Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

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.

Conditions

Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy

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Study Overview

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Study Details

Study overview

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.

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Condition
Congenital Myotonic Dystrophy
Intervention / Treatment

-

Contacts and Locations

Richmond

Virginia Commonwealth University, Richmond, Virginia, United States, 23298

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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
  • * Significant trauma within the past month
  • * Internal metal or devices (exclusion for DEXA component)
  • * Use of anticoagulants, such as warfarin or a direct oral anticoagulant (e.g., dabigatran) due to the increased risk of bleeding with biopsy
  • * Platelet count \<50,000
  • * History of a bleeding disorder
  • * Participation in a clinical trial involving an investigational product
  • * History of adverse reaction to lidocaine (if participating in muscle biopsy)

Ages Eligible for Study

3 Years to 17 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Virginia Commonwealth University,

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

Study Record Dates

2030-05