Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

A Randomized, Double-Blind, Placebo-Controlled, Multi-center Study to Examine the Effect of Nebivolol, a Beta-Blockade Drug, for the Prevention of Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

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.

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).

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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