3 completed, 2 active not recruiting, 2 recruiting, 1 terminated. 4 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 2, Completed 1, Terminated 1 |
| Phase 1 | 1 | Completed 1 |
| Phase 3 | 1 | Recruiting 1 |
Follow Facioscapulohumeral Muscular Dystrophy 1: new trials by email Literature Review of Facioscapulohumeral Muscular Dystrophy 1 trials Research Report on Facioscapulohumeral Muscular Dystrophy 1
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 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 |
| 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 to Evaluate the Efficacy and Safety of Satralizumab in FSHD1 Satralizumab Prefilled Syringe, Placebo Comparator - Facioscapulohumeral Muscular Dystrophy 1 | Phase 2 | Active not recruiting | Centre Hospitalier Universitaire de Nice | 46 | 2024-01-24 | 2025-03-26 |
| 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 | |
| Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) AOC 1020, Placebo - FSHD, FSHD1 | Phase 1 / Phase 2 | Completed | Avidity Biosciences, Inc. | 90 | 2023-04-04 | 2026-07-16 |
| Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory - Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1) | Completed | LMU Klinikum | 82 | 2021-04-15 | 2022-08-24 | |
| Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort Standard of care genetic testing group - Developmental Disability, Intellectual Disability | Unknown | Bionano Genomics | 1000 | 2020-11-30 | 2023-08-07 | |
| Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension Losmapimod - Facioscapulohumeral Muscular Dystrophy 1 | Phase 2 | Terminated | Fulcrum Therapeutics | 14 | 2019-08-23 | 2025-12-02 |
Muscular Dystrophy, Facioscapulohumeral (5) · Facioscapulohumeral Muscular Dystrophy 1B (4) · Facioscapulohumeral muscular dystrophy 1a (3) · Muscular Dystrophies (3) · Neuromuscular Diseases (2) · Autism Spectrum Disorder (1) · Congenital Abnormalities (1) · Developmental Disabilities (1) · Fragile X Syndrome (1) · Glycogen Storage Disease Type II (1) · Intellectual Disability (1) · Musculoskeletal Diseases (1) · Myositis, Inclusion Body (1) · Myotonic Dystrophy (1) · Nociceptive Pain (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (8) · Nervous System Diseases (8) · Muscular Diseases (7) · Muscular Disorders, Atrophic (7) · Musculoskeletal Diseases (6) · Neuromuscular Diseases (5) · Muscular Dystrophies (4) · Heredodegenerative Disorders, Nervous System (2) · Neurologic Manifestations (2) · Pathological Conditions, Signs and Symptoms (2) · Signs and Symptoms (2) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1)
Sodium Chloride (2) · 6-(5-((cyclopropylamino)carbonyl)-3-fluoro-2-methylphenyl)-N-(2,2-dimethylprpyl)-3-pyridinecarboxamide (1) · Vital Signs (1) · Walk Test (1) · Watchful Waiting (1)
Avidity Biosciences, Inc. (3) · Bionano Genomics (1) · Centre Hospitalier Universitaire de Nice (1) · FSHD Society (1) · Fulcrum Therapeutics (1) · Koç University (1) · LMU Klinikum (1)
United States (5) · Canada (4) · United Kingdom (3) · France (2) · Germany (2) · Netherlands (2) · Denmark (1) · Italy (1) · Japan (1) · Spain (1) · Turkey (Türkiye) (1)
Alka Chaubey, PhD, FACMG (1) · Amanda Hill, MBA (1) · Havvanur Albayrak (1) · Stephan Wenninger, PD Dr. med. (1)