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Duchenne Muscular Dystrophy | Ramipril Versus Carvedilol in Duchenne and Becker Patients

Duchenne Muscular Dystrophy research study

What is the primary objective of this study?

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.

Who is eligible to participate?

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).

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

Duchenne Muscular Dystrophy

Becker Muscular Dystrophy

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Drug:carvedilolcarvedilol vs ramipril

Drug:ramiprilcarvedilol vs ramipril

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

Ramipril

Carvedilol

Study Status

Unknown status

Start Date: December 2008

Completed Date: December 2016

Phase: Phase 4

Type: Interventional

Design:

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.

Study sponsors, principal investigator, and references

Principal Investigator: Vincenzo Giglio, MD, PhD

Lead Sponsor: Catholic University, Italy

Collaborator:

More information:https://clinicaltrials.gov/show/NCT00819845

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