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ClinicalTrials.gov public records Last synced Oct 2, 2026, 7:53 PM EDT

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Showing 1–24 of 52 matching trials from the live ClinicalTrials.gov search.
Filters: "gene therapy" Condition: Duchenne Muscular Dystrophy Clear all
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 24, 2023 · Synced Oct 2, 2026, 7:53 PM 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 4, 2025 · Synced Oct 2, 2026, 7:53 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2

NCT02704325
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2, PLACEBO (Saline)
Biological · Other
Lead sponsor
Kevin Flanigan
Other
Eligibility
9 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 6, 2018 · Synced Oct 2, 2026, 7:53 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 2, 2026 · Synced Oct 2, 2026, 7:53 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 22, 2026 · Synced Oct 2, 2026, 7:53 PM 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 4, 2020 · Synced Oct 2, 2026, 7:53 PM EDT
Completed Phase 1 Interventional

Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons

NCT00451074
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Gentamicin infusions twice a week for six months
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
5 Years to 20 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Scottsdale, Arizona • Kansas City, Kansas • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 23, 2012 · Synced Oct 2, 2026, 7:53 PM 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 5, 2013 · Synced Oct 2, 2026, 7:53 PM 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 21, 2025 · Synced Oct 2, 2026, 7:53 PM 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 8, 2025 · Synced Oct 2, 2026, 7:53 PM EDT
Completed Phase 2 Interventional Results available

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

NCT02167217
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisolone
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Month to 30 Months · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
6
States / cities
Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2018 · Synced Oct 2, 2026, 7:53 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 23, 2026 · Synced Oct 2, 2026, 7:53 PM EDT
Terminated Phase 1 Interventional

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)

NCT04184882
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Bocidelpar, Placebo
Drug
Lead sponsor
Astellas Pharma Inc
Industry
Eligibility
8 Years to 16 Years · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
5
States / cities
Sacramento, California • Kansas City, Kansas • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Oct 2, 2026, 7:53 PM EDT
Active, not 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 Sep 16, 2026 · Synced Oct 2, 2026, 7:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

NCT03769116
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Los Angeles, California • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 2, 2026, 7:53 PM 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 14, 2009 · Synced Oct 2, 2026, 7:53 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 Aug 10, 2026 · Synced Oct 2, 2026, 7:53 PM 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 27, 2025 · Synced Oct 2, 2026, 7:53 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 2, 2026, 7:53 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 8, 2026 · Synced Oct 2, 2026, 7:53 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 2, 2026, 7:53 PM EDT
Completed Phase 1 Interventional

Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy

NCT02376816
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.micro-Dystrophin
Biological
Lead sponsor
Jerry R. Mendell
Other
Eligibility
7 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 24, 2017 · Synced Oct 2, 2026, 7:53 PM 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 22, 2026 · Synced Oct 2, 2026, 7:53 PM 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 16, 2017 · Synced Oct 2, 2026, 7:53 PM EDT