Cardiac MRI Biomarkers and Genotype-Phenotype Correlations

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

Questions and Answers

What stage is this research?

This trial is actively recruiting participants.

What is the goal or purpose of this study?

The goal of this study is 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 Duchenne muscular dystrophy. The study aims: i) to identify and characterize several cardiac MRI biomarkers for boys with Duchenne; ii) to define the sensitivity of the well-characterized cardiac MRI biomarkers for detecting early cardiac involvement; and iii) to use these validated cardiac MRI biomarkers to better understand the genotype-phenotype correlation in boys with Duchenne.

Who is funding this study?

This study is funded by the National Institutes of Health.

Who is eligible to participate in this study?

To participate in this study you must be:

  • Healthy boy or pediatric patient with Duchenne age 7 to 21
  • Able and willing to complete an approximately 75 minute or less MRI exam without sedation or mechanical ventilation
  • On a stable drug regimen (if taking prescription drugs) for at least 3 months prior to participation

To participate in this study you may not have:
Renal insufficiency

  • 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 more than 5.0 mmol/L
  • Signs and symptoms of heart failure

What do I have to do if I decide to participate in this study?

This study involves three groups.

  • Group 1a boys are healthy volunteers who will complete a cardiac MRI without contrast.
  • Group 1b boys are boys with Duchenne who will complete a cardiac MRI with contrast, blood tests, 24 hour heart monitoring, and pulmonary function tests.
  • Group 2 boys are boys with Duchenne who will complete a cardiac MRI with contrast, blood tests, 24 hour heart monitoring, and pulmonary function test, then have a second MRI scan with contrast 6 months later.
  • Group 3 boys are boys with Duchenne who will complete a cardiac MRI with contrast, blood tests, 24 hour heart monitoring, pulmonary function tests, and genetic tests.

Special considerations: Some boys in Group 1a and 1b will be asked to have a repeat MRI scan at UCLA or at Children’s Hospital of Orange County (CHOC).

How long will this study last?

Depending upon your participation in the study, you will either have one visit, or you will return to the clinic for a second cardiac MRI in 6 months. You will not receive follow up information once your participation in the study has ended.

Where does this study take place?

This study takes place at Ronald Reagan UCLA Medical Center and Children’s Hospital of Orange County (CHOC).

How many visits to the study site are necessary?

Boys in Group 2 will come to the clinic for two visits. The first visit will be at the start of the study and the second visit will take place six months later.

All other boys (Group 1b and 3) will come to the clinic for a single visit, except for ten boys (Group 1b) who will have cardiac MRI scans repeated at CHOC or UCLA, depending upon where the initial MRI scan was obtained.

Can any visits be done locally?

No, the MRI equipment requires that the cardiac MRI exams take place at UCLA or CHOC hospitals.

Is there any funding to help pay for travel?

Yes, if you are a boy in Group 3 and you live a significant distance from the study site, you may be eligible for reimbursement for travel expenses related to accommodation, transportation, lodging and meals.

Will I get paid for participating in this study?

Yes, please contact the study group for specific information.

Why should I consider participating in this study?

While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to the latest cardiac MRI methods before they are widely available, and having access to medical specialists that may not be normally not available to you or your child, and helping others by contributing to the better understanding of Duchenne.

Where can I learn more about this study?

You can learn more about this study at www.ClinicalTrials.gov (NCT02834650).

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