Phase 1B/2A Study of the Safety and Tolerability of Human Neural Stem Cells for Huntington's Disease
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 6 and 12 weeks after dosing.
研究概览
简要总结
The purpose of this research study is to determine whether an implantation of hNSC-01 is a safe and tolerable study intervention for Huntington's disease. This study is the first time that hNSC-01 is being tested in people.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Please note this may not be a complete list of eligibility criteria.
- •Inclusion Criteria:
- •Have decision-making capacity and be able to provide written informed consent.
- •Are between 18 to 65 years of age
- •Have genetically confirmed diagnosis of Huntington's Disease
- •Able to undergo MRI scans, tolerate neurosurgical procedure, blood draws and lumbar puncture.
排除标准
- •Are pregnant
- •Test positive for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B at Screening
- •Have an implanted deep brain stimulation device.
研究组 & 干预措施
Cohort A
Unilateral, low dose
干预措施: hNSC-01 (Biological)
Cohort B
Bilateral, low dose
干预措施: hNSC-01 (Biological)
Cohort C
Bilateral, mid-dose
干预措施: hNSC-01 (Biological)
Cohort D
Bilateral, high dose
干预措施: hNSC-01 (Biological)
结局指标
主要结局
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 6 and 12 weeks after dosing.
时间窗: Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion
To determine the safety and tolerability of hNSC-01s implanted into the striatum of individuals affected with HD.
次要结局
- Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 12 months after dosing.(12 months)
- Change in Total Motor Score (TMS)(12 months)
- Change in Functional Imaging: FDG-PET(12 months)
- Change in structural MRI(12 months)
- Change in NfL in CSF.(12 months)
- Change in NfL in plasma.(12 months)
- Change in PENK levels in CSF.(12 months)
- Change in cognition using the MMSE.(12 months)
- Change in cognition using Stroop Word Reading Test.(12 months)
- Change in Total Functional Capacity (TFC)(12 months)
- Change in Problem Behaviors Assessment-short version(12 months)
研究者
Leslie Thompson
Bren Professor, Departments of Psychiatry and Human Behavior, Neurobiology and Behavior, and Biological Chemistry, Co-Director, UCI Sue & Bill Gross Research Center
University of California, Irvine