111. Congenital myopathy Clinical trials / Disease details
Clinical trials : 11 / Drugs : 17 - (DrugBank : 5) / Drug target gene : 1 - Drug target pathways : 9
| 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 | NCT05099107 (ClinicalTrials.gov) | October 25, 2021 | 15/10/2021 | Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment | COMPIS- Congenital Myopathy Intervention Study. An Open-label, Cross Over, Randomised, Controlled Study Using Oral Salbutamol | Congenital Myopathy;Neuromuscular Diseases;Musculoskeletal Diseases;Nemaline Myopathy;Centronuclear Myopathy;Myosin Storage Myopathy | Drug: Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet;Drug: Salbutamol Only Product in Oral Dose Form | Vastra Gotaland Region | NULL | Enrolling by invitation | 6 Years | 30 Years | All | 20 | N/A | Sweden |
| 2 | NCT04743557 (ClinicalTrials.gov) | October 2021 | 3/2/2021 | Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies | A 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 DNM2 | Centronuclear Myopathy | Drug: DYN101 | Dynacure | NULL | Not yet recruiting | 2 Years | 17 Years | All | 9 | Phase 1/Phase 2 | France;Germany |
| 3 | NCT04915846 (ClinicalTrials.gov) | December 18, 2020 | 6/5/2021 | Tamoxifen Therapy for Myotubular Myopathy | TAM4MTM: 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 Myopathy | Drug: ApoTamox 10mg;Drug: Placebo | James Dowling | Canadian 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;Sparks | Recruiting | 2 Years | N/A | Male | 16 | Phase 1/Phase 2 | United States;Canada;United Kingdom |
| 4 | EUCTR2019-001147-51-SE (EUCTR) | 31/03/2020 | 27/09/2019 | This is a treatment study for a muscle disorder known as congenital myopathy | Congenital 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 götalandsregionen | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 4 | Sweden | ||
| 5 | 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;United States |
| 6 | EUCTR2017-000876-27-DE (EUCTR) | 22/05/2018 | 22/09/2017 | First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients | ASPIRO: 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. | NULL | Not Recruiting | Female: no Male: yes | 26 | Phase 1;Phase 2 | France;United States;Canada;Germany;United Kingdom | ||
| 7 | EUCTR2017-000876-27-FR (EUCTR) | 23/04/2018 | 25/05/2018 | 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 | Audentes Therapeutics Inc. | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1;Phase 2 | United States;France;Germany;United Kingdom | ||
| 8 | EUCTR2017-000876-27-GB (EUCTR) | 06/12/2017 | 31/07/2017 | 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 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. | NULL | Authorised-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): no | France;United States;Germany;United Kingdom | ||
| 9 | NCT02362425 (ClinicalTrials.gov) | February 12, 2015 | 12/2/2015 | 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) | NULL | Completed | 7 Years | N/A | All | 63 | Phase 1/Phase 2 | United States |
| 10 | NCT02035501 (ClinicalTrials.gov) | January 2014 | 2/7/2013 | 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 | NULL | Enrolling by invitation | N/A | 20 Years | Both | 10 | Phase 2 | Israel |
| 11 | EUCTR2020-004608-32-DE (EUCTR) | 27/05/2021 | N/A | A 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 | Dynacure | NULL | NA | Female: yes Male: yes | 9 | Phase 1;Phase 2 | France;Spain;Netherlands;Germany |