Additional identifiers
EudraCT/CTIS number
2007-005932-10
IRAS 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(s)
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
Study setting(s)
Hospital
Study 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
Pharmaceutical study type(s)
Phase
Not Applicable
Drug/device/biological/vaccine name(s)
Perindopril, bisoprolol
Primary outcome measure
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 study start date
01/09/2007
Overall study end date
30/03/2019
Reason abandoned (if study stopped)
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(s)
Patient
Age group
Child
Lower age limit
7 Years
Upper age limit
12 Years
Sex
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
30/03/2018
Locations
Countries of recruitment
England, United Kingdom
Study 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
England
United Kingdom
Sponsor type
Hospital/treatment centre
Website
http://www.newcastle-hospitals.org.uk/
ROR
Funders
Funder type
Charity
Funder name
British Heart Foundation (UK)
Alternative name(s)
the_bhf, The British Heart Foundation, BHF
Funding Body Type
private sector organisation
Funding Body Subtype
Trusts, charities, foundations (both public and private)
Location
United Kingdom
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
31/10/2019
Individual participant data (IPD) sharing plan
IPD sharing plan summary
Not provided at time of registration
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Basic results | No | No | |||
| Protocol article | protocol | 19/12/2018 | 31/12/2020 | Yes | No |