Answer ALS -Creation of a Large Bio-repository of iPS Cells, Cell Lines, and Bio-fluid Samples, Combined With Clinical Information to Rapidly Advance Therapeutics That Could Treat ALS
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 1,049
- 试验地点
- 8
- 主要终点
- ALS Functional Rating Scale-Revised (ALSFRS-R)
研究概览
简要总结
Creation of a large repository of induced pluripotent stem cells (iPSC), bio-fluid samples (blood and spinal fluid (optional)), and cell lines for ALS gene identification. This will be combined carefully with collected measures of the pattern of the symptoms people with ALS have and how these change over time. People with other motor neuron diseases and healthy controls will be included as comparisons
详细描述
Patients will have 5 study visits; screening, 3, 6, 9 and 12 months. There will be a one year post-participation follow-up period, during which they will receive an email or phone call interview once every 3 months. During the first year, samples will be collected, breathing, muscle strength, spasticity, general function and cognitive behavior will be assessed. Healthy controls will have 2 study visits during which blood samples will be collected and questionnaires given.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Participants with familial or sporadic ALS diagnosed as possible, laboratory-supported probable, probable or definite according to the World Federation of Neurology (WFN) El Escorial criteria, Primary Lateral Sclerosis Flail Arm ALS, Progressive Muscular Atrophy, Monomelic Amyotrophy, Motor Neuron Disease, Asymptomatic ALS Gene Carriers
- •Participants who are ages 18-100, inclusive.
排除标准
- •Participants with Spinal-Bulbar Muscular Atrophy
- •Known diagnosis of HIV/AIDS, Hepatitis B, or Hepatitis C.
结局指标
主要结局
ALS Functional Rating Scale-Revised (ALSFRS-R)
时间窗: once every 3 months for one year
12 questions about patient's ability to function in certain activities of daily living. Each question is out of 4 with 4 being normal and 0 being completely impaired.
ALS Cognitive Behavioral Scale (ALS-CBS)
时间窗: once every 3 months for one year
short measure of cognition and behavior in patients with ALS. The cognitive portion consists of 8 tasks with a perfect score being 20. The behavioral portion measures changes in personality and behavior since the onset of ALS symptoms as well as mood, pseudobulbar affect and fatigue and is completed by a family member or caregiver. A normal score is 45.
Slow Vital Capacity (SVC)
时间窗: once every 3 months for one year
measurement of the maximum amount of air that can be exhaled following a deep breath.
Strength Testing with Hand Held Dynamometer (HHD)
时间窗: once every 3 months for one year
muscle strength testing performed on upper and lower limbs, ankles, wrists and fingers using a small hand held device. These measurements are followed over time and compared to measure decline.
次要结局
未报告次要终点