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Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD

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ClinicalTrials.gov Identifier: NCT02834650
Recruitment Status : Recruiting
First Posted : July 15, 2016
Last Update Posted : December 2, 2017
Sponsor:
Collaborators:
National Institutes of Health (NIH)
Children’s Hospital of Orange County
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Daniel Ennis, PhD, University of California, Los Angeles

July 13, 2016
July 15, 2016
December 2, 2017
February 1, 2017
January 2021   (Final data collection date for primary outcome measure)
  • Myocardial Tissue Characterization [ Time Frame: 6 months ]
    Focal and diffuse fibrosis, intra myocardial fat, edema plus water mobility
  • Myocardial Functional Characterization [ Time Frame: 6 months ]
    Strain imaging and rotational mechanics
  • Genomic Analysis [ Time Frame: 4 years ]
    Proposing mechanisms of cardiac dysfunction or protective phenotypes using genomic analysis
Same as current
Complete list of historical versions of study NCT02834650 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for Duchenne Muscular Dystrophy (DMD)

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.

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.
Interventional
Not Applicable
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Muscular Dystrophy, Duchenne
  • Other: Cardiac MRI with contrast
    Cardiac MRI
  • Other: Cardiac MRI without contrast
    Cardiac MRI
  • Other: Blood Test
    Hematocrit, Creatinine, Troponin, BNP
  • Other: Heart Rate
    Holter Monitor
  • Other: Pulmonary Function Test
    Pulmonary Function Test
  • Other: Genetic Testing
    Genetic Testing
  • Other: Repeat MRI scan
    Repeat MRI scan
  • Experimental: Group 1a
    Group 1a comprises healthy volunteers who will complete a Cardiac MRI without contrast. A subset of healthy volunteers will have a repeat MRI at Children's Hospital of Orange County.
    Interventions:
    • Other: Cardiac MRI without contrast
    • Other: Repeat MRI scan
  • Experimental: Group 1b

    Group 1b comprises boys with DMD who will complete a Cardiac MRI with contrast, a blood test, a heart rate test and a pulmonary function test.

    A subset of boys with DMD will have a repeat MRI with contrast at Children's Hospital of Orange County.

    Interventions:
    • Other: Cardiac MRI with contrast
    • Other: Blood Test
    • Other: Heart Rate
    • Other: Pulmonary Function Test
    • Other: Genetic Testing
    • Other: Repeat MRI scan
  • Experimental: Group 2
    Group 2 comprises boys with DMD who will complete a Cardiac MRI with contrast, a blood test, a heart rate test and a pulmonary function test and a repeat MRI scan with contrast at 6 Months.
    Interventions:
    • Other: Cardiac MRI with contrast
    • Other: Blood Test
    • Other: Heart Rate
    • Other: Pulmonary Function Test
    • Other: Genetic Testing
    • Other: Repeat MRI scan
  • Experimental: Group 3
    Group 3 comprises boys with DMD who will complete a Cardiac MRI with contrast, a blood test, a heart rate test and a pulmonary function test and a genetic testing.
    Interventions:
    • Other: Cardiac MRI with contrast
    • Other: Blood Test
    • Other: Heart Rate
    • Other: Pulmonary Function Test
    • Other: Genetic Testing
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
154
Same as current
October 2022
January 2021   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
Sexes Eligible for Study: Male
7 Years to 21 Years   (Child, Adult)
Yes
Contact: Stephanie Gilbert 310-794-0376 sgilbert@mednet.ucla.edu
Contact: Tammy Floore, RN 310-794-9202 tfloore@mednet.ucla.edu
United States
 
 
NCT02834650
DMD2016
1R01HL131975-01 ( U.S. NIH Grant/Contract )
Yes
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Daniel Ennis, PhD, University of California, Los Angeles
University of California, Los Angeles
  • National Institutes of Health (NIH)
  • Children’s Hospital of Orange County
  • National Heart, Lung, and Blood Institute (NHLBI)
Principal Investigator: Daniel Ennis, PhD University of California, Los Angeles
University of California, Los Angeles
November 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP