4 completed, 2 recruiting, 2 terminated, 1 not yet recruiting. 7 Phase 2, 3 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Completed 4, Not yet recruiting 1, Recruiting 1, Terminated 1 |
| Phase 1 | 3 | Completed 2, Recruiting 1 |
| Phase 3 | 1 | Terminated 1 |
Follow Facioscapulohumeral Muscular Dystrophy (FSHD): new trials by email Literature Review of Facioscapulohumeral Muscular Dystrophy (FSHD) trials Research Report on Facioscapulohumeral Muscular Dystrophy (FSHD)
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 | 2026-10-23 |
| BetterLife FSHD: A Patient-driven Health and Research Platform Observational - FSH, FSH Muscular Dystrophy | Recruiting | FSHD Society | 5000 | 2024-08-02 | 2026-02-20 | |
| A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy Placebo, RO7204239 - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 2 | Completed | Hoffmann-La Roche | 51 | 2023-02-07 | 2026-10-15 |
| Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH) Losmapimod, Placebo oral tablet - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 3 | Terminated | Fulcrum Therapeutics | 260 | 2022-06-16 | 2025-11-10 |
| Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE) Losmapimod - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 2 | Terminated | Fulcrum Therapeutics | 76 | 2020-02-13 | 2025-09-30 |
| Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) Losmapimod oral tablet, Placebo oral tablet - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 2 | Completed | Fulcrum Therapeutics | 80 | 2019-08-09 | 2024-07-10 |
| Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy ATYR1940 - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 1 / Phase 2 | Completed | aTyr Pharma, Inc. | 8 | 2016-03-30 | 2023-10-19 |
| Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy Placebo, ATYR1940 - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 1 / Phase 2 | Completed | aTyr Pharma, Inc. | 20 | 2014-09-04 | 2021-08-11 |
Muscular Dystrophy, Facioscapulohumeral (9) · Muscular Dystrophies (3) · Musculoskeletal Diseases (3) · Neuromuscular Diseases (3) · Facioscapulohumeral Muscular Dystrophy 1B (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Muscular Diseases (9) · Muscular Disorders, Atrophic (9) · Nervous System Diseases (9) · Muscular Dystrophies (6) · Musculoskeletal Diseases (6) · Neuromuscular Diseases (6)
6-(5-((cyclopropylamino)carbonyl)-3-fluoro-2-methylphenyl)-N-(2,2-dimethylprpyl)-3-pyridinecarboxamide (3) · Injections (1) · Watchful Waiting (1)
Fulcrum Therapeutics (3) · aTyr Pharma, Inc. (2) · FSHD Society (1) · Hoffmann-La Roche (1) · Satellos Bioscience, Inc. (1) · Soufflé Therapeutics, Inc. (1)
United States (7) · France (5) · Italy (4) · Canada (3) · Spain (3) · Denmark (2) · Netherlands (2) · United Kingdom (2) · Australia (1) · Germany (1) · New Zealand (1)
Amanda Hill, MBA (1) · Marie-Helene Jouvin, MD (1) · Michelle Mellion, MD (1) · Sanjay Shukla, MD (1)
PMID 35039091, PMID 38631764, PMID 41649965
2014 (2)