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

Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT05558813
Collaborator
(none)
150
Enrollment
1
Location
1
Arm
65
Anticipated Duration (Months)
2.3
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Diagnostic Test: Cardiac MRI
  • Biological: Blood assays
 N/A

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 Design

Study Type:
Interventional
Anticipated Enrollment :
150 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Diagnostic
Official Title:
Prospective Cardiac Magnetic Resonance Imaging Study in Duchenne Muscular Dystrophy (DMD-CMP)
Anticipated Study Start Date :
Nov 1, 2022
Anticipated Primary Completion Date :
Feb 1, 2028
Anticipated Study Completion Date :
Apr 1, 2028

Arms and Interventions

Arm Intervention/Treatment
Experimental: DMD-CMP cohort

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

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
Other Names:
  • Blood biobanking

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Late gadolinium enhancement burden on cardiac MRI [2 years]

    Secondary Outcome Measures

    1. Global T1 on cardiac MRI [2 years]

    2. Global T2 on cardiac MRI [2 years]

    3. Global extracellular volume on cardiac MRI [2 years]

    4. Left ventricular ejection fraction on cardiac MRI [2 years]

    5. Left ventricular systolic circumferentiel strain ejection fraction on cardiac MRI [2 years]

    6. Left ventricular systolic radial strain ejection fraction on cardiac MRI [2 years]

    7. Left ventricular systolic longitudinal strain on cardiac MRI [2 years]

    8. NTproBNP assay in the blood [2 years]

    9. Troponin I assay in the blood [2 years]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    Male
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Necker Hospital Paris France 75015

    Sponsors and Collaborators

    • Assistance Publique - Hôpitaux de Paris

    Investigators

    • Principal Investigator: Karim A WAHBI, MD, PhD, APHP

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Assistance Publique - Hôpitaux de Paris
    ClinicalTrials.gov Identifier:
    NCT05558813
    Other Study ID Numbers:
    • APHP220088
    • 2020-A03534-35
    First Posted:
    Sep 28, 2022
    Last Update Posted:
    Sep 28, 2022
    Last Verified:
    Sep 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Assistance Publique - Hôpitaux de Paris
    Duchenne
    dystrophin
    cardiomyopathy
    cardiac MRI
    Additional relevant MeSH terms:
    Muscular Dystrophies
    Muscular Dystrophy, Duchenne

    Study Results

    No Results Posted as of Sep 28, 2022