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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Myopathy; Hereditary Clear all
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, 1:55 PM 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, 1:55 PM EDT
Completed Not applicable Interventional

Clinical Trial of a Serious Game for Individuals With SCI/D

NCT02341950
View directory record Official record
Conditions
Spinal Cord Injury, Spinal Cord Involvement, Spina Bifida, Transverse Myelitis, Polio and Post-polio Syndrome, Syringomyelia, Spinal Stenosis, Spinal Neoplasms, Spinal Cord Diseases
Interventions
SCI Hard
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
13 Years to 29 Years
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Feb 21, 2019 · Synced Jul 22, 2026, 1:55 PM 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 7, 2019 · Synced Jul 22, 2026, 1:55 PM EDT
Terminated Phase 2 Interventional Results available

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy

NCT03400852
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
MNK-1411, Placebo
Drug · Other
Lead sponsor
Mallinckrodt ARD LLC
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
5
States / cities
Gulf Breeze, Florida • Atlanta, Georgia • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 15, 2021 · Synced Jul 22, 2026, 1:55 PM EDT
Withdrawn Phase 3 Interventional

A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants

NCT06952686
View directory record Official record
Conditions
Muscular Dystrophies, Limb-Girdle
Interventions
SRP-9005, Corticosteroid
Biological · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 5, 2025 · Synced Jul 22, 2026, 1:55 PM EDT
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 22, 2026, 1:55 PM 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 22, 2026, 1:55 PM 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 22, 2026, 1:55 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, 1:55 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

NCT04851873
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Up to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Jul 22, 2026, 1:55 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, 1:55 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, 1:55 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, 1:55 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, 1:55 PM 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 22, 2026, 1:55 PM EDT
Completed Phase 2 Interventional

Study of the Safety and Tolerability Associated With PPD10558 Versus Atorvastatin in Patients Previously Intolerant to Statins Due to Statin-associated Myalgia (SAM)

NCT01279590
View directory record Official record
Conditions
Myalgia, Hypercholesterolemia, Hyperlipidemia
Interventions
PPD10558, Atorvastatin, Placebo
Drug
Lead sponsor
Furiex Pharmaceuticals, Inc
Industry
Eligibility
40 Years and older
Enrollment
282 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
67
States / cities
Anniston, Alabama • Phoenix, Arizona • Huntington Park, California + 60 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2011 · Synced Jul 22, 2026, 1:55 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, 1:55 PM EDT
Recruiting Phase 1 Interventional

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

NCT06371417
View directory record Official record
Conditions
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP)
Interventions
RAY121
Drug
Lead sponsor
Chugai Pharmaceutical
Industry
Eligibility
18 Years to 85 Years
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
11
States / cities
Orange, California • Baltimore, Maryland • Lake Success, New York + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Jul 22, 2026, 1:55 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, 1:55 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, 1:55 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, 1:55 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, 1:55 PM EDT
Completed Phase 1Phase 2 Interventional

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

NCT06051448
View directory record Official record
Conditions
Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
Interventions
Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced Jul 22, 2026, 1:55 PM EDT