Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne, dystrophin, cardiomyopathy, cardiac MRI
Brief summary
The purpose of this study is to describe the progression of tissular and functional myocardial abnormalities in patients with Duchenne muscular dystrophy using cardiac magnetic resonance imaging and blood biomarkers assays.
Detailed description
This study is to describe the progression of tissular (late gadolinium enhancement, T1, T2, ECV assessments) and functional (segmental and global contractility, strain) myocardial abnormalities in patients with Duchenne muscular dystrophy using cardiac magnetic resonance imaging and blood biomarkers assays (troponin I, NTproBNP). Comparison between baseline and 2-years assessments will be conducted.
Interventions
two cardiac MRIs with Gadolinum injection at 2-year intervals
blood samples for the determination of blood biomarkers of heart failure (BNP, NTproBNP) and for the constitution of a biological collection
Sponsors
Study design
Eligibility
Inclusion criteria
: * Age \>= 6 years * Genetically proven Duchenne muscular dystrophy * Affiliation to French medical insurance * Informed consent provided
Exclusion criteria
: * Age \<6 years * Left ventricular ejection fraction \<30% * Tracheostomy of hospitalisation for acute respiratory failure \<1 year * Contraindication to MRI: claustrophobia, Gadolinum allergy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Late gadolinium enhancement burden on cardiac MRI | 2 years |
Secondary
| Measure | Time frame |
|---|---|
| Global T2 on cardiac MRI | 2 years |
| Global extracellular volume on cardiac MRI | 2 years |
| Left ventricular ejection fraction on cardiac MRI | 2 years |
| Left ventricular systolic circumferentiel strain ejection fraction on cardiac MRI | 2 years |
| Global T1 on cardiac MRI | 2 years |
| Left ventricular systolic longitudinal strain on cardiac MRI | 2 years |
| NTproBNP assay in the blood | 2 years |
| Troponin I assay in the blood | 2 years |
| Left ventricular systolic radial strain ejection fraction on cardiac MRI | 2 years |
Countries
France