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

Sponsor

InCor Heart Institute (Other)

Overall Status

Completed

CT.gov ID

NCT02432885

Collaborator

Federal University of Minas Gerais (Other), University of Sao Paulo (Other)

Enrollment

Arms

Duration (Months)

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Myocardial Fibrosis Muscular Dystrophies	Drug: Enalapril	Phase 3

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

76 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy

Study Start Date :

Jun 1, 2009

Actual Primary Completion Date :

Jun 1, 2012

Actual Study Completion Date :

Jun 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
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.	Drug: Enalapril up to 20mg bid
No Intervention: Control Patients with preserved EF (LVEF grater than 50%) and with no detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance

Arm

Intervention/Treatment

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.

Drug: Enalapril

up to 20mg bid

No Intervention: Control

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

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker

Exclusion Criteria:

Contraindications to cardiovascular magnetic resonance imaging

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

InCor Heart Institute
Federal University of Minas Gerais
University of Sao Paulo

Investigators

Principal Investigator: Carlos E Rochitte, MD, PhD, InCor, Heart Institute, University of Sao Paulo Medical School

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Carlos Eduardo Rochitte, Associate Professor of Cardiology, InCor Heart Institute

ClinicalTrials.gov Identifier:

NCT02432885

Other Study ID Numbers:

1095/08

First Posted:

May 4, 2015

Last Update Posted:

May 4, 2015

Last Verified:

Apr 1, 2015

Keywords provided by Carlos Eduardo Rochitte, Associate Professor of Cardiology, InCor Heart Institute

cardiac magnetic resonance

myocardial fibrosis

muscular dystrophies

Additional relevant MeSH terms:

Muscular Dystrophies

Muscular Dystrophy, Duchenne

Fibrosis

Enalapril

Study Results

No Results Posted as of May 4, 2015