混合控制的临床试验使用并发登记在肌缩侧面硬化症:一个可行性研究
Ruben P A van Eijk1,2, Leonard H van den Berg2, Kit C B Roes3
1Department of Biomedical Data Science and Centre for Innovative Study Design, School of Medicine, Stanford University, Stanford, California, USA.
Clinical pharmacology and therapeutics
|July 8, 2023
概括
使用并发注册表的混合临床试验设计提高了效率. 增加随机对照与外部数据可以减少患者数量和试验持续时间的肌缩性侧面硬化症 (ALS) 治疗.
科学领域:
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
- 神经退行性疾病 神经退行性疾病
背景情况:
- 混合试验设计将随机分组与外部控制相结合.
- 同时的患者登记册提供了高质量的数据,用于扩大临床试验.
- 肌缩侧面硬化症 (ALS) 药物开发在试验效率方面面临挑战.
研究的目的:
- 利用来自并发注册的患者级数据来增强临床试验.
- 评估外部控制对治疗效果估计,准确性和试验持续时间的影响.
- 为了说明在肌缩性侧面硬化症 (ALS) 试验设计中的应用.
主要方法:
- 利用一个明确的,基于人群的注册表与随机的,安慰剂对照试验并行运行.
- 识别并匹配与试验参与者同时非参与,符合条件的注册表患者.
- 将外部控制整合到统计分析中,以评估其影响.
主要成果:
- 从非参与注册患者中确定了一个匹配的外部对照群体.
- 增加外部对照可以使随机化减少12.8% (17名患者).
- 研究时间可以缩短25.0% (从30.1到22.6个月).
结论:
- 使用并发注册表和严格匹配的混合设计可以加速新的治疗开发.
- 仔细匹配可以最大限度地减少日历时间和护理标准差异的偏差.
- 这种方法提高了临床试验的效率,减少了不必要的随机化.
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