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Ramipril Versus Carvedilol in Duchenne and Becker Patients

Primary Purpose

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

Status

Unknown status

Phase

Phase 4

Locations

Italy

Study Type

Interventional

Intervention

carvedilol

ramipril

About this trial

This is an interventional prevention trial for Duchenne Muscular Dystrophy focused on measuring Duchenne, Becker muscular dystrophy, cardioprotective therapy, cardiac magnetic resonance, ultrasound tissue characterization

Eligibility Criteria

2 Years - 45 Years (Child, Adult)MaleAccepts Healthy Volunteers

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).

Sites / Locations

Unione Italiana lotta Distrofia MuscolareRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Ramipril

Carvedilol

Arm Description

Outcomes

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.

Secondary Outcome Measures

Prevalence of LGE in DMD and BMD patients,the effects of pharmacological therapy both on LGE evolution and myocardial UTC analysis.

Full Information

NCT ID

NCT00819845

First Posted

January 8, 2009

Last Updated

January 27, 2016

Sponsor

Catholic University, Italy

1. Study Identification

Unique Protocol Identification Number

NCT00819845

Brief Title

Ramipril Versus Carvedilol in Duchenne and Becker Patients

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 Type

Interventional

2. Study Status

Record Verification Date

January 2009

Overall Recruitment Status

Unknown status

Study Start Date

December 2008 (undefined)

Primary Completion Date

June 2009 (Actual)

Study Completion Date

December 2016 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor

Catholic University, Italy

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

Keywords

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

7. Study Design

Primary Purpose

Prevention

Study Phase

Phase 4

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

194 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Ramipril

Arm Type

Experimental

Arm Title

Carvedilol

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

carvedilol

Other Intervention Name(s)

Beta-Blocker

Intervention Description

carvedilol vs ramipril

Intervention Type

Drug

Intervention Name(s)

ramipril

Other Intervention Name(s)

ACE-inhibitor

Intervention Description

carvedilol vs ramipril

Primary Outcome Measure Information:

Title

Time Frame

1 year

Secondary Outcome Measure Information:

Title

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

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility 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).

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Vincenzo Giglio, MD, PhD

Phone

39-6-6604881

giglio.echo@libero.it

First Name & Middle Initial & Last Name or Official Title & Degree

Fortunato Mangiola, MD

Phone

39-6-6604881

fortunato.mangiola@uildmlazio.org

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Vincenzo Giglio, MD, PhD

Organizational Affiliation

Uildm, Rome

Official's Role

Principal Investigator

Facility Information:

Facility Name

Unione Italiana lotta Distrofia Muscolare

City

Rome

ZIP/Postal Code

00167

Country

Italy

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Vincenzo Giglio, MD, PhD

Phone

39-6-6604881

giglio.echo@libero.it

First Name & Middle Initial & Last Name & Degree

Fortunato Mangiola, MD

Phone

39-6-6604881

fortunato.mangiola@uildmlazio.org

First Name & Middle Initial & Last Name & Degree

Vincenzo Giglio, MD, PhD

12. IPD Sharing Statement

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Ramipril Versus Carvedilol in Duchenne and Becker Patients

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