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

Primary Purpose

Duchenne Muscular Dystrophy, Cardiomyopathy, Heart Failure

Status
Completed
Phase
Phase 3
Locations
France
Study Type
Interventional
Intervention
Nebivolol
Placebo
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Duchenne Muscular Dystrophy focused on measuring Nebivolol, beta-blockade treatment

Eligibility Criteria

10 Years - 15 Years (Child)MaleDoes not accept 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

Sites / Locations

  • Armand Trousseau Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Nebivolol

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Left ventricular systolic dysfunction
Development of left ventricular systolic dysfunction with an ejection fraction < 45%

Secondary Outcome Measures

Right ventricular ejection fraction
Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography
NT-ProBNP
NT-ProBNP
Left ventricular dysfunction
Development of left ventricular dysfunction
Hospitalizations
hospitalizations for heart failure
Mortality
Cardiovascular mortality

Full Information

First Posted
July 20, 2012
Last Updated
September 27, 2021
Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Association Française contre les Myopathies (AFM), Paris
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1. Study Identification

Unique Protocol Identification Number
NCT01648634
Brief Title
Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2021
Overall Recruitment Status
Completed
Study Start Date
February 13, 2012 (Actual)
Primary Completion Date
July 20, 2021 (Actual)
Study Completion Date
July 20, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Association Française contre les Myopathies (AFM), Paris

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy, Cardiomyopathy, Heart Failure
Keywords
Nebivolol, beta-blockade treatment

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
51 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Nebivolol
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Nebivolol
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
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)
Primary Outcome Measure Information:
Title
Left ventricular systolic dysfunction
Description
Development of left ventricular systolic dysfunction with an ejection fraction < 45%
Time Frame
at 5 years
Secondary Outcome Measure Information:
Title
Right ventricular ejection fraction
Description
Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography
Time Frame
at 5 years
Title
NT-ProBNP
Description
NT-ProBNP
Time Frame
at 1, 2, 3, 4, and 5 years
Title
Left ventricular dysfunction
Description
Development of left ventricular dysfunction
Time Frame
at 10 years
Title
Hospitalizations
Description
hospitalizations for heart failure
Time Frame
at 10 years
Title
Mortality
Description
Cardiovascular mortality
Time Frame
at 10 years ((5-years open label extension)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Henri-Marc BECANE, MD,PhD
Organizational Affiliation
Armand Trousseau Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Armand Trousseau Hospital
City
Paris
ZIP/Postal Code
75012
Country
France

12. IPD Sharing Statement

Learn more about this trial

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

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