NEBIDYS: Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
Study Details
Study Description
Brief Summary
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.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
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Phase 3 |
Detailed 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).
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: Nebivolol
|
Drug: Nebivolol
A 1.25mg-test dose will be administrated to assess the treatment tolerance before randomization. A forced titration of nebivolol will be performed with 2 weeks periods. Full dose of nebivolol is 5mg/day (7.5mg/day for patients whose weight is>60kg)
|
Placebo Comparator: Placebo
|
Drug: Placebo
A 1.25mg-test dose of nebivolol will be administrated to assess the treatment tolerance before randomization. A forced titration of placebo will be performed with 2 weeks periods. Full dose of placebo is 5mg/day (7.5mg/day for patients whose weight is>60kg)
|
Outcome Measures
Primary Outcome Measures
- Left ventricular systolic dysfunction [at 5 years]
Development of left ventricular systolic dysfunction with an ejection fraction < 45%
Secondary Outcome Measures
- Right ventricular ejection fraction [at 5 years]
Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography
- NT-ProBNP [at 1, 2, 3, 4, and 5 years]
NT-ProBNP
- Left ventricular dysfunction [at 10 years]
Development of left ventricular dysfunction
- Hospitalizations [at 10 years]
hospitalizations for heart failure
- Mortality [at 10 years ((5-years open label extension)]
Cardiovascular mortality
Eligibility Criteria
Criteria
Inclusion Criteria:
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Duchenne muscular dystrophy genetically proven
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Age between 10 and 15 years
-
Left ventricular ejection fraction assessed by radionuclide angiography or echocardiography ≥50% and measured within 3 months
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Systolic blood pressure ≥80 mmHg
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Diastolic blood pressure ≥70 mmHg
Exclusion Criteria:
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Heart rate <50 bpm
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2nd or 3rd degree atrioventricular blocks, sinus node dysfunction
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Asthma or bronchospasm
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Severe peripheral circulatory disease
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Hypersensitivity to nebivolol or excipients
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Metabolic acidosis
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Blood urea >7 mmol/l
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Liver transaminases enzymes >6 fold the upper limit of normal
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Formal indication for beta-blockade treatment
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Cardiac treatments except angiotensin-converting enzyme inhibitors
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Participation to another clinical trial within 3 months
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Armand Trousseau Hospital | Paris | France | 75012 |
Sponsors and Collaborators
- Assistance Publique - Hôpitaux de Paris
- Association Française contre les Myopathies (AFM), Paris
Investigators
- Principal Investigator: Henri-Marc BECANE, MD,PhD, Armand Trousseau Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- P090202
- 2010-020047-12