or planned intraocular surgery or procedure within 6 months
Optic neuropathy from causes other than LHON
History of recurrent or active eye inflammation
Unilateral or asymmetric ND4 LHON disease or amblyopia affecting treatment decision
Use of idebenone within 7 days before study start
Inability to tolerate anti-inflammatory treatment
Ocular or systemic disease or conditions affecting safety or study assessments
Use of investigational drugs or devices within 90 days before study or planned during study
Previous ocular gene therapy in either eye
Pregnancy or breastfeeding

Research Team

GenSight Biologics

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