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ClinicalTrials.gov public records Last synced Sep 17, 2026, 9:40 AM EDT

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Showing 1–24 of 473 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Congenital Myopathy Clear all
Recruiting No phase listed Observational

BetterLife FSHD: A Patient-driven Health and Research Platform

NCT07409142
View directory record Official record
Conditions
FSH, FSH Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1, FSHD, FSHD2, Facioscapulohumeral Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facioscapulohumeral Muscular Dystrophy (FSHD), Muscular Dystrophy, Facioscapulohumeral
Interventions
Observational
Other
Lead sponsor
FSHD Society
Other
Eligibility
1 Year and older
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2060
U.S. locations
1
States / cities
Randolph, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
No Longer Available No phase listed Expanded access

Extension Study of Drisapersen in DMD Subjects

NCT02636686
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Drisapersen
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years to 80 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 23, 2018 · Synced Sep 17, 2026, 9:40 AM EDT
Recruiting No phase listed Observational

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

NCT06564974
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone
Drug
Lead sponsor
Catalyst Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older · Male only
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
31
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Active, not recruiting Phase 2 Interventional

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

NCT06244082
View directory record Official record
Conditions
DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
Interventions
AOC 1044
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 27 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Active, not recruiting Phase 1 Interventional

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

NCT06817382
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
INS1201
Genetic
Lead sponsor
Insmed Gene Therapy LLC
Industry
Eligibility
2 Years to 4 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
10
States / cities
Little Rock, Arkansas • Davis, California • Los Angeles, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

NCT05230459
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
Interventions
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Genetic · Drug
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
6
States / cities
Irvine, California • Iowa City, Iowa • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Phase 1 Interventional

Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

NCT00428935
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAV2.5-CMV-minidystrophin (d3990)
Biological
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
5 Years to 15 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 4, 2013 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Phase 1 Interventional

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

NCT01128855
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968, 12 mg/kg GSK2402968, Placebo
Drug · Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
9 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 17, 2017 · Synced Sep 17, 2026, 9:40 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

NCT01484678
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
5 Years to 62 Years · Male only
Enrollment
550 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
3
States / cities
Gainesville, Florida • Portland, Oregon • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Sep 17, 2026, 9:40 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Wearable Technology to Assess Gait Function in SMA and DMD

NCT04193085
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2024 · Synced Sep 17, 2026, 9:40 AM EDT
Recruiting No phase listed Observational

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978
View directory record Official record
Conditions
Duchene Muscular Dystrophy
Interventions
Not listed
Lead sponsor
ITF Therapeutics LLC
Industry
Eligibility
6 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
9
States / cities
Little Rock, Arkansas • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy in RYR1-Related Congenital Myopathy

NCT02362425
View directory record Official record
Conditions
Neuromuscular Disease
Interventions
N-acetylcysteine, Placebo
Drug
Lead sponsor
National Institute of Nursing Research (NINR)
NIH
Eligibility
7 Years and older
Enrollment
63 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 23, 2019 · Synced Sep 17, 2026, 9:40 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

NCT05540860
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
4 Years to 9 Years · Male only
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
14
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 17, 2026, 9:40 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 17, 2026, 9:40 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Limb-Girdle Muscular Dystrophy

NCT00893334
View directory record Official record
Conditions
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 6, 2014 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT03375164
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
3 Months to 7 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Sep 17, 2026, 9:40 AM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

NCT06100887
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
6 Years to 17 Years · Male only
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 5, 2025 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)

NCT01831934
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Fluzone®
Biological
Lead sponsor
Stanford University
Other
Eligibility
13 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Sep 17, 2026, 9:40 AM EDT
Recruiting Phase 3 Interventional

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

NCT07608432
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
Interventions
Zeleciment Rostudirsen (DYNE-251), Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Hillsborough, North Carolina
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Phase 3 Interventional

Global Study of Del-desiran for the Treatment of DM1

NCT06411288
View directory record Official record
Conditions
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy
Interventions
AOC 1001 (del-desiran), Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years to 65 Years
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
18
States / cities
Stanford, California • Denver, Colorado • Gainesville, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Phase 1 Interventional

Liposomal Doxorubicin in Treating Children With Refractory Solid Tumors

NCT00019630
View directory record Official record
Conditions
Childhood Soft Tissue Sarcoma, Childhood Liver Cancer, Bone Cancer, Brain Tumor, Kidney Tumor
Interventions
doxorubicin HCl liposome
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Timeline
Started 1999
U.S. locations
2
States / cities
Bethesda, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 3, 2024 · Synced Sep 17, 2026, 9:40 AM EDT
Enrolling by invitation No phase listed Observational

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

NCT06270719
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Delandistrogene Moxeparvovec, Standard of Care
Genetic · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2038
U.S. locations
25
States / cities
Little Rock, Arkansas • Los Angeles, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced Sep 17, 2026, 9:40 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Burden of Access in Duchenne Muscular Dystrophy in the US

NCT03951675
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 99 Years
Enrollment
57 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 5, 2020 · Synced Sep 17, 2026, 9:40 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Movement Enhancing Device for Children

NCT01959581
View directory record Official record
Conditions
Upper Extremity Dysfunction
Interventions
Movement Enhancing Device
Device
Lead sponsor
University of Delaware
Other
Eligibility
1 Month to 6 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2016
U.S. locations
3
States / cities
Newark, Delaware • Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Jul 5, 2017 · Synced Sep 17, 2026, 9:40 AM EDT