一项为期40周的2B期随机,多中心,双盲,安慰剂控制的研究,评估了美曼丁在肌缩侧面硬化症的安全性和有效性
Salman Bhai1,2, Todd Levine3, Dan Moore4
1University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Muscle & nerve
|November 8, 2024
概括
孟坦丁在临床试验中没有减缓肌缩侧面硬化症 (ALS) 的进展,也没有改善认知症状. 远程招生策略可能会提高未来的ALS临床试验参与率.
科学领域:
- 神经科学是一个神经科学.
- 临床神经学 临床神经学
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,无法治愈,治疗选择有限.
- 对于ALS的疾病修饰疗法有显著的需求.
研究的目的:
- 调查孟坦丁在减缓ALS进展方面的疗效.
- 评估孟坦因对ALS患者认知和行为变化的影响.
主要方法:
- 一个随机,双盲,安慰剂对照试验,涉及89名ALS患者,随机分配给记忆剂或安慰剂36周.
- 使用ALSFRS-R测量疾病进展;还进行了包括NfL在内的生物标志物分析和神经精神病学测试.
- 这项研究利用了13个美国地点的面对面和远程注册.
主要成果:
- 与安慰剂相比,孟坦丁治疗没有导致疾病进展的显著差异 (ALSFRS-R).
- 在记忆剂和安慰剂组之间没有观察到生物标志物变化 (例如,NfL) 或神经精神病结局的显著差异.
- 基线NfL水平与ALSFRS-R下降率存在相关性.
结论:
- 在这项试验中,孟坦因对ALS疾病进展或相关的神经精神症状没有显著影响.
- 该研究表明,远程注册方法可能会提高未来ALS临床试验的参与率和成功率.
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