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Activity Number:
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418
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Type:
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Contributed
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Date/Time:
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Wednesday, August 1, 2007 : 10:30 AM to 12:20 PM
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Sponsor:
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Biopharmaceutical Section
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| Abstract - #309356 |
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Title:
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A Hybridized Two-Stage Design in an Ulcerative Colitis Pivotal Trial
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Author(s):
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Guohui Liu*+ and Mingxiu Hu
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Companies:
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Millennium Pharmaceuticals, Inc. and Millennium Pharmaceuticals, Inc.
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Address:
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40 Landsdowne Street, Cambridge, MA, 02139,
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Keywords:
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two-stage design ; type I error ; bias ; power
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Abstract:
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Ulcerative colitis (UC) is an inflammatory disease. A novel humanized antibody is under development as a treatment for active UC patients. This paper presents a hybridized two-stage design for a pivotal study to prove the efficacy of the antibody in the induction of remission of UC and to establish the optimal dose. Stage 1 serves as the confirmatory study for the overall population combining anti-TNF naïve and refractory populations, and also as the learning stage for anti-TNF refractory population. Stage 2 proceeds as the confirmatory study for the anti-TNF refractory population only if Stage 1 demonstrates a treatment effect for the subpopulation that passes a pre-specified threshold. Sample size for Stage 2 is based on Stage 1 results on the subpopulation. A simulation study has been done to explore the characteristics of this design including overall type I error, bias, and power.
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