Status:
COMPLETED
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
Lead Sponsor:
GenSight Biologics
Conditions:
Leber Hereditary Optic Neuropathy
Eligibility:
All Genders
15+ years
Phase:
PHASE3
Brief 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 mi...
Detailed Description
GS-LHON-CLIN-05 is a Phase III, global, multi-center randomized, double-masked for the primary analysis, placebo-controlled, clinical study. As LHON is a neurodegenerative disease, the goal is to admi...
Eligibility Criteria
Inclusion
- 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
- 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
- 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).
Key Trial Info
Start Date :
March 12 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 23 2024
Estimated Enrollment :
98 Patients enrolled
Trial Details
Trial ID
NCT03293524
Start Date
March 12 2018
End Date
July 23 2024
Last Update
August 26 2025
Active Locations (13)
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1
Doheny Eye Center UCLA Pasadena
Pasadena, California, United States, 91105
2
University of Colorado Health Eye Center
Aurora, Colorado, United States, 80045
3
Emory Healthcare - The Emory Clinic
Atlanta, Georgia, United States, 30322
4
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States, 02114