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

Overview

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.

Full Title of Study: “Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy – Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 2012

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.

Interventions

  • Drug: Enalapril
    • up to 20mg bid

Arms, Groups and Cohorts

  • Experimental: ACE inhibitor
    • 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.
  • No Intervention: Control
    • Patients with preserved EF (LVEF grater than 50%) and with no detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance

Clinical Trial Outcome Measures

Primary Measures

  • Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy
    • Time Frame: 2 years
    • Progression of myocardial fibrosis

Secondary Measures

  • Specific genetic mutations as predictors of cardiac involvement
    • Time Frame: 2 years
    • Relation of dystrophin gene site mutations in exons <45 relation and the extent of myocardial fibrosis measured by cardiac magnetic resonance

Participating in This Clinical Trial

Inclusion Criteria

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

  • Contraindications to cardiovascular magnetic resonance imaging

Gender Eligibility: All

Minimum Age: 6 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • InCor Heart Institute
  • Collaborator
    • Federal University of Minas Gerais
  • Provider of Information About this Clinical Study
    • Principal Investigator: Carlos Eduardo Rochitte, Associate Professor of Cardiology – InCor Heart Institute
  • Overall Official(s)
    • Carlos E Rochitte, MD, PhD, Principal Investigator, InCor, Heart Institute, University of Sao Paulo Medical School

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