Condition category
Nervous System Diseases
Date applied
12/06/2007
Date assigned
13/08/2007
Last edited
09/06/2015
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr John Bourke

ORCID ID

Contact details

Freeman Hospital
Newcastle upon Tyne
NE7 7DN
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

1.1

Study information

Scientific title

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 dysfunction

Acronym

DMD Heart

Study hypothesis

To determine whether the introduction of Angiotensin Converting Enzyme-inhibitor (ACE-inhibitor) (perindopril) combined with beta-blocker therapy (bisoprolol), before the onset of echo-detectable left ventricular dysfunction, can delay the age of onset and/or slow the rate of progression of cardiomyopathy in males with Duchenne Muscular Dystrophy (DMD).

Ethics approval

Ethics pending as of 12/06/2007. No patients will be recruited before ethics approval has been received.

Study design

Double-blind randomised multi-centre placebo-controlled trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

Duchenne muscular dystrophy

Intervention

Presentation of Investigational Medicinal Product (IMP):
Each participant will receive:
1. A one-month supply of perindopril 2 mg/bisoprolol 1.25 mg or placebo for the run-in period
2. Six-monthly supplies of perindopril 4 mg/bisoprolol 2.5 mg or placebo for the remainder of the trial

Introduction of IMP or placebo therapies:
The IMP or placebo therapy will be introduced in the following stepwise manner:
Step 1: combined capsule containing perindopril 2 mg/bisoprolol 1.25 mg or matching placebo to be administered by parent(s)/legal guardian(s) at bedtime
Step 2 (one month later): change to maintenance capsule containing perindopril 4 mg/bisoprolol 2.5 mg or matching placebo to be administered by parent(s)/legal guardian(s)at bedtime

Treatment period is for two years. Follow up is for up to 60 months.

Intervention type

Drug

Phase

Not Applicable

Drug names

Perindopril, bisoprolol

Primary outcome measures

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

1. Death from any cause
2. Development of symptoms and signs of congestive cardiac failure
3. Sufficient objective deterioration in cardiac function, without symptoms to make continued placebo therapy unethical

Secondary outcomes are measured at baseline and 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60 months.

Overall trial start date

01/09/2007

Overall trial end date

01/09/2012

Reason abandoned

Eligibility

Participant inclusion criteria

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)

Participant type

Patient

Age group

Child

Gender

Male

Target number of participants

140

Participant exclusion criteria

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)

Recruitment start date

01/09/2007

Recruitment end date

01/09/2012

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Freeman Hospital
Newcastle upon Tyne
NE7 7DN
United Kingdom

Sponsor information

Organisation

Newcastle upon Tyne Hospitals NHS Foundation Trust (UK)

Sponsor details

Research and Development Office
4th Floor Leazes Wing
Royal Victoria Infirmary
Newcastle upon Tyne
NE1 4LP
United Kingdom

Sponsor type

Government

Website

http://www.newcastle-hospitals.org.uk/

Funders

Funder type

Charity

Funder name

British Heart Foundation (UK)

Alternative name(s)

BHF

Funding Body Type

private sector organisation

Funding Body Subtype

foundation

Location

United Kingdom

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes