Hou Wei, Chang Myron N, Jung Sin-Ho, Li Yang
Department of Preventive Medicine, Stony Brook University Medical Center, Stony Brook, NY 11794-8036 USA.
Stat Med. 2013 Nov 10;32(25):4367-79. doi: 10.1002/sim.5829. Epub 2013 Apr 29.
The most common primary statistical end point of a phase II clinical trial is the categorization of a patient as either a 'responder' or 'nonresponder'. The primary objective of typical randomized phase II anticancer clinical trials is to evaluate experimental treatments that potentially will increase response rate over a historical baseline and select one to consider for further study. We propose single-stage and two-stage designs for randomized phase II clinical trials, precisely defining various type I error rates and powers to achieve this objective. We develop a program to compute these error rates and powers exactly, and we provide many design examples to satisfy pre-fixed requirements on error rates and powers. Finally, we apply our method to a randomized phase II trial in patients with relapsed non-Hodgkin's disease.
II期临床试验最常见的主要统计终点是将患者分类为“反应者”或“无反应者”。典型的随机II期抗癌临床试验的主要目标是评估可能比历史基线提高反应率的实验性治疗方法，并选择一种进行进一步研究。我们提出了随机II期临床试验的单阶段和两阶段设计，精确地定义了实现这一目标的各种I型错误率和检验效能。我们开发了一个程序来精确计算这些错误率和检验效能，并提供了许多设计示例，以满足对错误率和检验效能的预先设定要求。最后，我们将我们的方法应用于复发非霍奇金淋巴瘤患者的随机II期试验。
