16 completed, 7 recruiting, 4 active not recruiting, 2 terminated. 8 Phase 2, 6 Not applicable, 6 Phase 1, 1 Phase 3.
Follow Facioscapulohumeral Muscular Dystrophy: new trials by email Literature Review of Facioscapulohumeral Muscular Dystrophy trials Research Report on Facioscapulohumeral Muscular Dystrophy
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| BetterLife FSHD: A Patient-driven Health and Research Platform Observational - FSH, FSH Muscular Dystrophy | Recruiting | FSHD Society | 5000 | 2024-08-02 | 2026-02-20 | |
| Quantitative Assessment of Orofacial Muscle Function in FSHD Orofacial function assessment tests - Facioscapulohumeral Muscular Dystrophy | Not applicable | Completed | University of Mons | 22 | 2024-05-14 | 2026-02-27 |
| Unraveling Metabolic Involvement in Facioscapulohumeral Dystrophy Through Metabolomics metabolomic on plasma sample - Facioscapulohumeral Muscular Dystrophy | Unknown | University Hospital, Angers | 120 | 2024-01-01 | 2023-10-17 | |
| Ultrasound Detection of Early Facial Muscle Changes in FSHD: Thickness and Echo Intensity Findings Facioscapulohumeral Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy 1 | Completed | Koç University | 39 | 2023-09-01 | 2026-01-09 | |
| ADVANCED FSHD-COM: New Clinical Outcome Measures to Evaluate Non-ambulant FSHD Patients, a Pilot Study Validation of new COMs for non ambulant FSHD patients - Facioscapulohumeral Muscular Dystrophy | Not applicable | Active not recruiting | Centre Hospitalier Universitaire de Nice | 30 | 2023-04-03 | 2025-09-25 |
| Effectiveness of Upper Extremity Rehabilitation in pwFSHD (Patient With Facioscapulohumeral Dystrophia) upper extremity rehabilitation program, Results of assessment - Facioscapulohumeral Muscular Dystrophy | Completed | Istanbul University - Cerrahpasa | 36 | 2022-02-22 | 2023-10-12 | |
| Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) ACE-083 - Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease | Phase 2 | Terminated | Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA | 62 | 2019-05-10 | 2022-09-26 |
| Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD FSHD-specific functional rating scale, Electrical Impedance Myography - Facioscapulohumeral Muscular Dystrophy | Active not recruiting | University of Kansas Medical Center | 324 | 2018-03-05 | 2026-09-17 | |
| Study of Testosterone and rHGH in FSHD Testosterone Enanthate, Somatropin - Facioscapulohumeral Muscular Dystrophy | Phase 1 | Completed | University of Rochester | 20 | 2017-12-18 | 2023-05-16 |
| Arm Cycling in Facioscapulohumeral Dystrophy (FSHD) Patients Arm cycling - Facioscapulohumeral Muscular Dystrophy | Not applicable | Completed | Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust | 15 | 2016-12-14 | 2020-02-12 |
| Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) ACE-083, ACE-083 or placebo - Facioscapulohumeral Muscular Dystrophy | Phase 2 | Terminated | Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA | 95 | 2016-11-01 | 2022-09-26 |
| Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD) ATYR1940 - Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy | Phase 1 / Phase 2 | Completed | aTyr Pharma, Inc. | 8 | 2016-07-13 | 2023-12-22 |
| Rasch-analysis of Clinical Severity in FSHD Facioscapulohumeral Muscular Dystrophy | Completed | University of Kansas Medical Center | 44 | 2016-03-01 | 2018-04-12 | |
| Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD) ATYR1940, Placebo - Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy | Phase 1 / Phase 2 | Completed | aTyr Pharma, Inc. | 18 | 2015-11-30 | 2023-10-19 |
| Facioscapulohumeral Dystrophy in Children Neurological Observations, Facioscapulohumeral Muscular Dystrophy | Completed | University Medical Center Nijmegen | 32 | 2015-11-01 | 2019-09-26 | |
| Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension ATYR1940 - Facioscapulohumeral Muscular Dystrophy | Phase 1 / Phase 2 | Completed | aTyr Pharma, Inc. | 9 | 2015-08-13 | 2023-08-18 |
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Muscular Dystrophy, Facioscapulohumeral (30) · Muscular Dystrophies (6) · Myotonic Dystrophy (4) · Muscular Dystrophies, Limb-Girdle (3) · Muscular Dystrophy, Duchenne (3) · Neuromuscular Diseases (3) · Charcot-Marie-Tooth Disease (2) · Facioscapulohumeral Muscular Dystrophy 1B (2) · Acquired Immunodeficiency Syndrome (1) · Alopecia Areata (1) · Anemia, Sickle Cell (1) · Arthritis, Psoriatic (1) · Asthma (1) · Bipolar Disorder (1) · Breast Neoplasms (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (30) · Genetic Diseases, Inborn (30) · Muscular Diseases (30) · Muscular Disorders, Atrophic (30) · Musculoskeletal Diseases (30) · Nervous System Diseases (30) · Neuromuscular Diseases (27) · Muscular Dystrophies (24) · Heredodegenerative Disorders, Nervous System (5) · Neurodegenerative Diseases (5) · Myotonic Disorders (4) · Congenital Abnormalities (3) · Genetic Diseases, X-Linked (3) · Peripheral Nervous System Diseases (3) · Polyneuropathies (3)
Antioxidants (1) · Exercise (1) · Growth Hormone (1) · Human Growth Hormone (1) · Sodium Chloride (1) · Stamulumab (1) · Testosterone Propionate (1) · Transcutaneous Electric Nerve Stimulation (1) · Watchful Waiting (1) · apitegromab (1) · testosterone enanthate (1)
aTyr Pharma, Inc. (3) · Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA (2) · Centre Hospitalier Universitaire de Nice (2) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (2) · University of Kansas Medical Center (2) · University of Rochester (2) · Avidity Biosciences, Inc. (1) · Cooperative International Neuromuscular Research Group (1) · Epicrispr Biotechnologies, Inc. (1) · FSHD Society (1) · Istanbul University - Cerrahpasa (1) · Koç University (1) · Massachusetts General Hospital (1) · Newcastle University (1) · Oslo University Hospital (1)
United States (19) · France (6) · Canada (4) · Italy (4) · Netherlands (4) · United Kingdom (4) · Australia (3) · Denmark (3) · Spain (3) · Germany (2) · Turkey (Türkiye) (2) · Belgium (1) · Japan (1) · New Zealand (1) · Norway (1)
Jeffrey Statland, MD (2) · Amanda Hill, MBA (1) · Baziel van Engelen, MD, PhD (1) · Chad R Heatwole, MD, MS-CI (1) · Chiara Marini-Bettolo, MD, PhD (1) · Doris G Leung, MD, PhD (1) · Havvanur Albayrak (1) · Houman Hemmati, MD (1) · Ipek Yeldan Karagoz, PhD (1) · Jean K Mah, MD, MS (1) · Johann Hamel, MD (1) · Rabi Tawil, MD (1)
PMID 20434605, PMID 25246239, PMID 25641525, PMID 27159994, PMID 27530735, PMID 29053898, PMID 29266323, PMID 30895627, PMID 31506080, PMID 40900971
2015 (2), U01NS101944 (1), U54NS048843 (1), UL1TR000001 (1)