111. 先天性ミオパチー Congenital myopathy Clinical trials / Disease details
臨床試験数 : 11 / 薬物数 : 17 - (DrugBank : 5) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 9
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04743557 (ClinicalTrials.gov) | October 2021 | 3/2/2021 | Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies | A Phase 1/2, Multicenter, Open-label, Dose-confirmation Trial to Evaluate the Safety and Preliminary Efficacy of DYN101 in Participants 2 to 17 Years of Age With Centronuclear Myopathy Caused by Mutations in MTM1 or DNM2 | Centronuclear Myopathy | Drug: DYN101 | Dynacure | NULL | Not yet recruiting | 2 Years | 17 Years | All | 9 | Phase 1/Phase 2 | France;Germany |
| 2 | NCT04033159 (ClinicalTrials.gov) | January 9, 2020 | 12/6/2019 | Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients = 16 Years With Centronuclear Myopathies | A Phase 1/2 Trial on the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of DYN101 in Patients = 16 Years of Age With Centronuclear Myopathies Caused by Mutations in DNM2 or MTM1. | Centronuclear Myopathy | Drug: DYN101 | Dynacure | NULL | Recruiting | 16 Years | N/A | All | 18 | Phase 1/Phase 2 | Belgium;Denmark;France;Germany;Netherlands;United Kingdom;United States |
| 3 | EUCTR2020-004608-32-DE (EUCTR) | 27/05/2021 | N/A | A 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 | Dynacure | NULL | NA | Female: yes Male: yes | 9 | Phase 1;Phase 2 | France;Spain;Netherlands;Germany |