RECRUITING

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Talk to us

Conditions

Congenital Myotonic DystrophyChildhood Myotonic DystrophyMyotonic DystrophyDM1MyotoniaDystrophy MyotonicDMCRNTREAT-EXTTREAT CDM

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.

Official Title

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Quick Facts

Study Start:2025-05-14
Study Completion:2030-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06747884

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:3 Years to 17 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. * Any other non-DM1 illness that would interfere with the ability to undergo safe testing or would affect the interpretation of the results, in the opinion of the site investigator
  2. * Significant trauma within the past month
  3. * Internal metal or devices (exclusion for DEXA component)
  4. * Use of anticoagulants, such as warfarin or a direct oral anticoagulant (e.g., dabigatran) due to the increased risk of bleeding with biopsy
  5. * Platelet count \<50,000
  6. * History of a bleeding disorder
  7. * Participation in a clinical trial involving an investigational product
  8. * History of adverse reaction to lidocaine (if participating in muscle biopsy)

Contacts and Locations

Study Contact

Ruby Langeslay, MPH
CONTACT
804-828-6318
Ruby.Langeslay@vcuhealth.org

Principal Investigator

Nicholas Johnson, MD, MSCI, FAAN
PRINCIPAL_INVESTIGATOR
Virginia Commonwealth University

Study Locations (Sites)

Virginia Commonwealth University
Richmond, Virginia, 23298
United States

Collaborators and Investigators

Sponsor: Virginia Commonwealth University

  • Nicholas Johnson, MD, MSCI, FAAN, PRINCIPAL_INVESTIGATOR, Virginia Commonwealth University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-05-14
Study Completion Date2030-05

Study Record Updates

Study Start Date2025-05-14
Study Completion Date2030-05

Terms related to this study

Keywords Provided by Researchers

  • DM1
  • Childhood Myotonic Dystrophy
  • Congenital Myotonic Dystrophy
  • Myotonic Dystrophy
  • Myotonia
  • Dystrophy Myotonic
  • DMCRN
  • TREAT-EXT
  • TREAT CDM

Additional Relevant MeSH Terms

  • Congenital Myotonic Dystrophy
  • Childhood Myotonic Dystrophy
  • Myotonic Dystrophy