Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 7 - 21 |
| Updated: | 5/30/2018 |
| Start Date: | February 1, 2017 |
| End Date: | October 2022 |
| Contact: | Stephanie Gilbert |
| Email: | sgilbert@mednet.ucla.edu |
| Phone: | 310-794-0376 |
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.
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.
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.
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
We found this trial at
2
sites
1201 W La Veta Ave
Orange, California 92868
Orange, California 92868
(714) 997-3000
Phone: 714-509-3939
Children's Hospital of Orange County For more than 45 years, CHOC Children’s has been steadfastly...
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Los Angeles, California 90095
310-825-4321
Principal Investigator: Daniel Ennis, PhD
Phone: 310-481-7510
University of California at Los Angeles The University of California, Los Angeles (UCLA) is an...
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