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

NCT ID: NCT05558813

Last Updated: 2025-11-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

NA

Study Classification

INTERVENTIONAL

Study Start Date

2024-11-30

Study Completion Date

2029-04-30

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.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Study Groups

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DMD-CMP cohort

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

Group Type EXPERIMENTAL

Cardiac MRI

Intervention Type DIAGNOSTIC_TEST

two cardiac MRIs with Gadolinum injection at 2-year intervals

Blood assays

Intervention Type BIOLOGICAL

blood samples for the determination of blood biomarkers of heart failure (BNP, NTproBNP) and for the constitution of a biological collection

Interventions

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Cardiac MRI

two cardiac MRIs with Gadolinum injection at 2-year intervals

Intervention Type DIAGNOSTIC_TEST

Blood assays

blood samples for the determination of blood biomarkers of heart failure (BNP, NTproBNP) and for the constitution of a biological collection

Intervention Type BIOLOGICAL

Other Intervention Names

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Blood biobanking

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
Minimum Eligible Age

6 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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URC-CIC Paris Descartes Necker Cochin

OTHER

Sponsor Role collaborator

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Karim A WAHBI, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

APHP

Locations

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Necker Hospital

Paris, , France

Site Status

Countries

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France

Other Identifiers

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2020-A03534-35

Identifier Type: OTHER

Identifier Source: secondary_id

APHP220088

Identifier Type: -

Identifier Source: org_study_id

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