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

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

key information

study id #: NCT02432885

condition: Myocardial Fibrosis, Muscular Dystrophies

status: completed

purpose:

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.

intervention: Enalapril

mechanism of action: ACE inhibitor and beta-blocker to prevent cardiac function deterioration

results: https://clinicaltrials.gov/ct2/show/results/NCT02432885

last updated: November 22, 2018

study details

start date: June 2009

estimated completion: June 2012

phase of development: Phase 3

size / enrollment: 76

study 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.

primary outcomes:

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

secondary outcomes:

  • 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

inclusion criteria: Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker

exclusion criteria: Contraindications to cardiovascular magnetic resonance imaging

study contacts

sponsor: InCor Heart Institute

investigators: Carlos E Rochitte, MD, PhD