Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy

Completed · Not applicable · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT01521546

Run by

Subha Raman

People needed

42

Starts

2012-02-01

Expected to finish

2016-06-01

Last updated by the study team

2016-11-08

Who can join

Age: 7 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• DMD patients age 7 years and older (and able to complete cardiac MRI without sedation) with preserved left ventricular (LV) systolic function and abnormal heart muscle by late post-gadolinium imaging (LGE)

You may not qualify if…

• renal insufficiency (GFR <40 mL/min/m2)
• non-MR compatible implants (e.g. neurostimulator, AICD)
• severe claustrophobia
• allergy to gadolinium contrast
• prior use of or known allergy to epleronone
• use of potassium-sparing diuretics
• serum potassium level of >5.0 mmol/L

Where it is running

• Mattel Children's Hospital and David Geffen School of Medicine at UCLA — Los Angeles, California, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• The Ohio State University Medical Center — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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