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Long-term effect of leuprorelin acetate on the patients with spinal and bulbar muscular atrophy

Research Project

Project/Area Number 18K07523
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Review Section Basic Section 52020:Neurology-related
Research InstitutionNagoya University

Principal Investigator

Hashizume Atsushi  名古屋大学, 医学部附属病院, 病院講師 (00637689)

Co-Investigator(Kenkyū-buntansha) 勝野 雅央  名古屋大学, 医学系研究科, 教授 (50402566)
Project Period (FY) 2018-04-01 – 2021-03-31
Project Status Completed (Fiscal Year 2020)
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)
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
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)).

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。

Report

(4 results)
  • 2020 Annual Research Report   Final Research Report ( PDF )
  • 2019 Research-status Report
  • 2018 Research-status Report
  • Research Products

    (7 results)

All 2021 2019

All Journal Article (4 results) (of which Peer Reviewed: 4 results,  Open Access: 1 results) Presentation (3 results) (of which Invited: 2 results)

  • [Journal Article] Long-term Effects of Androgen Deprivation in a Patient with Spinal and Bulbar Muscular Atrophy - A Case Report with 14 Years of Follow-up2019

    • Author(s)
      Hijikata Yasuhiro、Hashizume Atsushi、Yamada Shinichiro、Ito Daisuke、Banno Haruhiko、Suzuki Keisuke、Sobue Gen、Katsuno Masahisa
    • Journal Title

      Internal Medicine

      Volume: 58 Issue: 15 Pages: 2231-2234

    • DOI

      10.2169/internalmedicine.1592-18

    • NAID

      130007686841

    • ISSN
      0918-2918, 1349-7235
    • Year and Date
      2019-08-01
    • Related Report
      2018 Research-status Report
    • Peer Reviewed / Open Access
  • [Journal Article] Efficacy and safety of leuprorelin acetate for subjects with spinal and bulbar muscular atrophy: pooled analyses of two randomized-controlled trials2019

    • Author(s)
      Hashizume Atsushi、JASMITT study group、Katsuno Masahisa、Suzuki Keisuke、Banno Haruhiko、Takeuchi Yu、Kawashima Motoshi、Suga Noriaki、Mano Tomoo、Araki Amane、Hijikata Yasuhiro、Hirakawa Akihiro、Sobue Gen
    • Journal Title

      Journal of Neurology

      Volume: 266 Issue: 5 Pages: 1211-1221

    • DOI

      10.1007/s00415-019-09251-x

    • Related Report
      2019 Research-status Report 2018 Research-status Report
    • Peer Reviewed
  • [Journal Article] Long-term Effects of Androgen Deprivation in a Patient with Spinal and Bulbar Muscular Atrophy - A Case Report with 14 Years of Follow-up2019

    • Author(s)
      Hijikata Yasuhiro、Hashizume Atsushi、Yamada Shinichiro、Ito Daisuke、Banno Haruhiko、Suzuki Keisuke、Sobue Gen、Katsuno Masahisa
    • Journal Title

      Intern Med

      Volume: 58 Pages: 2231-2234

    • NAID

      130007686841

    • Related Report
      2019 Research-status Report
    • Peer Reviewed
  • [Journal Article] Nasometric Scores in spinal and bulbar muscular atrophy - Effects of palatal lift prosthesis on dysarthria and dysphagia2019

    • Author(s)
      Tanaka Seiya、Hashizume Atsushi、Hijikata Yasuhiro、Yamada Shinichiro、Ito Daisuke、Nakayama Atsushi、Kurita Kenichi、Yogo Hiroaki、Banno Haruhiko、Suzuki Keisuke、Yamamoto Masahiko、Sobue Gen、Katsuno Masahisa
    • Journal Title

      Journal of the Neurological Sciences

      Volume: 407 Pages: 116503-116503

    • DOI

      10.1016/j.jns.2019.116503

    • Related Report
      2019 Research-status Report
    • Peer Reviewed
  • [Presentation] Longitudinal study of the subjects with SBMA using real-world data2021

    • Author(s)
      橋詰 淳
    • Organizer
      日本神経学会学術大会
    • Related Report
      2020 Annual Research Report
  • [Presentation] SBMAレジストリの現状と課題2019

    • Author(s)
      橋詰 淳
    • Organizer
      日本神経学会学術大会
    • Related Report
      2019 Research-status Report
    • Invited
  • [Presentation] 成功する医師主導治験:つまづきやすいポイントを確実に乗り越えろ!‐成功する試験デザインの作り方‐2019

    • Author(s)
      橋詰 淳
    • Organizer
      日本神経学会学術大会
    • Related Report
      2019 Research-status Report
    • Invited

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Published: 2018-04-23   Modified: 2022-01-27  

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