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
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