Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2016-06-01
Target enrollment:
Participant gender:
Summary
Duchenne muscular dystrophy (DMD), the most common muscular dystrophy, leads to skeletal and
cardiac muscle damage. Treatment of pulmonary complications has improved survival; however,
heart muscle disease or cardiomyopathy has emerged as a leading cause of death, typically by
the third decade. Although myocardial changes begin early, clinically significant heart
disease is rarely detected in the first decade of life. Consequently, DMD cardiomyopathy
frequently goes unrecognized (and untreated) until advanced (and irreversible).
Current DMD cardiovascular care guidelines recommend beta-blockers and angiotensin converting
enzyme inhibitors (ACEIs) when decreased ejection fraction (EF) is noted by echocardiography
(echo); however, this strategy has not significantly improved outcomes. Our team has recently
made a breakthrough in a mouse study, showing in a model that causes the same heart muscle
disease in humans with DMD adding an old medicine traditionally used for high blood pressure
and late-stage heart failure can actually prevent heart muscle damage. Because of this drug's
proven safety in both children and adults, it is ready to be studied immediately in an RCT in
patients with DMD to hopefully show, as we did in mice, that we can prevent the devastating
consequences of heart muscle damage.