Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
- Registration Number
- NCT02176733
- Lead Sponsor
- University Hospital, Angers
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 12
- patient with the mutation confirmed by molecular analysis
- patient with a recent loss of monocular vision (≤ 6 months)
- voluntarily Patient Consent
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description cyclosporine cyclosporine -
- Primary Outcome Measures
Name Time Method Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales at 9 months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Centre Hospitalier Universitaire
🇫🇷Angers, France