Group sequential design (GSD) is widely used in clinical trials in which correlated tests of multiple hypotheses are used. Multiple primary objectives resulting in tests with known correlations include evaluating 1) multiple experimental treatment arms, 2) multiple populations, 3) the combination of multiple arms and multiple populations, or 4) any asymptotically multivariate normal tests. In this paper, we focus on the first 3 of these and extend the framework of the weighted parametric multiple test procedure from fixed designs with a single analysis per objective to a GSD setting where different objectives may be assessed at the same or different times, each in a group sequential fashion. Pragmatic methods for design and analysis of weighted parametric group sequential design(WPGSD) under closed testing procedures are proposed to maintain the strong control of familywise Type I error rate (FWER) when correlations between tests are incorporated. This results in the ability to relax testing bounds compared to designs not fully adjusting for known correlations, increasing power or allowing decreased sample size. We illustrate the proposed methods using clinical trial examples and conduct a simulation study to evaluate the operating characteristics.
翻译:在临床试验中广泛使用小组顺序设计(GSD),在临床试验中采用多个假设的相关测试。多重主要目标,在已知关联性测试中,包括评估:(1) 多重实验处理武器,(2) 多重组群,(3) 多重武器与多重组群的组合,或(4) 任何非现式的多变正常测试。在本文中,我们侧重于其中的前3个,并将加权参数多重测试程序的框架从固定设计中从每个目标单项分析扩大到一个GSD设置,在这种设置中,不同目标可以在同一或不同时间,每个小组连续评估。提议在封闭测试程序下,设计和分析加权参数组序列设计(WPGSD)的实用方法,以便在将测试结合起来时保持对自家的I型误差率(FWER)的严格控制。这导致在设计上放松测试界限的能力,而不是对已知的相关性进行充分调整,增强能力或允许降低样本大小。我们用临床试验实例说明拟议的方法,并进行模拟研究,以评价操作特征。