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NCT05558813 | Not yet recruiting | Duchenne Muscular Dystrophy


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

Assistance Publique Hôpitaux de Paris

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.

Condition or disease

Duchenne Muscular Dystrophy

Intervention/treatment

Cardiac MRI

Blood assays

Phase

Not Applicable

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.}}

Study Type : Interventional
Estimated Enrollment : 150 participants
Masking : None (Open Label)
Primary Purpose : Diagnostic
Official Title : Prospective Cardiac Magnetic Resonance Imaging Study in Duchenne Muscular Dystrophy (DMD-CMP)
Actual Study Start Date : November 2024
Estimated Primary Completion Date : February 2029
Estimated Study Completion Date : April 2029
Arm Intervention/treatment

Experimental: DMD-CMP cohort

Minor (≥ 6 years) and major patients with genetically proven Duchenne myopathy

Diagnostic Test: Cardiac MRI

Biological: Blood assays

Ages Eligible for Study: 6 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No
Criteria
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

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

Location Details


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

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Locations


Not yet recruiting

France,

Necker Hospital

Paris, France, 75015

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