Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON P
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)
| Condition(s) | Leber Hereditary Optic Disease |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | 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) |
| Who can participate | Inclusion Criteria: 1. Aged 15 years or older at the time of vision loss onset 2. Clinically manifested vision loss due to ND4 LHON in both eyes 3. BCVA of at least LogMAR +2.39, measured at the FrACT scale, in both eyes 4. Documented results of genotyping showing the presence of pathogenic LHON-associated mutation(s) in the ND4 mitochondrial gene and the absence of other pathogenic LHON-associated mutations in the patient's mitochondrial DNA; absence of pathogenic mutations, other than the ND4 LHON-causing mutation(s), which are known to cause pathology of the optic nerve, retina or afferent visual system. Historic results of genetic analysis are acceptable upon Sponsor approval. 5. Vision loss duration from 6 months to 1.5 years in the first affected eye at the Inclusion visit (Day 1) 6. |
| Ages | 15 Years |
| Sex | All |
| Lead sponsor | GenSight Biologics |
| Locations | Paris, France |
| Start date | 2026-01-22 |
| NCT ID | NCT07303296 |
| Official listing | https://clinicaltrials.gov/study/NCT07303296 |