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

  
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PhaseCountries
1NCT05099107
(ClinicalTrials.gov)
October 25, 202115/10/2021Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no TreatmentCOMPIS- Congenital Myopathy Intervention Study. An Open-label, Cross Over, Randomised, Controlled Study Using Oral SalbutamolCongenital Myopathy;Neuromuscular Diseases;Musculoskeletal Diseases;Nemaline Myopathy;Centronuclear Myopathy;Myosin Storage MyopathyDrug: Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet;Drug: Salbutamol Only Product in Oral Dose FormVastra Gotaland RegionNULLEnrolling by invitation6 Years30 YearsAll20N/ASweden
2NCT04743557
(ClinicalTrials.gov)
October 20213/2/2021Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear MyopathiesA 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 DNM2Centronuclear MyopathyDrug: DYN101DynacureNULLNot yet recruiting2 Years17 YearsAll9Phase 1/Phase 2France;Germany
3NCT04915846
(ClinicalTrials.gov)
December 18, 20206/5/2021Tamoxifen Therapy for Myotubular MyopathyTAM4MTM: A Phase 1/2 Randomized, Placebo-Controlled, Double-Blinded, Single Crossover Study to Determine the Safety and Efficacy of Tamoxifen Therapy for Myotubular Myopathy (XLMTM)X Linked Myotubular MyopathyDrug: ApoTamox 10mg;Drug: PlaceboJames DowlingCanadian Institutes of Health Research (CIHR);Cures Within Reach;The Joshua Frase Foundation USA;Will Cure USA;Mogford Campbell Family Chair Fund;Myotubular Trust;Great Ormond Street Hospital Charity;SparksRecruiting2 YearsN/AMale16Phase 1/Phase 2United States;Canada;United Kingdom
4EUCTR2019-001147-51-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
5NCT04033159
(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;United States
6EUCTR2017-000876-27-DE
(EUCTR)
22/05/201822/09/2017First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) PatientsASPIRO: A Phase 1/2/3, 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
26Phase 1;Phase 2France;United States;Canada;Germany;United Kingdom
7EUCTR2017-000876-27-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
8EUCTR2017-000876-27-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]Product Name: rAAV8-Des-hMTM1
Product Code: AT132
Audentes Therapeutics Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
20 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Germany;United Kingdom
9NCT02362425
(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
10NCT02035501
(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
11EUCTR2020-004608-32-DE
(EUCTR)
27/05/2021N/AA 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
DynacureNULLNAFemale: yes
Male: yes
9Phase 1;Phase 2France;Spain;Netherlands;Germany