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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Not yet recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Cardiac MRI
Blood assays
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Duchenne Muscular Dystrophy focused on measuring Duchenne, dystrophin, cardiomyopathy, cardiac MRI

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Necker Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

DMD-CMP cohort

Arm Description

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

Outcomes

Primary Outcome Measures

Late gadolinium enhancement burden on cardiac MRI

Secondary Outcome Measures

Global T1 on cardiac MRI
Global T2 on cardiac MRI
Global extracellular volume on cardiac MRI
Left ventricular ejection fraction on cardiac MRI
Left ventricular systolic circumferentiel strain ejection fraction on cardiac MRI
Left ventricular systolic radial strain ejection fraction on cardiac MRI
Left ventricular systolic longitudinal strain on cardiac MRI
NTproBNP assay in the blood
Troponin I assay in the blood

Full Information

First Posted
April 15, 2022
Last Updated
September 26, 2022
Sponsor
Assistance Publique - Hôpitaux de Paris
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1. Study Identification

Unique Protocol Identification Number
NCT05558813
Brief Title
Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)
Acronym
DMD-CMP
Official Title
Prospective Cardiac Magnetic Resonance Imaging Study in Duchenne Muscular Dystrophy (DMD-CMP)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2022
Overall Recruitment Status
Not yet recruiting
Study Start Date
November 2022 (Anticipated)
Primary Completion Date
February 2028 (Anticipated)
Study Completion Date
April 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne, dystrophin, cardiomyopathy, cardiac MRI

7. Study Design

Primary Purpose
Diagnostic
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
150 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
DMD-CMP cohort
Arm Type
Experimental
Arm Description
Minor (≥ 6 years) and major patients with genetically proven Duchenne myopathy
Intervention Type
Diagnostic Test
Intervention Name(s)
Cardiac MRI
Intervention Description
two cardiac MRIs with Gadolinum injection at 2-year intervals
Intervention Type
Biological
Intervention Name(s)
Blood assays
Other Intervention Name(s)
Blood biobanking
Intervention Description
blood samples for the determination of blood biomarkers of heart failure (BNP, NTproBNP) and for the constitution of a biological collection
Primary Outcome Measure Information:
Title
Late gadolinium enhancement burden on cardiac MRI
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Global T1 on cardiac MRI
Time Frame
2 years
Title
Global T2 on cardiac MRI
Time Frame
2 years
Title
Global extracellular volume on cardiac MRI
Time Frame
2 years
Title
Left ventricular ejection fraction on cardiac MRI
Time Frame
2 years
Title
Left ventricular systolic circumferentiel strain ejection fraction on cardiac MRI
Time Frame
2 years
Title
Left ventricular systolic radial strain ejection fraction on cardiac MRI
Time Frame
2 years
Title
Left ventricular systolic longitudinal strain on cardiac MRI
Time Frame
2 years
Title
NTproBNP assay in the blood
Time Frame
2 years
Title
Troponin I assay in the blood
Time Frame
2 years

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Karim WAHBI, MD, PhD
Phone
+33 1 58 41 16 53
Email
karim.wahbi@aphp.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Karima MESBAHI
Phone
+33 1 58 41 12 11
Email
karima.mesbahi@aphp.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Karim A WAHBI, MD, PhD
Organizational Affiliation
APHP
Official's Role
Principal Investigator
Facility Information:
Facility Name
Necker Hospital
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Karim WAHBI, MD, PhD
Phone
+33 1 58 41 16 53
Email
karim.wahbi@aphp.fr

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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