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Development trial design in rare disease using registry data

Research Project

Project/Area Number 15K00067
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Statistical science
Research InstitutionNational Hospital Organization Nagoya Medical Center

Principal Investigator

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), その他部局等, 室長 (70399608)

Co-Investigator(Kenkyū-buntansha) 平川 晃弘  東京大学, 大学院医学系研究科(医学部), 特任准教授 (90609330)
Research Collaborator SAITO Akiko  
Project Period (FY) 2015-04-01 – 2019-03-31
Project Status Completed (Fiscal Year 2018)
Budget Amount *help
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2017: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Keywords疾患登録 / レジストリー / コホート研究 / 介入研究 / バイアス / 疾患レジストリー
Outline of Final Research Achievements

In this study, we considered the study design to establish treatment methods effectively using data of disease registry in the following three cases: (1) clinical trial within disease registry, (2) cohort study within disease registry, and (3) cohort study with prognostic survey within disease registry. Then we set external control groups and summarized the design and notes in the two clinical studies: (1) a clinical trial on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type II, and (2) a clinical trial on T-cell-replete haploidentical stem cell transplantation using low-dose antithymocyte globulin for relapsed and refractory pediatric acute leukemia.
In addition, we reviewed and summarized the master protocol trial focusing on oncology as a new clinical trial design.

Academic Significance and Societal Importance of the Research Achievements

希少疾患領域など対象者数が少ない疾患においては、治療法の開発が進みにくい現状がある。すでに疾患レジストリーが構築されている疾患群もある。疾患レジストリーを利用した効率的な臨床研究の設定ができれば、治療法開発の進展に寄与するものと考える。

Report

(5 results)
  • 2018 Annual Research Report   Final Research Report ( PDF )
  • 2017 Research-status Report
  • 2016 Research-status Report
  • 2015 Research-status Report
  • Research Products

    (10 results)

All 2019 2018 2017 2016 2015

All Journal Article (4 results) (of which Peer Reviewed: 2 results,  Open Access: 1 results) Presentation (6 results) (of which Int'l Joint Research: 2 results,  Invited: 2 results)

  • [Journal Article] A single-arm open-label clinical trial on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type II: a study protocol2019

    • Author(s)
      Akiko Kada, Jun Tohyama, Hideaki Shiraishi, Yukitoshi Takahashi, Eiji Nakagawa, Tomoyuki Akiyama, Akiko M Saito, Yushi Inoue, Mitsuhiro Kato
    • Journal Title

      The Kurume Medical Journal

      Volume: 印刷中

    • NAID

      130008066539

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Master protocol trials in oncology: Review and new trial designs2018

    • Author(s)
      33.Hirakawa A, Asano J, Sato H, Teramukai S
    • Journal Title

      Contemporary Clinical Trial Communications

      Volume: 12 Pages: 1-8

    • DOI

      10.1016/j.conctc.2018.08.009

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] 希少疾患レジストリーを用いた臨床研究デザイン2016

    • Author(s)
      嘉田晃子
    • Journal Title

      薬理と治療

      Volume: 44

    • Related Report
      2016 Research-status Report
  • [Journal Article] レジストリーデータは医薬品開発に活用できるか?2016

    • Author(s)
      平川晃弘
    • Journal Title

      薬理と治療

      Volume: 44

    • Related Report
      2016 Research-status Report
  • [Presentation] Evaluation of effect by using different control of internal clinical trial in rare disease registry2017

    • Author(s)
      Akiko Kada, Akiko M Saito
    • Organizer
      38th Annual Conference of the International Society for Clinical Biostatistics (ISCB2017)
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research
  • [Presentation] 個別化医療を目指した先進的試験デザイン2016

    • Author(s)
      平川晃弘
    • Organizer
      第13回DIA日本年会
    • Place of Presentation
      東京都江東区
    • Year and Date
      2016-11-13
    • Related Report
      2016 Research-status Report
  • [Presentation] 疾患レジストリを用いた医薬品開発の効率化2016

    • Author(s)
      平川晃弘
    • Organizer
      第13回DIA日本年会
    • Place of Presentation
      東京都江東区
    • Year and Date
      2016-11-13
    • Related Report
      2016 Research-status Report
  • [Presentation] 希少疾患レジストリーを用いた臨床研究デザイン2016

    • Author(s)
      嘉田晃子
    • Organizer
      日本臨床試験学会第7回学術集会総会
    • Place of Presentation
      名古屋
    • Year and Date
      2016-03-11
    • Related Report
      2015 Research-status Report
    • Invited
  • [Presentation] レジストリーデータは医薬品開発に活用できるか?2016

    • Author(s)
      平川晃弘
    • Organizer
      日本臨床試験学会第7回学術集会総会
    • Place of Presentation
      名古屋
    • Year and Date
      2016-03-11
    • Related Report
      2015 Research-status Report
    • Invited
  • [Presentation] Evaluation of the effect of selection pattern of internal cohort study from a disease registry2015

    • Author(s)
      Akiko Kada、Akiko M Saito
    • Organizer
      East Asia Regional Biometric Conference 2015
    • Place of Presentation
      Fukuoka
    • Year and Date
      2015-12-20
    • Related Report
      2015 Research-status Report
    • Int'l Joint Research

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Published: 2015-04-16   Modified: 2020-03-30  

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