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 : 17 - (DrugBank : 5) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 9
薬物ごとの開発者(Primary Sponsor)、臨床試験情報(抜粋)です。
AT132
Audentes Therapeutics Inc.
2018 Phase 1;Phase 2 EUCTR2017-000876-27-FR France;Germany;United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2017-000876-27-DE Canada;France;Germany;United Kingdom;United States;
2017 Phase 1;Phase 2 EUCTR2017-000876-27-GB France;Germany;United Kingdom;United States;
ApoTamox
James Dowling
2020 Phase 1/Phase 2 NCT04915846 Canada;United Kingdom;United States;
DYN101
Dynacure
2021 Phase 1/Phase 2 NCT04743557 France;Germany;
2020 Phase 1/Phase 2 NCT04033159 Belgium;Denmark;France;Germany;Netherlands;United Kingdom;United States;
- 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
Audentes Therapeutics Inc.
2018 Phase 1;Phase 2 EUCTR2017-000876-27-FR France;Germany;United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2017-000876-27-DE Canada;France;Germany;United Kingdom;United States;
2017 Phase 1;Phase 2 EUCTR2017-000876-27-GB France;Germany;United Kingdom;United States;
SALBUTAMOL SULFATE
Västra götalandsregionen
2020 Phase 4 EUCTR2019-001147-51-SE Sweden;
Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet
Vastra Gotaland Region
2021 - NCT05099107 Sweden;
Salbutamol Only Product in Oral Dose Form
Vastra Gotaland Region
2021 - NCT05099107 Sweden;
Ventoline oral solution 0,4mg/ml
Västra götalandsregionen
2020 Phase 4 EUCTR2019-001147-51-SE Sweden;
Ventoline tablet
Västra götalandsregionen
2020 Phase 4 EUCTR2019-001147-51-SE Sweden;
Audentes Therapeutics Inc.
2018 Phase 1;Phase 2 EUCTR2017-000876-27-FR France;Germany;United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2017-000876-27-DE Canada;France;Germany;United Kingdom;United States;
2017 Phase 1;Phase 2 EUCTR2017-000876-27-GB France;Germany;United Kingdom;United States;
ApoTamox
James Dowling
2020 Phase 1/Phase 2 NCT04915846 Canada;United Kingdom;United States;
DYN101
Dynacure
2021 Phase 1/Phase 2 NCT04743557 France;Germany;
2020 Phase 1/Phase 2 NCT04033159 Belgium;Denmark;France;Germany;Netherlands;United Kingdom;United States;
- 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
Audentes Therapeutics Inc.
2018 Phase 1;Phase 2 EUCTR2017-000876-27-FR France;Germany;United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2017-000876-27-DE Canada;France;Germany;United Kingdom;United States;
2017 Phase 1;Phase 2 EUCTR2017-000876-27-GB France;Germany;United Kingdom;United States;
SALBUTAMOL SULFATE
Västra götalandsregionen
2020 Phase 4 EUCTR2019-001147-51-SE Sweden;
Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet
Vastra Gotaland Region
2021 - NCT05099107 Sweden;
Salbutamol Only Product in Oral Dose Form
Vastra Gotaland Region
2021 - NCT05099107 Sweden;
Ventoline oral solution 0,4mg/ml
Västra götalandsregionen
2020 Phase 4 EUCTR2019-001147-51-SE Sweden;
Ventoline tablet
Västra götalandsregionen
2020 Phase 4 EUCTR2019-001147-51-SE Sweden;