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ClinicalTrials.gov public records Last synced Oct 9, 2026, 10:47 PM EDT

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Showing 73–96 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Muscular Dystrophies Clear all
Completed Phase 1Phase 2 Interventional

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

NCT02312011
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
IONIS-DMPKRx, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
20 Years to 55 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
8
States / cities
Stanford, California • Gainesville, Florida • Kansas City, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2022 · Synced Oct 9, 2026, 10:47 PM EDT
Terminated Phase 3 Interventional

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

NCT03917719
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Edasalonexent
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
15
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Oct 9, 2026, 10:47 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

NCT03675126
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
7
States / cities
Hartford, Connecticut • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 9, 2026, 10:47 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Where Does Hope Fit In? The Relationship Between Hope, Uncertainty, and Coping Efficacy in Mothers of Children With Duchenne/Becker Muscular Dystrophy

NCT02194725
View directory record Official record
Conditions
Stress
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
228 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 5, 2018 · Synced Oct 9, 2026, 10:47 PM EDT
Completed Phase 1 Interventional

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

NCT03375255
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
7
States / cities
Sacramento, California • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2022 · Synced Oct 9, 2026, 10:47 PM EDT
Completed Phase 2Phase 3 Interventional

Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy

NCT00016653
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Creatine Monohydrate, Glutamine
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
5 Years to 9 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 27, 2011 · Synced Oct 9, 2026, 10:47 PM EDT
Completed Phase 2 Interventional Results available

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

NCT01540409
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
AVI-4658 (Eteplirsen)
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 13 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
11
States / cities
Long Beach, California • Gainesville, Florida • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 9, 2026, 10:47 PM EDT
Completed No phase listed Observational

Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy

NCT02195999
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT), Metabolic Exercise Testing using stationary bicycle, Echocardiogram
Other
Lead sponsor
University of Florida
Other
Eligibility
5 Years to 15 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 8, 2019 · Synced Oct 9, 2026, 10:47 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Open-label Extension of the HOPE-2 Trial

NCT04428476
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deramiocel (CAP-1002)
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
5
States / cities
Sacramento, California • Aurora, Colorado • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 9, 2026, 10:47 PM EDT
Completed Phase 2 Interventional Results available

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

NCT02167217
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisolone
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Month to 30 Months · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
6
States / cities
Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2018 · Synced Oct 9, 2026, 10:47 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Influence of Endothelial Function on Central and Peripheral Causes Of Exercise Impairment in Type 2 Diabetes

NCT00786019
View directory record Official record
Conditions
Type 2 Diabetes
Interventions
Exercise program, Ascorbic Acid (Vitamin C)
Behavioral · Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
30 Years to 55 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2011
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 12, 2023 · Synced Oct 9, 2026, 10:47 PM EDT
Completed No phase listed Observational

Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy

NCT05280730
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
No intervention
Other
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Oct 9, 2026, 10:47 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Tadalafil as Adjuvant Therapy for DMD

NCT05195775
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Tadalafil
Drug
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 13 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 27, 2025 · Synced Oct 9, 2026, 10:47 PM EDT
Completed Phase 1 Interventional

Gentamicin Treatment of Muscular Dystrophy

NCT00005574
View directory record Official record
Conditions
Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
Interventions
Gentamicin
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 9, 2026, 10:47 PM EDT
Withdrawn Phase 2 Interventional

A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy

NCT02036463
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Prednisone, Placebo
Drug
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
3 Years to 6 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 18, 2015 · Synced Oct 9, 2026, 10:47 PM EDT
Recruiting Phase 1 Interventional

Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

NCT07172971
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
SGLT-2 inhibitor, SGLT2 inhibitor
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
8 Years to 18 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 9, 2026, 10:47 PM EDT
Terminated Phase 3 Interventional Results available

A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation

NCT03439514
View directory record Official record
Conditions
Dilated Cardiomyopathy, Lamin A/C Gene Mutation
Interventions
ARRY-371797 (PF-07265803), Placebo
Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
56
States / cities
Birmingham, Alabama • Mobile, Alabama • Chandler, Arizona + 26 more
Source: ClinicalTrials.gov public record
Updated Jan 9, 2024 · Synced Oct 9, 2026, 10:47 PM EDT
Terminated Phase 2 Interventional Results available

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

NCT02310763
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06252616, Placebo
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
6 Years to 15 Years · Male only
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
30
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2020 · Synced Oct 9, 2026, 10:47 PM EDT
Terminated Phase 2 Interventional Results available

Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

NCT04264442
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Losmapimod
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
12
States / cities
Irvine, California • Los Angeles, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2025 · Synced Oct 9, 2026, 10:47 PM EDT
Terminated No phase listed Observational

FSHD Molecular Characterization

NCT06096441
View directory record Official record
Conditions
Facio-Scapulo-Humeral Dystrophy
Interventions
Not listed
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
13 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 18, 2025 · Synced Oct 9, 2026, 10:47 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 Oct 9, 2026, 10:47 PM EDT
Recruiting Phase 2Phase 3 Interventional

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

NCT03373968
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Givinostat
Drug
Lead sponsor
Italfarmaco
Industry
Eligibility
7 Years and older · Male only
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
10
States / cities
Sacramento, California • San Diego, California • Hartford, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 9, 2026, 10:47 PM EDT
Terminated Phase 1 Interventional

Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

NCT06747273
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Interventions
SRP-9004
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
2
States / cities
Columbus, Ohio • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Oct 9, 2026, 10:47 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults

NCT04585464
View directory record Official record
Conditions
Healthy Volunteer, Becker Muscular Dystrophy
Interventions
EDG-5506, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
127 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jun 28, 2022 · Synced Oct 9, 2026, 10:47 PM EDT