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ClinicalTrials.gov public records Last synced Oct 22, 2026, 2:34 PM EDT

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Showing 1–24 of 51 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Myotonic Disorders Clear all
Recruiting No phase listed Observational

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

NCT07700225
View directory record Official record
Conditions
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type-1, Myotonic Dystrophy, Type 1 (DM1), Myotonic Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years to 70 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 2:34 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 24, 2025 · Synced Oct 22, 2026, 2:34 PM EDT
Completed No phase listed Observational

Multicenter Observational Study of Myotonic Dystrophy Type 1

NCT02308657
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 70 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
6
States / cities
Stanford, California • Gainesville, Florida • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2017 · Synced Oct 22, 2026, 2:34 PM 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 27, 2026 · Synced Oct 22, 2026, 2:34 PM EDT
Terminated No phase listed Observational

Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

NCT03867435
View directory record Official record
Conditions
Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
11 Years to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 22, 2026, 2:34 PM EDT
Completed No phase listed Observational

Characteristics of Nondystrophic Myotonias

NCT00244413
View directory record Official record
Conditions
Nondystrophic Myotonias, Myotonia Congenita, Myotonic Disorders
Interventions
Not listed
Lead sponsor
Richard Barohn, MD
Other
Eligibility
6 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
4
States / cities
Kansas City, Kansas • Boston, Massachusetts • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2013 · Synced Oct 22, 2026, 2:34 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

NCT04886518
View directory record Official record
Conditions
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Interventions
Pitolisant Oral Tablet, Placebo oral tablet
Drug
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
Irvine, California • Aurora, Colorado • Tampa, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 22, 2026, 2:34 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy

NCT06089018
View directory record Official record
Conditions
Myotonic Dystrophy 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
12 Years to 90 Years
Enrollment
125 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 18, 2023 · Synced Oct 22, 2026, 2:34 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 14, 2018 · Synced Oct 22, 2026, 2:34 PM EDT
Recruiting No phase listed Observational

A Natural History Study of RYR1-Related Disorders

NCT06287762
View directory record Official record
Conditions
Ryanodine Receptor 1-Related Myopathy, Ryanodine Receptor 1 Related Disorders
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
7 Years to 100 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 22, 2026, 2:34 PM 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
5
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 22, 2026, 2:34 PM EDT
Not yet recruiting No phase listed Observational

Nemaline Myopathy Clinical Research Network (NM-CTRN)

NCT06774703
View directory record Official record
Conditions
Nemaline Myopathy
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
0 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Palo Alto, California • Bethesda, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 14, 2025 · Synced Oct 22, 2026, 2:34 PM EDT
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 21, 2014 · Synced Oct 22, 2026, 2:34 PM EDT
Recruiting No phase listed Observational

Molecular and Genetic Studies of Congenital Myopathies

NCT00272883
View directory record Official record
Conditions
Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease, Myotubular Myopathy, Nemaline Myopathy, Rigid Spine Muscular Dystrophy, Undefined Congenital Myopathy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2050
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 22, 2026, 2:34 PM EDT
Completed Phase 1Phase 2 Interventional Results available

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 Sep 28, 2026 · Synced Oct 22, 2026, 2:34 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)

NCT00233519
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
SomatoKine/IPLEX
Drug
Lead sponsor
University of Rochester
Other
Eligibility
21 Years to 60 Years
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2008
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 25, 2012 · Synced Oct 22, 2026, 2:34 PM EDT
Recruiting No phase listed Observational

Long-Term Development of Muscular Dystrophy Outcome Assessments

NCT05989620
View directory record Official record
Conditions
LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 50 Years
Enrollment
1,000 participants
Timeline
2023 – 2029
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Oct 22, 2026, 2:34 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 10, 2026 · Synced Oct 22, 2026, 2:34 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 11, 2025 · Synced Oct 22, 2026, 2:34 PM EDT
Recruiting Phase 2Phase 3 Interventional

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

NCT05004129
View directory record Official record
Conditions
Congenital Myotonic Dystrophy
Interventions
Tideglusib
Drug
Lead sponsor
AMO Pharma Limited
Industry
Eligibility
6 Years to 45 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
10
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated May 28, 2025 · Synced Oct 22, 2026, 2:34 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 23, 2026 · Synced Oct 22, 2026, 2:34 PM EDT
Enrolling by invitation Phase 2 Interventional

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

NCT06926621
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
VX-670
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
3
States / cities
St Louis, Missouri • Winston-Salem, North Carolina • Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 22, 2026, 2:34 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
3
States / cities
Little Rock, Arkansas • Kansas City, Kansas • Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 22, 2026, 2:34 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 10, 2026 · Synced Oct 22, 2026, 2:34 PM EDT