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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Muscular Dystrophies Clear all
Completed Phase 3 Interventional Results available

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren, PLACEBO
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
360 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
22
States / cities
Phoenix, Arizona • Los Angeles, California • Oakland, California + 19 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Jul 23, 2026, 6:07 AM EDT
Recruiting No phase listed Observational

Myotonic Dystrophy Family Registry

NCT02398786
View directory record Official record
Conditions
Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Dystrophia Myotonica, Dystrophia Myotonica 1, Dystrophia Myotonica 2, Myotonia Dystrophica, Myotonic Dystrophy, Congenital, Myotonic Myopathy, Proximal, PROMM (Proximal Myotonic Myopathy), Proximal Myotonic Myopathy, Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease, Myotonia Atrophica
Interventions
Patient-entered data
Other
Lead sponsor
Myotonic Dystrophy Foundation
Other
Eligibility
Not listed
Enrollment
3,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Nov 20, 2024 · Synced Jul 23, 2026, 6:07 AM EDT
Recruiting No phase listed Observational

The Duchenne Registry

NCT02069756
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier, Dystrophinopathy Female Carrier
Interventions
Not listed
Lead sponsor
The Duchenne Registry
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2047
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Jul 23, 2026, 6:07 AM EDT
Completed No phase listed Observational

Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy

NCT02153970
View directory record Official record
Conditions
Cerebral Palsy, Neuromuscular Disease
Interventions
Not listed
Lead sponsor
National Institute of Nursing Research (NINR)
NIH
Eligibility
8 Years and older
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 13, 2019 · Synced Jul 23, 2026, 6:07 AM 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 27, 2018 · Synced Jul 23, 2026, 6:07 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Sodium Nitrate to Improve Blood Flow

NCT02847975
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Sodium nitrate
Dietary Supplement
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
15 Years to 55 Years · Male only
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 5, 2018 · Synced Jul 23, 2026, 6:07 AM EDT
Completed No phase listed Observational

Evaluation and Diagnosis of People With Pain and Fatigue Syndromes

NCT00677157
View directory record Official record
Conditions
Fatigue
Interventions
Not listed
Lead sponsor
National Institute of Nursing Research (NINR)
NIH
Eligibility
18 Years and older
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 21, 2020 · Synced Jul 23, 2026, 6:07 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 9, 2025 · Synced Jul 23, 2026, 6:07 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Open-label Extension of the HOPE-2 Trial

NCT04428476
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deramiocel (CAP-1002)
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Sacramento, California • Aurora, Colorado • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 2, 2025 · Synced Jul 23, 2026, 6:07 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 10, 2025 · Synced Jul 23, 2026, 6:07 AM EDT
Recruiting No phase listed Observational

Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy

NCT07630389
View directory record Official record
Conditions
Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
0 Years to 17 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Jul 23, 2026, 6:07 AM 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, 6:07 AM 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 22, 2022 · Synced Jul 23, 2026, 6:07 AM EDT
Completed No phase listed Observational

Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

NCT01506518
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
5 Years to 13 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 1, 2021 · Synced Jul 23, 2026, 6:07 AM EDT
Active, not recruiting No phase listed Observational

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

NCT05683379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
AAV8 DetectCDx
Diagnostic Test
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
0 Years to 25 Years · Male only
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 23, 2026, 6:07 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 – 2029
U.S. locations
11
States / cities
La Jolla, California • Los Angeles, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2025 · Synced Jul 23, 2026, 6:07 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

NCT02972580
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Genetic characterization
Genetic
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
250 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 26, 2024 · Synced Jul 23, 2026, 6:07 AM EDT
Terminated No phase listed Observational

A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy

NCT04254172
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Activity Monitor
Device
Lead sponsor
Pfizer
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 14, 2021 · Synced Jul 23, 2026, 6:07 AM EDT
Completed No phase listed Observational

NSAA NON-Interventional Study Protocol

NCT06054971
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Remote Administration of NSAA
Other
Lead sponsor
Red Nucleus Enterprise Solutions, LLC
Industry
Eligibility
4 Years to 12 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Malvern, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Jul 23, 2026, 6:07 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Innovative Ultrasound Technology in Neuromuscular Disease

NCT02104921
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Muscular Dystrophy, Radiculopathy, Myopathy, Polyneuropathy and Mononeuropathies, Trauma Injury, Orthopedic Disorder
Interventions
Not listed
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 90 Years
Enrollment
223 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 10, 2022 · Synced Jul 23, 2026, 6:07 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Tadalafil as Adjuvant Therapy for DMD

NCT05195775
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Tadalafil
Drug
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 13 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 26, 2025 · Synced Jul 23, 2026, 6:07 AM EDT
Completed Phase 2 Interventional Results available

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03038399
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
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 19, 2021 · Synced Jul 23, 2026, 6:07 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Optimizing BCI-FIT: Brain Computer Interface - Functional Implementation Toolkit

NCT04468919
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Brainstem Stroke, Muscular Dystrophies, Parkinson's Disease and Parkinsonism, Multiple System Atrophy, Brain Tumor Adult, Spinal Cord Injuries, Locked-in Syndrome
Interventions
BCI-FIT multi-modal access, BCI-FIT adaptive signal modeling, BCI-FIT active querying, BCI-FIT language modeling
Behavioral
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years to 89 Years
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 15, 2025 · Synced Jul 23, 2026, 6:07 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 29, 2020 · Synced Jul 23, 2026, 6:07 AM EDT