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

• Status: Active, not recruiting
• Trial end date: 2024-06-30
• Target enrollment: 0
• Participant gender: All

Summary

The goal of this clinical trial is to assess the safety and efficacy of GS010, a gene therapy, in improving the retina functional & structural outcomes in subjects with LHON due to the G11778A ND4 mitochondrial mutation when vision loss duration is present up to one year.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: GenSight Biologics
• Treatments: Central Nervous System Depressants

Criteria

Main Selection Criteria:

• Age 15 years or older on the date of signed informed consent.

• Clinically manifested vision loss due to ND4 LHON, to any extent, in at least one eye.

• Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2).

Main Non-Selection Criteria:

• Contraindication to intravitreal injection in any eye.

• Subjects refusing to discontinue idebenone.

• Previous vitrectomy in either eye.

• Narrow angle in any eye contra-indicating pupillary dilation.

• Presence of known/documented mutations, other than the G11778A ND4 LHON-causing mutation, which are known to cause pathology of the optic nerve, retina or afferent visual system.

• History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation.

Main Inclusion Criteria:

• Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2).

• Each eye of the subject must maintain at least Hand Motion (HM) visual acuity, as defined by the study's SOP for visual acuity testing.

• Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject's mitochondrial DNA.

Main Exclusion Criteria:

• Light Perception (LP) or No Light Perception (NLP) visual acuity in any eye, as defined by the study's standard operating procedure (SOP) for visual acuity testing.

• Presence of active infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye.

• Presence of alcoholism, alcohol dependence, or alcohol or drug abuse (excluding nicotine).