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
| ISRCTN | ISRCTN50395346 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN50395346 |
| EudraCT/CTIS number | 2007-005932-10 |
| Secondary identifying numbers | 1.1 |
- Submission date
- 12/06/2007
- Registration date
- 13/08/2007
- Last edited
- 31/12/2020
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English Summary
Not provided at time of registration
Contact information
Dr John Bourke
Scientific
Scientific
Freeman Hospital
Newcastle upon Tyne
NE7 7DN
United Kingdom
Study information
| 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 |
| 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 |
| Study 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. |
| 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 |
Eligibility
| 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 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 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
NE7 7DN
United Kingdom
Sponsor information
Newcastle upon Tyne Hospitals NHS Foundation Trust (UK)
Hospital/treatment centre
Hospital/treatment centre
Research and Development Office
4th Floor Leazes Wing
Royal Victoria Infirmary
Newcastle upon Tyne
NE1 4LP
England
United Kingdom
| Website | http://www.newcastle-hospitals.org.uk/ |
|---|---|
"ROR" |
https://ror.org/05p40t847 |
Funders
Funder type
Charity
British Heart Foundation (UK)
Private sector organisation / Trusts, charities, foundations (both public and private)
Private sector organisation / Trusts, charities, foundations (both public and private)
- Alternative name(s)
- the_bhf, The British Heart Foundation, BHF
- Location
- United Kingdom
Results and Publications
| Intention to publish date | 31/10/2019 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
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 |
Editorial Notes
31/12/2020: The following changes have been made:
1. Publication reference added.
2. Added EudraCT/clinicaltrials.gov link to basic results (scientific).
17/01/2019: The following changes have been made to the trial record:
1. The overall trial end date has been changed from 01/09/2012 to 30/03/2019
2. The recruitment end date has been changed from 01/09/2012 to 30/03/2018
3. The intention to publish date has been added
04/10/2017: No publications found in PubMed, verifying study status with principal investigator
