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ClinicalTrials.gov public records Last synced Oct 1, 2026, 10:33 PM EDT

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Showing 25–48 of 237 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Duchenne/Becker Muscular Dystrophy Clear all
Enrolling by invitation No phase listed Observational

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

NCT06606340
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Eteplirsen, Golodirsen, Casimersen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2033
U.S. locations
20
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Oct 1, 2026, 10:33 PM EDT
Completed Phase 2Phase 3 Interventional

Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

NCT01126697
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions
Coenzyme Q10 and Lisinopril
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
8 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Chicago, Illinois • Charlotte, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2018 · Synced Oct 1, 2026, 10:33 PM EDT
Recruiting Phase 1Phase 2 Interventional

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

NCT04906460
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-N531
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
2
States / cities
Little Rock, Arkansas • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 1, 2026, 10:33 PM EDT
Completed No phase listed Observational

The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

NCT01182324
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 5, 2018 · Synced Oct 1, 2026, 10:33 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03439670
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone, Prednisone, Placebo
Drug · Other
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
10
States / cities
Davis, California • Los Angeles, California • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2023 · Synced Oct 1, 2026, 10:33 PM EDT
Completed Phase 2 Interventional Results available

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

NCT04179409
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Amondys 45, Exondys 51, Vyondys 53
Drug
Lead sponsor
Kevin Flanigan
Other
Eligibility
6 Months and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 26, 2023 · Synced Oct 1, 2026, 10:33 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 29, 2020 · Synced Oct 1, 2026, 10:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Limb-Girdle Muscular Dystrophy

NCT00893334
View directory record Official record
Conditions
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 1, 2026, 10:33 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 1, 2026, 10:33 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants

NCT07317063
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
GRT6019
Drug
Lead sponsor
Grünenthal GmbH
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 13, 2026 · Synced Oct 1, 2026, 10:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

NCT03680365
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management, Impairment, Rare Diseases
Interventions
Not listed
Lead sponsor
Jett Foundation, Inc.
Other
Eligibility
11 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 1, 2019 · Synced Oct 1, 2026, 10:33 PM EDT
Recruiting Phase 2 Interventional

Vasodilator and Exercise Study for DMD (VASO-REx)

NCT06290713
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children, Vasodilation, Exercise, DMD
Interventions
Tadalafil, Placebo, Exercise Training
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
6 Years and older · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 1, 2026, 10:33 PM EDT
Completed Phase 1 Interventional

Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons

NCT00451074
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Gentamicin infusions twice a week for six months
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
5 Years to 20 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Scottsdale, Arizona • Kansas City, Kansas • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 23, 2012 · Synced Oct 1, 2026, 10:33 PM EDT
Completed No phase listed Observational

Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

NCT01506518
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 2, 2021 · Synced Oct 1, 2026, 10:33 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 1, 2026, 10:33 PM EDT
Completed No phase listed Observational

NSAA NON-Interventional Study Protocol

NCT06054971
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Remote Administration of NSAA
Other
Lead sponsor
Red Nucleus Enterprise Solutions, LLC
Industry
Eligibility
4 Years to 12 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Malvern, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 21, 2025 · Synced Oct 1, 2026, 10:33 PM EDT
Completed Phase 1 Interventional

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

NCT01128855
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968, 12 mg/kg GSK2402968, Placebo
Drug · Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
9 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 18, 2017 · Synced Oct 1, 2026, 10:33 PM EDT
Completed Phase 2 Interventional Results available

(-)- Epicatechin Becker Muscular Dystrophy

NCT03236662
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(-)-Epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 70 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2021 · Synced Oct 1, 2026, 10:33 PM EDT
Active, not recruiting Phase 4 Interventional

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

NCT04687020
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Sacramento, California • Chicago, Illinois • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2022 · Synced Oct 1, 2026, 10:33 PM EDT
Terminated Phase 3 Interventional

Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

NCT03603288
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
idebenone 150 mg film-coated tablets
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
11 Years and older · Male only
Enrollment
161 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
13
States / cities
Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2021 · Synced Oct 1, 2026, 10:33 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy

NCT07642635
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Semaglutide (Rybelsus®), Placebo
Drug · Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Oct 1, 2026, 10:33 PM EDT
Recruiting Phase 1 Interventional

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

NCT04626674
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
2 Years and older · Male only
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
12
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 1, 2026, 10:33 PM EDT
Terminated Phase 2 Interventional

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
2 Years to 3 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 1, 2026, 10:33 PM EDT
Completed Phase 2 Interventional

An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy

NCT00033189
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 16, 2010 · Synced Oct 1, 2026, 10:33 PM EDT