A Single Patient Treatment Protocol for Autologous Mesenchymal Stem Cell Intraspinal Therapy in Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 1
- 试验地点
- 2
- 主要终点
- Safety
研究概览
简要总结
The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.
详细描述
A single patient safety study for harvesting, expanding ex vivo and injecting autologous mesenchymal stem cells (MSC's) into the subarachnoid space of a patient with amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue by subcutaneous biopsy and expanded using an FDA-approved protocol. They will then be injected by lumbar puncture into the cerebrospinal fluid. Injection will be completed in the in- patient clinical research unit (CRU). the patient will be followed for two years.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •age greater than 18 years, if female, must be menopausal or had hysterectomy
- •resident and citizen of the United States
- •history of a chronic onset of a progressive motor weakness
- •able to comply with protocol requirements
- •can provide written consent
排除标准
- •does not have renal disease (Creatine > 2.0)
- •does not have active systemic disease
- •does not have any clinically significant abnormalities on prestudy laboratory evaluation
- •does not have any clinically significant medical condition (e.g.,within 6 months of baseline, had a myocardial infarct, angina pectoris, and/or congestive heart failure), that in the opinion of the investigator, would compromise the safety of the patient
- •does not have a history of cancer including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion,or re-occurrence within 3 years of baseline).
- •has not used an investigational drug within 30 days of baseline visit
- •does not have a tracheostomy
- •does not have a Beck's Depression Inventory score >16
结局指标
主要结局
Safety
时间窗: 2 year follow-up
Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.
次要结局
- Neurologic disability score(Two year follow-up)