A Historical Placebo Controlled Screening Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 2 期
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- The slope of decline of the ALS Functional Rating Scale-Revised (ALSFRS-R) score
研究概览
简要总结
This study will examine whether fasudil is effective and safe in treating patients with amyotrophic lateral sclerosis (ALS).
详细描述
This is an open labeled, single center clinical study with placebo of historical researches as control. 10 patients will be enrolled in the study. The basic treatment is riluzole, 50mg twice a day. For the procedure, patients will take fasudil treatment for 14 days (30mg twice a day, intravenous). 3 months later, patients will repeat the fasudil treatment. All the patients will be followed up for 6 months. The primary outcome was the decline rate of ALSFRS-R. The secondary outcomes are the survival time, endpoint time(death, tracheotomy and continuous ventilator-dependent), forced vital capacity (FVC), the short form health survey (SF-36), evaluation of cognitive function (verbal fluency and Frontal Behavioral Inventory Scale) and safety.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of laboratory-supported probable, probable, or definite ALS
- •Age: 18-70 years
- •Disease duration: 3-36 months
- •Forced vital capacity: at least 60% of predicted
- •ALSFRS-R: at least 30, respiratory items: at least 10
- •Decline of ALSFRS-R in the last 3 months before enrollment: 1-8
- •Must take riluzole, on a stable dose for at least 30 days prior to baseline visit with no serious side effects. They must continue the riluzole treatment for at least 6 months after enrollment.
- •Patients of childbearing potential must be using an effective method of birth control
- •Willing and able to give informed consent
排除标准
- •Familial ALS
- •Pregnant or nursing women
- •Patients after tracheotomy or continuous ventilator-dependent (time with non-invasive ventilator more than 22 hours per day for 7 consecutive days.)
- •After percutaneous endoscopic gastrostomy
- •Alanine Transaminase (ALT) or Aspartate Transaminase (AST): at least 3 times the upper limit of normal
- •Abnormal creatinine or urea nitrogen
- •Severe cardiac disease, pulmonary disease, hematic disease, autoimmune disease, mental disease, dementia and substance abuse
- •History of malignancy
- •History of intracranial hemorrhage
- •History of severe bleeding of digestive tract, lungs, nose and skin
- •Allergic to fasudil
- •Participating in other clinical studies or using other investigational drugs at present
研究组 & 干预措施
Fasudil
All the patients will take the fasudil treatment for 14 days (30mg twice a day, intravenous). 3 months later they will repeat the treatment mentioned before.
干预措施: Fasudil (Drug)
结局指标
主要结局
The slope of decline of the ALS Functional Rating Scale-Revised (ALSFRS-R) score
时间窗: Month 3, 6
次要结局
- Survival time(2 years)
研究者
Fan Dongsheng
Chairman of the department of neurology of Peking University Third Hospital
Peking University Third Hospital