Long-Term Follow-Up Safety and Preliminary Efficacy Study of Human Central Nervous System Stem Cell (HuCNS-SC®) Transplantation in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.
研究概览
简要总结
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
详细描述
Only subjects who underwent HuCNS-SC transplantation under Protocol CL-N01-PMD will be enrolled in this long term follow-up study.
Subjects will return to the site six months and one year after completion of the Phase I study and then annually for a total study duration of four years. Phone calls will also be made by the Investigator to the subject's parent/legal guardian bi-annually to conduct a phone visit through the four-year duration of the study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects who received HuCNS-SC cells under Protocol CL-N01-PMD
排除标准
- •Subjects who received off-protocol immunosuppressive medications.
- •Subjects who are concurrently enrolled in another investigational study.
结局指标
主要结局
Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.
时间窗: 4 years
次要结局
- Preliminary efficacy using Bayley-III and Callier-Azusa Scale.(4 years)
- Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).(4 years)