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

Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Overall Status
Completed
CT.gov ID
NCT01648634
Collaborator
Association Française contre les Myopathies (AFM), Paris (Other)
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Study Details

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.

Condition or Disease Intervention/Treatment Phase
Phase 3

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 Design

Study Type:
Interventional
Actual Enrollment :
51 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Prevention
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
Actual Study Start Date :
Feb 13, 2012
Actual Primary Completion Date :
Jul 20, 2021
Actual Study Completion Date :
Jul 20, 2021

Arms and Interventions

Arm Intervention/Treatment
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)

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)

Outcome Measures

Primary Outcome Measures

  1. Left ventricular systolic dysfunction [at 5 years]

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

Secondary Outcome Measures

  1. Right ventricular ejection fraction [at 5 years]

    Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography

  2. NT-ProBNP [at 1, 2, 3, 4, and 5 years]

    NT-ProBNP

  3. Left ventricular dysfunction [at 10 years]

    Development of left ventricular dysfunction

  4. Hospitalizations [at 10 years]

    hospitalizations for heart failure

  5. Mortality [at 10 years ((5-years open label extension)]

    Cardiovascular mortality

Eligibility Criteria

Criteria

Ages Eligible for Study:
10 Years to 15 Years
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Armand Trousseau Hospital Paris France 75012

Sponsors and Collaborators

  • Assistance Publique - Hôpitaux de Paris
  • Association Française contre les Myopathies (AFM), Paris

Investigators

  • Principal Investigator: Henri-Marc BECANE, MD,PhD, Armand Trousseau Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01648634
Other Study ID Numbers:
  • P090202
  • 2010-020047-12
First Posted:
Jul 24, 2012
Last Update Posted:
Sep 28, 2021
Last Verified:
Sep 1, 2021
Keywords provided by Assistance Publique - Hôpitaux de Paris
Additional relevant MeSH terms:

Study Results

No Results Posted as of Sep 28, 2021