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Study on reseach design and statistical inferences for exploratory clinical researches

Research Project

Project/Area Number 23500349
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Statistical science
Research InstitutionOsaka University

Principal Investigator

UESAKA Hiroyuki  大阪大学, 臨床医工学融合研究教育センター, 招聘教授 (60446250)

Co-Investigator(Renkei-kenkyūsha) KADA Akiko  独立行政法人国立病院機構, 名古屋医療センター臨床研究センター, 室長 (70399608)
Research Collaborator MORIKAWA Toshihiko  元久留米大学, バイオ統計センター
Project Period (FY) 2011 – 2013
Project Status Completed (Fiscal Year 2013)
Budget Amount *help
¥5,200,000 (Direct Cost: ¥4,000,000、Indirect Cost: ¥1,200,000)
Fiscal Year 2013: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2012: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2011: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000)
Keywords臨床試験デザイン / sample size reestimation / adaptive design / multiple comparison / gatekeeping procedure / group sequential design / gate keeping procedure / 臨床研究デザイン
Research Abstract

We developed two-phase and three-stage designs for a situation where information on sample size estimation was very scarce. In these designs sample size is re-estimated after the first phase. The second phase consists of two stages and one interim analysis is performed. Sample size is not re-estimated at this phase. Theses designs were proved to give about 85 to 95 percent of the power of the single stage design and much less average sample sizes than that design.
We also investigated multiple testing problems. When we wish to test a family of several elementary hypotheses which are structured according to the importance and logical relationships among them. We obtained explicit formulae of adjusted p-values of each test of hypothesis. Furthermore, we defined multiple parallel/fixed sequence hypothesis structure, which consists of several ordered treatments and equally important multiple endpoints. We also developed explicit formulae for the adjusted p-values of elementary hypotheses.

Report

(4 results)
  • 2013 Annual Research Report   Final Research Report ( PDF )
  • 2012 Research-status Report
  • 2011 Research-status Report
  • Research Products

    (15 results)

All 2013 2012 2011

All Presentation (15 results)

  • [Presentation] 2相3段階適応型試験デザインのデザインパラメータの選択2013

    • Author(s)
      上坂浩之, 森川敏彦, 嘉田晃子
    • Organizer
      2013年度 統計関連学会連合大会
    • Place of Presentation
      大阪大学
    • Related Report
      2013 Final Research Report
  • [Presentation] Multiple Testing on Multiple/Parallel Fixed Sequence Hypothesis Structures2013

    • Author(s)
      Morikawa, T. and Uesaka, H
    • Organizer
      34th Annual Conference of the International Society for Clinical Biostatistics
    • Place of Presentation
      Ludwig- Maximilians-University Munich, Germany
    • Related Report
      2013 Final Research Report
  • [Presentation] Standardization of a gate keeping procedures2013

    • Author(s)
      Morikawa, T. and Uesaka, H
    • Organizer
      26th International Biometrics Conference
    • Place of Presentation
      Kobe
    • Related Report
      2013 Final Research Report
  • [Presentation] 2相3段階適応型試験デザインのデザインパラメータの選択2013

    • Author(s)
      上坂浩之、嘉田晃子、森川敏彦
    • Organizer
      統計関連学会連合大会
    • Place of Presentation
      大阪大学
    • Related Report
      2013 Annual Research Report
  • [Presentation] 被験者数再推定を伴う群増量デザインの検討2013

    • Author(s)
      嘉田晃子、上坂浩之、森川敏彦
    • Organizer
      統計関連学会連合大会
    • Place of Presentation
      大阪大学
    • Related Report
      2013 Annual Research Report
  • [Presentation] Multiple Testing on Multiple/Parallel Fixed Sequence Hypothesis Structures2013

    • Author(s)
      Morikawa, T. and Uesaka, H.
    • Organizer
      34th Annual Conference of the International Society for Clinical Biostatistics
    • Place of Presentation
      Ludwig-Maximilians- University Munich, Germany
    • Related Report
      2013 Annual Research Report
  • [Presentation] Some considerations on use of two-phase three-stage adaptive designs2013

    • Author(s)
      Uesaka, T., Kada, A., and Morikawa, T.
    • Organizer
      34th Annual Conference of the International Society for Clinical Biostatistics.
    • Place of Presentation
      Ludwig-Maximilians- University Munich, Germany
    • Related Report
      2013 Annual Research Report
  • [Presentation] ゲートキーピング法における調整p値の陽表現2013

    • Author(s)
      森川敏彦、上坂浩之
    • Organizer
      日本軽量生物学会念会
    • Place of Presentation
      福島市飯坂温泉観光会館
    • Related Report
      2013 Annual Research Report
  • [Presentation] Some considerations on use of two-phase three-stage adaptive designs2012

    • Author(s)
      Uesaka, H., Morikawa, T., and kada, A
    • Organizer
      34th Annual Conference of the International Society for Clinical Biostatistics
    • Place of Presentation
      Ludwig-Maximilians- University Munich, Germany
    • Related Report
      2013 Final Research Report
  • [Presentation] Comparisons of three stage adaptive designs for a clinical trial2012

    • Author(s)
      Uesaka, H., Morikawa, T., and kada, A
    • Organizer
      26th International Biometrics Conference
    • Place of Presentation
      Kobe
    • Related Report
      2013 Final Research Report 2012 Research-status Report
  • [Presentation] Standardization of a gate keeping procedures2012

    • Author(s)
      Morikawa, T. and Uesaka, H.
    • Organizer
      26th International Biometrics Conference
    • Place of Presentation
      Kobe
    • Related Report
      2012 Research-status Report
  • [Presentation] 既存情報が極めて乏しい状況における探索-検証型試験のデザインと被験者数設定2012

    • Author(s)
      上坂浩之、森川敏彦、嘉田晃子
    • Organizer
      2012年度統計関連学会連合大会
    • Place of Presentation
      北海道大学
    • Related Report
      2012 Research-status Report
  • [Presentation] Some evaluations of hybrid adaptive designs for an exploratory-confirmatory trial2012

    • Author(s)
      Hiroyuki Uesaka, Toshihiko Morikawa, Akiko Kada
    • Organizer
      East Asia Regional Biometric Conference 2012
    • Place of Presentation
      Seoul Seoul National Univ.
    • Related Report
      2011 Research-status Report
  • [Presentation] 臨床試験における解析対象集団再考2011

    • Author(s)
      上坂浩之
    • Organizer
      日本計量生物学会
    • Place of Presentation
      大阪大学
    • Related Report
      2011 Research-status Report
  • [Presentation] 医師主導型の臨床試験のデザイン上の問題-初期探索研究を中心に-.  7階 大会議室 2011年9月30日2011

    • Author(s)
      上坂浩之
    • Organizer
      久留米大学バイオ統計フォーラム
    • Place of Presentation
      福岡市 アクロス福岡
    • Related Report
      2011 Research-status Report

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Published: 2011-08-05   Modified: 2019-07-29  

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