ABSTRACT
This chapter describes study designs that are commonly used in phase II trials in oncology and discusses when each of these might be relevant. It focuses on design considerations for these studies. The chapter explores design considerations for single-arm versus randomized trials, various biomarker-based designs; and design considerations for immunotherapy trials. A single-arm trial is designed using a historical control for comparison—all patients receive the same intervention and results are generally compared to a historical control. A simple two-arm study includes one arm that receives the intervention of interest and a separate “control” arm that receives the control intervention. In the randomized phase II setting, one may also wish to consider randomized screening designs, which involve the non-definitive comparison of one or more experimental regimes against the standard of care. In umbrella trials, patients with a specific disease/histology are first screened for a set of targeted biomarkers and divided into biomarker-defined subpopulations.
