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Duchenne Muscular Dystrophy clinical trials

Directory of U.S. ClinicalTrials.gov public records for Duchenne Muscular Dystrophy. Browse Duchenne Muscular Dystrophy recruiting trials, filter by phase, sponsor, location, or intervention, and open the official ClinicalTrials.gov study record for each result.

By Clinical Trials Finder Guidance last reviewed June 10, 2026 Data synced October 25, 2026 Report an issue

ClinicalTrials.gov public records Last synced Oct 25, 2026, 7:40 PM EDT

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

Official matching trials
237
Shown on this page
12
Current sort
Recently updated
Condition scope
Duchenne Muscular Dystrophy
Additional filters apply within this condition.

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

How Duchenne Muscular Dystrophy studies are organized in the registry

Duchenne records are often titled with just “Duchenne” or the abbreviation DMD, and much of the portfolio is organized around a therapeutic platform — gene transfer, exon skipping — which appears in titles. Because Duchenne is diagnosed in childhood, the age filter meaningfully changes what you see.

Terms that often appear in related listings
DMD exon skipping gene therapy ambulatory

These describe how studies tend to be titled and grouped on ClinicalTrials.gov — they are search vocabulary, not medical guidance.

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These controls apply only within Duchenne Muscular Dystrophy. Default order is Recently updated. Add location, sponsor, treatment, or recruitment status to narrow this condition page further.

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Showing 181–192 of 237 matching trials for Duchenne Muscular Dystrophy.
Live ClinicalTrials.gov results.
Local aggregate panels are unavailable or hidden for filtered views. The result list above identifies its data source.

Live official records

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Terminated Phase 2 Interventional

HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

NCT02525302
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
HT-100
Drug
Lead sponsor
Akashi Therapeutics
Industry
Eligibility
6 Years to 20 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
5
States / cities
Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2019 · Synced Oct 25, 2026, 7:40 PM EDT
Completed Not applicable Interventional

Strength Training in Duchenne Muscular Dystrophy

NCT02421523
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Aim 2 Exercise group, Aim 1 Exercise Dosing, Aim 2 Control group
Procedure
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 10 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 6, 2019 · Synced Oct 25, 2026, 7:40 PM EDT
Terminated Phase 2 Interventional Results available

Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy

NCT01168908
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 25, 2026, 7:40 PM EDT
Completed No phase listed Observational

Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03319030
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
2 Years to 17 Years · Male only
Enrollment
43 participants
Timeline
2017 – 2018
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 4, 2019 · Synced Oct 25, 2026, 7:40 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02760264
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day, Vamorolone 6.0 mg/kg/day
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 6 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 2, 2019 · Synced Oct 25, 2026, 7:40 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 25, 2026, 7:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Sodium Nitrate to Improve Blood Flow

NCT02847975
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Sodium nitrate
Dietary Supplement
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
15 Years to 55 Years · Male only
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 6, 2018 · Synced Oct 25, 2026, 7:40 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 25, 2026, 7:40 PM EDT
Not listed No phase listed Observational

Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

NCT01539772
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years and older · Male only
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
13
States / cities
Sacramento, California • Washington D.C., District of Columbia • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2018 · Synced Oct 25, 2026, 7:40 PM EDT
Terminated Phase 3 Interventional

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

NCT01803412
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
Drisapersen
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
10
States / cities
Sacramento, California • Tampa, Florida • Iowa City, Iowa + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2018 · Synced Oct 25, 2026, 7:40 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 25, 2026, 7:40 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 25, 2026, 7:40 PM EDT

About this Duchenne Muscular Dystrophy clinical trials directory page

What this page lists
U.S. ClinicalTrials.gov public records that list Duchenne Muscular Dystrophy as a study condition, including recruiting, not yet recruiting, and active not recruiting trials across phase 1, phase 2, and phase 3.
How to narrow results
Use the filters above to scope by intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, age, or NCT ID within Duchenne Muscular Dystrophy.
Where the data comes from
Records are sourced from the official ClinicalTrials.gov public API. Each result links back to the official study page, which is the source of truth for eligibility, enrollment, and contact information.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, ranking of trials by quality, or AI-generated medical summaries.

Related: all condition pages, browse by drug or therapy, browse by U.S. city, browse by sponsor, or open the full search for Duchenne Muscular Dystrophy.