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

Phase 3

Completed

• Conditions: Duchenne Muscular Dystrophy; Heart Failure; Cardiomyopathy
• Interventions: Drug: Placebo; Drug: Nebivolol

• Registration Number: NCT01648634
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

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.

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 Timeline

• Study Start: 2012-02-13
• Study First Submitted: 2012-07-20
• Study First Submitted QC: 2012-07-23
• Study First Posted: 2012-07-24
• Primary Completion: 2021-07-20
• Study Completion: 2021-07-20
• Status Verified: 2021-09
• Last Update Submitted: 2021-09-27
• Last Update Posted: 2021-09-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 51

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
Nebivolol	Nebivolol	-

Primary Outcome Measures

Name	Time	Method
Left ventricular systolic dysfunction	at 5 years	Development of left ventricular systolic dysfunction with an ejection fraction \< 45%

Secondary Outcome Measures

Name	Time	Method
Hospitalizations	at 10 years	hospitalizations for heart failure
Left ventricular dysfunction	at 10 years	Development of left ventricular dysfunction
NT-ProBNP	at 1, 2, 3, 4, and 5 years	NT-ProBNP
Mortality	at 10 years ((5-years open label extension)	Cardiovascular mortality
Right ventricular ejection fraction	at 5 years	Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography

Trial Locations

Locations (1)

Armand Trousseau Hospital; 🇫🇷 Paris, France