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

Duchenne Muscular Dystrophy Clinical Trial

— DMD-CMP  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05558813
• Other study ID #: APHP220088
• Secondary ID: 2020-A03534-35
• Status: Not yet recruiting
• Phase: N/A
• Start date: November 2024
• Est. completion date: April 2029

Study information

• Verified date: October 2023
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Karim WAHBI, MD, PhD
• Phone: +33 1 58 41 16 53
• Email: karim.wahbi[@]aphp.fr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 150
• Est. completion date: April 2029
• Est. primary completion date: February 2029
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years and older

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Diagnostic Test:
• Cardiac MRI
two cardiac MRIs with Gadolinum injection at 2-year intervals

Biological:
• Blood assays
blood samples for the determination of blood biomarkers of heart failure (BNP, NTproBNP) and for the constitution of a biological collection

Locations

Country	Name	City	State
France	Necker Hospital	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Late gadolinium enhancement burden on cardiac MRI		2 years
Secondary	Global T1 on cardiac MRI		2 years
Secondary	Global T2 on cardiac MRI		2 years
Secondary	Global extracellular volume on cardiac MRI		2 years
Secondary	Left ventricular ejection fraction on cardiac MRI		2 years
Secondary	Left ventricular systolic circumferentiel strain ejection fraction on cardiac MRI		2 years
Secondary	Left ventricular systolic radial strain ejection fraction on cardiac MRI		2 years
Secondary	Left ventricular systolic longitudinal strain on cardiac MRI		2 years
Secondary	NTproBNP assay in the blood		2 years
Secondary	Troponin I assay in the blood		2 years