NCT04680065进行中（未招募）1 期
Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 9
- 试验地点
- 7
- 主要终点
- The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
研究概览
简要总结
The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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 35-75 years of age (inclusive)
- •Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and > 30 years of age
- •Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years
- •Stable anti-parkinsonian medication regimen
- •Ability to walk a distance of 25 feet with or without an assistive device
排除标准
- •Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases
- •Presence of dementia, psychosis, substance abuse or poorly controlled depression
- •Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities
- •History of cancer or poorly controlled medical conditions that would increase surgical risk
- •Received investigational agent within 12 weeks
- •Inability to tolerate laying flat in an MRI and/or allergy to gadolinium
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Active Treatment
Experimental
干预措施: AAV2-GDNF gene therapy (Biological)
Placebo Surgery
Sham Comparator
干预措施: Sham (Placebo) Surgery (Procedure)
结局指标
主要结局
The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
时间窗: 3 years
Number of TEAE and SAE's reported post-treatment.
次要结局
- MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)(12 months)
- Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)(12 months)
- Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane(12 months)
研究者
研究点 (7)
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