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

Heart Failure Clinical Trial

— NEBIDYS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01648634
• Other study ID #: P090202
• Secondary ID: 2010-020047-12
• Status: Completed
• Phase: Phase 3
• Start date: February 13, 2012
• Est. completion date: July 20, 2021

Study information

• Verified date: September 2021
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 51
• Est. completion date: July 20, 2021
• Est. primary completion date: July 20, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 10 Years to 15 Years

Eligibility

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

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy
• Duchenne Muscular Dystrophy
• Heart Failure
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

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
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)

Locations

Country	Name	City	State
France	Armand Trousseau Hospital	Paris

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Association Française contre les Myopathies (AFM), Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Left ventricular systolic dysfunction	Development of left ventricular systolic dysfunction with an ejection fraction < 45%	at 5 years
Secondary	Right ventricular ejection fraction	Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography	at 5 years
Secondary	NT-ProBNP	NT-ProBNP	at 1, 2, 3, 4, and 5 years
Secondary	Left ventricular dysfunction	Development of left ventricular dysfunction	at 10 years
Secondary	Hospitalizations	hospitalizations for heart failure	at 10 years
Secondary	Mortality	Cardiovascular mortality	at 10 years ((5-years open label extension)