Intravenous Administration of Mesenchymal Stem Cells (IV-MSC) for the Treatment of Cerebral Adrenoleukodystrophy (cALD)
试验速览
- 阶段
- 1 期
- 状态
- 暂停
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
研究概览
简要总结
This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will occur in the time between diagnosis of active CALD and transplant which is currently 8-12 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •age ≥ 3 years
- •diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation
- •evidence of active cerebral disease as determined by the presence of gadolinium enhancement
- •ALD MRI (Loes) score ≥ 1
- •Patients who have not received prior gene therapy or transplant
- •Life expectancy of > 6 months as determined by the enrolling researcher
- •Have adequate organ function confirmed by laboratory values obtained within 28 days prior to enrollment
排除标准
- •Inability to undergo sedation or MRI studies for any reason
- •Other concurrent life-threatening disease (life expectancy <6 months) or eligible for hospice care
- •Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.
研究组 & 干预措施
IV-MSC for cALD (Early Disease/Bridge Therapy)
Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.
干预措施: Mesenchymal stem cells (IV-MSC) (Biological)
IV-MSC for cALD (Advanced Disease)
Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.
干预措施: Mesenchymal stem cells (IV-MSC) (Biological)
结局指标
主要结局
Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
时间窗: 6 months
Safety and tolerability of cerebral adrenoleukodystrophy (cALD) patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC) assessed by incidence of adverse events.
次要结局
- Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).(6 months)