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ClinicalTrials.gov public records Last synced Sep 30, 2026, 2:00 PM EDT

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Showing 1–24 of 34 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Dystrophy, Facioscapulohumeral Muscular Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarker Development for Muscular Dystrophies

NCT05019625
View directory record Official record
Conditions
Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
465 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2028
U.S. locations
5
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 24, 2025 · Synced Sep 30, 2026, 2:00 PM EDT
Completed No phase listed Observational

Rasch-analysis of Clinical Severity in FSHD

NCT02766985
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Kansas City, Kansas • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 12, 2018 · Synced Sep 30, 2026, 2:00 PM EDT
Active, not recruiting Phase 2 Interventional

A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy

NCT05548556
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Placebo, RO7204239
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 65 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
4
States / cities
Orange, California • Aurora, Colorado • Fairway, Kansas + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy

NCT02239224
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Placebo, ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Rochester, New York • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 11, 2021 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 1Phase 2 Interventional

Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT05747924
View directory record Official record
Conditions
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
Interventions
AOC 1020, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years to 70 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
14
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension

NCT02531217
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 18, 2023 · Synced Sep 30, 2026, 2:00 PM EDT
Completed No phase listed Observational Results available

Bone Health in Facioscapulohumeral Muscular Dystrophy

NCT02413190
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
18 Years to 100 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 5, 2019 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Not applicable Interventional

Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)

NCT00027391
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
Albuterol, Oxandrolone
Drug
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years to 80 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Sep 30, 2026, 2:00 PM EDT
Recruiting Phase 1 Interventional

Clenbuterol to Target DUX4 in FSHD

NCT06721299
View directory record Official record
Conditions
Muscular Dystrophy, Facioscapulohumeral
Interventions
Clenbuterol
Drug
Lead sponsor
Jeffrey Statland
Other
Eligibility
18 Years to 75 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
Kansas City, Kansas • Rochester, New York • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Feb 25, 2026 · Synced Sep 30, 2026, 2:00 PM EDT
Recruiting Not applicable Interventional

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT07478172
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 30, 2026, 2:00 PM EDT
Recruiting Phase 1 Interventional

Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT07086521
View directory record Official record
Conditions
FSHD - Facioscapulohumeral Muscular Dystrophy
Interventions
ULSC, Placebo
Biological
Lead sponsor
Restem, LLC.
Industry
Eligibility
15 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 1Phase 2 Interventional

Study Evaluating MYO-029 in Adult Muscular Dystrophy

NCT00104078
View directory record Official record
Conditions
Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy
Interventions
MYO-029
Drug
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
18 Years and older
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
9
States / cities
Washington D.C., District of Columbia • Kansas City, Kansas • Baltimore, Maryland + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2007 · Synced Sep 30, 2026, 2:00 PM EDT
Terminated Phase 2 Interventional Results available

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)

NCT03943290
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Interventions
ACE-083
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
19
States / cities
Orange, California • Sacramento, California • Aurora, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2022 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02836418
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions
ATYR1940
Drug
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
16 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
3
States / cities
Irvine, California • Stanford, California • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Dec 22, 2023 · Synced Sep 30, 2026, 2:00 PM EDT
Completed No phase listed Observational

A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD

NCT01437345
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
7
States / cities
Sacramento, California • Washington D.C., District of Columbia • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 11, 2017 · Synced Sep 30, 2026, 2:00 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

NCT00082108
View directory record Official record
Conditions
Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 2, Congenital Myotonic Dystrophy, PROMM (Proximal Myotonic Myopathy), Steinert's Disease, Myotonic Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2028
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Sep 30, 2026, 2:00 PM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT06547216
View directory record Official record
Conditions
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
Interventions
AOC 1020
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years to 72 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
14
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT04003974
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Losmapimod oral tablet, Placebo oral tablet
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
12
States / cities
Irvine, California • Los Angeles, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

NCT02603562
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
16 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
4
States / cities
Stanford, California • Iowa City, Iowa • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2023 · Synced Sep 30, 2026, 2:00 PM EDT
Terminated Phase 2 Interventional Results available

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02927080
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
ACE-083, ACE-083 or placebo
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
18
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 15 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2022 · Synced Sep 30, 2026, 2:00 PM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

NCT05397470
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Losmapimod, Placebo oral tablet
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
260 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
14
States / cities
Irvine, California • Los Angeles, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 10, 2025 · Synced Sep 30, 2026, 2:00 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

NCT06907875
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
EPI-321
Biological
Lead sponsor
Epicrispr Biotechnologies, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 30, 2026, 2:00 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Sep 30, 2026, 2:00 PM EDT
Completed Phase 1 Interventional Results available

Study of Testosterone and rHGH in FSHD

NCT03123913
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Testosterone Enanthate, Somatropin
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated May 16, 2023 · Synced Sep 30, 2026, 2:00 PM EDT