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Showing 1–24 of 232 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Duchenne Disease Clear all
No Longer Available No phase listed Expanded access

Extension Study of Drisapersen in DMD Subjects

NCT02636686
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Drisapersen
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years to 80 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 23, 2018 · Synced Sep 18, 2026, 1:21 AM EDT
Recruiting No phase listed Observational

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

NCT06564974
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone
Drug
Lead sponsor
Catalyst Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older · Male only
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
31
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 18, 2026, 1:21 AM EDT
Active, not recruiting Phase 2 Interventional

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

NCT06244082
View directory record Official record
Conditions
DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
Interventions
AOC 1044
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 27 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Sep 18, 2026, 1:21 AM 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 15, 2026 · Synced Sep 18, 2026, 1:21 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 Sep 18, 2026, 1:21 AM EDT
Completed Phase 1 Interventional

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

NCT01128855
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968, 12 mg/kg GSK2402968, Placebo
Drug · Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
9 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 17, 2017 · Synced Sep 18, 2026, 1:21 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

NCT01484678
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
5 Years to 62 Years · Male only
Enrollment
550 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
3
States / cities
Gainesville, Florida • Portland, Oregon • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Sep 18, 2026, 1:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Wearable Technology to Assess Gait Function in SMA and DMD

NCT04193085
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2024 · Synced Sep 18, 2026, 1:21 AM 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 Sep 18, 2026, 1:21 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

NCT05540860
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
4 Years to 9 Years · Male only
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
14
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 18, 2026, 1:21 AM EDT
Active, not recruiting Phase 3 Interventional

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT05126758
View directory record Official record
Conditions
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
Interventions
Deramiocel (CAP-1002), Placebo
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older · Male only
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
20
States / cities
Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 17 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 18, 2026, 1:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Limb-Girdle Muscular Dystrophy

NCT00893334
View directory record Official record
Conditions
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 6, 2014 · Synced Sep 18, 2026, 1:21 AM 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 Sep 18, 2026, 1:21 AM 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 Sep 18, 2026, 1:21 AM EDT
Recruiting Phase 3 Interventional

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

NCT07608432
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
Interventions
Zeleciment Rostudirsen (DYNE-251), Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Hillsborough, North Carolina
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Sep 18, 2026, 1:21 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 Sep 18, 2026, 1:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Burden of Access in Duchenne Muscular Dystrophy in the US

NCT03951675
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 99 Years
Enrollment
57 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 5, 2020 · Synced Sep 18, 2026, 1:21 AM EDT
Completed Phase 3 Interventional

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT02500381
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SRP-4045, SRP-4053, Placebo
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
6 Years to 13 Years · Male only
Enrollment
228 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
23
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2025 · Synced Sep 18, 2026, 1:21 AM EDT
Terminated Phase 2 Interventional

HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

NCT02525302
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
HT-100
Drug
Lead sponsor
Akashi Therapeutics
Industry
Eligibility
6 Years to 20 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
5
States / cities
Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2019 · Synced Sep 18, 2026, 1:21 AM EDT
Completed Phase 2 Interventional Results available

Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

NCT01396239
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
AVI-4658 (Eteplirsen), Placebo
Drug · Other
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 13 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 29, 2020 · Synced Sep 18, 2026, 1:21 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 Sep 18, 2026, 1:21 AM EDT
Completed Phase 2 Interventional Results available

An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02760277
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day, Vamorolone 6.0 mg/day/day
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2019 · Synced Sep 18, 2026, 1:21 AM EDT
Completed No phase listed Observational

Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

NCT02034305
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
6 Years to 21 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 4, 2024 · Synced Sep 18, 2026, 1:21 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 2, 2020 · Synced Sep 18, 2026, 1:21 AM EDT