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

NCT05558813 | Withdrawn

• 2 other identifiers: APHP2200882020-A03534-35
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne; dystrophin; cardiomyopathy; cardiac MRI

Outcome Measures

Primary Outcomes (1)

• Late gadolinium enhancement burden on cardiac MRI

  2 years

Secondary Outcomes (9)

• Global T1 on cardiac MRI

  2 years

• Global T2 on cardiac MRI

  2 years

• 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

Study Arms (1)

DMD-CMP cohort

EXPERIMENTAL

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

Diagnostic Test: Cardiac MRI; Biological: Blood assays

Interventions

Cardiac MRI DIAGNOSTIC_TEST

two cardiac MRIs with Gadolinum injection at 2-year intervals

DMD-CMP cohort

Blood assays BIOLOGICAL

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

Also known as: Blood biobanking

DMD-CMP cohort

Eligibility Criteria

• Age: 6 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>= 6 years
• Genetically proven Duchenne muscular dystrophy
• Affiliation to French medical insurance
• Informed consent provided

You may not qualify if:

• Age \<6 years
• Left ventricular ejection fraction \<30%
• Tracheostomy of hospitalisation for acute respiratory failure \<1 year
• Contraindication to MRI: claustrophobia, Gadolinum allergy

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Necker Hospital

Paris, 75015, France

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Cardiomyopathies

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heart Diseases; Cardiovascular Diseases

Study Officials

• Karim A WAHBI, MD, PhD

  APHP

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2022
• First Posted: September 28, 2022
• Study Start: November 1, 2024
• Primary Completion (Estimated): February 1, 2029
• Study Completion (Estimated): April 1, 2029
• Last Updated: November 20, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)