Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
Conditions
Brief summary
BCVA change from baseline to 1.5 years post-treatment in the study eyes.
Detailed description
BCVA responder rates from baseline to 1.5 years post-treatment in the study eyes, defined as an improvement of at least -0.2 LogMAR., BCVA change from baseline to 1.5 years post-treatment in both eyes., Difference between ARM H and ARM L in BCVA change from baseline to 1.5 years post-treatment in the study eyes., Frequency and severity of ocular adverse events (AEs)., Frequency and severity of ocular AEs of special interest (AESIs) post-GS010 administration: o Intraocular inflammation; evolution with time and response to corticosteroid. o Increase in intraocular pressure (IOP); evolution with time and response to IOP lowering treatment., Frequency and severity of non-ocular AEs.
Interventions
DRUGLumevoq
Sponsors
Gensight Biologics
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| BCVA change from baseline to 1.5 years post-treatment in the study eyes. | — |
Secondary
| Measure | Time frame |
|---|---|
| BCVA responder rates from baseline to 1.5 years post-treatment in the study eyes, defined as an improvement of at least -0.2 LogMAR., BCVA change from baseline to 1.5 years post-treatment in both eyes., Difference between ARM H and ARM L in BCVA change from baseline to 1.5 years post-treatment in the study eyes., Frequency and severity of ocular adverse events (AEs)., Frequency and severity of ocular AEs of special interest (AESIs) post-GS010 administration: o Intraocular inflammation; evolution with time and response to corticosteroid. o Increase in intraocular pressure (IOP); evolution with time and response to IOP lowering treatment., Frequency and severity of non-ocular AEs. | — |
Countries
France
Outcome results
None listed