A double-blind randomised multi-centre, placebo-controlled trial of combined angiotensin converting enzyme-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in genetically characterised males with Duchenne Muscular Dystrophy without echo-detectable left ventricular dysfunctio
- Conditions
- Duchenne muscular dystrophyNervous System DiseasesDiseases of myoneural junction and muscle
- Registration Number
- ISRCTN50395346
- Lead Sponsor
- ewcastle upon Tyne Hospitals NHS Foundation Trust (UK)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- 140
1. Boys aged 7 to 12 years
2. Genetically confirmed DMD with normal left ventricular function on trans-thoracic echocardiography (i.e., left ventricular ejection fraction by Simpson's biplane method greater than 55% [normal mean + SD = 63 + 5%], no global or regional wall motion abnormalities)
1. Contraindication to ACE-inhibitor or beta-blocker therapy
2. Patients, whose initial echo is of insufficient quality to allow reliable measurements of ejection fraction or wall motion
3. Patients with abnormal echocardiograms at baseline
4. Patients with abnormal renal function (creatinine greater than upper limit of local laboratory range; typically greater than 120 mmol/l) or consistently abnormally high serum potassium level (K greater than upper limit of local laboratory range; typically 5 mmol/l)
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in left ventricular ejection fraction by Simpson's biplane disk method, compared to baseline, after a minimum of two years of combination therapy or placebo. To assess robustness of ejection fraction result, similar comparisons will be made for parameters of left ventricular end-systolic volume and wall motion index.
- Secondary Outcome Measures
Name Time Method <br> 1. Death from any cause<br> 2. Development of symptoms and signs of congestive cardiac failure<br> 3. Sufficient objective deterioration in cardiac function, without symptoms to make continued placebo therapy unethical<br><br> Secondary outcomes are measured at baseline and 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60 months.<br>