A generalized Dunnett test for multi-arm multi-stage clinical studies with treatment selection

• 成果类型: Article
• 署名作者: Magirr, D.; Jaki, T.; Whitehead, J.
• 署名单位: Lancaster University
• 刊物名称: BIOMETRIKA
• ISSN/ISSBN: 0006-3444
• DOI: 10.1093/biomet/ass002
• 发表日期: 2012
• 页码: 494501
• 关键词: trials; DESIGN; implementation

摘要

We generalize the Dunnett test to derive efficacy and futility boundaries for a flexible multi-arm multi-stage clinical trial for a normally distributed endpoint with known variance. We show that the boundaries control the familywise error rate in the strong sense. The method is applicable for any number of treatment arms, number of stages and number of patients per treatment per stage. It can be used for a wide variety of boundary types or rules derived from alpha-spending functions. Additionally, we show how sample size can be computed under a least favourable configuration power requirement and derive formulae for expected sample sizes.