Ramipril Versus Carvedilol in Duchenne and Becker Patients

NCT ID: NCT00819845

Last Updated: 2016-01-28

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE4
• Total Enrollment: 194 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-12-31
• Study Completion Date: 2016-12-31

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.

Conditions

Duchenne Muscular Dystrophy; Becker Muscular Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Ramipril

Group Type: EXPERIMENTAL

ramipril

Intervention Type: DRUG

carvedilol vs ramipril

Carvedilol

Group Type: EXPERIMENTAL

carvedilol

Intervention Type: DRUG

carvedilol vs ramipril

Interventions

carvedilol

carvedilol vs ramipril

Intervention Type: DRUG

ramipril

carvedilol vs ramipril

Intervention Type: DRUG

Other Intervention Names

Beta-Blocker; ACE-inhibitor

Eligibility 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).

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).

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: Yes

Sponsors

Catholic University, Italy

OTHER

Sponsor Role: lead

Responsible Party

Uildm of Rome

Principal Investigators

Vincenzo Giglio, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Uildm, Rome

Locations

Unione Italiana lotta Distrofia Muscolare

Rome, , Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Vincenzo Giglio, MD, PhD

Role: CONTACT

giglio.echo@libero.it

39-6-6604881

Fortunato Mangiola, MD

Role: CONTACT

fortunato.mangiola@uildmlazio.org

39-6-6604881

Facility Contacts

Vincenzo Giglio, MD, PhD

Role: primary

giglio.echo@libero.it

39-6-6604881

Fortunato Mangiola, MD

Role: backup

fortunato.mangiola@uildmlazio.org

39-6-6604881

Other Identifiers

Uildm Rome

Identifier Type: -

Identifier Source: org_study_id