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Clinical trial first to test heart drug regimen for Duchenne muscular dystrophy
Date:6/26/2012

in mice treated earlier at 4 weeks.

"The results of that research indicated that earlier treatment with this medication offers an excellent chance of preserving heart function, decreasing fibrosis and reducing cardiac death in DMD," said Hor.

The Phase 2/3 double-blind randomized clinical trial will treat 40 patients initially for one year, focusing on patients older than 7 years of age with evidence of heart muscle scarring but normal heart function as determined by LVEF.

The study is supported in part by Ballou Skies, Parent Project Muscular Dystrophy (PPMD), and The Lindner Center for Research & Education at The Christ Hospital Foundation. Long time research collaborator, Dr. Wojciech Mazur from The Christ Hospital in Cincinnati, is also assisting with the trial.

Hor said treatment with eplerenone will be compared to a placebo and current standard drug treatments. Depending on trial results, the research could move into a larger trial to include more patients at different stages of heart disease. Hor said he expects the current trial to take about 12-18 months to enroll patients and a little over two years to complete.


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Contact: Nick Miller
nicholas.miller@cchmc.org
513-803-6035
Cincinnati Children's Hospital Medical Center
Source:Eurekalert

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