A two-stage design for randomized trials with binary outcomes

Sam Wieand; Terry Therneau

doi:10.1016/0197-2456(87)90022-5

A two-stage design for randomized trials with binary outcomes

Sam Wieand, Terry Therneau

Quantitative Health Sciences

Research output: Contribution to journal › Article › peer-review

22 Scopus citations

Abstract

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 language	English (US)
Pages (from-to)	20-28
Number of pages	9
Journal	Controlled Clinical Trials
Volume	8
Issue number	1
DOIs	https://doi.org/10.1016/0197-2456(87)90022-5
State	Published - Mar 1987

Keywords

arcsin statistic
binomial response
stopping rule

ASJC Scopus subject areas

Pharmacology

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10.1016/0197-2456(87)90022-5

Cite this

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abstract = "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.",

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note = "Funding Information: We would like to thank Miss Rebecca Pierce and Mr. Stephan Cha for assistance in programming and Mrs. Deb Strauss for the typing. We would also like to thank Dr. Susan Ellenberg for several useful suggestions and calling our attention to some articles addressing the fact that different sample sizes are required for the Fisher exact test than for the unconditionalt ests. The authors were supported in part by the Mayo Comprehensive Cancer Center Grant, CA-15083, NCI, DHHS.",

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KW - stopping rule

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A two-stage design for randomized trials with binary outcomes

Abstract

Keywords

ASJC Scopus subject areas

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