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 Protectio

Phase 1

• Conditions: Duchenne muscular dystrophy; MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy

• Registration Number: EUCTR2007-005932-10-GB
• Lead Sponsor: ewcastle upon Tyne Hospitals NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2009-04-14
• Study Completion: 2018-03-23
• Last Update Posted: 1 February 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 85

Inclusion Criteria

• 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

Exclusion Criteria

• 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified