Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2016-06-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Subha Raman
• Collaborator: Ballou Skies
• Treatments: Eplerenone; Spironolactone

Criteria

Inclusion Criteria:

• 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)

Exclusion Criteria:

• 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