A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 21
- 试验地点
- 4
- 主要终点
- The safety and tolerability of SNUG01 administered by intrathecal.
研究概览
简要总结
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).
详细描述
This is a 52-week, multicenter, open-label, single-arm, dose escalation and expansion Phase Ⅰ/Ⅱa study of SNUG01 in adults with ALS. Safety will be the primary focus, with secondary emphasis on immunogenicity, PK and preliminary clinical efficacy of SNUG01.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
3 Ascending Dose Levels, Single Injection by Intrathecal(IT)
干预措施: SNUG01 (Drug)
结局指标
主要结局
The safety and tolerability of SNUG01 administered by intrathecal.
时间窗: up to 1 year after administration
Dose-limiting toxicity (DLT), adverse events (AEs), clinically significant changes in vital signs, physical examination, clinical laboratory tests (hematology, biochemistry, urinalysis, coagulation, cardiac enzymes, etc.) and 12-lead ECG, etc.
To recommend the optimal expansion dose of SNUG01 that demonstrates acceptable safety with maximum preliminary efficacy administered by IT.
时间窗: up to 1 years
次要结局
- Immunogenicity of SNUG01(up to 1 years)
- PK of SNUG01 (Biodistribution and Viral Shedding)(up to 1 years)
- Preliminary Clinical Efficacy of SNUG01(up to 1 years)