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

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ClinicalTrials.gov Identifier: NCT01648634
Recruitment Status : Active, not recruiting
First Posted : July 24, 2012
Last Update Posted : March 29, 2018
Sponsor:
Collaborator:
Association Française contre les Myopathies (AFM), Paris
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

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
Duchenne Muscular Dystrophy Cardiomyopathy Heart Failure Drug: Nebivolol Drug: Placebo 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 Type : Interventional  (Clinical Trial)
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 : February 13, 2012
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021


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)




Primary Outcome Measures :
  1. Left ventricular systolic dysfunction [ Time Frame: at 5 years ]
    Development of left ventricular systolic dysfunction with an ejection fraction < 45%


Secondary Outcome Measures :
  1. Right ventricular ejection fraction [ Time Frame: at 5 years ]
    Right ventricular ejection fraction assessed by radionuclide angiography or echocardiography

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

  3. Left ventricular dysfunction [ Time Frame: at 10 years ]
    Development of left ventricular dysfunction

  4. Hospitalizations [ Time Frame: at 10 years ]
    hospitalizations for heart failure

  5. Mortality [ Time Frame: at 10 years ((5-years open label extension) ]
    Cardiovascular mortality



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Ages Eligible for Study:   10 Years to 15 Years   (Child)
Sexes 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 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01648634


Locations
France
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

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01648634     History of Changes
Other Study ID Numbers: P090202
First Posted: July 24, 2012    Key Record Dates
Last Update Posted: March 29, 2018
Last Verified: March 2018

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

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