Operating characteristics of a Simon two-stage phase II clinical trial design incorporating continuous toxicity monitoring.
Phase II clinical trials are usually designed to measure efficacy, but safety is also an important end point. Previous authors recommended a method to monitor toxic events after each patient is enrolled, which is also known as continuously monitoring the toxicity. In this work, we investigate combining the usual Simon two-stage design to monitor response with the continuous toxicity monitoring methodology. Theoretical justification is given for the nominal size, probability of early termination, and average sample size under the null hypothesis of the combined testing procedure. A series of simulations are performed to investigate the performance of the combined procedure.
Department of Bioinformatics and Biostatistics, School of Public Health and Information Sciences, University of Louisville, Louisville, KY, USA. hray8@kennesaw.edu
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D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D003198:Computer Simulation; D016903:Drug Monitoring / Q000379:methods*; D064420:Drug-Related Side Effects and Adverse Reactions; D057239:Early Termination of Clinical Trials; D023381:Endpoint Determination; D006801:Humans; D012107:Research Design; D018401:Sample Size