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

Assistance Publique - Hôpitaux de Paris

Status: Withdrawn

Conditions: Duchenne Muscular Dystrophy

Treatments: Biological: Blood assays; Diagnostic Test: Cardiac MRI

Study type: Interventional

Funder types: Other

Identifiers

NCT05558813

2020-A03534-35 (Other Identifier)

APHP220088

Details and patient eligibility

About

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.

Full 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.

Sex: Male

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Diagnostic

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

DMD-CMP cohort

Experimental group

Description:

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

Treatment:

Diagnostic Test: Cardiac MRI

Biological: Blood assays