4 recruiting, 3 not yet recruiting, 2 completed, 1 active not recruiting. 4 Not applicable, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 4 | Completed 2, Not yet recruiting 1, Recruiting 1 |
| Phase 2 | 1 | Unknown 1 |
Follow Nemaline Myopathy: new trials by email Literature Review of Nemaline Myopathy trials Research Report on Nemaline Myopathy
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 | |
| 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 | |
| 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 |
| Inspiratory Muscle Training in Nemaline Myopathy Threshold IMT, Philips Respironics, Murrysville, PA, USA - Nemaline Myopathy, Inspiratory Muscle Training | Not applicable | Completed | Radboud University Medical Center | 42 | 2018-10-10 | 2024-12-05 |
| Treatment of TNNT1-Myopathy With L-Tyrosine. L-Tyrosine, Placebo - Nemaline Myopathy | Phase 2 | Unknown | Hadassah Medical Organization | 10 | 2014-01-01 | 2014-01-14 |
| Molecular and Genetic Studies of Congenital Myopathies Central Core Disease, Centronuclear Myopathy | Recruiting | Boston Children's Hospital | 4000 | 2003-08-01 | 2026-03-25 |
Myopathies, Nemaline (11) · Myopathies, Structural, Congenital (5) · Myotonia Congenita (5) · Neuromuscular Diseases (4) · Minicore Myopathy with External Ophthalmoplegia (3) · Myopathy, Central Core (3) · Muscular Diseases (2) · Amyotrophic Lateral Sclerosis (1) · Charcot-Marie-Tooth Disease (1) · Glycogen Storage Disease Type II (1) · Lambert-Eaton Myasthenic Syndrome (1) · Malignant Hyperthermia (1) · Mitochondrial Myopathies (1) · Motor Neuron Disease (1) · Muscular Atrophy, Spinal (1)
Nervous System Diseases (11) · Musculoskeletal Diseases (10) · Muscular Diseases (9) · Neuromuscular Diseases (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, Inborn (6) · Heredodegenerative Disorders, Nervous System (6) · Myopathies, Structural, Congenital (6) · Neurodegenerative Diseases (6) · Myotonic Disorders (5) · Muscular Disorders, Atrophic (2) · Muscular Dystrophies (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2) · Autoimmune Diseases (1)
Albuterol (1) · Dosage Forms (1) · Nutritional Status (1) · Respiratory Rate (1) · Tablets (1) · Tyrosine (1)
Radboud University Medical Center (2) · Boston Children's Hospital (1) · Centre Hospitalier Universitaire de Liege (1) · Hadassah Medical Organization (1) · Hospital Universitari Vall d'Hebron Research Institute (1) · King's College London (1) · Stanford University (1) · University of Missouri-Columbia (1) · University of Oxford (1) · Vastra Gotaland Region (1)
United States (3) · Netherlands (2) · United Kingdom (2) · Belgium (1) · Canada (1) · Israel (1) · Spain (1) · Sweden (1)
Alan Beggs, PhD (1) · Alan H. Beggs, Ph.D. (1) · Baziel van Engelen, Prof. PhD (1) · Carolina Tesi-Rocha, MD (1) · Carsten Bonnemann, MD, PhD (1) · Jim Dowling, MD, PhD (1) · John W Day, MD, PhD (1) · Kaitlin Batley, MD (1) · Laurent Servais, MD (1) · Leslie Hayes, MD (1) · Nicol Voermans, MD PhD (1) · Niklas Darin, M.D (1) · Prof Laurent Servais (1) · Richard Finkel, MD (1) · Tina Duong, PT, 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 21357678, PMID 42551994
2019 (1)