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
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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.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Muscular Dystrophy, Duchenne | Other: Cardiac MRI with contrast Other: Cardiac MRI without contrast Other: Blood Test Other: Heart Rate Other: Pulmonary Function Test Other: Genetic Testing Other: Repeat MRI scan | Not Applicable |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 154 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Diagnostic |
Official Title: | Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for Duchenne Muscular Dystrophy (DMD) |
Actual Study Start Date : | February 1, 2017 |
Estimated Primary Completion Date : | January 2021 |
Estimated Study Completion Date : | October 2022 |

Arm | Intervention/treatment |
---|---|
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.
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Other: Cardiac MRI without contrast
Cardiac MRI
Other: Repeat MRI scan
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. |
Other: Cardiac MRI with 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 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.
|
Other: Cardiac MRI with 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 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.
|
Other: Cardiac MRI with 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
|
- 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

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Ages Eligible for Study: | 7 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | Yes |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02834650
Contact: Stephanie Gilbert | 310-794-0376 | sgilbert@mednet.ucla.edu | |
Contact: Tammy Floore, RN | 310-794-9202 | tfloore@mednet.ucla.edu |
United States, California | |
University of California, Los Angeles | Recruiting |
Los Angeles, California, United States, 90024 | |
Contact: Tammy Floore, BSN 310-481-7510 tfloore@mednet.ucla.edu | |
Contact: Saima Chaabane, PhD 310-794-8995 schaabane@mednet.ucla.edu | |
Principal Investigator: Daniel Ennis, PhD | |
Children's Hospital of Orange County | Recruiting |
Orange, California, United States, 92868 | |
Contact: Pierangelo Renella, MD 714-509-3939 prenella@choc.org | |
Contact: Ofelia Vargas, BS 714-509-8735 Ovargas@choc.org |
Principal Investigator: | Daniel Ennis, PhD | University of California, Los Angeles |
Responsible Party: | Daniel Ennis, PhD, PhD, University of California, Los Angeles |
ClinicalTrials.gov Identifier: | NCT02834650 History of Changes |
Other Study ID Numbers: |
DMD2016 1R01HL131975-01 ( U.S. NIH Grant/Contract ) |
First Posted: | July 15, 2016 Key Record Dates |
Last Update Posted: | December 2, 2017 |
Last Verified: | November 2017 |
Studies a U.S. FDA-regulated Drug Product: | No | |
Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |