Development trial design in rare disease using registry data
| Project/Area Number |
15K00067
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Statistical science
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| Research Institution | National Hospital Organization Nagoya Medical Center |
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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)
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| 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)
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| Research Collaborator |
SAITO Akiko
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| Project Period (FY) |
2015-04-01 – 2019-03-31
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| Project Status |
Completed (Fiscal Year 2018)
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| 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)
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| 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 |
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| 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.
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| 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。
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Report
(5 results)
Research Products
(10 results)
