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One- and two-stage design proposals for a phase II trial comparing three active treatments with control using an ordered categorical endpoint.

Whitehead, John and Jaki, Thomas (2009) One- and two-stage design proposals for a phase II trial comparing three active treatments with control using an ordered categorical endpoint. Statistics in Medicine, 28 (5). pp. 828-847.

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Abstract

Phase II 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.

Item Type: Article
Journal or Publication Title: Statistics in Medicine
Uncontrolled Keywords: interim analysis ; one-stage design ; ordinal data ; phase II trial ; two-stage design
Subjects: Q Science > QA Mathematics
Departments: Faculty of Science and Technology > Mathematics and Statistics
ID Code: 26752
Deposited By: Dr Thomas Jaki
Deposited On: 13 Jul 2009 09:27
Refereed?: Yes
Published?: Published
Last Modified: 09 Oct 2013 14:49
Identification Number:
URI: http://eprints.lancs.ac.uk/id/eprint/26752

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