Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne, Becker muscular dystrophy, cardioprotective therapy, cardiac magnetic resonance, ultrasound tissue characterization
Brief summary
Data on preventive therapy in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) affected individuals without cardiac involvement are very limited and currently lacking regard both ACE-inhibitors and Beta-Blockers in Becker Muscular Dystrophy and for the latter even in Duchenne Muscular Dystrophy patients. Thus, the study aim is to compare the efficacy of carvedilol vs ramipril on myocardial tissue properties and heart function, performing CMR and myocardial Ultrasound Tissue Characterisation analysis.
Detailed description
This protocol represent an open randomized and prospective trial, designed to answer the specific question regarding the role of the cardioprotective therapy in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy patients. In this light, CMR could provide relevant data, reinforcing the scientific background, to start early (particularly in BMD patients in whom this is still a debated question) a cardioprotective treatment with carvedilol or ramipril.Finally,this clinical trial will clarify whether a preventive therapy may be helpful on the clinical outcome, both in reducing myocardial fibrosis and preventing the progression towards the cardiomyopathy.
Interventions
DRUGcarvedilol
carvedilol vs ramipril
DRUGramipril
carvedilol vs ramipril
Sponsors
Catholic University, Italy
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
2 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
1. Immunohystochemical and molecular diagnosis of Duchenne and Becker muscular dystrophy. 2. Not evidence of clinical cardiomyopathy,normal 2D-echocardiography with normal systolic,WMSI = 1) and diastolic function. 3. DMD patients treated with steroid therapy. 4. All DMD and BMD patients are not treated with cardiological therapy (ACE-inhibitors, ARBs or Beta-Blockers). 5. Written informed consent to study participation (with serial visit, CMR and echocardiographic study) is required from all patients themselves, as well as their parent or guardian and healthy-control subjects.
Exclusion criteria
1. Failure to obtain informed consent from patients, parents or guardians. 2. Any controindications to carvedilol or ramipril treatment (bronchial asthma, diabetes, any degree of renal failure (all patients are required to have a normal creatinine level and clearance). 3. in BMD patients ECG changes suggestive of ischemic heart disease, left bundle-branch block, atrial flutter/fibrillation, ventricular arrhythmias, any degree of atrioventricular block and left ventricular (LV) hypertrophy. Aspecific ST changes will be not considered as electrocardiographic
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Left ventricular Ejection Fraction, systolic and diastolic left ventricular volumes and LGE (as a quantitative measure) detected by MRI and myocardial Ultrasound Tissue Characterisation data by Echocardiography. | 1 year |
Secondary
| Measure | Time frame |
|---|---|
| Prevalence of LGE in DMD and BMD patients,the effects of pharmacological therapy both on LGE evolution and myocardial UTC analysis. | 1 year |
Countries
Italy
Contacts
Primary ContactVincenzo Giglio, MD, PhD
Backup ContactFortunato Mangiola, MD
Outcome results
None listed