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

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 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: June 25, 2014
Last verified: June 2014
  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:


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 2020
Estimated Primary Completion Date: September 2019 (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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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: June 25, 2014
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 August 28, 2014