111. 先天性ミオパチー
［臨床試験数：7，薬物数：13（DrugBank：4），標的遺伝子数：1，標的パスウェイ数：8］
Searched query = "Congenital myopathy", "Nemaline myopathy", "Central core disease", "Minicore myopathy", "Myotubular myopathy", "Centronuclear myopathy", "Congenital fiber-type disproportion myopathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | 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 |