Phase 3
N=98
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
Leber Hereditary Optic Neuropathy
Bottom Line
View on ClinicalTrials.gov: NCT03293524 ↗Enrolled (actual)
98
Serious AEs
11.2%
Results posted
Apr 2026
Primary outcome: Primary: Change From Baseline of the Best Corrected Visual Acuity (BCVA) Reported With Log of the Minimal Angle of Resolution (LogMAR) at 1.5 Years Post-treatment, in the Second Affected/Not-yet Affected Eyes — -0.09; -0.04 logMAR — p== 0.608
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- GS010 (Genetic); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 15+ yrs
- Sex
- All
- Sponsor
- GenSight Biologics
- Primary completion
- Jul 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline of the Best Corrected Visual Acuity (BCVA) Reported With Log of the Minimal Angle of Resolution (LogMAR) at 1.5 Years Post-treatment, in the Second Affected/Not-yet Affected Eyes |
-0.09; -0.04 | = 0.608 |
| SECONDARY Change From Baseline of the BCVA Reported With LogMAR at 5 Years Post-treatment, in the Second Affected/Not-yet Affected Eyes |
-0.13; -0.05 | = 0.5086 |
| SECONDARY Proportion of Patients Who Switched From Off-chart Eyes to On-chart Eyes at 5 Years Post-treatment |
61.54; 33.33 | — |
| SECONDARY Responder Analyses - Improvements From Nadir (Gainer Eyes) at 5 Years |
68.8; 66.0 | = 0.7605 |
| SECONDARY Responder Analyses - Clinically Relevant Recovery From Nadir at 5 Years |
75.0; 60.0 | = 0.1352 |
| SECONDARY Responder Analyses- Clinically Relevant Benefit at 5 Years |
75.0; 62.0 | = 0.1995 |
| SECONDARY Quality of Life Questionnaire: VFQ-25 - Composite Score |
11.8; 15.0 | = 0.3605 |
Summary
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
Eligibility 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).
Data sourced from ClinicalTrials.gov (NCT03293524). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.