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

University of São Paulo (USP)

Status and phase: Completed; Phase 3

Conditions: Myocardial Fibrosis; Muscular Dystrophies

Treatments: Drug: Enalapril

Study type: Interventional

Funder types: Other

Identifiers

NCT02432885

1095/08

Details and patient eligibility

About

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 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.

Enrollment: 76 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

• Contraindications to cardiovascular magnetic resonance imaging

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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76 participants in 2 patient groups

ACE inhibitor

Experimental group

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.

Treatment:

Drug: Enalapril

Control

No Intervention group

Description:

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