An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 1
- 主要终点
- Lymphocyte frataxin level
研究概览
简要总结
The purpose of this study is to determine the effect of two doses of resveratrol taken for a 12 week period, on frataxin levels in individuals with Friedreich ataxia. This study will also measure the effect of resveratrol on markers of oxidative stress, clinical measures of ataxia, and cardiac parameters.
详细描述
Resveratrol shows promise as an agent for the treatment of Friedreich ataxia due to its antioxidant properties, neuroprotective effects, and ability to increase frataxin levels in vitro and in vivo. This clinical pilot study aims to determine the effect of two doses of resveratrol (1g/day and 5g/day) taken for 12 weeks, on frataxin levels in individuals with Friedreich ataxia. Additional outcome measures include the effect of resveratrol on markers of oxidative stress, clinical measures of ataxia , and cardiac parameters (including relative wall thickness and left ventricular mass index).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 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
- •Adults with Friedreich ataxia due to homozygosity for the GAA repeat expansion in intron 1 of the FXN gene
- •Functional stage on the Ataxia subscale of the FARS of 1 or higher
排除标准
- •Women who are pregnant or lactating
- •Active arrythmias or significant cardiac insufficiency
- •Use of idebenone, Coenzyme Q or vitamin E within 30 days prior to enrolment
- •Use of amiodarone or other medications which may have clinically significant drug interactions that cannot be safely monitored
研究组 & 干预措施
Resveratrol, 1g daily
15 participants will receive resveratrol 1g daily
干预措施: Resveratrol (Drug)
Resveratrol, 5g daily
15 participants will receive resveratrol, 5g daily
干预措施: Resveratrol (Drug)
结局指标
主要结局
Lymphocyte frataxin level
时间窗: 12 weeks
Change in lymphocyte frataxin levels at 12 weeks compared to baseline
次要结局
- Pharmacokinetic studies of resveratrol(First 2 hours post dose)
- Oxidative stress markers(12 weeks)
- Clinical rating scales of ataxia(12 weeks)
- Echocardiogram measures(12 weeks)
研究者
Martin Delatycki
Prof Martin Delatycki
Murdoch Childrens Research Institute