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

Not Applicable

Withdrawn

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Diagnostic Test: Cardiac MRI; Biological: Blood assays

• Registration Number: NCT05558813
• Lead 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.

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 Timeline

• Study First Submitted: 2022-04-15
• Study First Submitted QC: 2022-09-26
• Study First Posted: 2022-09-28
• Study Start: 2024-11
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-30
• Last Update Posted: 2025-02-03
• Primary Completion: 2029-02
• Study Completion: 2029-04

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: Male
• Target Recruitment: 150

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
DMD-CMP cohort	Blood assays	Minor (≥ 6 years) and major patients with genetically proven Duchenne myopathy
DMD-CMP cohort	Cardiac MRI	Minor (≥ 6 years) and major patients with genetically proven Duchenne myopathy

Primary Outcome Measures

Name	Time	Method
Late gadolinium enhancement burden on cardiac MRI	2 years

Secondary Outcome Measures

Name	Time	Method
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
Global T2 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

Trial Locations

Locations (1)

Necker Hospital; 🇫🇷 Paris, France