Mayo Matthew S, Mahnken Jonathan D, Soong Seng-Jaw
Department of Biostatistics, University of Kansas Medical Center, Kansas City, Kansas 66160, USA.
J Biopharm Stat. 2010 Jan;20(1):106-24. doi: 10.1080/10543400903280597.
Multi-stage Phase II trials are often employed in practice but may not be the best approach when the endpoint of interest is not obtained soon after enrollment and/or when a control arm is desired. We present a new design in which sample size determination includes a control arm and allows for the estimation of response for each treatment as well as estimation of the difference in the response rates. We evaluate this design under varying allocation schemes to treatment arms and response rates for each treatment.
多阶段II期试验在实践中经常被采用，但当入组后不久未获得感兴趣的终点且/或需要一个对照臂时，可能不是最佳方法。我们提出了一种新设计，其中样本量的确定包括一个对照臂，并允许估计每种治疗的反应以及反应率的差异估计。我们在不同的治疗组分配方案和每种治疗的反应率下评估这种设计。
