Summary

for males ages 7-21 (full criteria)
healthy people welcome
at UCLA
study started
estimated completion
Daniel Ennis (ucla)

Description

Summary

This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in heart health over time in boys with DMD. Currently, there is a lack of sufficiently well characterized cardiac MRI biomarkers that can serve as endpoints for detecting on-target and/or off-target cardiac effects during clinical drug trials for boys with DMD. Consequently, the first objective is to identify and characterize several cardiac MRI biomarkers for boys with DMD.

Official Title

Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for Duchenne Muscular Dystrophy (DMD)

Details

The second objective is to use their well-characterized cardiac MRI biomarkers and define their sensitivity for detecting early cardiac involvement. The final objective is to use these validated cardiac MRI biomarkers to better understand the genotype-phenotype correlation in boys with DMD, which to date remain tenuous. The investigators propose a pilot study to explore cardiac genotype-phenotype correlations in boys with DMD and outlier phenotypes using approaches they have pioneered for skeletal muscle.

Keywords

Muscular Dystrophy, DuchenneMuscular DystrophiesCardiac MRI with contrastCardiac MRI without contrastBlood TestHeart RatePulmonary Function TestGenetic TestingRepeat MRI scan

Eligibility

You can join if…

Open to males ages 7-21

  • Healthy boys or pediatric patients with DMD age 7 to 21
  • Able & willing to complete an approximately 75-minute (or less) MRI exam without sedation or mechanical ventilation
  • Drug regimen (if applicable) stable for at least 3 months prior to participation

You CAN'T join if...

  • Renal insufficiency (GFR<40 mL/min/m2)
  • Non-MRI compatible implants (e.g. neurostimulator, pacemaker, implanted cardioverter defibrillator)
  • Claustrophobia that prevents an MRI exam
  • Known allergy to MRI contrast agents
  • Serum potassium level of >5.0 mmol/L
  • Signs and symptoms of heart failure

Locations

  • University of California, Los Angeles accepting new patients
    Los AngelesCalifornia90024United States
  • Children's Hospital of Orange County accepting new patients
    OrangeCalifornia92868United States

Lead Scientist

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
University of California, Los Angeles
ID
NCT02834650
Study Type
Interventional
Last Updated