| Project/Area Number |
18K07523
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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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| Review Section |
Basic Section 52020:Neurology-related
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| Research Institution | Nagoya University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
勝野 雅央 名古屋大学, 医学系研究科, 教授 (50402566)
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| Project Period (FY) |
2018-04-01 – 2021-03-31
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| Project Status |
Completed (Fiscal Year 2020)
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| Budget Amount *help |
¥4,420,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥1,020,000)
Fiscal Year 2020: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2019: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2018: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
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| Keywords | 球脊髄性筋萎縮症 / 医師主導治験 / リアルワールドデータ / slope analysis / 神経変性疾患 / 疾患修飾療法 / バイオマーカー / リュープロレリン酢酸塩 / real-world data / 疾患修飾薬 / long-term effect / リュープリロレリン酢酸塩 |
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| Outline of Final Research Achievements |
Spinal and bulbar muscular atrophy (SBMA) is one of rare neuromuscular diseases. In 2017, leuprorelin acetate which has disease-modifying effect on SBMA was approved in Japan, however, there is not enough information to demonstrate its efficacy or appropriate usage of this drug. The purpose of the study is to develop a real-world database to accumulate more information to demonstrate its efficacy.The principal outcome measurement to evaluate disease severity was ALSFRS-R.A total of 392 SBMA patients were enrolled. Although the results of a slope analysis for total population presented that there was no significant difference between pre- and post-treatment of leuprorelin acetate, the analysis for subpopulation who have enough observational records (more than two years before and after administration) revealed that the drug might slow disease progression (disease progression slope before intervention; -0.528 ± 0.634/ year: slope after intervention; -0.292 ± 1.471/ year (p = 0.3185)).
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| Academic Significance and Societal Importance of the Research Achievements |
医薬品承認申請を目的とした治験は、限られた期間で、限られた被験者を対象に実施されるものであり、特に球脊髄性筋萎縮症のような緩徐進行性の神経筋疾患に対する疾患修飾効果を十分に検討することは困難である。本研究では、2017年に薬事承認を受けたリュープロレリン酢酸塩に対するリアルワールド（実臨床下）での有効性を検討するために、承認前から収集してきた臨床データに対する継続的縦断的データをさらに収集し、有効性の推定を行うものである。疾患修飾効果は数年以上をかけて明確になることが病態メカニズムから推定され、引き続き縦断的データを収集することで、リュープロレリン酢酸塩の有効性を実臨床下で評価できると考える。
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