多发性硬化症临床试验失败的决定因素:来自ClinicalTrials.gov的见解
Alejandro Rivero-de-Aguilar1, Mónica Pérez-Ríos1, Joseph S Ross2
1University of Santiago de Compostela.
Research square
|September 26, 2025
概括
大约四分之一的多发性硬化症 (MS) 临床试验失败,通常是由于招聘或商业决策的低水平. 增加研究地点和改善招募可能会提高多发性硬化试验的成功率.
科学领域:
- 神经学 神经学
- 临床试验 临床试验
- 药学研究 药学研究
背景情况:
- 临床试验失败,与安全性或疗效无关,浪费资源并带来风险.
- 多发性硬化症 (MS) 药物开发面临的重大挑战是试验耗尽.
研究的目的:
- 分析III期和IV期多发性硬化症药物临床试验中的失败率和相关因素.
- 了解MS研究中临床试验终止背后的主要原因.
主要方法:
- 在ClinicalTrials.gov搜索了从2008年到2024年进行的多发性硬化症药物试验 (III-IV期).
- 根据招募和报告的原因,将试验归类为正常结束或失败,并与出版物和PI进行验证.
- 采用卡普兰-梅尔和多变量后勤回归来评估试验结束的时间,并确定失败预测因素.
主要成果:
- 在分析的282个多发性硬化症临床试验中,25.2%的试验失败,失败的试验平均早10个月结束.
- 关键的失败原因包括低招聘率 (28.2%),未披露的业务决策 (26.8%) 和后勤问题 (12.7%).
- 专注于药物安全性的试验 (OR 0.35) 和具有≥50个中心 (OR 0.10) 的试验显示出较低的失败率.
结论:
- 四分之一的多发性硬化症临床试验未能成功完成.
- 增加研究中心数量和增强患者招募的战略对于提高多发性硬化试验成功率至关重要.
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