Duchenne muscular dystrophy Cardiomyopathy Tachycardia MedDRA version: 27.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Betaloc ZOK 25, 23,75 mg
Pharmaceutical Form: Prolonged-release tablet
INN or Proposed INN: Metoprolol
Other descriptive name: METOPROLOL SUCCINATE
Concentration unit: mg milligram(s)
Conc
Sponsors
Medical University of Gdansk
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to requirements (>16 years old) 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Male, = 8 years and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: An individual who meets any of the following criteria will be excluded from participation in this study: 1. Current or previous permanent use of any beta-blocker medication 2. Treatment with another investigational drug or other intervention within 3 months prior to screening 3. Clinically significant bradycardia at rest or by Holter ECG, based on age and sex adjusted normal values, atrioventricular block higher than first degree at rest, or second degree Wenckebach at night, pauses longer than 2.5 seconds 4. Presence of pacemaker or ICD 5. Clinical signs or symptoms of heart failure 6. Left ventricular Ejection Fraction (LVEF) <57% (assessed by Teichholtz echocardiography) 7. Inability to obtain adequate quality echocardiography images (necessary to monitor for primary endpoint and safety) 8. Known allergic reactions to components of the IMPs
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate whether metoprolol succinate in addition to standard of care cardiac treatment introduced before the onset of echocardiography detectable left ventricular dysfunction, can slow the rate of progression left ventricular ejection fraction drop that leads to cardiomyopathy in males with DMD compared to placebo.;Secondary Objective: The secondary objectives of this study are to assess if metoprolol succinate in addition to standard of care treatment introduced before the onset of echocardiography detectable left ventricular dysfunction and compared to placebo: - can delay the occurrence of cardiomyopathy in children with DMD - can delay the occurrence of heart failure in children with DMD - can delay the age of onset and/or slow the rate of progression of myocardial fibrosis assessed by cardiac magnetic resonance (CMR) - can reduce the prevalence of sinus tachycardia assessed by Holter ECG - is safe and well tolerated in children with DMD ;Primary end point(s): The primary endpoint is change in left ventricular ejection fraction (LVEF) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.;Timepoint(s) of evaluation of this end point: Interim Analysis and Final Analysis | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Disease Free Survival (DFS) i.e., the time to develop cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method. • prevalence of patients with cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method • Disease Free Survival (DFS) i.e., the time to develop clinically evident Heart Failure (HF) • prevalence of patients with myocardial fibrosis assessed by cardiac magnetic resonance (CMR) • prevalence of patients with sinus tachycardia defined as mean daily heart rate above 95th percentile for age and sex and/or heart rate variability abnormalities assessed by Holter ECG ;Timepoint(s) of evaluation of this end point: All secondary endpoints assessed at Interim Analysis and Final Analysis | — |
Countries
Poland
Contacts
Public ContactDepartment of Pediatric Cardiology
Joanna Kwiatkowska
Outcome results
None listed