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Clinical trial condition

Myotonic Disorders

Explore clinical trials studying Myotonic Disorders. Study availability and eligibility vary by location and protocol.

17 public trials 4 recruiting studies
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Myotonic Disorders trial results

Recruiting

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

NCT07486934

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +18 more
Locations
  • UCSD - Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • IU Health Neuroscience Center Indianapolis, Indiana, United States
  • Roy Blunt NextGen Precision Health Institute Columbia, Missouri, United States
View all 21 locations
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Recruiting

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

NCT06185764

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +15 more
Locations
  • Stanford Neuromuscular Research San Carlos, California, United States
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • University of Kansas Medical Center Fairway, Kansas, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View all 26 locations
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Enrolling by Invitation

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07008469

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +19 more
Locations
  • University of California, Los Angeles (UCLA) Los Angeles, California, United States
  • Stanford University Stanford, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View all 35 locations
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Enrolling by Invitation

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

NCT06926621

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +15 more
Locations
  • Wesley Research Institute Auchenflower, Australia
  • Neuroscience Clinical Trials Unit, Alfred Brain Melbourne, Australia
  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology Leuven, Belgium
  • Altasciences Montreal Montreal, Canada
View all 13 locations
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Completed

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

NCT05027269

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Dystrophy Myotonic, +15 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View Trial Details
Completed

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

NCT06204809

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • UCI Center for Clinical Research Irvine, California, United States
  • Stanford University Palo Alto, California, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • University of Kansas Medical Center Fairway, Kansas, United States
View Trial Details
Completed

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

NCT05479981

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +11 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View Trial Details

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