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Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial

Primary Purpose

Myocardial Fibrosis, Muscular Dystrophies

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Enalapril
Sponsored by
InCor Heart Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myocardial Fibrosis focused on measuring cardiac magnetic resonance, myocardial fibrosis, muscular dystrophies

Eligibility Criteria

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

Inclusion Criteria:

  • Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker

Exclusion Criteria:

  • Contraindications to cardiovascular magnetic resonance imaging

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    No Intervention

    Arm Label

    ACE inhibitor

    Control

    Arm Description

    ACE inhibitor (enalapril up to 20mg BID), in patients with preserved EF (LVEF grater than 50%) and with detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance, randomized to therapy or not.

    Patients with preserved EF (LVEF grater than 50%) and with no detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance

    Outcomes

    Primary Outcome Measures

    Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy
    Progression of myocardial fibrosis

    Secondary Outcome Measures

    Specific genetic mutations as predictors of cardiac involvement
    Relation of dystrophin gene site mutations in exons <45 relation and the extent of myocardial fibrosis measured by cardiac magnetic resonance

    Full Information

    First Posted
    March 24, 2015
    Last Updated
    May 1, 2015
    Sponsor
    InCor Heart Institute
    Collaborators
    Federal University of Minas Gerais, University of Sao Paulo
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02432885
    Brief Title
    Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial
    Official Title
    Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2015
    Overall Recruitment Status
    Completed
    Study Start Date
    June 2009 (undefined)
    Primary Completion Date
    June 2012 (Actual)
    Study Completion Date
    June 2013 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    InCor Heart Institute
    Collaborators
    Federal University of Minas Gerais, University of Sao Paulo

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    This trial intends to evaluate myocardial Fibrosis progression in Duchenne and Becker Muscular Dystrophy, as well the influence of ACE inhibitors in fibrosis progression. Additionally, this study aims to determine genetic predictors of cardiac involvement in these dystrophies.
    Detailed Description
    Duchenne and Becker muscular dystrophies (DMD/BMD) are diseases characterized by progressive skeletal muscle degeneration and replacement by fibrofatty tissue. Data on cardiac involvement (defined as myocardial fibrosis), effect of ACE-inhibitors and specific genetic mutations on myocardial involvement detected by cardiac magnetic resonance (CMR) is lacking. The study will include 76 patients with DMD/BMD. All patients will be referred to two CMRs for assessment of ventricular function and myocardial fibrosis. Patients with myocardial fibrosis and normal left ventricle ejection fraction (LVEF) will be randomized into two groups, each group receiving ACE-inhibitor treatment or no treatment for cardiomyopathy. A genetic profile will be performed in every patient to identify possible mutations related to cardiac involvement.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Myocardial Fibrosis, Muscular Dystrophies
    Keywords
    cardiac magnetic resonance, myocardial fibrosis, muscular dystrophies

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    76 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    ACE inhibitor
    Arm Type
    Experimental
    Arm Description
    ACE inhibitor (enalapril up to 20mg BID), in patients with preserved EF (LVEF grater than 50%) and with detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance, randomized to therapy or not.
    Arm Title
    Control
    Arm Type
    No Intervention
    Arm Description
    Patients with preserved EF (LVEF grater than 50%) and with no detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance
    Intervention Type
    Drug
    Intervention Name(s)
    Enalapril
    Intervention Description
    up to 20mg bid
    Primary Outcome Measure Information:
    Title
    Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy
    Description
    Progression of myocardial fibrosis
    Time Frame
    2 years
    Secondary Outcome Measure Information:
    Title
    Specific genetic mutations as predictors of cardiac involvement
    Description
    Relation of dystrophin gene site mutations in exons <45 relation and the extent of myocardial fibrosis measured by cardiac magnetic resonance
    Time Frame
    2 years

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    6 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker Exclusion Criteria: Contraindications to cardiovascular magnetic resonance imaging
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Carlos E Rochitte, MD, PhD
    Organizational Affiliation
    InCor, Heart Institute, University of Sao Paulo Medical School
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    27926769
    Citation
    Silva MC, Magalhaes TA, Meira ZM, Rassi CH, Andrade AC, Gutierrez PS, Azevedo CF, Gurgel-Giannetti J, Vainzof M, Zatz M, Kalil-Filho R, Rochitte CE. Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial. JAMA Cardiol. 2017 Feb 1;2(2):190-199. doi: 10.1001/jamacardio.2016.4801.
    Results Reference
    derived

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    Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial

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