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 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. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04033159November 201928 October 2019Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients = 16 Years With Centronuclear MyopathiesA 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 MyopathyDrug: DYN101Dynacure SASNot recruiting16 YearsN/AAll18Phase 1/Phase 2Belgium;Denmark;France;Germany;Netherlands;United Kingdom
2EUCTR2017-000876-27-DE22/05/201815 October 2018First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) PatientsASPIRO: 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 - ASPIROX-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.AuthorisedFemale: no
Male: yes
12Phase 1;Phase 2United Kingdom;Germany;United States;France
3EUCTR2017-000876-27-FR28 May 2018First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) PatientsASPIRO: 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 - ASPIROX-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 AvailableFemale: no
Male: yes
12Phase 1;Phase 2United States;France;Germany;United Kingdom
4NCT02362425February 12, 201516 July 2018Antioxidant Therapy in RYR1-Related Congenital MyopathyAntioxidant Therapy in RYR1-Related Congenital MyopathyNeuromuscular DiseaseDrug: N-acetylcysteine;Drug: PlaceboNational Institute of Nursing Research (NINR)Not recruiting7 YearsN/AAll56Phase 1/Phase 2United States
5NCT02035501January 201419 February 2015Treatment of TNNT1-Myopathy With L-Tyrosine.Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.Nemaline MyopathyDrug: L-Tyrosine;Drug: PlaceboHadassah Medical OrganizationRecruitingN/A20 YearsBoth10Phase 2Israel

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