A Phase I, Open Label, Single Arm, Dose Escalation and Dose Expansion Clinical Trial to Evaluate the Safety, Tolerability and Efficacy of Human Induced Pluripotent Stem Cell Derived Dopaminergic Progenitor Cells (NCR201) Injection in the Treatment of Subjects With Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 48
- 试验地点
- 1
- 主要终点
- Incidence and severity of adverse events.
研究概览
简要总结
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy that NCR201 has on Parkinson's disease (PD) patients.
详细描述
Parkinson's disease (PD) is a common neurodegenerative disease in the middle-aged and elderly. It is the "third killer" of the middle-aged and elderly after tumors and cardiovascular and cerebrovascular diseases. Its main clinical manifestations are resting tremor, reduced voluntary movement, muscle rigidity, postural reflex impairment, and autonomic dysfunction, which seriously affect patients' work ability and quality of life. It is estimated that nearly 100,000 people in China become new Parkinson's patients every year. Experts from the World Health Organization predict that the number of Parkinson's patients in China will reach 5 million in 2030, which will be more than half of the world's total. As the disease progresses, the symptoms of Parkinson's patients will become increasingly severe. The high prevalence and high disability rate of Parkinson's disease bring heavy burdens to individuals, families, and society.
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
- 40 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages between 40 and 75 years;
- •Diagnosed to be Parkinson's disease according to Parkinson's disease diagnostic criteria;
- •Disease history over 5 years;
- •Stable dose of dopamine treatment;
- •Able to undergo PET/CT/MRI detection;
排除标准
- •Patients who have previously undergone brain surgery;
- •Past use of stem cell therapy or participation in stem cell clinical research;
- •Cognitive impairment;
- •History of mental disorders;
- •Patients with other serious systemic diseases;
- •Past or current metastatic malignant tumors.
研究组 & 干预措施
Low Dose
MRI-guided bilateral stereotactic cell implantation
干预措施: Allogeneic dopaminergic neural precursor cell(NCR201) (Drug)
High Dose
MRI-guided bilateral stereotactic cell implantation
干预措施: Allogeneic dopaminergic neural precursor cell(NCR201) (Drug)
结局指标
主要结局
Incidence and severity of adverse events.
时间窗: Within 24 weeks post-transplantation
Safety and tolerability
次要结局
- Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part III, in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Hamilton Anxiety Scale (HAMA)-14 in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Parkinson's Disease Questionnaire-39 (PDQ-39) in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Hoehn & Yahr scale in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part I,II,IV, in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Hamilton Depression Scale (HAMD)-17 in comparison with baseline values.(Within 24 months post-transplantation)
- Bilateral putamen standardized uptake value as demonstrated on positron emission tomography(PET) compared with baseline in the 'off' state.(Within 24 months post-transplantation)
- Patient L-dopa equivalent dose compared with baseline.(Within 24 months post-transplantation)
- Incidence and severity of adverse events.(Within 24 months post-transplantation)