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ClinicalTrials.gov public records Last synced Oct 23, 2026, 7:38 PM EDT

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Showing 169–192 of 237 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Duchenne Muscular Dystrophy Clear all
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 23, 2026, 7:38 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 Sep 28, 2026 · Synced Oct 23, 2026, 7:38 PM EDT
Completed No phase listed Observational

Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy

NCT05280730
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
No intervention
Other
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Oct 23, 2026, 7:38 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Pediatric Radio Frequency Coils Generic

NCT01633866
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2031
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 23, 2026, 7:38 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

NCT03675126
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
7
States / cities
Hartford, Connecticut • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 23, 2026, 7:38 PM EDT
Completed Phase 1 Interventional

Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)

NCT02235844
View directory record Official record
Conditions
Duchenne's Muscular Dystrophy
Interventions
Umbilical Cord Mesenchymal Stem Cells
Biological
Lead sponsor
Allergy and Asthma Consultants, Wichita, Kansas
Other
Eligibility
28 Years to 31 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Wichita, Kansas
Source: ClinicalTrials.gov public record
Updated Sep 16, 2019 · Synced Oct 23, 2026, 7:38 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 24, 2005 · Synced Oct 23, 2026, 7:38 PM EDT
Terminated Phase 3 Interventional

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

NCT01803412
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
Drisapersen
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
10
States / cities
Sacramento, California • Tampa, Florida • Iowa City, Iowa + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2018 · Synced Oct 23, 2026, 7:38 PM EDT
Completed Phase 2 Interventional Results available

Weekly Steroids in Muscular Dystrophy

NCT04054375
View directory record Official record
Conditions
Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Prednisone
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Oct 23, 2026, 7:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

NCT02439216
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Edasalonexent, Placebo
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Orlando, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2022 · Synced Oct 23, 2026, 7:38 PM 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 18, 2017 · Synced Oct 23, 2026, 7:38 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 6, 2025 · Synced Oct 23, 2026, 7:38 PM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

NCT04060199
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 23, 2026, 7:38 PM EDT
Completed Phase 1 Interventional

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

NCT03375255
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
7
States / cities
Sacramento, California • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2022 · Synced Oct 23, 2026, 7:38 PM 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 18, 2017 · Synced Oct 23, 2026, 7:38 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 23, 2026, 7:38 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

NCT03433807
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years and older
U.S. locations
12
States / cities
Phoenix, Arizona • New Haven, Connecticut • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2023 · Synced Oct 23, 2026, 7:38 PM EDT
Recruiting Phase 1 Interventional

Trial of Cell Based Therapy for DMD

NCT06692426
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
MyoPAXon, Tacrolimus
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Oct 23, 2026, 7:38 PM EDT
Approved For Marketing No phase listed Expanded access

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

NCT02592941
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older
U.S. locations
42
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 36 more
Source: ClinicalTrials.gov public record
Updated Feb 28, 2018 · Synced Oct 23, 2026, 7:38 PM EDT
Active, not recruiting Phase 2 Interventional

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT05996003
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Exon 44, DMD
Interventions
NS-089/NCNP-02
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 14 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
11
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 23, 2026, 7:38 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 21, 2026 · Synced Oct 23, 2026, 7:38 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

NCT05524883
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
DYNE-251, Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
4 Years to 16 Years · Male only
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
11
States / cities
La Jolla, California • Los Angeles, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 23, 2026, 7:38 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Exon 44
Interventions
AOC 1044, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 55 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2025 · Synced Oct 23, 2026, 7:38 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants

NCT07317063
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
GRT6019
Drug
Lead sponsor
Grünenthal GmbH
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 13, 2026 · Synced Oct 23, 2026, 7:38 PM EDT