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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 5, 2026 Report an issue

ClinicalTrials.gov public records Last synced Oct 5, 2026, 3:23 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
35
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 13–24 of 35 matching trials for Duchenne Muscular Dystrophy.
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Live official records

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Completed Phase 3 Interventional

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT02500381
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SRP-4045, SRP-4053, Placebo
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
6 Years to 13 Years · Male only
Enrollment
228 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
23
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Oct 5, 2026, 3:23 PM EDT
Completed Phase 1Phase 2 Interventional Results available

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

NCT04240314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
scAAV9.U7.ACCA
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
6 Months to 13 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 5, 2026, 3:23 PM EDT
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 5, 2026, 3:23 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Exon 44
Interventions
AOC 1044, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 55 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2025 · Synced Oct 5, 2026, 3:23 PM EDT
Terminated Phase 2 Interventional

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

NCT04004065
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced Oct 5, 2026, 3:23 PM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

NCT04060199
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 5, 2026, 3:23 PM EDT
Terminated Phase 3 Interventional Results available

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

NCT03532542
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Casimersen, Golodirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 23 Years · Male only
Enrollment
171 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
25
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 21 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 5, 2026, 3:23 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

NCT03675126
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 19, 2024 · Synced Oct 5, 2026, 3:23 PM EDT
Completed Phase 2 Interventional Results available

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

NCT04956289
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Oct 5, 2026, 3:23 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 5, 2026, 3:23 PM EDT
Completed Phase 1 Interventional

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

NCT03375255
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
7
States / cities
Sacramento, California • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2022 · Synced Oct 5, 2026, 3:23 PM EDT
Completed Phase 2 Interventional Results available

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02740972
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 7, 2021 · Synced Oct 5, 2026, 3:23 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.