Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01, a Human Embryonic Stem Cell-Derived Midbrain Dopaminergic Neuronal Cell Therapy
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- Total SAEs and total number of subjects with at least 1 SAE
研究概览
简要总结
This is a multicenter study to evaluate the safety and clinical outcomes of BRT-DA01 in subjects with PD who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101.No investigational therapy will be administered in this study.
详细描述
All subjects who received BRT-DA01 transplantation in Study MSK-DA01-101 (ie, parent study) will be asked to enroll in Study BRT-DA01-501. Subjects enrolled in this study are to be monitored for safety and clinical outcomes through 5 years post-BRT-DA01 transplantation. For individual participating subjects, the Baseline visit for the BRT-DA01-501 study will be defined as the last observation recorded in the parent study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Subject received BRT-DA01 in Study MSK-DA01-101 (ie, parent study) and will have completed or discontinued the parent study
- •Subject must be willing and able to provide written informed consent for the BRT-DA0-501 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures
排除标准
- •No exclusion criteria
结局指标
主要结局
Total SAEs and total number of subjects with at least 1 SAE
时间窗: Baseline through 5 years post-transplantation
Total SAEs and total number of subjects with at least 1 SAE related to cellular drug product
次要结局
- Change in the Unified Dyskinesia Rating Scale (UDysRS)(Baseline through 5 years post-transplantation)
- Change in striatal 18F-DOPA uptake using positron emission tomography (PET)(Baseline through 5 years post-transplantation)
- Change in the International Parkinson and Movement Disorder Society (MDS)-Unified Parkinson's Disease Rating Scale (UPDRS)(Baseline through 5 years post-transplantation)
- Change in levodopa equivalent daily dose (LEDD)(Baseline through 5 years post-transplantation)
- Change in Hauser PD diary measurements(Baseline through 5 years post-transplantation)
- Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index(Baseline through 5 years post-transplantation)