The Clinical Study on the Use of Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Nerve functional evaluation
研究概览
简要总结
Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intrathecal injection of umbilical cord mesenchymal stem cells can secret trophic factors that keep the motorneurons functional. The investigators have designed a phase I/II clinical trial to check the feasibility of this approach in humans.
详细描述
A total of 30 diagnosed ALS patients. The patients would got these symptoms such as gait difficulty and tremor, hand incoordination or speech difficulties.
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
- 20 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnose established following the World Federation of Neurology criteria
- •More than 6 and less than 36 months of evolution of the disease
- •Medullar onset of the disease
- •More than 20 and less than 65 years old
- •Forced Vital Capacity equal or superior to 50%
- •Total time of oxygen saturation <90% inferior to 2% of the sleeping time
- •Signed informed consent
排除标准
- •Neurological or psychiatric concomitant disease
- •Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube
- •Concomitant systemic disease
- •Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months
- •Inclusion in other clinical trials
- •Unability to understand the informed consent
结局指标
主要结局
Nerve functional evaluation
时间窗: within one week before,1month,6months,12months and 24months after transplantation
1. Medical Research Council scale：To determine the strength of bilateral little finger abductor muscle and anterior tibial. 2. To evaluate the situation of upper motor neuron impairment through the Ellis reflex scale 3. The severity evaluation: NorrisALS score and ALS functional rating scale, ALSFRS 4. Speed of disease progression by the following formula：Progress rate = (40-ALSFRS score) / course 5. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI) and memory (Mini Mental State Examination)
Forced vital capacity
时间窗: within one week before,1month,6months,12months and 24months after transplantation
vital capacity(VC）、forced vital capacity( FVC）、forced expiratory volune(FEV1）、FEV1/FVC、maximal voluntary ventilation(MVV）、peak expiratory flow(PEF）
次要结局
- Blood test(within one week before,1month,6months,12months and 24months after transplantation)
- Urinal test(within one week before,1month,6months,12months and 24months after transplantation)
- Electrophysiology examination(within one week before,1month,6months,12months and 24months after transplantation)