39 completed, 17 recruiting, 5 active not recruiting, 5 terminated. 23 Not applicable, 18 Phase 2, 13 Phase 1, 2 Phase 3.
Follow Muscular Dystrophy, Facioscapulohumeral: new trials by email Literature Review of Muscular Dystrophy, Facioscapulohumeral trials Research Report on Muscular Dystrophy, Facioscapulohumeral
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 Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) forazapadin, Placebo Comparator - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 2 | Not yet recruiting | Satellos Bioscience, Inc. | 51 | 2026-12-15 | 2026-10-13 |
| A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD SFL-0821 for injection, Placebo - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 1 / Phase 2 | Recruiting | Soufflé Therapeutics, Inc. | 90 | 2026-09-01 | 2026-10-16 |
| Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD Apitegromab, Placebo - Facioscapulohumeral Muscular Dystrophy, FSHD | Phase 2 | Recruiting | Scholar Rock, Inc. | 60 | 2026-07-30 | 2026-09-02 |
| Amino Acids and Exercise in FSHD Essential Amino Acids Supplementation intervention, Exercise, Diet - FSH Muscular Dystrophy, Muscular Dystrophy | Not applicable | Recruiting | University of Pavia | 48 | 2026-04-08 | 2026-04-21 |
| 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 |
| Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD) ULSC, Placebo - FSHD - Facioscapulohumeral Muscular Dystrophy | Phase 1 | Recruiting | Restem, LLC. | 16 | 2025-12-01 | 2026-10-21 |
| The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. Assistive gait devices combined with physiotherapy - Inclusion Body Myositis, Myotonic Dystrophy 1 | Not applicable | Enrolling by invitation | LMU Klinikum | 30 | 2025-09-01 | 2025-07-18 |
| Clenbuterol to Target DUX4 in FSHD Clenbuterol - Muscular Dystrophy, Facioscapulohumeral | Phase 1 | Recruiting | Jeffrey Statland | 30 | 2025-06-25 | 2026-02-25 |
| A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD AOC-1020, Placebo - Facioscapulohumeral Muscular Dystrophy, FSHD | Phase 3 | Recruiting | Avidity Biosciences, Inc. | 200 | 2025-06-10 | 2026-07-27 |
| Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds) Muscular Dystrophy, Facioscapulohumeral | Recruiting | University of Kansas Medical Center | 80 | 2025-05-22 | 2026-02-25 | |
| A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy EPI-321 - Facioscapulohumeral Muscular Dystrophy | Phase 1 / Phase 2 | Active not recruiting | Epicrispr Biotechnologies, Inc. | 12 | 2025-05-08 | 2026-08-19 |
| Muscle MRI Outlining of Neuromuscular Diseases Using Artificial Intelligence No intervention - Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy | Not yet recruiting | Copenhagen University Hospital, Rigshospitalet | 120 | 2025-05-01 | 2025-04-08 | |
| Dietary Protein Requirements in Adults With Facioscapulohumeral Muscular Dystrophy Amino Acid Intake - Fascioscapulohumeral Muscular Dystrophy | Not applicable | Completed | McGill University | 10 | 2025-01-19 | 2026-07-09 |
| Personalized Training for People With Rare Neuromuscular Disorders Exercise, Usual Care - Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT) | Not applicable | Recruiting | Oslo University Hospital | 120 | 2024-12-13 | 2026-06-18 |
| Imaging and Gait Analysis in FSHD Patients FSHD - Facioscapulohumeral Muscular Dystrophy | Enrolling by invitation | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 40 | 2024-11-30 | 2025-09-10 | |
| Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders FBIndex - Inclusion Body Myositis, Myotonic Dystrophy | Enrolling by invitation | LMU Klinikum | 108 | 2024-09-09 | 2025-09-10 | |
| Ten Year Follow-up in FSHD: the FOCUS 3 Study FSHD - Facioscapulohumeral Muscular Dystrophy | Recruiting | Radboud University Medical Center | 200 | 2024-09-01 | 2025-04-04 | |
| BetterLife FSHD: A Patient-driven Health and Research Platform Observational - FSH, FSH Muscular Dystrophy | Recruiting | FSHD Society | 5000 | 2024-08-02 | 2026-02-20 | |
| Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) AOC 1020 - FSHD, FSHD1 | Phase 2 | Active not recruiting | Avidity Biosciences, Inc. | 84 | 2024-07-25 | 2026-07-14 |
| Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1 SRP-1001 for Injection, Placebo - Muscular Dystrophy, Facioscapulohumeral | Phase 1 / Phase 2 | Enrolling by invitation | Sarepta Therapeutics, Inc. | 60 | 2024-06-19 | 2026-10-05 |
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Muscular Dystrophies (17) · Neuromuscular Diseases (12) · Myotonic Dystrophy (10) · Muscular Dystrophy, Duchenne (8) · Muscular Dystrophies, Limb-Girdle (6) · Charcot-Marie-Tooth Disease (5) · Motor Activity (5) · Muscular Atrophy, Spinal (5) · Myositis, Inclusion Body (5) · Facioscapulohumeral Muscular Dystrophy 1B (4) · Lambert-Eaton Myasthenic Syndrome (4) · Myasthenia Gravis (4) · Polyradiculoneuropathy, Chronic Inflammatory Demyelinating (4) · Amyotrophic Lateral Sclerosis (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (75) · Muscular Diseases (75) · Muscular Disorders, Atrophic (75) · Nervous System Diseases (75) · Genetic Diseases, Inborn (73) · Musculoskeletal Diseases (72) · Neuromuscular Diseases (63) · Muscular Dystrophies (58) · Neurodegenerative Diseases (13) · Heredodegenerative Disorders, Nervous System (12) · Pathological Conditions, Signs and Symptoms (11) · Myotonic Disorders (10) · Congenital Abnormalities (8) · Genetic Diseases, X-Linked (8) · Pathologic Processes (8)
6-(5-((cyclopropylamino)carbonyl)-3-fluoro-2-methylphenyl)-N-(2,2-dimethylprpyl)-3-pyridinecarboxamide (3) · Exercise (2) · Injections (2) · Sodium Chloride (2) · Acceptance and Commitment Therapy (1) · Albuterol (1) · Antioxidants (1) · Clenbuterol (1) · Creatine (1) · Diet (1) · Electromyography (1) · Growth Hormone (1) · Hematologic Tests (1) · High-Energy Shock Waves (1) · Human Growth Hormone (1)
Centre Hospitalier Universitaire de Nice (5) · aTyr Pharma, Inc. (5) · Avidity Biosciences, Inc. (3) · Fulcrum Therapeutics (3) · LMU Klinikum (3) · University of Kansas Medical Center (3) · University of Rochester (3) · Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA (2) · Centre Hospitalier Universitaire de Liege (2) · Centre Hospitalier Universitaire de Saint Etienne (2) · Grete Andersen, MD (2) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (2) · Istanbul University - Cerrahpasa (2) · Radboud University Medical Center (2) · Centre for Human Drug Research, Netherlands (1)
United States (32) · France (16) · Italy (12) · Canada (11) · Netherlands (10) · United Kingdom (10) · Germany (8) · Australia (7) · Denmark (7) · Spain (7) · Belgium (3) · New Zealand (3) · Turkey (Türkiye) (3) · China (1) · Czechia (1)
Jeffrey Statland, MD (3) · Aleksandar Radunovic (1) · Amanda Hill, MBA (1) · Baziel van Engelen, MD, PhD (1) · Chad R Heatwole, MD, MS-CI (1) · Chiara Marini-Bettolo, MD, PhD (1) · Christopher Graham (1) · Doris G Leung, MD, PhD (1) · Enzo Ricci (1) · Havvanur Albayrak (1) · Houman Hemmati, MD (1) · Ian R Woodcock, MBBS (1)
PMID 16267188, PMID 16849828, PMID 16900450, PMID 20434605, PMID 22003934, PMID 25246239, PMID 25641525, PMID 27159994, PMID 27530735, PMID 28168524, PMID 29050397, PMID 29053898, PMID 29266323, PMID 30287669, PMID 30895627, PMID 30979860, PMID 31506080, PMID 32611642, PMID 34479574, PMID 35039091, PMID 35192788, PMID 36098990, PMID 36920465, PMID 38631764, PMID 39891610, PMID 40366059, PMID 40900971, PMID 41649965, PMID 41739307
2014 (2), 2015 (2), 2023 (1), P50AR065139 (1), U01NS101944 (1), U01NS137702 (1), U54NS048843 (1), UL1TR000001 (1)