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ClinicalTrials.gov public records Last synced Oct 5, 2026, 6:21 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–44 of 44 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Myotonia Atrophica Clear all

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

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

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

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

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

Conditions: Myotonic Dystrophy Type 1 (DM1)
Interventions: DYNE-101, Placebo; Drug
Lead sponsor: Dyne Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 127 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2029
U.S. locations: 8
States / cities: Stanford, California • Gainesville, Florida • Indianapolis, Indiana + 5 more

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

Conditions: Myotonic Dystrophy 1
Interventions: ATX-01, Placebo; Drug
Lead sponsor: ARTHEx Biotech S.L.; Industry
Eligibility: 18 Years to 64 Years
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 5
States / cities: Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 2 more

Conditions: Myotonic Dystrophy Type 1 (DM1)
Interventions: VX-670, Placebo; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 18 Years to 64 Years
Enrollment: 47 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

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

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

Conditions: Myotonic Dystrophy Type 1
Interventions: rhIGF-I/rhIGFBP-3, placebo; Drug
Lead sponsor: Insmed Incorporated; Industry
Eligibility: 21 Years to 65 Years
Enrollment: 69 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 12
States / cities: Orange, California • Sacramento, California • Kansas City, Kansas + 9 more

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

Conditions: Myotonic Dystrophy 2
Interventions: Exercise; Other
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years to 70 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Boston, Massachusetts

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

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

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

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

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

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

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Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2

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

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

Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy

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

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

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Study of ATX-01 in Participants With DM1

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

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

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

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Long-Term Development of Muscular Dystrophy Outcome Assessments

Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

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

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

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

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

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

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension