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

This study is currently recruiting participants.
Verified April 2012 by Assistance Publique - Hôpitaux de Paris
Sponsor:
Collaborator:
Association Française contre les Myopathies (AFM), Paris
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01648634
First received: July 20, 2012
Last updated: July 23, 2012
Last verified: April 2012
History of Changes
  Purpose

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 Intervention Phase
Duchenne Muscular Dystrophy
Cardiomyopathy
Heart Failure
 Drug: Nebivolol
Drug: Placebo
 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • Left ventricular systolic dysfunction [ Time Frame: at 5 years ] [ Designated as safety issue: Yes ]
    Development of left ventricular systolic dysfunction with an ejection fraction < 45%


Secondary Outcome Measures:
  • Right ventricular ejection fraction [ Time Frame: at 5 years ] [ Designated as safety issue: Yes ]
    Right ventricular ejection fraction assessed by radionuclide angiography

  • NT-ProBNP [ Time Frame: at 1, 2, 3, 4, and 5 years ] [ Designated as safety issue: Yes ]
    NT-ProBNP

  • Left ventricular dysfunction [ Time Frame: at 10 years ] [ Designated as safety issue: No ]
    Development of left ventricular dysfunction

  • Hospitalizations [ Time Frame: at 10 years ] [ Designated as safety issue: No ]
    hospitalizations for heart failure

  • Mortality [ Time Frame: at 10 years ((5-years open label extension) ] [ Designated as safety issue: No ]
    Cardiovascular mortality


Estimated Enrollment: 60
Study Start Date: March 2012
Estimated Study Completion Date: March 2019
Estimated Primary Completion Date: September 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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)

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).

  Eligibility

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

Inclusion Criteria:

  • Duchenne muscular dystrophy genetically proven
  • Age between 10 and 15 years
  • Left ventricular ejection fraction assessed by radionuclide angiography ≥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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01648634

Contacts
Contact: Henri-Marc BECANE, MD, PhD +33144 73 54 87 henri-marc.becane@croix-rouge.fr
Contact: Laurence LECOMTE, PhD ++33171196494 laurence.lecomte@nck.aphp.fr

Locations
France
Armand Trousseau Hospital Recruiting
Paris, France, 75012
Contact: Henri-Marc BECANE, MD, PhD     +331 44 73 54 87     henri-marc.becane@croix-rouge.fr    
Contact: Laurence LECOMTE, PhD     ++33171196494     laurence.lecomte@nck.aphp.fr    
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
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01648634     History of Changes
Other Study ID Numbers: P090202
Study First Received: July 20, 2012
Last Updated: July 23, 2012
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Nebivolol
beta-blockade treatment

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Heart Failure
Muscular Dystrophies
Cardiomyopathies
Ventricular Dysfunction, Left
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heart Diseases
Cardiovascular Diseases
 Ventricular Dysfunction
Nebivolol
Antihypertensive Agents
Cardiovascular Agents
Therapeutic Uses
Pharmacologic Actions
Vasodilator Agents
Adrenergic beta-1 Receptor Antagonists
Adrenergic beta-Antagonists
Adrenergic Antagonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 22, 2013