Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patient with the mutation confirmed by molecular analysis
- •patient with a recent loss of monocular vision (≤ 6 months)
- •voluntarily Patient Consent
排除标准
- •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
研究组 & 干预措施
cyclosporine
干预措施: cyclosporine (Drug)
结局指标
主要结局
Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales
时间窗: at 9 months
次要结局
未报告次要终点