在阿尔茨海默病的第二阶段试验的风景
Alina Lee1, Di Shan1, David Castle2,3
1Centre for Addiction and Mental Health, Toronto, Ontario, Canada.
Journal of Alzheimer's disease : JAD
|October 16, 2023
概括
很少有阿尔茨海默病 (AD) 试验使用适应性设计,尽管它们有可能加速药物开发. 这一审查强调了在AD研究中创新的试验设计的低采用.
科学领域:
- 临床试验 临床试验
- 神经科学是一个神经科学.
- 药物开发 药物开发
背景情况:
- 阿尔茨海默病 (AD) 药物开发面临着高失败率的重大挑战.
- 新的临床试验设计,包括适应性设计,为提高效率提供了潜在的解决方案.
研究的目的:
- 在阿尔茨海默氏病II期试验中评估设计特征.
- 分析试验设计中的时间趋势.
- 在各类赞助商之间比较设计差异.
主要方法:
- 从ClinicalTrials.gov (1996-2021) 中对I/II,II和II/III期AD试验进行系统审查.
- 描述性统计和回归模型用于评估时间趋势和赞助商类型差异.
- 包括药物或生物干预.
主要成果:
- 确定了474项试验;随机并行组设计是最常见的 (72%).
- 只有2.5% (12项试验) 使用了适应性设计 (例如,早期停止,适应性剂量发现).
- 非随机和开放标签设计随着时间的推移而增加;适应性设计没有时间趋势.
- 行业赞助的试验倾向于随机设计和更大的样本大小.
结论:
- 在II期AD试验中采用适应性试验设计的比例仍然很低.
- 实施创新设计对于加速AD药物开发至关重要.
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