Queen Square Centre for Neuromuscular Diseases


A trial of combined ACE-inhibitor and beta-blocker therapy for heart protection in genetically characterised DMD males with without echo-detectable left ventricular dysfunction

Sponsor Newcastle upon Tyne NHS Hospitals Foundation Trust
Funder British Heart Foundation
CI Dr John Burke / Professor Francesco Muntoni
sites Newcastle, London GOSH
Contact trial coordinator on 020 7905 2639020 7905 2639
More information


DMD is an X-linked inherited neuromuscular disorder. It results from abnormal expression of the protein dystrophin.

Clinical features include progressive weakness of proximal limb-girdle muscles and calf muscle hypertrophy.

20 to 30% of DMD patients have evidence of left ventricular dysfunction (LVD) on echocardiography by age 10 years. But this kind of heart failure is often ignored by cardiologists because it only happens terminally in DMD patients.

Even now, drug treatment for the heart is only started in DMD patients when heart failure is evident. It is then only used for symptom control without any expectation that it can prolong life.

Trial information

Primary objective: to determine if ACE inhibitor combined with beta-blocker therapy, before the onset of echo-detectable LVD, can:

  • delay the age of onset 
  • and/or slow the rate of progression of cardiomyopathy.

This is a double-blind randomised, placebo-controlled Phase III trial of combined ACE inhibitor and beta-blocker therapy (perindopril and bisoprolol).

It will last at least three years but no more than five years.


140 participants (70 per arm)