A Randomized, Double-Blind, Sham-Controlled Study to Evaluate the Safety and Tolerability of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 12
- 主要终点
- Incidence of Adverse Events Related to the Treatment
研究概览
简要总结
The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's Disease.
详细描述
The planned length of participation in the study for each participant was approximately 7 months, including a screening period of up to 40 days, randomization, surgery, and a follow-up period of 26 weeks.
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
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 25 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed Parkinson's Disease
- •Levodopa responsiveness for at least 12 months
- •UPDRS Part 3 score of ≥25 points in the "off" state
排除标准
- •History of brain surgery to treat Parkinson's Disease
- •Any history of cerebral insult or central nervous system infection
- •Atypical Parkinson's Disease
- •Focal or lateralized neurologic deficits
- •Evidence of significant medical or psychiatric disorders
- •Cognitive impairment as defined by the Montreal Cognitive Assessment (MoCA) ≤ 20
- •Beck Depression Inventory-II score of ≥ 20
结局指标
主要结局
Incidence of Adverse Events Related to the Treatment
时间窗: Baseline to Week 26
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.
Incidence of Serious Adverse Events Related to the Treatment
时间窗: Baseline to Week 26
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.
次要结局
未报告次要终点