Heart Drug Being Tested in DMD

Eplerenone, a drug commonly used to prevent scarring after a heart attack, is being tested in a phase 2-3 clinical trial to determine whether it can stop or slow heart damage in people with Duchenne muscular dystrophy (DMD).  

Eplerenone blocks scarring that over time can turn heart muscle into nonfunctioning fatty tissue.

A key trial element will be the use of enhanced cardiac magnetic resonance (CMR), an imaging technique that can detect very small declines in heart function much earlier than the current heart imaging technique used in DMD, an echocardiogram. The investigators expect that the use of CMR will help them determine the best time to detect early changes in heart function and start cardiac therapy.

Recruitment under way

The trial is being conducted at two study sites in Ohio: Cincinnati Children's Hospital Medical Center in Cincinnati (view press release), and the Ohio State University Medical Center in Columbus.

Trial investigators are seeking 40 boys with DMD, age 7 years or older, who are able to undergo complete cardiac magnetic resonance testing without sedation, and who meet other study criteria.

Participants will be assigned to receive a 25-milligram tablet of eplerenone, once daily for 12 months; or a placebo tablet (made of an inactive substance) once daily for 12 months.

Eplerenone may reduce DMD-related heart strain

In addition to causing skeletal muscles to weaken, lack of dystrophin protein (the underlying molecular cause of DMD) can weaken the heart muscle, resulting in a condition called cardiomyopathy. Over time, the damage done by DMD to the heart can become life-threatening.

Eplerenone belongs to a class of drugs called aldosterone antagonists, or aldosterone blockers, which work by blocking receptors for the aldosterone hormone inside cells. The kidneys respond by holding onto potassium and ridding the body of fluid through increased urination. This results in a decrease in blood pressure and total blood volume, which eases strain and reduces the heart's workload.

Eplerenone has shown promise in man and mouse

Eplerenone has been successfully used to treat fibrosis in adult heart patients.

Additionally, in mouse models of DMD, treatment with the medication resulted in improved function and reduced scarring of the heart. Researchers treated two groups of mice, one at 8 weeks of age and a second at 4 weeks. As the mice aged, the researchers noted improved heart function in both groups. Benefits were more pronounced in the mice that were treated earlier, an indication that earlier treatment is likely to be more beneficial.

For details and contact information on the new study, see Early Treatment of Cardiomyopathy in Duchenne Muscular Dystrophy; or go to ClinicalTrials.gov and enter NCT01521546 into the search box.


About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to ClinicalTrials.gov.

20 521