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Showing 25–46 of 22 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Myotonic Dystrophy Clear all
Not listed No phase listed Observational Accepts healthy volunteers

Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy

NCT02269865
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
8 Years to 17 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 20, 2014 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

NCT06185764
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
VX-670, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 64 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
8
States / cities
San Carlos, California • Gainesville, Florida • Fairway, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jul 21, 2026, 4:52 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 21, 2026, 4:52 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2

NCT05854433
View directory record Official record
Conditions
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Interventions
Non-interventional study
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
30 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 2 Interventional Results available

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

NCT01406873
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Mexiletine, Placebo
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 80 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 18, 2018 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting Phase 3 Interventional

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

NCT07486934
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy, Steinert Disease, Steinert
Interventions
zeleciment basivarsen (DYNE-101), Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
16 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
7
States / cities
La Jolla, California • Atlanta, Georgia • Indianapolis, Indiana + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 1Phase 2 Interventional

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05027269
View directory record Official record
Conditions
DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
Interventions
AOC 1001, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
8
States / cities
Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarker Development for Muscular Dystrophies

NCT05019625
View directory record Official record
Conditions
Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
465 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2028
U.S. locations
5
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting No phase listed Observational

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

NCT05224778
View directory record Official record
Conditions
Congenital Myotonic Dystrophy, CDM
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
Up to 59 Months
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
Los Angeles, California • Fairway, Kansas • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy

NCT03059264
View directory record Official record
Conditions
Congenital Myotonic Dystrophy
Interventions
Natural history
Other
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
0 Years to 15 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2025
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced Jul 21, 2026, 4:52 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 21, 2026, 4:52 PM EDT
Completed Phase 2 Interventional

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05479981
View directory record Official record
Conditions
DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
Interventions
Placebo, AOC 1001
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
18 Years to 66 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
8
States / cities
Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting No phase listed Observational

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

NCT06075693
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
88 participants
Timeline
2022 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy

NCT07362875
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Active, not recruiting 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 Jan 21, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting No phase listed Observational

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

NCT06747884
View directory record Official record
Conditions
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years to 17 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Extracellular RNA Biomarkers of Myotonic Dystrophy

NCT05020002
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
215 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2027
U.S. locations
3
States / cities
Boston, Massachusetts • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 1Phase 2 Interventional

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

NCT02312011
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
IONIS-DMPKRx, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
20 Years to 55 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
8
States / cities
Stanford, California • Gainesville, Florida • Kansas City, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2022 · Synced Jul 21, 2026, 4:52 PM 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 Jul 21, 2026, 4:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

NCT05481879
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
DYNE-101, Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
8
States / cities
Stanford, California • Gainesville, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 1 Interventional

Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1

NCT02251457
View directory record Official record
Conditions
Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
Interventions
Ranolazine
Drug
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 100 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 4, 2019 · Synced Jul 21, 2026, 4:52 PM EDT
Recruiting No phase listed Observational

Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

NCT03981575
View directory record Official record
Conditions
Myotonic Dystrophy 1, DM1
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years to 70 Years
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
10
States / cities
La Jolla, California • Los Angeles, California • Denver, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jul 21, 2026, 4:52 PM EDT