A Sequential Dose-Escalation, Randomized, Active-Controlled, Multi-Center, Phase 1/2a Clinical Trial to Evaluate the Safety, Tolerability and Efficacy of SNR1611 in Patients With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- Genuv Inc.
- 入组人数
- 23
- 试验地点
- 5
- 主要终点
- Safety and tolerability of SNR1611: adverse events
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability and efficacy of trametinib (SNR1611) in the treatment of amyotrophic lateral sclerosis.
详细描述
Trametinib (SNR1611) is a MEK inhibitor that downregulates the MAPK/ERK pathway. In this study, the potential of MAPK/ERK pathway downregulation through trametinib (SNR1611) as a therapeutic treatment for amyotrophic lateral sclerosis (ALS) will be evaluated.
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
- Single (Outcomes Assessor)
盲法说明
K-ALSFRS-R score will be measured by independent outcome assessors.
入排标准
- 年龄范围
- 19 Years 至 75 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
Trametinib (0.5 mg)
One tablet of trametinib 0.5 mg per day
干预措施: Trametinib (0.5 mg) (Drug)
Trametinib (1 mg)
Two tablets of trametinib 0.5 mg per day
干预措施: Trametinib (1 mg) (Drug)
Riluzole (100 mg)
One tablet of riluzole 50 mg taken twice per day
干预措施: Riluzole (100 mg) (Drug)
结局指标
主要结局
Safety and tolerability of SNR1611: adverse events
时间窗: 24-week (24-week extension and additional 48-week are optional)
Observation of adverse events
次要结局
- K-ALSFRS-R score(24-week (24-week extension and additional 48-week are optional))
- FVC(24-week (24-week extension and additional 48-week are optional))
- Plasma trough concentrations of SNR1611(24-week (24-week extension and additional 48-week are optional))
- Milestone(Additional 48-week (optional))
- CSF trough concentrations of SNR1611(24-week (24-week extension and additional 48-week are optional))