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

NCT ID: NCT01648634

Last Updated: 2025-11-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

51 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-02-13

Study Completion Date

2021-07-20

Brief Summary

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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

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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).

Conditions

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Duchenne Muscular Dystrophy Cardiomyopathy Heart Failure

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Nebivolol

Group Type EXPERIMENTAL

Nebivolol

Intervention Type DRUG

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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)

Interventions

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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)

Intervention Type 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)

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

10 Years

Maximum Eligible Age

15 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Association Française contre les Myopathies (AFM), Paris

OTHER

Sponsor Role collaborator

URC-CIC Paris Descartes Necker Cochin

OTHER

Sponsor Role collaborator

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Henri-Marc BECANE, MD,PhD

Role: PRINCIPAL_INVESTIGATOR

Armand Trousseau Hospital

Locations

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Armand Trousseau Hospital

Paris, , France

Site Status

Countries

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France

Other Identifiers

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2010-020047-12

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

P090202

Identifier Type: -

Identifier Source: org_study_id

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