Phase 1 Study To Assess the Safety and Tolerability of Human Embryonic Stem Cell-Derived Midbrain Dopamine Neuron Cell Therapy (MSK-DA01) For Advanced Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- Safety and Tolerability
研究概览
简要总结
This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and to monitor for potential side effects.
详细描述
Subjects will undergo surgical transplantation of the dopamine-producing cells under general anesthesia into a part of the brain called the putamen. Subjects then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of cell survival (using MRI and PET scans of the brain), and effect on Parkinson's disease symptoms are assessed for 2 years post-transplant.
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
- 50 Years 至 78 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 50-78 years old (Canada)
- •Age 60-78 years old (United States)
- •Diagnosis of Parkinson's Disease made between 3 to 20 years ago
- •Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia
- •Able to participate in all study visits and evaluations, including brain MRI and PET scan
- •Existence of a study partner who may act as potential surrogate over long term for ongoing consent
排除标准
- •Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis or other neurodegenerative diseases such as Alzheimer's disease
- •Prior Deep Brain Stimulation , lesion therapy, or gene therapy for PD
- •Prior surgical or radiation therapy to the brain or spinal cord
- •Any medical condition resulting in high risk of immunosuppressive drugs, including any active infectious disease
- •Inability to temporarily stop anti-platelet agents or other anti-coagulant medications without serious risk
- •Previous or currently active malignant disease within the past 5 years, except basal cell carcinoma or in situ uterine cervical carcinoma that have been treated
- •Severe obesity (>350 lbs) or any condition that prevents use of PET/MRI
- •Pregnancy or breastfeeding
- •Contraindication to surgery or general anesthesia
- •In the opinion of the investigator, any other condition regarded as making subject unsuitable for trial
研究组 & 干预措施
MSK-DA01
干预措施: MSK-DA01 (Biological)
MSK-DA01
干预措施: MSK-DA01 Cell Delivery Device (Device)
结局指标
主要结局
Safety and Tolerability
时间窗: Baseline to 1 Year Post-Transplant
The incidence of Serious Adverse Events (SAEs) at 1 year post-transplant. or abnormal tissue overgrowth related to presence of transplanted cells;
次要结局
- Changes in Waking Hours in "Off" State(Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant)
- Continued Safety and Tolerability(Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant)
- Evidence of Cell Survival(Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant)
- Changes in Motor Function(Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant)