Ramipril Versus Carvedilol in Duchenne and Becker Patients
Information source: Catholic University, Italy
Information obtained from ClinicalTrials.gov on October 19, 2009
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Intervention: carvedilol (Drug); ramipril (Drug)
Phase: Phase 4
Status: Recruiting
Sponsored by: Catholic University, Italy Official(s) and/or principal investigator(s):
Vincenzo Giglio, MD, PhD, Principal Investigator, Affiliation: Uildm, Rome
Overall contact:
Vincenzo Giglio, MD, PhD, Phone: 39-6-6604881, Email: giglio.echo@libero.it
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.
Clinical Details
Official title: Effects of Cardioprotective Therapy, Carvedilol vs Ramipril, in Patients Affected by Duchenne and Becker Muscular Dystrophy. Clinical Significance and Prognostic Value of Cardiac Magnetic Resonance Study.
Study design: Prevention, Randomized, Open Label, Parallel Assignment, Efficacy Study
Primary outcome: 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.
Secondary outcome: Prevalence of LGE in DMD and BMD patients,the effects of pharmacological therapy both on LGE evolution and myocardial UTC 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.
Eligibility
Minimum age: 2 Years.
Maximum age: 45 Years.
Gender(s): Male.
Criteria:
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 exclusion criteria both in DMD and BMD
patients.
4. In BMD patients exclusion criteria will be also hypertension and valvular heart
disease other than trivial.
5. DMD and BMD patients requiring ventilatory (non-invasive or invasive) assistance.
6. Presence of systolic and/or diastolic dysfunction detected by 2D-Echocardiography.
7. Presence of any contraindications to CMR (including any history of claustrophobia).
8. Patients under the age of 2 years.
9. Renal failure, even mild.
10. Patient unable or unwilling to attend the follow-up and tests, in the opinion of
local study principal investigator, (children not willing to perform CMR will not be
enrolled).
Locations and Contacts
Vincenzo Giglio, MD, PhD, Phone: 39-6-6604881, Email: giglio.echo@libero.it
Unione Italiana lotta Distrofia Muscolare, Rome 00167, Italy; Recruiting
Vincenzo Giglio, MD, PhD, Phone: 39-6-6604881, Email: giglio.echo@libero.it
Fortunato Mangiola, MD, Phone: 39-6-6604881, Email: fortunato.mangiola@uildmlazio.org
Vincenzo Giglio, MD, PhD, Principal Investigator
Additional Information
Starting date: December 2008
Ending date: December 2011
Last updated: January 8, 2009
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