RECRUITING

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

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Conditions

Congenital Myotonic DystrophyCDMClinical ResearchMyotonic dystrophy

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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

The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.

Official Title

Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)

Quick Facts

Study Start:2022-08-24
Study Completion:2026-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05224778

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:Not specified to 59 Months
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Age neonate to 3 years 11 months at enrollment.
  2. * A diagnosis of CDM, which is defined as children having symptoms of myotonic dystrophy in the newborn period (\<30 days), such as hypotonia, feeding or respiratory difficulty, requiring hospitalization to a ward or to the neonatal intensive care unit for more than 72 hours; and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. An expanded CTG repeat size in the child is considered greater than 200 repeats or E1-E4 classification (E1= 200-500, E2=500-1,000, E3=1,000-1,500, E4\>1,500).
  3. * Guardian is willing and able to sign consent and follow study procedures
  1. * Any other non-DM1 illness that would interfere with the ability or results of the study in the opinion of the site investigator
  2. * Significant trauma within one month
  3. * Internal metal or devices (exclusion for DEXA component)
  4. * History of bleeding disorder or platelet count \<50,000
  5. * History of reaction to local anesthetic

Contacts and Locations

Study Contact

Ruby Langeslay
CONTACT
804-828-8481
ruby.langeslay@vcuhealth.org
Jennifer Raymond
CONTACT
804-828-6318
jennifer.raymond@vcuhealth.org

Principal Investigator

Nicholas E. Johnson, MD
PRINCIPAL_INVESTIGATOR
Virginia Commonwealth University

Study Locations (Sites)

University of California, Los Angeles
Los Angeles, California, 90095
United States
University of Kansas Medical Center
Fairway, Kansas, 66205
United States
University of Rochester Medical Center
Rochester, New York, 14642
United States
Virginia Commonwealth University
Richmond, Virginia, 23298
United States

Collaborators and Investigators

Sponsor: Virginia Commonwealth University

  • Nicholas E. Johnson, MD, 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 Date2022-08-24
Study Completion Date2026-12

Study Record Updates

Study Start Date2022-08-24
Study Completion Date2026-12

Terms related to this study

Keywords Provided by Researchers

  • Clinical Research
  • Myotonic dystrophy
  • Congenital Myotonic Dystrophy
  • CDM

Additional Relevant MeSH Terms

  • Congenital Myotonic Dystrophy
  • CDM