111. Congenital myopathy
7 clinical trials,   13 drugs   (DrugBank: 4 drugs),   1 drug target gene,   8 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1EUCTR2019-001147-51-SE
(EUCTR)
31/03/202027/09/2019This is a treatment study for a muscle disorder known as congenital myopathyCongenital myopathy intervention study - COMPIS Congenital myopathy
MedDRA version: 20.0;Level: PT;Classification code 10062547;Term: Congenital myopathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Ventoline tablet 2mg
INN or Proposed INN: SALBUTAMOL SULFATE
Trade Name: Ventoline oral solution 0,4mg/ml
INN or Proposed INN: SALBUTAMOL SULFATE
Västra götalandsregionenNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 4Sweden
2NCT04033159
(ClinicalTrials.gov)
January 9, 202012/6/2019Early 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: DYN101DynacureNULLRecruiting16 YearsN/AAll18Phase 1;Phase 2Belgium;Denmark;France;Germany;Netherlands;United Kingdom
3EUCTR2017-000876-27-DE
(EUCTR)
22/05/201822/09/2017First-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 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
Audentes Therapeutics Inc.NULLNot RecruitingFemale: no
Male: yes
20Phase 1;Phase 2France;United States;Germany;United Kingdom
4EUCTR2017-000876-27-FR
(EUCTR)
23/04/201825/05/2018First-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 - ASPIRO X-linked Myotubular Myopathy (XLMTM);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: rAAV8-Des-hMTM1
Product Code: AT132
Audentes Therapeutics Inc.NULLNot RecruitingFemale: no
Male: yes
12Phase 1;Phase 2United States;France;Germany;United Kingdom
5EUCTR2017-000876-27-GB
(EUCTR)
06/12/201731/07/2017First-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 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]Audentes Therapeutics Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
20Phase 1;Phase 2France;United States;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02362425
(ClinicalTrials.gov)
February 12, 201512/2/2015Antioxidant Therapy in RYR1-Related Congenital MyopathyAntioxidant Therapy in RYR1-Related Congenital MyopathyNeuromuscular DiseaseDrug: N-acetylcysteine;Drug: PlaceboNational Institute of Nursing Research (NINR)NULLCompleted7 YearsN/AAll63Phase 1;Phase 2United States
7NCT02035501
(ClinicalTrials.gov)
January 20142/7/2013Treatment 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 OrganizationNULLEnrolling by invitationN/A20 YearsBoth10Phase 2Israel