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

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

key information

study id #: NCT01648634

condition: Duchenne Muscular Dystrophy, Cardiomyopathy, Heart Failure

status: active, not recruiting

purpose:

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

results: https://clinicaltrials.gov/ct2/show/results/NCT01648634

last updated: November 22, 2018

study details

start date: February 13, 2012

estimated completion: June 2021

phase of development: Phase 3

size / enrollment: 51

study 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).

primary outcomes:

  • Left ventricular systolic dysfunction [Time Frame: at 5 years]
    Development of left ventricular systolic dysfunction with an ejection fraction < 45%

secondary outcomes:

  • 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]
    NT-ProBNP
  • 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)]
    Cardiovascular mortality

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

study contacts

sponsor: Assistance Publique - Hôpitaux de Paris

investigators: Henri-Marc BECANE, MD,PhD

locations: France