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

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

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.

Study Overview

• Status: Completed
• Conditions: Heart Failure; Duchenne Muscular Dystrophy; Cardiomyopathy
• Intervention / Treatment: Drug: Nebivolol; Drug: Placebo

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 Type: Interventional
• Enrollment (Actual): 51
• Phase: Phase 3

Contacts and Locations

Study Locations

• France
  • Paris, France, 75012
    • Armand Trousseau Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 15 years (Child)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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)
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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Left ventricular systolic dysfunction	Development of left ventricular systolic dysfunction with an ejection fraction < 45%	at 5 years

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: URC-CIC Paris Descartes Necker Cochin; Association Française contre les Myopathies (AFM), Paris
• Investigators: Principal Investigator: Henri-Marc BECANE, MD,PhD, Armand Trousseau Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 13, 2012
• Primary Completion (Actual): July 20, 2021
• Study Completion (Actual): July 20, 2021

Study Registration Dates

• First Submitted: July 20, 2012
• First Submitted That Met QC Criteria: July 23, 2012
• First Posted (Estimated): July 24, 2012

Study Record Updates

• Last Update Posted (Actual): November 20, 2025
• Last Update Submitted That Met QC Criteria: November 17, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: Nebivolol; beta-blockade treatment
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Cardiovascular Diseases; Muscular Diseases; Heart Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heart Failure; Cardiomyopathies; Muscular Dystrophy, Duchenne; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Pyrans; Amines; Alcohols; Amino Alcohols; Ethanolamines; Benzopyrans; Nebivolol
• Other Study ID Numbers: P090202; 2010-020047-12 (EudraCT Number)