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

Assistance Publique - Hôpitaux de Paris

Status and phase: Completed; Phase 3

Conditions: Cardiomyopathy; Heart Failure; Duchenne Muscular Dystrophy

Treatments: Drug: Nebivolol; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT01648634

2010-020047-12 (EudraCT Number)

P090202

Details and patient eligibility

About

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.

Full description

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).

Enrollment: 51 patients

Sex: Male

Ages: 10 to 15 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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51 participants in 2 patient groups, including a placebo group

Nebivolol

Experimental group

Treatment:

Drug: Nebivolol

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo