Duchenne muscular dystrophy is an X-linked inherited disorder which primarily affects skeletal muscle through a mutation in dystrophin which maps to the long arm of the X chromosome (Xp21). Patients with skeletal myopathy such as DMD eventually progress to develop dilated cardiomyopathy as dystrophin is expressed in all forms of muscle including cardiac and smooth muscle. Often the cause of death in patients with DMD is cardiac failure and arrhythmia.
Conditions
Interventions
Trade Name: Eucardic
Product Name: Eucardic
Product Code: No code
Pharmaceutical Form: Tablet
INN or Proposed INN: CARVEDILOL
CAS Number: 72956093
Concentration unit: mg/kg milligram(s)/kilogram
Conce
Sponsors
Department Of Paediatric Cardiology
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Diagnosis of confirmed DMD with either dilated LV (>2Z scores) and depressed LV systolic function (LVEF =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Refusal to participate. 2.Severe asthma which would be exacerbated by beta-blockade 3.Glucose intolerance or diabetes mellitus. 4.Severe claustrophobia which would prevent MR. 5.Any major contra-indication to cardiac MRI
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1.Determine baseline left ventricular systolic function using cardiac MRI, neurohormonal status (B natriuretic peptide levels, secreted by ventricular myocardium in congestive heart failure), two-dimensional echocardiographic and tissue Doppler imaging characteristics of a cohort of patients (children / adolescents 7-18 years age) with DMD. 2.Determine the effects of carvedilol on LV ejection fraction, LV end-diastolic and systolic volumes, BNP levels, and tissue Doppler imaging velocities. The effects on these parameters (LV dimensions, LVEF, tissue Doppler velocities) will be measured at baseline and at 6 months after commencing therapy. The effects on BNP levels will be assessed at baseline, 1 and 6 months after initiation of therapy. ;Secondary Objective: ;Primary end point(s): 1. Left ventricular ejection fraction. 2. Left ventricular shortening fraction. 3. Left ventricular end diastolic and systolic volumes. 4. Tissue Doppler velocities. 5. BNP levels. 6. Clinical improvement of symptoms (NYHA status) and signs of heart failure. | — |
Countries
Ireland
Outcome results
None listed