肌缩侧面硬化症中的Reldesemtiv:来自COURAGE-ALS随机临床试验的结果
Jeremy M Shefner1,2, Merit E Cudkowicz3, Angela Genge4
1Department of Neurology, Barrow Neurological Institute, University of Arizona, Phoenix.
JAMA neurology
|March 24, 2025
概括
雷尔德塞莫维没有减缓肌缩侧面硬化症 (ALS) 患者的功能衰退. 第三阶段的COURAGE-ALS试验因徒劳而提前停止,与安慰剂相比没有显著的益处.
科学领域:
- 神经科学是一个神经科学.
- 临床试验 临床试验
- 药物开发 药物开发
背景情况:
- 肌缩侧面硬化症 (ALS) 是一种进展性神经退行性疾病,治疗选择有限.
- 以前的研究表明,reldesemtiv可能会减缓ALS的进展,需要进一步调查.
研究的目的:
- 为了评估与安慰剂相比,reldesemtiv在ALS患者中的疗效和安全性.
- 评估reldesemtiv对ALS患者功能结果的影响.
主要方法:
- COURAGE-ALS试验是一个第三阶段,双盲,安慰剂控制,随机临床试验.
- 参与者接受了口服reldesemtiv (300毫克每天两次) 或安慰剂24周.
- 主要终点是从基线到第24周,ALS功能评级量表修订 (ALSFRS-R) 评分的变化.
主要成果:
- 在24周的临时分析后,试验因徒劳而提前终止.
- 在reldesemtiv和安慰剂组之间ALSFRS-R得分的变化没有显著差异 (P=.04,有利于安慰剂).
- 综合评估也未能证明对reldesemtiv的治疗益处.
结论:
- 雷尔德塞莫维在ALS患者的功能衰退减缓方面没有表现出有效性.
- 这些发现表明,根据这项试验,reldesemtiv不是一种有效的ALS治疗方法.
- 对ALS的新型治疗策略进行进一步的研究是有必要的.
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