Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)

Study Overview
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Brief Summary
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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
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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.

Official Title
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Prospective Cardiac Magnetic Resonance Imaging Study in Duchenne Muscular Dystrophy (DMD-CMP)

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Diagnostic Test: Cardiac MRI
* Biological: Blood assays


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: DMD-CMP cohort<br>Minor (≥ 6 years) and major patients with genetically proven Duchenne myopathy | Diagnostic Test: Cardiac MRI<br>* two cardiac MRIs with Gadolinum injection at 2-year intervals<br>Biological: Blood assays<br>* blood samples for the determination of blood biomarkers of heart failure (BNP, NTproBNP) and for the constitution of a biological collection<br>* Other names: Blood biobanking;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Late gadolinium enhancement burden on cardiac MRI |  | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Global T1 on cardiac MRI |  | 2 years | 
| 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 | 
| Left ventricular systolic radial strain ejection fraction 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne, dystrophin, cardiomyopathy, cardiac MRI