在骨髓缩侧面硬化症中使用普里多:HEALEY ALS平台试验
, Jeremy M Shefner1, Björn Oskarsson2
1Barrow Neurological Institute, Phoenix, Arizona.
JAMA
|March 11, 2025
概括
作为西格玛- 1受体激动剂的多平在24周内没有减缓肌缩侧面硬化症 (ALS) 患者的疾病进展. 这项研究没有发现 pridopidine 和安慰剂组之间的功能或生存结果有显著差异.
科学领域:
- 神经科学
- 药理学
- 临床试验
背景情况:
- 肌缩侧面硬化 (ALS) 是一种进展性神经退行性疾病,治疗选择有限.
- 西格玛-1 (σ1) 受体是ALS中神经保护的潜在治疗点.
研究的目的:
- 在ALS患者中评估多平素的疗效和安全性,1受体激动剂.
- 评估 pridopidine 对功能状态 (ALSFRS-R) 和生存的影响.
主要方法:
- 进行了2/ 3期多中心随机双盲,安慰剂控制平台试验 (HEALEY ALS).
- 163名患有ALS的参与者被随机分配给24周的口服普里多匹丁 (45毫克每天两次) 或安慰剂.
- 主要结局是使用贝叶斯共享参数模型,分析功能 (ALSFRS- R) 和生存率的变化.
主要成果:
- 普里多平没有显著改变ALS疾病进展的初级终点 (DRR,0. 99;DRR的概率<1. 0.55).
- 在 pridopidine 和安慰剂组之间没有观察到 ALSFRS- R 评分或存活率的显著差异.
- 最常见的不良反应是跌倒和肌肉衰弱,两组的发病率相似.
结论:
- 在这项为期24周的研究中,普里多匹丁在减缓疾病进展或改善ALS患者的结果方面没有显著的益处.
- 可能需要进一步的研究来探索 σ1受体调节在ALS中的作用,可能与不同的药物或患者群体有关.
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