Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy (CICLO-NOHL)

The Leber Hereditary Optic Neuropathy is a genetic disorder caused by maternal transmission of mitochondrial DesoxiroboNucleid Acid mutations. It is manifested by a rapidly progressive blindness, profound, due to atrophic optic nerve. The visual loss is primarily unilateral bilateralisation taking place in the vast majority of cases in weeks or months. The neuro-cardio-protective properties of cyclosporine (and its analogs specifically targeting the anti-apoptotic mechanisms) are particularly promising. The investigators hypothesis is that cyclosporine may limit apoptosis during the acute phase of the disease process and would limit the loss of visual acuity and improve the visual prognosis of these patients.

Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales

Inclusion Criteria: patient with the mutation confirmed by molecular analysis patient with a recent loss of monocular vision (≤ 6 months) voluntarily Patient Consent Exclusion Criteria: patient who have not given their written and informed consent signed against indication of cyclosporine no drug compliance to previous inclusion no national health insurance affiliation pregnant women or lactating women who could become pregnant during the study period and with no contraception private patients of their liberty by judicial or administrative decision, or patients under supervision

Leruez S, Verny C, Bonneau D, Procaccio V, Lenaers G, Amati-Bonneau P, Reynier P, Scherer C, Prundean A, Orssaud C, Zanlonghi X, Rougier MB, Tilikete C, Milea D. Cyclosporine A does not prevent second-eye involvement in Leber's hereditary optic neuropathy. Orphanet J Rare Dis. 2018 Feb 17;13(1):33. doi: 10.1186/s13023-018-0773-y.