Optimal two-stage designs for phase II clinical trials.

Controlled Clinical Trials
R Simon

Abstract

The primary objective of a phase II clinical trial of a new drug or regimen is to determine whether it has sufficient biological activity against the disease under study to warrant more extensive development. Such trials are often conducted in a multi-institution setting where designs of more than two stages are difficult to manage. This paper presents two-stage designs that are optimal in the sense that the expected sample size is minimized if the regimen has low activity subject to constraints upon the size of the type 1 and type 2 errors. Two-stage designs which minimize the maximum sample size are also determined. Optimum and "minimax" designs for a range of design parameters are tabulated. These designs can also be used for pilot studies of new regimens where toxicity is the endpoint of interest.

Associated Clinical Trials

Jan 24, 2013·Sanjiv Harpavat

References

Mar 1, 1986·Controlled Clinical Trials·M N Chang, P C O'Brien
Jan 1, 1982·Experientia. Supplementum·K McPherson

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