A double-blind randomised multi-centre, placebo-controlled trial of combined ACE-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in genetically characterised males with DMD without echo-detectable left ventricular dysfunction

• Status: Closed
• Sponsor: The Newcastle upon Tyne Hospitals NHS Foundation Trust
• Funder: British Heart Foundation
• Therapeutic area: Cardiovascular
• Type of study: CTIMP

Aim: Determine whether the introduction of ACE-inhibitor combined with beta-blocker therapy, before the onset of echo-detectable left ventricular dysfunction, can delay the age of onset and/or slow the rate of progression of cardiomyopathy compared to placebo in males with DMD.

Primary Outcome:
Change in left ventricular ejection fraction (LVEF %), compared to baseline after a minimum of three years of combination therapy or placebo.
To assess the robustness of ejection fraction result, similar comparisons will be made for parameters of left ventricular end-systolic dimension, wall motion index and left ventricular fractional shortening (%) and mitral flow to left ventricular tissue Doppler ratios (E/E’ ratios)

• Clinical Phase: III
• Population: Paediatrics (males)
• Design: RCT
• Setting: Secondary Care
• Planned Sample Size: 140