Statistical analysis for two-stage seamless design with different study endpoints.

Published

Journal Article

In the pharmaceutical industry, it is desirable to apply an adaptive seamless trial design to combine two separate clinical studies that are normally conducted for achieving separate objectives such as a Phase II study for dose finding and a Phase III confirmatory study for efficacy. As a result, an adaptive seamless Phase II/III trial design consisting of two phases, namely a learning phase and a confirmatory phase, is commonly considered in pharmaceutical development. In some cases, however, the study endpoints for the two separate studies may be different due to long treatment duration. In this case, test statistics for the final analysis based on the combined data are necessary developed. In this paper, a test statistic utilizing data collected from both phases is proposed assuming that there is a well established relationship between the two different study endpoints. Formula for sample size calculation based on the proposed test statistic is derived. Sample size allocation at the two phases is also discussed.

Full Text

Duke Authors

Cited Authors

  • Chow, S-C; Lu, Q; Tse, S-K

Published Date

  • 2007

Published In

Volume / Issue

  • 17 / 6

Start / End Page

  • 1163 - 1176

PubMed ID

  • 18027223

Pubmed Central ID

  • 18027223

International Standard Serial Number (ISSN)

  • 1054-3406

Digital Object Identifier (DOI)

  • 10.1080/10543400701645249

Language

  • eng

Conference Location

  • England