Ramipril Versus Carvedilol in Duchenne and Becker Patients

NCT00819845 | Unknown Phase 4

• 1 other identifier: Uildm Rome
• Study Type: interventional
• Target: 194
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy; Becker Muscular Dystrophy

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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.

  1 year

Secondary Outcomes (1)

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

  1 year

Study Arms (2)

Ramipril

EXPERIMENTAL

Drug: ramipril

Carvedilol

EXPERIMENTAL

Drug: carvedilol

Interventions

carvedilol DRUG

carvedilol vs ramipril

Also known as: Beta-Blocker

Carvedilol

ramipril DRUG

carvedilol vs ramipril

Also known as: ACE-inhibitor

Ramipril

Eligibility Criteria

• Age: 2 Years - 45 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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).
• 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).

Sponsors & Collaborators

• Catholic University, Italy: lead

Study Sites (1)

Unione Italiana lotta Distrofia Muscolare

Rome, 00167, Italy

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Carvedilol; Adrenergic beta-Antagonists; Ramipril; Angiotensin-Converting Enzyme Inhibitors

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Propanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Propanols; Amines; Carbazoles; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 3-Ring; Adrenergic Antagonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Physiological Effects of Drugs; Protease Inhibitors; Enzyme Inhibitors

Study Officials

• Vincenzo Giglio, MD, PhD

  Uildm, Rome

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Vincenzo Giglio, MD, PhD

CONTACT

39-6-6604881; giglio.echo@libero.it

Fortunato Mangiola, MD

CONTACT

39-6-6604881; fortunato.mangiola@uildmlazio.org

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: January 8, 2009
• First Posted: January 9, 2009
• Study Start: December 1, 2008
• Primary Completion: June 1, 2009
• Study Completion: December 1, 2016
• Last Updated: January 28, 2016

Record last verified: 2009-01

Locations

IT (1)