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Clinical trial condition

Myotonic Disorders

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

117 public trials 42 recruiting studies
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Myotonic Disorders trial results

Recruiting

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

NCT07587242

Conditions
Congenital, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DMD, +22 more
Locations
  • AP-HP Hospital Armand-Trousseau Paris, France
  • Universitaetsklinikum Essen Essen, Germany
  • Universitaetsklinikum Heidelberg Heidelberg, Germany
  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen München, Germany
View Trial Details
Recruiting

VirtualPark_Pediatric

NCT07580365

Conditions
Congenital Myopathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dystrophinopathy, +11 more
Locations
  • IRCCS E. Medea - La Nostra Famiglia Bosisio Parini, LC, Italy
  • IRCCS Fondazione Stella Maris Pisa, PI, Italy
  • IRCCS Fondazione Mondino Pavia, PV, Italy
  • Fondazione Don Gnocchi - Centro "S. Maria al Mare" Salerno, SA, Italy
View Trial Details
Recruiting

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

NCT06747884

Conditions
Childhood Myotonic Dystrophy, Congenital Myotonic Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +16 more
Locations
  • Virginia Commonwealth University Richmond, Virginia, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • Hospital das Clínicas, University of São Paulo School of Medicine (HCFMUSP) Cerqueira César, São Paulo, Brazil
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Recruiting

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

NCT06138743

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • Liverpool Hospital Liverpool, New South Wales, Australia
  • Sunshine Coast University Hospital Birtinya, Queensland, Australia
  • Royal Brisbane And Women's Hospital Herston, Queensland, Australia
  • Alfred Health Melbourne, Victoria, Australia
View all 35 locations
View Trial Details
Recruiting

Personalized Training for People With Rare Neuromuscular Disorders

NCT06708468

Conditions
Charcot Marie Tooth Disease (CMT), Charcot-Marie-Tooth Disease, Congenital Abnormalities, +20 more
Locations
  • Haukeland University Hospital Bergen, Norway
  • Helse Møre and Romsdal HF (Molde Hospital) Molde, Norway
  • Namsos Hospital Namsos, Norway
  • Oslo University Hospital Oslo, Norway
View Trial Details
Recruiting

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

NCT05224778

Conditions
CDM, Congenital Myotonic Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations
  • University of California, Los Angeles Los Angeles, California, United States
  • University of Kansas Medical Center Fairway, Kansas, United States
  • University of Rochester Medical Center Rochester, New York, United States
  • Virginia Commonwealth University Richmond, Virginia, United States
View Trial Details
Recruiting

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

NCT03981575

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +11 more
Locations
  • University of California, San Diego La Jolla, California, United States
  • University of California, Los Angeles Los Angeles, California, United States
  • University of Colorado - Denver Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View all 17 locations
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Recruiting

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

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +9 more
Locations
  • University of Florida, 2004 Mowry Road - Site Number: 8400005 Gainesville, Florida, United States
  • University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 Tampa, Florida, United States
  • Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 New York, United States
  • Virginia Commonwealth University Medical Center- Site Number : 8400006 Richmond, Virginia, United States
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