Ramipril Versus Carvedilol in Duchenne and Becker Patients - Full Text View

Sponsor:	Catholic University, Italy
Information provided by:	Catholic University, Italy
ClinicalTrials.gov Identifier:	NCT00819845

Purpose

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.

Condition	Intervention	Phase
Duchenne Muscular Dystrophy Becker Muscular Dystrophy	Drug: carvedilol Drug: ramipril	Phase IV

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Prevention
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.

Resource links provided by NLM:

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy

MedlinePlus related topics: Muscular Dystrophy Ultrasound

Drug Information available for: Carvedilol Ramipril

U.S. FDA Resources

Further study details as provided by Catholic University, Italy:

Primary Outcome Measures:

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. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Prevalence of LGE in DMD and BMD patients,the effects of pharmacological therapy both on LGE evolution and myocardial UTC analysis. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment:	194
Study Start Date:	December 2008
Estimated Study Completion Date:	December 2011
Estimated Primary Completion Date:	June 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Ramipril	Drug: ramipril carvedilol vs ramipril Other Name: ACE-inhibitor
Experimental: Carvedilol	Drug: carvedilol carvedilol vs ramipril Other Name: Beta-Blocker

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

Ages Eligible for Study:	2 Years to 45 Years
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	Yes

Criteria

Inclusion Criteria:

Immunohystochemical and molecular diagnosis of Duchenne and Becker muscular dystrophy.
Not evidence of clinical cardiomyopathy,normal 2D-echocardiography with normal systolic,WMSI = 1) and diastolic function.
DMD patients treated with steroid therapy.
All DMD and BMD patients are not treated with cardiological therapy (ACE-inhibitors, ARBs or Beta-Blockers).
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:

Failure to obtain informed consent from patients, parents or guardians.
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).
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.
In BMD patients exclusion criteria will be also hypertension and valvular heart disease other than trivial.
DMD and BMD patients requiring ventilatory (non-invasive or invasive) assistance.
Presence of systolic and/or diastolic dysfunction detected by 2D-Echocardiography.
Presence of any contraindications to CMR (including any history of claustrophobia).
Patients under the age of 2 years.
Renal failure, even mild.
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).

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00819845

Contacts

Contact: Vincenzo Giglio, MD, PhD	39-6-6604881	giglio.echo@libero.it
Contact: Fortunato Mangiola, MD	39-6-6604881	fortunato.mangiola@uildmlazio.org

Locations

Italy
Unione Italiana lotta Distrofia Muscolare	Recruiting
Rome, Italy, 00167
Contact: Vincenzo Giglio, MD, PhD 39-6-6604881 giglio.echo@libero.it
Contact: Fortunato Mangiola, MD 39-6-6604881 fortunato.mangiola@uildmlazio.org
Principal Investigator: Vincenzo Giglio, MD, PhD

Sponsors and Collaborators

Catholic University, Italy

Investigators

Principal Investigator:

Vincenzo Giglio, MD, PhD

Uildm, Rome

More Information

No publications provided

Responsible Party:	Vincenzo Giglio MD, PhD, Uildm of Rome
ClinicalTrials.gov Identifier:	NCT00819845 History of Changes
Other Study ID Numbers:	Uildm Rome
Study First Received:	January 8, 2009
Last Updated:	January 8, 2009
Health Authority:	Italy: The Italian Medicines Agency

Keywords provided by Catholic University, Italy:

Duchenne
Becker muscular dystrophy
cardioprotective therapy
cardiac magnetic resonance
ultrasound tissue characterization

Additional relevant MeSH terms:

Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Angiotensin-Converting Enzyme Inhibitors
Ramipril
Carvedilol
Protease Inhibitors
Enzyme Inhibitors

Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Adrenergic beta-Antagonists
Adrenergic Antagonists
Adrenergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Antihypertensive Agents
Cardiovascular Agents
Therapeutic Uses
Vasodilator Agents
Adrenergic alpha-1 Receptor Antagonists
Adrenergic alpha-Antagonists

ClinicalTrials.gov processed this record on February 09, 2012