A two-stage design for randomized trials with binary outcomes

Sam Wieand, Terry Therneau

Research output: Contribution to journalArticlepeer-review

22 Scopus citations


A two-stage rule for a randomized clinical trial with a dichotomous outcome is presented. It is based on a rule discussed by Ellenberg and Eisenberger (Cancer Treat Rep 69:1147-1154, 1985). The rule is shown to lead to a reduction in expected sample size if the test treatment is ineffective, with almost no loss in power. All the mathematical formulas required to design such a rule are given, as well as some time-saving approximations. The accuracy of the approximations is shown.

Original languageEnglish (US)
Pages (from-to)20-28
Number of pages9
JournalControlled Clinical Trials
Issue number1
StatePublished - Mar 1987


  • arcsin statistic
  • binomial response
  • stopping rule

ASJC Scopus subject areas

  • Pharmacology


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