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

Phase 3

Completed

• Conditions: Myocardial Fibrosis; Muscular Dystrophies
• Interventions: Drug: Enalapril

• Registration Number: NCT02432885
• Lead Sponsor: InCor Heart Institute

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.

Study Timeline

• Study Start: 2009-06
• Primary Completion: 2012-06
• Study Completion: 2013-06
• Study First Submitted: 2015-03-24
• Status Verified: 2015-04
• Study First Submitted QC: 2015-05-01
• Last Update Submitted: 2015-05-01
• Study First Posted: 2015-05-04
• Last Update Posted: 2015-05-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 76

Inclusion Criteria

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

Exclusion Criteria

• Contraindications to cardiovascular magnetic resonance imaging

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ACE inhibitor	Enalapril	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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