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Showing 1–24 of 474 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Myopathy; Hereditary Clear all
Completed No phase listed Observational

A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases

NCT01417533
View directory record Official record
Conditions
GNE Myopathy, GNE Related Diseases
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 22, 2026, 4:56 AM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Jul 22, 2026, 4:56 AM EDT
Not listed No phase listed Observational

Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

NCT01772043
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years and older · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
8
States / cities
Sacramento, California • Stanford, California • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2015 · Synced Jul 22, 2026, 4:56 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 22, 2026, 4:56 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias

NCT02413450
View directory record Official record
Conditions
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 85 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Jul 22, 2026, 4:56 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926, Placebo
Genetic · Other
Lead sponsor
Pfizer
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2039
U.S. locations
17
States / cities
Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Jul 22, 2026, 4:56 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study - Mitochondrial Disease

NCT01532791
View directory record Official record
Conditions
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
4 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jul 22, 2026, 4:56 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
SAR446268
Biological
Lead sponsor
Sanofi
Industry
Eligibility
10 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
4
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 22, 2026, 4:56 AM EDT
Terminated No phase listed Observational

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

NCT01753804
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Observational study
Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Sacramento, California • Cincinnati, Ohio • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Jul 22, 2026, 4:56 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

NCT02603562
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
16 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
4
States / cities
Stanford, California • Iowa City, Iowa • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2023 · Synced Jul 22, 2026, 4:56 AM EDT
Completed Phase 3 Interventional

High-dose Prednisone in Duchenne Muscular Dystrophy

NCT00110669
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisone
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years to 10 Years · Male only
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 26, 2011 · Synced Jul 22, 2026, 4:56 AM EDT
Terminated Phase 3 Interventional Results available

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

NCT03783923
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
8
States / cities
Atlanta, Georgia • Iowa City, Iowa • Kansas City, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2022 · Synced Jul 22, 2026, 4:56 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

NCT06907875
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
EPI-321
Biological
Lead sponsor
Epicrispr Biotechnologies, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Jul 22, 2026, 4:56 AM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jul 22, 2026, 4:56 AM EDT
Not listed Phase 1 Interventional

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2023 · Synced Jul 22, 2026, 4:56 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Tadalafil in Becker Muscular Dystrophy

NCT01070511
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 19, 2013 · Synced Jul 22, 2026, 4:56 AM EDT
Completed No phase listed Observational

The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

NCT01182324
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Jul 22, 2026, 4:56 AM EDT
Available No phase listed Expanded access

Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease

NCT06931834
View directory record Official record
Conditions
Mitochondrial Disease, Chronic Kidney Disease
Interventions
Probucol
Drug
Lead sponsor
RiboNova Inc.
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 16, 2025 · Synced Jul 22, 2026, 4:56 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 Jul 22, 2026, 4:56 AM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

NCT07038200
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1, FSHD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Dystrophy, Facio-Scapulo-Humeral Dystrophy, Facioscapulohumeral Muscular Dystrophy 2, Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1), FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Landouzy-Dejerine Syndrome
Interventions
AOC-1020, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years to 70 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
14
States / cities
Orange, California • Palo Alto, California • Denver, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jul 22, 2026, 4:56 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT06138639
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
0 Years to 17 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
12
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 22, 2026, 4:56 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 Jul 22, 2026, 4:56 AM EDT
Terminated Phase 3 Interventional

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

NCT03917719
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Edasalonexent
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
15
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 22, 2020 · Synced Jul 22, 2026, 4:56 AM EDT
Completed Not applicable Interventional Results available

Ultrasound in Muscle Biopsy

NCT01790178
View directory record Official record
Conditions
Myopathy, Myositis, Muscular Dystrophy
Interventions
Ultrasound
Procedure
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 1, 2017 · Synced Jul 22, 2026, 4:56 AM EDT