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ClinicalTrials.gov public records Last synced Oct 2, 2026, 6:42 AM EDT

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Showing 1–24 of 35 matching trials from the live ClinicalTrials.gov search.
Filters: "exon skipping" Condition: Duchenne Muscular Dystrophy Clear all
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 2, 2026, 6:42 AM EDT
Available No phase listed Expanded access

Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

NCT07250737
View directory record Official record
Conditions
Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Dystrophies, Muscular Dystrophy, Duchenne
Interventions
delpacibart zotadirsen
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
2 Years and older · Male only
U.S. locations
21
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Irvine, California + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 2, 2026, 6:42 AM EDT
Terminated Phase 3 Interventional Results available

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

NCT03532542
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Casimersen, Golodirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 23 Years · Male only
Enrollment
171 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
25
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 21 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 2, 2026, 6:42 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 2, 2026, 6:42 AM EDT
Completed Phase 2 Interventional Results available

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

NCT04956289
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Oct 2, 2026, 6:42 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 27, 2026 · Synced Oct 2, 2026, 6:42 AM EDT
Completed Phase 1 Interventional

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

NCT03508947
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-210201, Placebo
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
5 Years to 18 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 8, 2019 · Synced Oct 2, 2026, 6:42 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 16, 2017 · Synced Oct 2, 2026, 6:42 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-050/NCNP-03, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 15 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Oct 2, 2026, 6:42 AM EDT
Enrolling by invitation No phase listed Observational

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

NCT06606340
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Eteplirsen, Golodirsen, Casimersen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2033
U.S. locations
20
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Oct 2, 2026, 6:42 AM EDT
Recruiting Phase 1Phase 2 Interventional

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

NCT04906460
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-N531
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
2
States / cities
Little Rock, Arkansas • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 2, 2026, 6:42 AM 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, 6:42 AM EDT
Recruiting No phase listed Observational

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

NCT07092540
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
0 Days to 3 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Rochester, New York • Stony Brook, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 2, 2026, 6:42 AM 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 25, 2021 · Synced Oct 2, 2026, 6:42 AM EDT
Terminated Phase 4 Interventional

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

NCT04708314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
Drug
Lead sponsor
Rare Disease Research, LLC
Other
Eligibility
7 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 3, 2026 · Synced Oct 2, 2026, 6:42 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

NCT03992430
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 13 Years · Male only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
3
States / cities
Birmingham, Alabama • Gainesville, Florida • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 2, 2026, 6:42 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 2, 2026 · Synced Oct 2, 2026, 6:42 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 11, 2025 · Synced Oct 2, 2026, 6:42 AM EDT
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 24, 2018 · Synced Oct 2, 2026, 6:42 AM 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 2, 2026, 6:42 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03907072
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-210201 (suvodirsen), Placebo
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
5 Years to 12 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
6
States / cities
New Haven, Connecticut • Atlanta, Georgia • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated May 20, 2021 · Synced Oct 2, 2026, 6:42 AM 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 27, 2026 · Synced Oct 2, 2026, 6:42 AM 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 2, 2026, 6:42 AM EDT
Terminated Phase 2 Interventional

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

NCT04004065
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vesleteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
11
States / cities
Sacramento, California • Farmington, Connecticut • Gulf Breeze, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced Oct 2, 2026, 6:42 AM EDT