111. 先天性ミオパチー Disease details / Clinical trials / Drug dev / DR info


臨床試験数 : 10 / 薬物数 : 13 - (DrugBank : 2) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 11

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)
Apotamox 10MG
   James Dowling
      2020   Phase 1/Phase 2   NCT04915846   Canada;United Kingdom;United States
AT132
   Astellas Gene Therapies
      2017   Phase 2/Phase 3   NCT03199469   Canada;France;Germany;United States
   Astellas Gene Therapies, Inc.
      2018   Phase 1;Phase 2;Phase 3   EUCTR2017-000876-27-DE   Canada;France;Germany;United Kingdom;United States
   Audentes Therapeutics Inc.
      2018   Phase 1;Phase 2   EUCTR2017-000876-27-FR   France;Germany;United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2017-000876-27-GB   France;Germany;United Kingdom;United States
DYN101
   Dynacure
      -   Phase 1;Phase 2   EUCTR2020-004608-32-DE   France;Germany;Netherlands;Spain
L-tyrosine
   Hadassah Medical Organization
      2014   Phase 2   NCT02035501   Israel
N-acetylcysteine
   National Institute of Nursing Research (NINR)
      2015   Phase 1/Phase 2   NCT02362425   United States
RAAV8-DES-HMTM1
   Astellas Gene Therapies, Inc.
      2018   Phase 1;Phase 2;Phase 3   EUCTR2017-000876-27-DE   Canada;France;Germany;United Kingdom;United States
   Audentes Therapeutics Inc.
      2018   Phase 1;Phase 2   EUCTR2017-000876-27-FR   France;Germany;United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2017-000876-27-GB   France;Germany;United Kingdom;United States
Salbutamol (AS salbutamol sulfate) 2 MG oral tablet
   Vastra Gotaland Region
      2021   -   NCT05099107   Sweden
Salbutamol 2MG
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Salbutamol 4MG
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Salbutamol only product IN oral dose form
   Vastra Gotaland Region
      2021   -   NCT05099107   Sweden
Salbutamol sulfate
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Ventoline oral solution 0,4MG/ML
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Ventoline tablet 2MG
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden