A Phase I/II, Open-Label Study to Assess the Safety and Efficacy of Striatum Transplantation of Human Embryonic Stem Cells-derived Neural Precursor Cells in Patients With Parkinson's Disease
试验速览
- 阶段
- 1 期
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination
研究概览
简要总结
This study will evaluate the safety and efficacy of intracerebral transplantation of human embryonic stem cells-derived neural precursor cells in patients with Parkinson's Disease.
详细描述
This study is a Phase I/II, open-label, non randomized clinical trial. The study will enroll 50 patients for cell injection, administering a single dose of neural precursor cells by stereotaxic intra-striatal injection.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 50 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Primary Parkinson's disease patients，a history over 5 years，females or males;
- •Cannot effectively control the PD or tolerate the side effects of drugs;
- •Hoehn and Yahr Stage 3 or 4 in the off state at screening
- •Age between 50 and 80 years;
- •Dopamine is effective or once;
- •Sign the informed consent
排除标准
- •Atypical Parkinsonian syndrome or only having tremor syndrome;
- •Having been done pallidotomy, DBS, striatum or extrapyramidal surgery;
- •Subjects are using apomorphine or anticoagulant;
- •Subjects used immunosuppressant or antipsychotic drugs in last 3 months;
- •Subjects used botulinum toxin, phenol, or other drugs for the treatment of dystonia or muscle cramps in last 6 months;
- •During the period of active epilepsy preventing epilepsy with antiepileptic;
- •Coagulant function abnormality or other obviously abnormal laboratory test results;
- •Having skin basal cell carcinoma or cervical cancer and other pre-cancerous lesions;
- •Subject has a history of chronic alcohol or drug abuse ;
- •Pregnancy or lactation;
- •Subjects participated in other clinical trials in recent 3 months;
- •Subject is considered as dementia, a serious mental disorder (depression or mania), and personality or behavioral disorders through cognitive and behavioral test;
- •Cannot cooperate on the research;
- •Severe brain atrophy, or existing brain injury such as cerebral infarction, cerebral vascular malformation or trauma;
- •Severe systemic diseases;
- •Severe dyskinesia or frequent "OFF" or "ON" states
- •Severe infectious diseases (eg HIV, HCV, HBV, syphilis positive)
- •Not suitable to participate in this clinical trial assessed by other physicians
研究组 & 干预措施
NPC transplantation
The patients will receive Levodopa combined with a regular neural precursor cell (NPC) transplantation
干预措施: NPC transplantation (Biological)
NPC transplantation
The patients will receive Levodopa combined with a regular neural precursor cell (NPC) transplantation
干预措施: Levodopa (Drug)
HLA-matched NPC transplantation
The patients will receive Levodopa combined with a HLA-matched neural precursor cell (NPC) transplantation
干预措施: NPC transplantation (Biological)
HLA-matched NPC transplantation
The patients will receive Levodopa combined with a HLA-matched neural precursor cell (NPC) transplantation
干预措施: Levodopa (Drug)
HLA-non-matched NPC transplantation
The patients will receive Levodopa combined with a HLA-non-matched neural precursor cell (NPC) transplantation
干预措施: NPC transplantation (Biological)
HLA-non-matched NPC transplantation
The patients will receive Levodopa combined with a HLA-non-matched neural precursor cell (NPC) transplantation
干预措施: Levodopa (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination
时间窗: 6 months
Number of subjects with adverse events such as the evidence of graft failure or rejection
次要结局
- Change in Unified Parkinson's Disease Rating Scale (UPDRS) score from baseline(Baseline and 12 months)
- Change in DATscan from baseline(Baseline and 12 months)
- Change in Hoehn and Yahr Stage from baseline(Baseline and 12 months)
研究者
Qi Zhou
Deputy director of Institute of zoology, Chinese academy of sciences, and vice president of medical school， University of Chinese academy of sciences
Chinese Academy of Sciences