Department of Biostatistics, University at Buffalo, 3435 Main Street, Buffalo, NY 14214, USA.
Contemp Clin Trials. 2012 Mar;33(2):332-41. doi: 10.1016/j.cct.2011.10.008. Epub 2011 Oct 30.
Features common to phase II clinical trials include limited knowledge of the experimental treatment being evaluated, design components reflecting ethical considerations, and small to moderate sample sizes as a result of resource constraints. It is for these reasons that there exist many two-stage designs proposed in the literature for use in this context. The majority of these designs are for binary endpoints and based on exact probability calculations, or are for continuous endpoints and rooted in asymptotic approximations to the null distribution. We present exact two-stage Mann-Whitney designs in the context of two-arm randomized clinical trials. In addition to describing the designs, we present tables of decision rules under a variety of assumed realities for use in trial planning.
对所评估的实验性治疗方法了解有限、设计成分反映伦理考虑、以及由于资源限制导致样本量小到中等。正是由于这些原因，文献中提出了许多用于这种情况下的两阶段设计。这些设计大多数是针对二分类结局的，基于精确的概率计算，或者是针对连续结局的，基于对零分布的渐近逼近。我们在双臂随机临床试验的背景下提出了精确的两阶段 Mann-Whitney 设计。除了描述设计外，我们还根据各种假设的现实情况列出了决策规则表，以供试验计划使用。
