Safety and Efficacy of Autologous Induced Neural Stem Cell-derived Dopaminergic Precursor Cells in the Treatment of Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Assessment of changes during the medication "off" time in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part III, in comparison with baseline values.
研究概览
简要总结
This is a phase I, interventional, single arm, open-label, clinical study to evaluate the safety and efficacy of the striatal transplantation of autologous induced neural stem cell-derived DA precursor cells in Parkinson's Disease patients.
详细描述
Parkinson's Disease (PD) is the second most common neurodegenerative disease, caused by progressive depletion of midbrain dopaminergic neurons in the substantia nigra pars compacta. This clinical study will include the preparation of dopaminergic neural precursor cells derived from neural stem cells through reprogramming of patient's peripheral blood mononuclear cells (PBMCs), and transplantation of the obtained cells into the brains of PD patients by stereotaxic injection. Safety, tolerability, evidence of cell survival (using PET scan), and the efficacy on PD symptoms will be assessed at different time points up to 12 months post-transplantation.
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
- 30 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages between 30 and 85 years, males or females; Diagnosed to be Parkinson's disease patients according to MDS Parkinson's disease diagnostic criteria; Disease history over 3 years; Hoehn and Yahr Stage less than or equal to 4 during the medication "on" time; Responsive to levodopa treatment (Maximum rate of improvement in MDS-UPDRS, part 3, is over 30%).
排除标准
- •Atypical Parkinsonian syndrome or secondary Parkinsonian syndrome; Accompanied with other central nervous system diseases; With other severe systemic diseases or dysfunction; With severe psychiatric disorders; Subjects are using hormone or cytotoxic drugs and cannot stop taking the drug during the trial; With cognitive disorders (MMSE<24); With severe dyskinesia (MDS-UPDRS part 4, score in 4.1/4.2 ≥ 2); Subjects have undergone previous brain surgery; Subjects are long-term user of anticoagulant; Subjects have intracranial lesions which may affect the surgery or follow-up studies as assessed by imaging; Subjects are unable to undergo MRI or AV133 PET examination; Pregnancy or in preparation for pregnancy; Not suitable to participate in this clinical trial as assessed by the study investigators/physicians.
研究组 & 干预措施
Autologous induced neural stem cell-derived DA precursor cells
The patients will receive transplantation of autologous induced neural stem cell-derived DA precursor cells.
干预措施: Autologous induced neural stem cell-derived DA precursor cells (Drug)
结局指标
主要结局
Assessment of changes during the medication "off" time in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part III, in comparison with baseline values.
时间窗: Within 12 months post-transplantation
Minimum score: 0; Maximum score: 132; Higher scores mean a worse outcome.
Incidence and severity of transplant-related adverse events.
时间窗: Within 12 months post-transplantation
次要结局
- Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part I, in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part II, in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part IV, in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes during the medication "off" time in motor function by using Purdue Pegboard test and gait analyzer, in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in the daily total medication "off" time in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in Unified Dyskinesia Rating Scale in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in daily levodopa-equivalent dose in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in Parkinson's Disease Questionnaire (PDQ-39) in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in AV133 PET imaging in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes in International Parkinson and Movement Disorder Society - Non-Motor Rating Scale (NMSS), in comparison with baseline values.(Within 12 months post-transplantation)
- Assessment of changes from baseline in total daily OFF time (or ratio of OFF time to daytime waking hours) at 12 months post-surgery.(Within 12 months post-transplantation)