111. 先天性ミオパチー ［臨床試験数：5，薬物数：7（DrugBank：2），標的遺伝子数：0，標的パスウェイ数：0］
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 of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04033159 | November 2019 | 28 October 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 SAS | Not recruiting | 16 Years | N/A | All | 18 | Phase 1/Phase 2 | Belgium;Denmark;France;Germany;Netherlands;United Kingdom | |
| 2 | EUCTR2017-000876-27-DE | 22/05/2018 | 15 October 2018 | First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients | ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO | X-linked Myotubular Myopathy (XLMTM) MedDRA version: 20.0 Level: HLGT Classification code 10029317 Term: Neuromuscular disorders System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: rAAV8-Des-hMTM1 Product Code: AT132 Pharmaceutical Form: Solution for infusion | Audentes Therapeutics Inc. | Authorised | Female: no Male: yes | 12 | Phase 1;Phase 2 | United Kingdom;Germany;United States;France | |||
| 3 | EUCTR2017-000876-27-FR | 28 May 2018 | First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients | ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO | X-linked Myotubular Myopathy (XLMTM);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: rAAV8-Des-hMTM1 Product Code: AT132 Pharmaceutical Form: Solution for infusion | Audentes Therapeutics Inc. | Not Available | Female: no Male: yes | 12 | Phase 1;Phase 2 | United States;France;Germany;United Kingdom | ||||
| 4 | NCT02362425 | February 12, 2015 | 16 July 2018 | Antioxidant Therapy in RYR1-Related Congenital Myopathy | Antioxidant Therapy in RYR1-Related Congenital Myopathy | Neuromuscular Disease | Drug: N-acetylcysteine;Drug: Placebo | National Institute of Nursing Research (NINR) | Not recruiting | 7 Years | N/A | All | 56 | Phase 1/Phase 2 | United States | |
| 5 | NCT02035501 | January 2014 | 19 February 2015 | Treatment of TNNT1-Myopathy With L-Tyrosine. | Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial. | Nemaline Myopathy | Drug: L-Tyrosine;Drug: Placebo | Hadassah Medical Organization | Recruiting | N/A | 20 Years | Both | 10 | Phase 2 | Israel |