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ClinicalTrials.gov public records Last synced May 21, 2026, 11:55 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 398 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Muscular Dystrophy Clear all
Terminated Phase 1 Interventional Results available
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Spironolactone, Prednisolone
Drug
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years to 7 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
4
States / cities
Iowa City, Iowa • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2023 · Synced May 21, 2026, 11:55 PM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 21, 2026, 11:55 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced May 21, 2026, 11:55 PM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Pentoxifylline
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years to 7 Years · Male only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Rochester, Minnesota • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2011 · Synced May 21, 2026, 11:55 PM EDT
Available No phase listed Expanded access
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
Biological
Lead sponsor
MED Institute Inc.
Industry
Eligibility
5 Years to 10 Years · Male only
U.S. locations
1
States / cities
Denton, Texas
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced May 21, 2026, 11:55 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Genetic Disorder
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced May 21, 2026, 11:55 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Muscular Dystrophies
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
4 Years to 65 Years
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Aurora, Colorado • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced May 21, 2026, 11:55 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-065/NCNP-01, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 9 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Sacramento, California • Gainesville, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2021 · Synced May 21, 2026, 11:55 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked in Syndrome, Muscular Dystrophy
Interventions
Placement of the BrainGate2 sensor(s) into the motor-related cortex
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2038
U.S. locations
5
States / cities
Sacramento, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced May 21, 2026, 11:55 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1), DM1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 88 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced May 21, 2026, 11:55 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
6 Years to 18 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
3
States / cities
Washington D.C., District of Columbia • St Louis, Missouri • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 10, 2013 · Synced May 21, 2026, 11:55 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
(+)- Epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
8 Years to 17 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 20, 2021 · Synced May 21, 2026, 11:55 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 21, 2026, 11:55 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 13 Years · Male only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
3
States / cities
Birmingham, Alabama • Gainesville, Florida • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced May 21, 2026, 11:55 PM EDT
Withdrawn No phase listed Observational
View directory record Official record
Conditions
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
Interventions
Not listed
Lead sponsor
Cure CMD
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
San Pedro, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2018 · Synced May 21, 2026, 11:55 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Bladder Cancer
Interventions
Gemcitabine, Carboplatin, Panitumumab, radical cystectomy
Drug · Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
5
States / cities
Basking Ridge, New Jersey • Commack, New York • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2016 · Synced May 21, 2026, 11:55 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Cardiomyopathy, Hypertrophic, Muscular Dystrophy, Oculopharyngeal
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 11:55 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
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 18, 2024 · Synced May 21, 2026, 11:55 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced May 21, 2026, 11:55 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome, Muscular Dystrophies
Interventions
BrainGate Neural Interface System
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Nov 30, 2025 · Synced May 21, 2026, 11:55 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years to 17 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced May 21, 2026, 11:55 PM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
Conditions
Congenital Muscular Dystrophy
Interventions
Omigapil
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
5 Years to 16 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 23, 2021 · Synced May 21, 2026, 11:55 PM EDT
Completed Phase 1Phase 2 Interventional Results available
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 18, 2023 · Synced May 21, 2026, 11:55 PM EDT