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ClinicalTrials.gov public records Last synced Jul 22, 2026, 11:12 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Progressive Muscular Dystrophy Clear all
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 22, 2026, 11:12 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 27, 2018 · Synced Jul 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 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 22, 2026, 11:12 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 22, 2022 · Synced Jul 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 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 – 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM 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 22, 2026, 11:12 PM EDT
Recruiting No phase listed Observational

Natural History Study for Charcot Marie Tooth Disease

NCT05902351
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease
Interventions
Not listed
Lead sponsor
Hereditary Neuropathy Foundation
Government
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 30, 2024 · Synced Jul 22, 2026, 11:12 PM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 22, 2026, 11:12 PM EDT
Terminated Phase 2 Interventional

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
2 Years to 3 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Jul 22, 2026, 11:12 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced Jul 22, 2026, 11:12 PM EDT
Completed Phase 2 Interventional Results available

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

NCT00592553
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
15
States / cities
Sacramento, California • Aurora, Colorado • Pensacola, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2020 · Synced Jul 22, 2026, 11:12 PM EDT
Completed No phase listed Observational

A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD

NCT01437345
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
7
States / cities
Sacramento, California • Washington D.C., District of Columbia • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 10, 2017 · Synced Jul 22, 2026, 11:12 PM EDT