The Duchenne muscular dystrophy heart protection study - a randomised trial of ACE-inhibitor and beta-blocker therapy in preventing cardiomyopathy
Boys with Duchenne muscular dystrophy (DMD) begin to show heart muscle weakness in their teens. Ultimately heart weakness causes symptoms and contributes to premature death from DMD. It may be possible to protect the heart using existing drugs, ACE-inhibitors and beta-blockers, although the benefits of these treatments in DMD have not been scientifically tested in a clinical trial. This research will determine whether combining the two drugs in boys with DMD prevents heart deterioration. In total, around 140 boys aged 7 to 12 years with DMD and normal heart scans will be invited to take part in a clinical trial. Half of the boys will be given an ACE-inhibitor and a beta-blocker and the other half a dummy drug or placebo. Heart function, quality of life and any bad effects from therapy will be measured repeatedly for 5 years. Results will establish whether and when people with DMD should start treatments and should help to find this out for other related inherited heart conditions.
Project details
| Grant amount | £805,038 |
|---|---|
| Grant type | Chairs & Programme Grants |
| Application type | Special Project |
| Start Date | 26 March 2011 |
| Duration | 5 years |
| Reference | SP/05/001/18616 |
| Status | Complete |
