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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 the Clinical Trials Finder editorial team Guidance last reviewed June 10, 2026 Data synced July 23, 2026 Report an issue

ClinicalTrials.gov public records Last synced Jul 23, 2026, 4:26 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
52
Shown on this page
12
Current sort
Recently updated
Condition scope
Duchenne Muscular Dystrophy
Additional filters apply within this condition.
The trial list below is retrieved from the live ClinicalTrials.gov search API for Duchenne Muscular Dystrophy. Results on this page are currently sorted by Recently updated.

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 1–12 of 52 matching trials for Duchenne Muscular Dystrophy.
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Live official records

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Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926, Placebo
Genetic · Other
Lead sponsor
Pfizer
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2039
U.S. locations
17
States / cities
Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05693142
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
RGX-202
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Year and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
19
States / cities
Little Rock, Arkansas • Orange, California • Palo Alto, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Recruiting Phase 3 Interventional

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT07160634
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003, Placebo
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
7 Years to 11 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
3
States / cities
Little Rock, Arkansas • Flower Mound, Texas • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT06138639
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
0 Years to 17 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
12
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCT03368742
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-001
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
4 Years to 17 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
2
States / cities
Los Angeles, California • Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Active, not recruiting No phase listed Observational

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

NCT05683379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
AAV8 DetectCDx
Diagnostic Test
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
0 Years to 25 Years · Male only
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Recruiting No phase listed Observational

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978
View directory record Official record
Conditions
Duchene Muscular Dystrophy
Interventions
Not listed
Lead sponsor
ITF Therapeutics LLC
Industry
Eligibility
6 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
9
States / cities
Little Rock, Arkansas • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Jul 23, 2026, 4:26 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
7
States / cities
Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Recruiting Phase 1Phase 2 Interventional

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

NCT07429240
View directory record Official record
Conditions
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Interventions
PBGENE-DMD (IV)
Biological
Lead sponsor
Precision BioSciences, Inc.
Industry
Eligibility
2 Years to 7 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
2
States / cities
Little Rock, Arkansas • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Recruiting Phase 1 Interventional

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

NCT06817382
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
INS1201
Genetic
Lead sponsor
Insmed Gene Therapy LLC
Industry
Eligibility
2 Years to 4 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
10
States / cities
Little Rock, Arkansas • Davis, California • Los Angeles, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Recruiting No phase listed Observational

Duchenne Electronic Health Record Study

NCT07609394
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
Interventions
Observational study with patients who may be treated with various disease-modifying therapies
Other
Lead sponsor
The Duchenne Registry
Other
Eligibility
Not listed
Enrollment
2,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2072
U.S. locations
10
States / cities
Little Rock, Arkansas • Sacramento, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Jul 23, 2026, 4:26 PM EDT
Active, not recruiting Phase 3 Interventional

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

NCT05881408
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
14
States / cities
Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Jul 23, 2026, 4:26 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.