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ClinicalTrials.gov public records Last synced Sep 24, 2026, 3:57 PM EDT

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Showing 1–24 of 240 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years to 15 Years · Male only
Enrollment
219 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
21
States / cities
Los Angeles, California • Sacramento, California • Stanford, California + 18 more
Completed Phase 4 Interventional Accepts healthy volunteers

Tadalafil in Becker Muscular Dystrophy

NCT01070511
Conditions
Becker Muscular Dystrophy
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
2
States / cities
Los Angeles, California
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
0 Years to 17 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
12
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 9 more
Conditions
Muscular Dystrophy, Duchenne
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
5 Years to 13 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Conditions
Duchenne Muscular Dystrophy
Interventions
Vesleteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
11
States / cities
Sacramento, California • Farmington, Connecticut • Gulf Breeze, Florida + 8 more
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Sildenafil
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.micro-Dystrophin
Biological
Lead sponsor
Jerry R. Mendell
Other
Eligibility
7 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Completed Not applicable Interventional Accepts healthy volunteers

Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD

NCT02834650
Conditions
Muscular Dystrophy, Duchenne
Interventions
Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test, Heart Rate, Pulmonary Function Test, Genetic Testing, Repeat MRI scan
Other
Lead sponsor
Stanford University
Other
Eligibility
7 Years to 21 Years · Male only
Enrollment
89 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2022
U.S. locations
3
States / cities
Los Angeles, California • Orange, California • Stanford, California
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
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT03836300
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Completed Not applicable Interventional Accepts healthy volunteers Results available

DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls

NCT01523964
Conditions
Duchenne Muscular Dystrophy
Interventions
Testing with EIM
Other
Lead sponsor
Dart Therapeutics. LLC
Other
Eligibility
3 Years to 12 Years · Male only
Enrollment
92 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012
U.S. locations
6
States / cities
Aurora, Colorado • Boston, Massachusetts • St Louis, Missouri + 3 more
Conditions
Augmentation, Augmentation Revision, General Breast Enlargement, Post-lactational Involution, Asymmetry
Interventions
Mentor Spectra/Becker 80 Breast Implant
Device
Lead sponsor
Mentor Worldwide, LLC
Industry
Eligibility
22 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
Santa Barbara, California
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2, PLACEBO (Saline)
Biological · Other
Lead sponsor
Kevin Flanigan
Other
Eligibility
9 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
7 Years to 22 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
St Louis, Missouri
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
8 Years to 18 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
5
States / cities
Sacramento, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2
Biological
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Muscular Dystrophy, Duchenne
Interventions
Oxatomide (tinset)
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
5 Years to 10 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
DMD-Associated Dilated Cardiomyopathy
Interventions
SRD-001
Genetic
Lead sponsor
Sardocor Corp.
Industry
Eligibility
18 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Kansas City, Kansas • Cincinnati, Ohio • Columbus, Ohio
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 30 Years · Male only
Enrollment
73 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Duchenne Muscular Dystrophy
Interventions
Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
4 Years to 9 Years · Male only
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
14
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 11 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Ketamine, Dexmedetomidine
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Muscular Dystrophy, Duchenne
Interventions
Coenzyme Q10
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
5 Years to 11 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
2
States / cities
Washington D.C., District of Columbia • St Louis, Missouri
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06252616
Biological
Lead sponsor
Pfizer
Industry
Eligibility
6 Years to 18 Years · Male only
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
26
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 9 more
Conditions
Genetic Disease, Communication
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
13 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland