Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 76
- 主要终点
- Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker
排除标准
- •Contraindications to cardiovascular magnetic resonance imaging
研究组 & 干预措施
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.
干预措施: Enalapril (Drug)
结局指标
主要结局
Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy
时间窗: 2 years
Progression of myocardial fibrosis
次要结局
- Specific genetic mutations as predictors of cardiac involvement(2 years)
研究者
Carlos Eduardo Rochitte
Associate Professor of Cardiology
InCor Heart Institute