Futility Study of R(+) Pramipexole in Early Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- ALS-FRSr score taken each month for 3 months during lead-in and for 6 months during treatment
研究概览
简要总结
The hypothesis of this study is that treatment with R(+) pramipexole at 30 mg/day will alter the slope of decline in ALS functional rating scale over the course of 6 months. ALS patients at an early stage of disease will be observed for 3 months after enrollment and then treated with drug for 6 months.
详细描述
This is a futility design Phase II study using ALS-FRSr as the primary variable to monitor progression of disease in patients with early ALS. The drug to be tested is R(+) pramipexole, an antioxidant that concentrates into brain and mitochondria. R(+)PPX will be administered at 30 mg/day over 6 months, following a 3 month lead-in period without drug therapy. For purposes of this study, futility is defined as failure to decrease the slope of ALS-FRSr decline by less than 40%.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •established diagnosis of ALS FVC>60% of predicted not being ventilated no difficulty swallowing ambulatory (can use assistance devices)
排除标准
- •ALS duration >3 years advanced ALS with survival predicted <6 months dementia (MMSE<22) prior exposure to R(+) pramipexole orthostatic hypotension >30 mmHg history of psychosis or hallucinations abnormal baseline safety lab values
结局指标
主要结局
ALS-FRSr score taken each month for 3 months during lead-in and for 6 months during treatment
时间窗: -3 -2 -1 0 1 2 3 4 5 6 months
次要结局
- FVC taken each month(-3 -2 -1 0 1 2 3 4 5 6 months)
- hand dynamometry taken each month(-3 -2 -1 0 1 2 3 4 5 6)