NCT04167540进行中（未招募）1 期
Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 11
- 试验地点
- 3
- 主要终点
- The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations
研究概览
简要总结
The objective of this Phase 1b investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a recent or a long-standing diagnosis of PD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 35 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female adults 35-75 years of age (inclusive)
- •Diagnosed with Parkinson's disease
- •Modified Hoehn and Yahr stage I-III OFF medication
- •Time since receiving a clinical diagnosis of PD and disease severity consistent with one of the following:
- •EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate UPDRS III OFF score
- •OR: At least 4 years since clinical diagnosis of PD and moderate to severe UPDRS III OFF score
- •Responsiveness to levodopa
排除标准
- •Atypical parkinsonism
- •Severe dyskinesia
- •Presence of dementia, psychosis, substance abuse or qualify as "severe depression"
- •Prior brain surgery (i.e. deep brain stimulator or DBS implantation) or other brain imaging abnormalities
- •Receiving an investigational drug
- •History of cancer or poorly controlled medical conditions that would increase surgical risk
- •Inability to tolerate laying flat in an MRI or allergy to gadolinium
研究组 & 干预措施
Earlier stage PD
Experimental
干预措施: AAV2-GDNF (Biological)
Later stage PD
Experimental
干预措施: AAV2-GDNF (Biological)
结局指标
主要结局
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations
时间窗: 5 years
Evaluation of the safety and tolerability through the assessment of incidence of TEAE, identified by MedDRA preferred term and grouped by MedDRA System Organ Class, as well as clinically meaningful changes in clinical exams or laboratory assays.
次要结局
- Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)(18 months)
- Brain dopaminergic cell integrity as measured by DaTscan(18 months)
- Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)(18 months)
研究者
研究点 (3)
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