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    Summary
    EudraCT Number:2020-004901-29
    Sponsor's Protocol Code Number:NBK154/1/2020
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-01-20
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-004901-29
    A.3Full title of the trial
    The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled
    study.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Metoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD).
    A.3.2Name or abbreviated title of the trial where available
    MeDMD
    A.4.1Sponsor's protocol code numberNBK154/1/2020
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMedical University of Gdańsk
    B.1.3.4CountryPoland
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMedical Research Agency
    B.4.2CountryPoland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJoanna Kwiatkowska
    B.5.2Functional name of contact pointDepartment of Pediatric Cardiology
    B.5.3 Address:
    B.5.3.1Street AddressDębinki 7
    B.5.3.2Town/ cityGdańsk
    B.5.3.3Post code80-952
    B.5.3.4CountryPoland
    B.5.4Telephone number004858349 28 82
    B.5.5Fax number004858349 28 95
    B.5.6E-mailjoannak@gumed.edu.pl
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Betaloc ZOK 25, 23,75 mg
    D.2.1.1.2Name of the Marketing Authorisation holderAstraZeneca AB
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Prolonged-release tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMetoprolol
    D.3.9.3Other descriptive nameMETOPROLOL SUCCINATE
    D.3.9.4EV Substance CodeSUB03274MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number23.75
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Betaloc ZOK 100, 95 mg
    D.2.1.1.2Name of the Marketing Authorisation holderAstraZeneca AB
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Prolonged-release tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMetoprolol
    D.3.9.3Other descriptive nameMETOPROLOL SUCCINATE
    D.3.9.4EV Substance CodeSUB03274MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number95
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboProlonged-release tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboProlonged-release tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne muscular dystrophy
    Cardiomyopathy
    Tachycardia
    E.1.1.1Medical condition in easily understood language
    DMD is a genetic disorder characterized by progressive degeneration and muscle weakness.
    Cardiomyopathy is a progressive disease of the heart muscle.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 27.1
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    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.
    E.2.2Secondary objectives of the trial
    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
    E.2.3Trial contains a sub-study No
    E.3Principal 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 <17 years of age at time of enrolment in the study
    4. Ability to take oral medication and be willing to adhere to the study intervention regimen
    5. Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with typical DMD and:
    • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, or
    • Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-offrame'
    or
    • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion/duplication
    leading to a downstream stop codon)
    6. Taking ACEi treatment at minimum required doses as outlined in the protocol for at least 30 days
    E.4Principal 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
    E.5 End points
    E.5.1Primary 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.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Interim Analysis and Final Analysis
    E.5.2Secondary 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
    E.5.2.1Timepoint(s) of evaluation of this end point
    All secondary endpoints assessed at Interim Analysis and Final Analysis
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of study is defined as completion of the last visit or procedure in the trial globally.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 144
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 64
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 80
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state144
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Continuation of IMP if positive results. Otherwise, current standard of care.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-06-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-21
    P. End of Trial
    P.End of Trial StatusOngoing
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