Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protoc

This paper describes the design of a double-blind, multicenter, randomized, placebo-controlled study to evaluate whether combination therapy with an ACE inhibitor and a beta-blocker delays the age of onset and/or slows the rate of progression of cardiomyopathy in male children with Duchenne muscular dystrophy. The primary endpoint is change in left ventricular ejection fraction; secondary endpoints include cardiac MRI measures of left ventricular size and function, as well as the presence, extent, and distribution of intramyocardial fibrosis. Baseline FEV1 and FVC values were collected using a Vitalograph spirometer.