NCT01005004已完成1 期
Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Safety assessment through clinical neurological and MRI evaluation.
研究概览
简要总结
The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).
详细描述
Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 5 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Confirmed clinical diagnosis of connatal PMD
- •Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
- •MRI consistent with PMD as interpreted by a qualified neuroradiologist
排除标准
- •Other significant congenital brain abnormality not related to PMD
- •Previous participation in gene transfer or cell transplant trial
- •Presence of neurological signs and symptoms not consistent with PMD
- •Current or prior malignancy
- •Prior organ, tissue or bone marrow transplant
结局指标
主要结局
Safety assessment through clinical neurological and MRI evaluation.
时间窗: one year post transplant
次要结局
- MRI examination for post-transplant myelination(one year post transplant)
研究者
研究点 (2)
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