11 recruiting, 9 completed, 6 not yet recruiting, 2 active not recruiting. 8 Not applicable, 7 Phase 2, 5 Phase 1, 1 Phase 3.
Follow Myopathies, Structural, Congenital: new trials by email Literature Review of Myopathies, Structural, Congenital trials Research Report on Myopathies, Structural, Congenital
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957 ASP2957 - X-linked Myotubular Myopathy | Phase 1 / Phase 2 | Not yet recruiting | Astellas Gene Therapies | 9 | 2026-10-31 | 2026-10-15 |
| Reducing Risk of Stroke and DementIa In patientS With COVert cERebrovascular Disease Functional and Cognitive scores - Covert Cerebrovascular Disease | Not yet recruiting | University of Edinburgh | 100 | 2026-10-01 | 2026-09-09 | |
| Natural History Study for Patients With Nemaline Myopathy in Belgium Motor Function Assessments, Syde, Respiratory Assessments - Nemaline Myopathy | Not applicable | Not yet recruiting | Centre Hospitalier Universitaire de Liege | 10 | 2026-08-01 | 2026-03-25 |
| Natural History Study for Patients With Nemaline Myopathy in Spain Muscle Ultrasound, Motor function scales, Complete physical examination - Nemaline Myopathy, Myopathies | Recruiting | Hospital Universitari Vall d'Hebron Research Institute | 100 | 2026-06-01 | 2026-03-23 | |
| Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease Whole-body Electrical Muscle Stimulation Exercise - Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis | Not applicable | Recruiting | University of Missouri-Columbia | 50 | 2026-03-10 | 2026-03-17 |
| Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators ASP2957, Methylprednisolone, Prednisolone - X-Linked Myotubular Myopathy | Phase 1 / Phase 2 | Recruiting | Astellas Gene Therapies | 9 | 2025-12-15 | 2026-09-03 |
| Smoking Cessation Intervention for Young Adults Using MTM: A Randomized Controlled Trial Multi-Theory Model (MTM)-Based Smoking Cessation Program - Tobacco Use Disorder/Cigarette Smoking | Not applicable | Not yet recruiting | University of Nevada, Las Vegas | 144 | 2025-11-01 | 2025-08-11 |
| Multispectral Optoacoustic Tomography for Advanced Imaging of Centronuclear Myopathy MSOT - Centronuclear Myopathy | Recruiting | University of Erlangen-Nürnberg Medical School | 40 | 2025-05-29 | 2025-06-15 | |
| A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition No Intervention - X-Linked Myotubular Myopathy | Recruiting | Astellas Gene Therapies | 50 | 2025-05-19 | 2026-09-30 | |
| The Prevalence of RYR1-related Disease Neuromuscular Disease, Malignant Hyperthermia | Not yet recruiting | King's College London | 2000 | 2025-02-01 | 2025-01-24 | |
| Nemaline Myopathy Clinical Research Network (NM-CTRN) Nemaline Myopathy | Not yet recruiting | Stanford University | 50 | 2025-01-01 | 2025-01-14 | |
| A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD reSEES - Intermediate AMD | Not applicable | Recruiting | Oculox Technologies SA | 30 | 2024-11-01 | 2024-11-15 |
| Natural History Study for Patients With Nemaline Myopathy in the UK Nemaline Myopathy | Active not recruiting | University of Oxford | 45 | 2024-10-07 | 2026-04-13 | |
| The Natural History and Muscle Fatigability of Patients With Congenital Myopathies. Natural history and non therapeutical therapy - Central Core Disease, Multi-Minicore Disease | Recruiting | Radboud University Medical Center | 100 | 2024-03-28 | 2024-08-19 | |
| Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies DYN101 - Centronuclear Myopathy | Phase 1 / Phase 2 | Withdrawn | Dynacure | 2024-01-01 | 2022-07-11 | |
| Natural History Study of Patients With Centronuclear Myopathies Centronuclear Myopathy | Withdrawn | Dynacure | 2022-09-01 | 2022-07-11 | ||
| Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet, Salbutamol Only Product in Oral Dose Form - Congenital Myopathy, Neuromuscular Diseases | Not applicable | Completed | Vastra Gotaland Region | 18 | 2021-10-25 | 2025-06-19 |
| Tamoxifen Therapy for Myotubular Myopathy ApoTamox 10mg, Placebo - X Linked Myotubular Myopathy | Phase 1 / Phase 2 | Terminated | James Dowling | 6 | 2020-12-18 | 2024-09-19 |
| Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study ActiMyo/Syde - Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy | Not applicable | Recruiting | Centre Hospitalier Universitaire de Liege | 300 | 2020-07-10 | 2025-05-20 |
| Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies DYN101 - Centronuclear Myopathy | Phase 1 / Phase 2 | Terminated | Dynacure | 14 | 2020-01-09 | 2023-06-27 |
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Myopathies, Nemaline (12) · Neuromuscular Diseases (7) · Muscular Diseases (6) · Myotonia Congenita (6) · Myopathy, Central Core (5) · Minicore Myopathy with External Ophthalmoplegia (3) · Musculoskeletal Diseases (3) · Charcot-Marie-Tooth Disease (2) · Glycogen Storage Disease Type II (2) · Muscular Dystrophy, Facioscapulohumeral (2) · Nervous System Diseases (2) · Amyotrophic Lateral Sclerosis (1) · Cigarette Smoking (1) · Hyperthermia (1)
Nervous System Diseases (31) · Musculoskeletal Diseases (30) · Muscular Diseases (27) · Neuromuscular Diseases (26) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Heredodegenerative Disorders, Nervous System (8) · Neurodegenerative Diseases (8) · Myotonic Disorders (7) · Pathological Conditions, Signs and Symptoms (5) · Muscular Disorders, Atrophic (3) · Muscular Dystrophies (3) · Pathologic Processes (3) · Signs and Symptoms (3)
Respiratory Rate (2) · Albuterol (1) · Base Sequence (1) · Dosage Forms (1) · Functional Status (1) · Genetic Testing (1) · Health Records, Personal (1) · Methylprednisolone (1) · Nutritional Status (1) · Prednisolone (1) · Restraint, Physical (1) · Sirolimus (1) · Tablets (1) · Tamoxifen (1) · Tyrosine (1)
Astellas Gene Therapies (6) · Dynacure (3) · Centre Hospitalier Universitaire de Liege (2) · Cure CMD (2) · Radboud University Medical Center (2) · Boston Children's Hospital (1) · Clinic for Special Children (1) · Hadassah Medical Organization (1) · Hospital Universitari Vall d'Hebron Research Institute (1) · Institut de Myologie, France (1) · James Dowling (1) · King's College London (1) · Newcastle-upon-Tyne Hospitals NHS Trust (1) · Oculox Technologies SA (1) · Stanford University (1)
United States (15) · United Kingdom (8) · Canada (7) · Germany (7) · France (6) · Belgium (5) · Italy (3) · Netherlands (3) · Spain (3) · Czechia (1) · Denmark (1) · Egypt (1) · Hungary (1) · Israel (1) · Poland (1)
Laurent Servais, MD (2) · Alan Beggs, PhD (1) · Alan H. Beggs, Ph.D. (1) · Anne Rutkowski, MD (1) · Barbara K Smith, PT, PhD (1) · Baziel van Engelen, Prof. PhD (1) · Carolina Tesi-Rocha, MD (1) · Carsten Bonnemann, MD, PhD (1) · Chiara Marini Bettolo (1) · Chris Freitag, MD (1) · Elizabeth DeChene, MS, GCG (1) · Ferdinand Knieling, PD Dr. med. habil. Dr. rer. b (1) · Hal Landy, MD (1) · Jame J Dowling, MD, PhD (1)
PMID 10051637, PMID 10192379, PMID 10508519, PMID 11558787, PMID 12196661, PMID 12601110, PMID 12677001, PMID 15056467, PMID 15236405, PMID 16042307, PMID 17160903, PMID 17537630, PMID 19232495, PMID 19346529, PMID 19562689, PMID 19944167, PMID 19953533, PMID 20434914, PMID 21357678, PMID 30902907, PMID 31484632, PMID 37977713, PMID 42551994
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