Project Leader(s): Dr John P Bourke
Contact: Contact: Mr Chris Speed (firstname.lastname@example.org) 0191 222 6054; Miss Gillian Watson (email@example.com) 0191 222 8813
Sponsors: The Newcastle upon Tyne Hospitals NHS Foundation Trust
Partners: Newcastle University
Funder: British Heart Foundation
A double-blind, randomised, multi-centre, placebo-controlled trial to determine the effect of combined ACE-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in 140 genetically-characterised males with Duchenne Muscular Dystrophy, without echo-detectable left ventricular (LV) dysfunction, aged 7-12 years, at five UK sites.
All boys will receive one capsule containing 2mg perindopril and 1.25mg bisoprolol, or placebo, once daily for the first month. Boys then receive one or two capsules per day, dependent on weight, for between 36 and 60 months.
Study objectives are to determine whether this combined therapy delays the age of onset and/or slows the rate of progression of cardiomyopathy. The primary outcome measure is change in LV ejection fraction compared to baseline, after at least two years’ combination therapy/placebo. The robustness of EF result will be assessed by comparing LV end-systolic dimension, wall motion index and LV fractional shortening, and mitral flow to LV tissue Doppler ratios.