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ClinicalTrials.gov public records Last synced Sep 10, 2026, 5:13 AM EDT

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Showing 1–24 of 389 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Congenital Muscular Dystrophy Clear all
Active, not recruiting No phase listed Observational

Genetic and Physical Study of Childhood Nerve and Muscle Disorders

NCT01568658
View directory record Official record
Conditions
Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies, Inherited Neuromuscular Conditions
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Day to 100 Years
Enrollment
2,586 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 10, 2026, 5:13 AM EDT
Terminated Phase 1 Interventional Results available

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

NCT03362502
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
4 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
22
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 14, 2025 · Synced Sep 10, 2026, 5:13 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 10, 2026, 5:13 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect

NCT03627494
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
GSK3439171A, Placebo
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2020 · Synced Sep 10, 2026, 5:13 AM EDT
Not listed No phase listed Observational

Clinical Outcome Study for Dysferlinopathy

NCT01676077
View directory record Official record
Conditions
Dysferlinopathy, LGMD2B Now Classified as LGMDR2, Miyoshi Myopathy
Interventions
Not listed
Lead sponsor
Newcastle-upon-Tyne Hospitals NHS Trust
Other
Eligibility
10 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2024
U.S. locations
6
States / cities
Orange, California • Palo Alto, California • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 25, 2022 · Synced Sep 10, 2026, 5:13 AM EDT
Completed No phase listed Observational

Arrhythmias in Myotonic Muscular Dystrophy

NCT00622453
View directory record Official record
Conditions
Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
Interventions
Screening
Other
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
448 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2015
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 13, 2018 · Synced Sep 10, 2026, 5:13 AM 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 13, 2025 · Synced Sep 10, 2026, 5:13 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Home Based Assessments on Participants With DMD

NCT05657938
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Wearable Device, Duchenne Video Assessment (DVA)
Device · Other
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 13, 2023 · Synced Sep 10, 2026, 5:13 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 10, 2026, 5:13 AM EDT
Completed Phase 1 Interventional Results available

Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)

NCT01805024
View directory record Official record
Conditions
Congenital Muscular Dystrophy
Interventions
Omigapil
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
5 Years to 16 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 23, 2021 · Synced Sep 10, 2026, 5:13 AM EDT
Completed No phase listed Observational

Clinical Studies of Progeria

NCT00094393
View directory record Official record
Conditions
Progeria
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
6 Months to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 14, 2019 · Synced Sep 10, 2026, 5:13 AM EDT
Not yet recruiting Phase 4 Interventional

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

NCT07542314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 10, 2026, 5:13 AM EDT
Completed No phase listed Observational

Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03319030
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
2 Years to 17 Years · Male only
Enrollment
43 participants
Timeline
2017 – 2018
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 3, 2019 · Synced Sep 10, 2026, 5:13 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

NCT06839469
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 10, 2026, 5:13 AM EDT
Terminated Phase 2 Interventional Results available

Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

NCT04264442
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Losmapimod
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
12
States / cities
Irvine, California • Los Angeles, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Sep 10, 2026, 5:13 AM EDT
No Longer Available No phase listed Expanded access

HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.

NCT05154851
View directory record Official record
Conditions
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
Interventions
HB-adMSCs
Biological
Lead sponsor
Hope Biosciences Research Foundation
Industry
Eligibility
Not listed
U.S. locations
1
States / cities
Sugar Land, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Sep 10, 2026, 5:13 AM EDT
Active, not recruiting Not applicable Interventional

COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis

NCT05035823
View directory record Official record
Conditions
Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Spinal Cord Injuries, Stroke, Lacunar, Stroke Brainstem, Cervical Spinal Cord Injury, Spinal Muscular Atrophy, Tetraplegic; Paralysis, Quadriplegia/Tetraplegia
Interventions
Motor Neuroprosthesis (MNP)
Device
Lead sponsor
Synchron Medical, Inc.
Industry
Eligibility
21 Years to 75 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
3
States / cities
Buffalo, New York • New York, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 7, 2026 · Synced Sep 10, 2026, 5:13 AM EDT
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 – 2026
U.S. locations
10
States / cities
Aurora, Colorado • Kissimmee, Florida • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Sep 10, 2026, 5:13 AM EDT
Terminated Phase 3 Interventional Results available

A Study of Tadalafil for Duchenne Muscular Dystrophy

NCT01865084
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
7 Years to 14 Years · Male only
Enrollment
331 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
23
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 20 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2019 · Synced Sep 10, 2026, 5:13 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Enhance Wellness for Individuals With Long-Term Physical Disabilities

NCT02815878
View directory record Official record
Conditions
Multiple Sclerosis, Muscular Dystrophy, Post-polio Syndrome, Spinal Cord Injury
Interventions
Enhance Wellness
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
45 Years and older
Enrollment
240 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 2, 2018 · Synced Sep 10, 2026, 5:13 AM EDT
Active, not recruiting Phase 2 Interventional

Study of BBP-418 in Patients With LGMD2I

NCT04800874
View directory record Official record
Conditions
LGMD2I
Interventions
BBP-418
Drug
Lead sponsor
ML Bio Solutions, Inc.
Industry
Eligibility
12 Years to 55 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Sep 10, 2026, 5:13 AM EDT
Completed No phase listed Observational

Rasch-analysis of Clinical Severity in FSHD

NCT02766985
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Kansas City, Kansas • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 11, 2018 · Synced Sep 10, 2026, 5:13 AM EDT
Recruiting Phase 2 Interventional

Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

NCT07435129
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy, FSHD
Interventions
Apitegromab, Placebo
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
18 Years to 60 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 10, 2026, 5:13 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetic and Family Studies of Inherited Muscle Diseases

NCT00001331
View directory record Official record
Conditions
Dermatomyositis, Glycogen Storage Disease Type II, Glycogen Storage Disease Type VII, Myositis, Polymyositis
Interventions
Not listed
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Sep 10, 2026, 5:13 AM EDT