Phase II trials are an important component of drug development in oncology. Their objective is to screen for preliminary signals of efficacy of new anticancer agents and to provide further information on drug toxicity. Many different phase II trial designs have been envisioned and utilized according to cancer behavior, type of drug effects on tumors, study endpoints, and treatment settings. The trials are generally classified as either single- or multiple-arm non-randomized trials or randomized phase II trials. Within each group, there are numerous different designs, with the most common ones being single-arm phase II trials, biomarker-driven trials, randomized “pick the winner” trials, randomized controlled phase II studies, randomized discontinuation trials, and crossover studies. The aim of this chapter is to provide an overview of the different designs of phase II cancer trials and to discuss the advantages and disadvantages of each design.
Phase II clinical trials Randomized clinical trials Oncology Cancer
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