One-sample multiple testing procedure for phase II clinical trials.

Abstract

Commonly, the central objective of Phase II clinical trials is the assessment of the antitumor 'therapeutic efficacy' of a specific treatment regimen. It is of interest to formulate test procedures which can be employed in these trials to decide whether or not this therapeutic efficacy warrants further investigation. For ethical reasons, these procedures should allow for early termination if initial results are extreme. In this paper, a one-sample multiple testing procedure is proposed which employs the standard single-stage test procedure at the last test, and which both allows for early termination and essentially preserves the size, power and simplicity of the single-stage procedure.