A three-outcome design for phase II clinical trials

Daniel J. Sargent, Victor Chan, Richard M. Goldberg

Research output: Contribution to journalArticle

56 Scopus citations

Abstract

The goal of a phase II trial is to make a preliminary determination regarding the activity and tolerability of a new treatment and thus to determine whether the treatment warrants further study in the phase III setting. Phase II clinical trials are typically designed in the hypothesis testing framework with two possible outcomes, either reject the null hypothesis H0 or reject the alternative hypothesis Ha, based on the observed activity level. However, in cases where the observed activity is "borderline," the decision regarding the future of the agent is not as clear as the prespecified hypothesis test would indicate. In this paper we propose an alternative design that allows for three outcomes: reject H0, reject Ha, or reject neither. We describe the theoretical properties of this design and illustrate it with several examples. We focus on the clinical implications of the three-outcome design.

Original languageEnglish (US)
Pages (from-to)117-125
Number of pages9
JournalControlled Clinical Trials
Volume22
Issue number2
DOIs
StatePublished - Apr 23 2001

Keywords

  • Minimax design
  • Optimal design
  • Sequential testing

ASJC Scopus subject areas

  • Pharmacology

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