30. 遠位型ミオパチー Disease details / Clinical trials / Drug dev / DR info

臨床試験数 : 13 / 薬物数 : 11 - (DrugBank : 2) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 1

薬物、開発者（Primary Sponsor）、臨床試験情報（症例登録開始年（Enrollment year）、フェーズ、ID、実施国）
Aceneuramic acid
   Suzuki Ryoichi
      2021   Phase 3   JPRN-jRCT2021200030   -
   ULTRAGENYX PHARMACEUTICAL INC.
      2017   Phase 3   EUCTR2016-000360-42-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom
Aceneuramic acid extended-release
   Ultragenyx Pharmaceutical Inc
      2016   Phase 2   NCT02731690   Bulgaria;Canada;United States
      2015   Phase 3   NCT02377921   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
Aceneuramic acid extended-release tablets
   Ultragenyx Pharmaceutical Inc
      2016   Phase 3   NCT02736188   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
Common name: sialic acid
   ULTRAGENYX PHARMACEUTICAL INC.
      2017   Phase 3   EUCTR2016-000360-42-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000360-42-GB   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
Deflazacort
   Ludwig-Maximilians - University of Munich
      2003   Phase 2/Phase 3   NCT00527228   Germany
INN: aceneuramic acid
   ULTRAGENYX PHARMACEUTICAL INC.
      2015   Phase 3   EUCTR2014-005432-33-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
      2017   Phase 2   EUCTR2015-004553-41-BG   Bulgaria;Canada;United States
      2016   Phase 3   EUCTR2016-000360-42-GB   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
      2016   Phase 3   EUCTR2014-005432-33-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
N-acetylneuraminic acid
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 2   EUCTR2015-004553-41-BG   Bulgaria;Canada;United States
NPC-09
   Nobelpharma
      2021   Phase 3   NCT04671472   Japan
   Suzuki Ryoichi
      2021   Phase 3   JPRN-jRCT2021200030   -
   Tohoku University
      2010   Phase 1   NCT01236898   Japan
Sialic acid
   ULTRAGENYX PHARMACEUTICAL INC.
      2015   Phase 3   EUCTR2014-005432-33-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 2   EUCTR2015-004553-41-BG   Bulgaria;Canada;United States
      2016   Phase 3   EUCTR2014-005432-33-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
Sialic acid, N-acetylneuraminic acid
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
UX001
   ULTRAGENYX PHARMACEUTICAL INC.
      2017   Phase 3   EUCTR2016-000360-42-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom
      2015   Phase 3   EUCTR2014-005432-33-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
      2017   Phase 2   EUCTR2015-004553-41-BG   Bulgaria;Canada;United States
      2016   Phase 3   EUCTR2016-000360-42-GB   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
      2016   Phase 3   EUCTR2014-005432-33-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States