DYN101 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
111先天性ミオパチー1

111. 先天性ミオパチー


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
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1EUCTR2020-004608-32-DE
(EUCTR)
27/05/2021N/AA Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate the safety and preliminary efficacy of DYN101 in subjects 2 to 17 years of age with centronuclear myopathy caused by mutations in MTM1 or DNM2. - Research Using an Investigational Treatment for CNM (DyNaMic) Centronuclear myopathy (CNM), in subjects 2 to 17 years of age (all ages inclusive)caused by mutations in the myotubularin1 (MTM1) or dynamin 2 (DNM2) gene
MedDRA version: 20.0;Level: HLT;Classification code 10028640;Term: Myopathies;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: DYN101
Product Code: DYN101
INN or Proposed INN: DYN101
DynacureNULLNAFemale: yes
Male: yes
9Phase 1;Phase 2France;Spain;Netherlands;Germany