NCT01238315撤回1 期
A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis
适应症
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 试验地点
- 2
- 主要终点
- number of participants with adverse events.
研究概览
简要总结
The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.
详细描述
Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.
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
- 6 Months 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 6 months to 6 years
- •Male or female
- •Clinical diagnosis of Infantile neuronal ceroid lipofuscinosis or late infantile neuronal ceroid lipofuscinosis
- •CLN1 or CLN2 mutation
排除标准
- •Previously received an organ, tissue or bone marrow transplantation
- •Previously participated in any gene or cell therapy study
- •Infection with hepatitis virus, cytomegalovirus, Epstein-Barr virus, or Human Immunodeficiency Virus (HIV)
- •Current or prior cancer
- •Bleeding disorder
- •Unable to have an MRI scan
结局指标
主要结局
number of participants with adverse events.
时间窗: one year following transplantation
Safety will be measured by number of adverse events and changes in neurological status.
次要结局
- Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS(one year following transplantation)
研究者
研究点 (2)
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