临床试验的方法质量在肌缩侧面硬化症:一个系统的审查
Elisabetta Pupillo1, Ammar Al-Chalabi2,3,4, Serena Sassi1
1Department of Neuroscience, Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Research Center for ALS, Milan, Italy.
Journal of neuromuscular diseases
|May 17, 2024
概括
尽管对肌缩侧面硬化症 (ALS) 进行了大量的临床试验,但尚未出现有效的治疗方法. 本次审查强调了试验方法的显著变化和问题,表明需要改进未来ALS研究的研究设计和报告.
科学领域:
- 神经学 神经学
- 临床试验方法论 临床试验方法论
- 药物开发 药物开发
背景情况:
- 在全球范围内,已经进行了200多项关于肌缩侧面硬化症 (ALS) 的临床试验.
- 没有任何治疗方法在减缓ALS疾病进展方面表现出显著的有效性.
研究的目的:
- 系统评估到2022年12月31日公布的ALS临床试验的方法质量.
- 确定在未来ALS研究的设计和执行方面需要改进的关键领域.
主要方法:
- 按照"系统性审查和元分析首选报告项目" (PRISMA) 准则进行了系统性审查.
- 213个ALS临床试验被纳入分析.
主要成果:
- 在各个试验中,方法质量差异很大.
- 在报告临床前数据,研究设计 (例如,平行臂与交叉),随机化,盲目化,参与者纳入标准 (呼吸能力和疾病持续时间) 和学率方面发现了不一致.
- 很大一部分试验缺乏有关呼吸功能和基线疾病持续时间等关键参数的详细报告.
结论:
- 对于ALS临床试验的方法质量是高度可变的.
- 未来的ALS试验需要标准化的临床前毒理学和I期研究.
- 需要改善研究设计,为II-III期研究分配资源,以及透明的结果报告,以推进ALS治疗的发展.
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