Journal
STATISTICS IN MEDICINE
Volume 28, Issue 5, Pages 828-847Publisher
WILEY
DOI: 10.1002/sim.3508
Keywords
interim analysis; one-stage design; ordinal data; phase II trial; two-stage design
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Funding
- MRC [G0800792] Funding Source: UKRI
- Medical Research Council [G0800792] Funding Source: researchfish
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Phase If clinical trials are performed to investigate whether a novel treatment shows sufficient promise of efficacy to justify its evaluation in a subsequent definitive phase III trial, and they are often also used to select the dose to take forward. In this paper we discuss different design proposals for a phase II trial in which three active treatment doses and a placebo control are to be compared in terms of a single-ordered categorical endpoint. The sample size requirements for one-stage and two-stage designs are derived, based on an approach similar to that of Dunnett. Detailed computations are prepared for an illustrative example concerning a study in stroke. Allowance for early stopping for futility is made. Simulations are used to verify that the specified type I error and power requirements are valid, despite certain approximations used in the derivation of sample size. The advantages and disadvantages of the different designs are discussed, and the scope for extending the approach to different forms of endpoint is considered. Copyright (C) 2008 John Wiley & Sons, Ltd.
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