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 - DMD-Heart Protection

ewcastle upon Tyne Hospitals NHS Foundation Trust0 个研究点目标入组 85 人开始时间: 2009年4月14日最近更新: 2020年2月1日

适应症

试验速览

暂无简介。

•Boys with proven DMD (defined out of frame deletion OR frame-shifting point mutation OR absent/<3% dystrophin on muscle biopsy);
•Age 7-12 years
•Normal left ventricular function on trans-thoracic echocardiography [defined as left ventricular ejection fraction > 60% [as calculated from 16 segment wall motion score method (see appendix C of trial protocol ID NCTU:ISRCTN50395346)]
•Left ventricular fractional shortening > 28% [for calculation – see appendix C of trial protocol ID NCTU:ISRCTN50395346]
•no global or regional wall motion abnormalities – refer appendix C of trial protocol ID NCTU:ISRCTN50395346 for use of 16-segment wall motion scoring system.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Current or recent inclusion in another trial will not exclude participation in the DMD Heart Protection study.
•Contraindication to ACE-inhibitor or beta-blocker therapy (see SmPCs for perindopril (Appendix A of trial protocol) and bisoprolol (Appendix B of trial protocol ID NCTU:ISRCTN50395346) for contra-indications to these drugs).
•An initial echocardiogram of insufficient quality (see appendix C of trial protocol ID NCTU:ISRCTN50395346) to allow reliable measurements of ejection fraction, fractional shortening or wall motion (one repeat echocardiogram is permitted at baseline) may not enter the Trial.
•Renal function should be measured by plasma urea, creatinine and potassium prior to study entry. Patients with abnormal renal function [creatinine > upper limit of local laboratory range; typically > 120 mmol/l] or consistently abnormally high serum potassium level [K > upper limit of local laboratory range; typically 5 mmol/l] will also be excluded from the trial.
•Inability to swallow a placebo capsule of the size to be used in the trial

发起方

ewcastle upon Tyne Hospitals NHS Foundation Trust