DMD Heart Protection - long term follow-up study (V 1.2)
Research type
Research Study
Full title
Long-term follow-up of heart function in participants of DMD Heart Protection Study (EudraCT number of original study: 2007-005932-10)
IRAS ID
269110
Contact name
John Bourke
Contact email
Sponsor organisation
Newcastle Joint Research Office, Newcastle upon Tyne NHS Hospitals Foundation Trust
Duration of Study in the UK
0 years, 9 months, 31 days
Research summary
Almost all boys with Duchenne muscular dystrophy develop progressive heart muscle weakness. The DMD Heart Protection study (2011-’18) recruited 85 boys, 5-13 years old, with Duchenne muscular dystrophy without evidence of heart muscle weakness (left ventricular dysfunction) and randomised them to receive either perindopril and bisoprolol or matched placebo in a double-blind study at 4- UK NHS hospitals. The study ended in 2018 and results did not show a group-difference between study arms in the primary end-point - change in left ventricular ejection fraction from baseline to 36 months. Because of the young age of participants, variability in the age of onset of cardiomyopathy in DMD and the fact that 88% were taking concomitant steroid therapy, which delays DMD-cardiomyopathy onset by an average of 2-years, the negative result is probably explained by an inadequate length of follow up to show differences. Furthermore, the negative finding is consistent with that of the only other previous randomised trial of heart therapy in DMD (Duboc et al, 2005), whose participants did not receive steroid therapy. As in the DMD-Heart Protection study, all participants in the French study received active therapy at study exit. Over 5-year follow-up, the French group reported a survival benefit in the active treatment arm (Duboc et al, 2007). The object of this follow-on study is to collect, collate and analyse results of heart and heart-related assessments, performed as part of normal NHS-care ('Standard of Care’ management) on the original participants since they exited the study. The null hypothesis is that the early introduction of the perindopril-bisoprolol combination does not delay the onset or slow the rate of decline in left ventricular function over 5-6-year follow-up. The results will provide the evidence to inform clinicians about the benefits or otherwise of prophylactic, combination, heart medications in young boys with DMD.
REC name
London - Brent Research Ethics Committee
REC reference
21/PR/0595
Date of REC Opinion
13 May 2021
REC opinion
Favourable Opinion