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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 23, 2026 Report an issue

ClinicalTrials.gov public records Last synced Oct 23, 2026, 6:14 AM 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

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 49–60 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

Open in full search
Recruiting Not applicable Interventional Accepts healthy volunteers

Urinary Titin Biomarker in DMD

NCT07332013
Conditions
Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
Interventions
Descending stair walk
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 10 Years · Male only
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Duchenne Muscular Dystrophy
Interventions
Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
Drug
Lead sponsor
Rare Disease Research, LLC
Other
Eligibility
7 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Pittsburgh, Pennsylvania
Enrolling by invitation No phase listed Observational

Long Term Follow-up for RGX-202

NCT06491927
Conditions
Duchenne Muscular Dystrophy
Interventions
No Intervention
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
Little Rock, Arkansas • Palo Alto, California • Chicago, Illinois + 2 more
Conditions
Duchenne Muscular Dystrophy Cardiomyopathy, Cardiomyopathy, Dilated
Interventions
Ifetroban, Placebo
Drug
Lead sponsor
Cumberland Pharmaceuticals
Industry
Eligibility
7 Years and older · Male only
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
10
States / cities
Little Rock, Arkansas • Los Angeles, California • Washington D.C., District of Columbia + 7 more
Completed Phase 2 Interventional Results available

Twice Weekly Steroids and Exercise as Therapy for DMD

NCT04322357
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
Drug · Behavioral
Lead sponsor
University of Florida
Other
Eligibility
5 Years to 9 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
2 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rockville, Maryland
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren, PLACEBO
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
360 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
22
States / cities
Phoenix, Arizona • Los Angeles, California • Oakland, California + 19 more
Conditions
Duchenne Muscular Dystrophy
Interventions
MyoPAXon, Tacrolimus
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
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
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
22
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 19 more
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

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.