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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, 9:42 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
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 145–156 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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Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

NCT02420379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 6 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced Oct 25, 2026, 9:42 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

NCT01207908
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
IGF-1
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years and older · Male only
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 20, 2021 · Synced Oct 25, 2026, 9:42 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

NCT03039686
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
RO7239361, Placebo for RO7239361
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
6 Years to 11 Years · Male only
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
17
States / cities
Phoenix, Arizona • Palo Alto, California • Sacramento, California + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Oct 25, 2026, 9:42 AM EDT
Terminated Phase 2 Interventional Results available

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

NCT02310763
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06252616, Placebo
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
6 Years to 15 Years · Male only
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
30
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2020 · Synced Oct 25, 2026, 9:42 AM EDT
Terminated Phase 2 Interventional Results available

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

NCT02907619
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Oct 25, 2026, 9:42 AM EDT
Terminated Phase 3 Interventional

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

NCT03917719
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Edasalonexent
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
15
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Oct 25, 2026, 9:42 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

NCT02515669
View directory record Official record
Conditions
Muscular Dystrophy (DMD)
Interventions
RO7239361, Placebo
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
5 Years to 10 Years · Male only
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
10
States / cities
Los Angeles, California • Palo Alto, California • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2020 · Synced Oct 25, 2026, 9:42 AM 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 25, 2026, 9:42 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase I/II Study of SRP-4053 in DMD Patients

NCT02310906
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Placebo, SRP-4053
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
6 Years to 15 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 19, 2020 · Synced Oct 25, 2026, 9:42 AM EDT
Completed No phase listed Observational

Constipation and Gut Transit in DMD Patients

NCT01890616
View directory record Official record
Conditions
Constipation, DMD
Interventions
SmartPill ingestion
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years and older · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 16, 2020 · Synced Oct 25, 2026, 9:42 AM EDT
Terminated Phase 1Phase 2 Interventional

Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

NCT01847573
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
HT-100
Drug
Lead sponsor
Processa Pharmaceuticals
Industry
Eligibility
6 Years to 20 Years · Male only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2020 · Synced Oct 25, 2026, 9:42 AM EDT
Terminated Phase 2 Interventional

Open Label Extension Study of HT-100 in Patients With DMD

NCT01978366
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
HT-100
Drug
Lead sponsor
Processa Pharmaceuticals
Industry
Eligibility
6 Years to 20 Years · Male only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2020 · Synced Oct 25, 2026, 9:42 AM 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.