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Showing 1–24 of 52 matching trials from the live ClinicalTrials.gov search.
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Filters: "gene therapy" Condition: Duchenne Muscular Dystrophy Clear all
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 13, 2024 · Synced Jul 23, 2026, 6:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

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

NCT03333590
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2
Biological
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 12, 2025 · Synced Jul 23, 2026, 6:48 PM EDT
Completed Phase 3 Interventional

A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)

NCT00018109
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
glutamine, creatine monohydrate
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
5 Years to 10 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 23, 2026, 6:48 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 22, 2012 · Synced Jul 23, 2026, 6:48 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, 6:48 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Urinary Titin Biomarker in DMD

NCT07332013
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
Interventions
Descending stair walk
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 10 Years · Male only
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Jul 23, 2026, 6:48 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, 6:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

NCT02354781
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAV1.CMV.huFollistin344
Biological
Lead sponsor
Jerry R. Mendell
Other
Eligibility
7 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 10, 2023 · Synced Jul 23, 2026, 6:48 PM EDT
Terminated Phase 1 Interventional Results available

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

NCT03362502
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
4 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
22
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 14, 2025 · Synced Jul 23, 2026, 6:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

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

NCT03375164
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
3 Months to 7 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Jul 23, 2026, 6:48 PM EDT
Completed Phase 1 Interventional Results available

Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

NCT00873782
View directory record Official record
Conditions
Muscular Dystrophies, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy
Interventions
Retrograde high pressure transvenous perfusion with normal saline
Other
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
21 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 8, 2015 · Synced Jul 23, 2026, 6:48 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 25, 2023 · Synced Jul 23, 2026, 6:48 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 5, 2018 · Synced Jul 23, 2026, 6:48 PM EDT
Completed Phase 3 Interventional Results available

Study of Eteplirsen in DMD Patients

NCT02255552
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 16 Years · Male only
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
37
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 32 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2021 · Synced Jul 23, 2026, 6:48 PM EDT
Terminated Phase 2 Interventional Results available

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

NCT00847379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
15
States / cities
Sacramento, California • Aurora, Colorado • Gulf Breeze, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2020 · Synced Jul 23, 2026, 6:48 PM EDT
Enrolling by invitation No phase listed Observational

Long Term Follow-up for RGX-202

NCT06491927
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
No Intervention
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
Little Rock, Arkansas • Palo Alto, California • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Jul 23, 2026, 6:48 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 23, 2017 · Synced Jul 23, 2026, 6:48 PM 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 28, 2020 · Synced Jul 23, 2026, 6:48 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, 6:48 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, 6:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

NCT01856868
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(-)-epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 21, 2021 · Synced Jul 23, 2026, 6:48 PM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

NCT06100887
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
6 Years to 17 Years · Male only
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 5, 2025 · Synced Jul 23, 2026, 6:48 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 20, 2018 · Synced Jul 23, 2026, 6:48 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, 6:48 PM EDT