Clinical Trial
NCT02064569 Completed
An open label dose escalation clinical trial to evaluate the safety and the tolerability of gs010 (rAAV2/2-ND4) in patients with leber hereditary optic neuropathy due to mutations in the mitochondrial nadh dehydrogenase 4 gene
An open label dose escalation clinical trial to evaluate the safety and the tolerability of gs010 (rAAV2/2-ND4) in patients with leber hereditary optic neuropathy due to mutations in the mitochondrial nadh dehydrogenase 4 gene.
Disease
Disease type
Leber Hereditary Optic Neuropathy (LHON)
Orphan drug recognition
NA
Patient type
Adult
Children
Inclusion / Exclusion
Funding
industry
Members involved
Main investigators

Dr Catherine Vignal-Clermont
France
Neuro-Ophthalmology Rare Diseases (WG2)
Working Group Leader
Others investigators

Pr Isabelle Audo
Representative
France
Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), National Integration (TWG9), Neuro-Ophthalmology Rare Diseases (WG2), Pediatric Ophthalmology Rare Diseases (WG3), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)
ERN EYE member investigating site
HCP : Principal investigators

RÉFÉRET National Hospital of Ophthalmology of Quinze-Vingts, Paris, France
Adress
28 Rue de Charenton
75012 Paris
France