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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: "gene therapy" Condition: Duchenne Muscular Dystrophy Clear all
Terminated Phase 3 Interventional

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

NCT05689164
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
fordadistrogene movaparvovec
Biological
Lead sponsor
Pfizer
Industry
Eligibility
0 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
12
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Jul 24, 2026, 5:44 AM 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 24, 2026, 5:44 AM 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 24, 2026, 5:44 AM EDT
Completed Phase 1 Interventional

Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

NCT00428935
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAV2.5-CMV-minidystrophin (d3990)
Biological
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
5 Years to 15 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 4, 2013 · Synced Jul 24, 2026, 5:44 AM EDT
Recruiting Phase 1 Interventional

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

NCT06224660
View directory record Official record
Conditions
DMD-Associated Dilated Cardiomyopathy
Interventions
SRD-001
Genetic
Lead sponsor
Sardocor Corp.
Industry
Eligibility
18 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Kansas City, Kansas • Cincinnati, Ohio • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 26, 2025 · Synced Jul 24, 2026, 5:44 AM 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 24, 2026, 5:44 AM EDT
Completed Phase 3 Interventional Results available

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

NCT05096221
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced Jul 24, 2026, 5:44 AM EDT
Terminated Phase 1 Interventional

A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

NCT06597656
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced Jul 24, 2026, 5:44 AM EDT
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 24, 2026, 5:44 AM EDT
Completed Phase 2 Interventional Results available

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

NCT01462292
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
GSK2402968 3mg/kg/week, GSK2402968 6 mg/kg/week, Placebo to match GSK2402968 3 mg/kg/week, Placebo to match GSK2402968 6 mg/kg/week
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
5 Years and older · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
14
States / cities
Sacramento, California • Stanford, California • Gulf Breeze, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2017 · Synced Jul 24, 2026, 5:44 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

NCT00264888
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PTC124
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jan 13, 2009 · Synced Jul 24, 2026, 5:44 AM EDT
Completed No phase listed Observational

This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy

NCT01098708
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
7 Years to 22 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 17, 2017 · Synced Jul 24, 2026, 5:44 AM EDT
Terminated Phase 2 Interventional

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
2 Years to 3 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Jul 24, 2026, 5:44 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

NCT02972580
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Genetic characterization
Genetic
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
250 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 26, 2024 · Synced Jul 24, 2026, 5:44 AM EDT
Terminated Phase 2 Interventional Results available

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

NCT01009294
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Ataluren, Chronic Corticosteroid Therapy
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years and older · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
5
States / cities
Davis, California • Boston, Massachusetts • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2020 · Synced Jul 24, 2026, 5:44 AM EDT
Completed Not applicable Interventional Results available

Cardiomyopathy in DMD: Lisinopril vs. Losartan

NCT01982695
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Cardiomyopathy
Interventions
Losartan, Lisinopril
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
6
States / cities
Davis, California • Kansas City, Kansas • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2017 · Synced Jul 24, 2026, 5:44 AM EDT
Enrolling by invitation No phase listed Observational

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

NCT06270719
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Delandistrogene Moxeparvovec, Standard of Care
Genetic · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2038
U.S. locations
25
States / cities
Little Rock, Arkansas • Los Angeles, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced Jul 24, 2026, 5:44 AM EDT
Recruiting No phase listed Observational

Molecular Analysis of Patients With Neuromuscular Disease

NCT00390104
View directory record Official record
Conditions
Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Week to 100 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 23, 2023 · Synced Jul 24, 2026, 5:44 AM EDT
Completed Phase 2 Interventional Results available

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00592553
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
15
States / cities
Sacramento, California • Aurora, Colorado • Pensacola, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2020 · Synced Jul 24, 2026, 5:44 AM EDT
Completed Phase 3 Interventional Results available

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT01826487
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years to 16 Years · Male only
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
21
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 17 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2020 · Synced Jul 24, 2026, 5:44 AM 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 10, 2025 · Synced Jul 24, 2026, 5:44 AM 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 24, 2026, 5:44 AM 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 24, 2026, 5:44 AM EDT
Not listed Phase 1 Interventional

Treatment of a Single Patient With CRD-TMH-001

NCT05514249
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
CRD-TMH-001
Drug
Lead sponsor
Cure Rare Disease, Inc
Other
Eligibility
18 Years to 28 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 31, 2022 · Synced Jul 24, 2026, 5:44 AM EDT