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

Background

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.

Recruitment

140 participants (70 per arm)