Ramipril Versus Carvedilol in Duchenne and Becker Patients

The recruitment status of this study is unknown because the information has not been verified recently.
Verified January 2009 by Catholic University, Italy.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Catholic University, Italy
ClinicalTrials.gov Identifier:
NCT00819845
First received: January 8, 2009
Last updated: NA
Last verified: January 2009
History: No changes posted
  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 4

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:


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:

  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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 Dystrophies
Muscular Dystrophy, Duchenne
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases
Carvedilol
Ramipril
Adrenergic Agents
Adrenergic alpha-1 Receptor Antagonists
Adrenergic alpha-Antagonists
Adrenergic Antagonists
Adrenergic beta-Antagonists
Angiotensin-Converting Enzyme Inhibitors
Antihypertensive Agents
Cardiovascular Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Pharmacologic Actions
Physiological Effects of Drugs
Protease Inhibitors
Therapeutic Uses
Vasodilator Agents

ClinicalTrials.gov processed this record on October 22, 2014