A dose-ranging randomized, open-label study evaluating the effect of bilateral intravitreal injection of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy – The REVISE Study

Condition studied

Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene

Investigational medicinal product(s)

Eligibility

Primary endpoint

BCVA change from baseline to 1.5 years post-treatment in the study eyes.

Endpoint detail

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.