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Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
study id #: NCT01648634
condition: Duchenne Muscular Dystrophy, Cardiomyopathy, Heart Failure
status: active, not recruitingpurpose:
The objective is to determine whether nebivolol, a beta-blockade drug, can prevent the development of heart disease in patients with Duchenne muscular dystrophy aged 10 to 15 year-old.
intervention: Nebivolol, Placebo
mechanism of action: Beta-blocker to prevent cardiac function deterioration
start date: February 13, 2012
estimated completion: June 2021
phase of development: Phase 3
size / enrollment: 51
A 1.25 mg-test dose will be administrated to assess the treatment tolerance before randomization. A forced titration of nebivolol and placebo will be performed with 2 weeks periods. Full dose of nebivolol and placebo is 5mg/day (7.5mg/day for patients whose weight is >60kg).
- Left ventricular systolic dysfunction [Time Frame: at 5 years]
Development of left ventricular systolic dysfunction with an ejection fraction < 45%
- Right ventricular ejection fraction [Time Frame: at 5 years]
Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography
- NT-ProBNP [Time Frame: at 1, 2, 3, 4, and 5 years]
- Left ventricular dysfunction [Time Frame: at 10 years]
Development of left ventricular dysfunction
- Hospitalizations [Time Frame: at 10 years]
hospitalizations for heart failure
- Mortality [Time Frame: at 10 years ((5-years open label extension)]
• Duchenne muscular dystrophy genetically proven
• Age between 10 and 15 years
• Left ventricular ejection fraction assessed by radionuclide angiography or echocardiography >=50% and measured within 3 months
• Systolic blood pressure >=80 mmHg
• Diastolic blood pressure >=70 mmHg
• Heart rate <50 bpm
• 2nd or 3rd degree atrioventricular blocks, sinus node dysfunction
• Asthma or bronchospasm
• Severe peripheral circulatory disease
• Hypersensitivity to nebivolol or excipients
• Metabolic acidosis
• Blood urea >7 mmol/l
• Liver transaminases enzymes >6 fold the upper limit of normal
• Formal indication for beta-blockade treatment
• Cardiac treatments except angiotensin-converting enzyme inhibitors
• Participation to another clinical trial within 3 months
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