Generalized optimal design for two-arm, randomized phase II clinical trials with endpoints from the exponential dispersion family

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• 作者：Wei Jiang ; Jonathan D. Mahnken ; Jianghua He and Matthew S. Mayo
• 刊名：Pharmaceutical Statistics
• 出版年：2016
• 出版时间：November/December 2016
• 年：2016
• 卷：15
• 期：6
• 页码：459-470
• 全文大小：396K
• ISSN：1539-1612

文摘

For two-arm randomized phase II clinical trials, previous literature proposed an optimal design that minimizes the total sample sizes subject to multiple constraints on the standard errors of the estimated event rates and their difference. The original design is limited to trials with dichotomous endpoints. This paper extends the original approach to be applicable to phase II clinical trials with endpoints from the exponential dispersion family distributions. The proposed optimal design minimizes the total sample sizes needed to provide estimates of population means of both arms and their difference with pre-specified precision. Its applications on data from specific distribution families are discussed under multiple design considerations. Copyright