title: Designs for randomized phase II clinical trials with two treatment arms.
abstract: 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.
authors: Department of Preventive Medicine, Stony Brook University Medical Center, Stony Brook, NY 11794-8036 USA. wei.hou@stonybrookmedicine.edu
authors: Wei
authors: W
authors: Hou
authors: Myron N
authors: MN
authors: Chang
authors: Sin-Ho
authors: SH
authors: Jung
authors: Yang
authors: Y
authors: Li
mesh_terms: D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D015982:Bias; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D023381:Endpoint Determination; D006801:Humans; D000077269:Lenalidomide; D008228:Lymphoma, Non-Hodgkin / Q000188:drug therapy*; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012008:Recurrence; D012107:Research Design; D000069283:Rituximab; D013792:Thalidomide / Q000031:analogs & derivatives / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D000970:Antineoplastic Agents; D000069283:Rituximab; D013792:Thalidomide; D000077269:Lenalidomide
keywords: average sample size; minimizing the maximum sample size; power; single-stage design; threshold; two-stage design; type I error rate