The Phase II/III transition toward the proof of efficacy in cancer clinical trials

Melissa Fazzari, Glenn Heller, Howard I. Scher

Research output: Contribution to journalArticlepeer-review

36 Scopus citations


Few phase III investigations show a benefit for an experimental treatment when compared to a standard therapy or placebo. This illustrates the need for more reliable estimates of treatment effects from the phase II investigations used to design the more definitive phase III trials. In this manuscript, we examine four aspects of phase II clinical trial designs: (1) selecting endpoints; (2) defining the patient population for evaluation; (3) determining a level of activity that would justify a phase III trial; and (4) estimating sample sizes. In each area, problems with the conventional approaches are discussed and alternatives for the successful transition of phase II results to a phase III setting are suggested. An application of the design for patients with androgen-independent prostate cancer is illustrated. Control Clin Trials 2000;21:360-368 Copyright (C) 2000 Elsevier Science Inc.

Original languageEnglish (US)
Pages (from-to)360-368
Number of pages9
JournalControlled Clinical Trials
Issue number4
StatePublished - Aug 2000
Externally publishedYes


  • Historical data
  • Patient population
  • Phase II
  • Sample size
  • Surrogate endpoint

ASJC Scopus subject areas

  • Pharmacology

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