Stereotactic Intracerebral Injection of Allogenic Induced Pluripotent Stem Cell-derived Dopamine Progenitor Cells in Patients With Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Incidence and servility of Treatment-Emergent Adverse Events
研究概览
简要总结
Parkinson's disease is a progressive neurodegenerative disorder characterized by high morbidity due to the limited regenerative capacity of dopaminergic neurons in the brain. Current drug treatments primarily manage symptoms but do not halt or reverse neuronal loss. Cellular replacement therapy has emerged as a potential strategy to restore dopaminergic function and address the underlying neuronal deficits. This study aims to evaluate the safety, feasibility, and efficacy of transplanting dopaminergic neurons into the brain to improve motor function and quality of life in patients with advanced Parkinson's disease.
详细描述
Patients with Parkinson's disease will be treated with allogenic induced pluripotent stem cell-derived dopamine progenitor cells (iPSC-DAPs). These cells will be transplanted directly into the striatum to restore dopamine-producing capacity. Patients will be evaluated at 1, 3, 6, 9 and 12 months after transplantation for safety, feasibility, and efficacy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 55 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Able to sign informed consent and comply with the study protocol
- •55-75 years of age, at the time of signing informed consent
- •Diagnosed to be Parkinson's disease patients over 5 years
- •Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia
- •At least 3 hours accumulative "off" time per day
- •Hoehn and Yahr Stage 3 - 4 in the off state at screening
- •Dopamine drug responsiveness demonstrated by a positive "on/off" test with at least a 30% improvement on UPDRS III (motor) scale
排除标准
- •Patients with the following concomitant conditions or disorders: Epilepsy；Multiple sclerosis；Unable to give consent due to dementia；Atypical Parkinsonism；Genetic Parkinson's disease；Suicidal ideation associated with intent or plan in the past 12 months；History of psychosis；History of subarachnoid hemorrhage；History of stroke or transient ischemic attack
- •Patient with unstable vital sign at screening and/or prior to the surgery
- •Estimated Glomerular Filtration Rate (eGFR) < 60 ml/min/1.73m2
- •Liver dysfunction, as evidenced by enzymes (AST and ALT) greater than three times the ULN.
- •Hematologic abnormality: hemoglobin <10 mg/dL or platelet count < 100,000/mL
- •International normalized ratio (INR) ≥ 1.3 not due to a reversible cause
- •Patients with autoimmune disorders
- •Patients with HIV and/or active HBV or HCV
- •Patients who are unable to undergo MRI and PET/CT
- •Patients with an expected life expectancy of <1 year
- •Patients who have had active malignancies
- •Patients currently receive levodopa-carbidopa intestinal gel or apomorphine treatment
- •Patients who have history of pallidotomy or thalamotomy or deep brain stimulation (DBS) surgery
- •Received cell or gene therapy (autologous or allogeneic) within the previous 12 months
- •Participation in an investigational therapeutic or device trial within 30 days of consent
- •Women who are pregnant or breast-feeding
- •Other conditions that researchers consider not suitable to participate in this study
研究组 & 干预措施
Low Dose ALC01 therapy
Stereotactic Intracerebral Injection of iPSC-DAPs into the putamen on each side of the brain
干预措施: ALC01 therapy (Biological)
High Dose ALC01 therapy
Stereotactic Intracerebral Injection of iPSC-DAPs into the putamen on each side of the brain
干预措施: ALC01 therapy (Biological)
结局指标
主要结局
Incidence and servility of Treatment-Emergent Adverse Events
时间窗: From baseline to 12 months post surgery
Incidence of adverse event (AE), serious adverse event(SAE) is defined as the composite of number and severity of adverse events, regardless of causality, clinical laboratory abnormalities, clinical meaningful changes from baseline
次要结局
- Changes in motor function(From baseline to 12 months post surgery)
- Changes in the Hoehn and Yahr scale(From baseline to 12 months post surgery)
- Changes in quality of life (QoL)(From baseline to 12 months post surgery)
- Change in PD medication usage(From baseline to 12 months post surgery)
- 18-F DAT PET uptake(Baseline, 6 and 12 months post surgery)