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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.