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

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01648634

Acronym

NEBIDYS

Enrollment

Registered

2012-07-24

Start date

2012-02-13

Completion date

2021-07-20

Last updated

2025-11-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cardiomyopathy, Duchenne Muscular Dystrophy, Heart Failure

Keywords

Nebivolol, beta-blockade treatment

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).

Interventions

DRUGNebivolol

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)

DRUGPlacebo

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)

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

PREVENTION

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

MALE

Age

10 Years to 15 Years

Healthy volunteers

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

Design outcomes

Primary

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

Secondary

Measure	Time frame	Description
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

Countries

France

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026