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    The EU Clinical Trials Register currently displays   43845   clinical trials with a EudraCT protocol, of which   7282   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2014-002277-11
    Sponsor's Protocol Code Number:20120123
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-02-15
    Trial results View 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 ConcernedGreece - EOF
    A.2EudraCT number2014-002277-11
    A.3Full title of the trial
    Double-blind, Randomized, Multicenter, Placebo-Controlled, Parallel Group Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for Low Density Lipoprotein-Cholesterol (LDL-C) Reduction, as Add-On to Diet and Lipid-Lowering Therapy, in Pediatric Subjects 10 to 17 Years
    of Age With Heterozygous Familial Hypercholesterolemia (HeFH)
    «Διπλά τυφλή, τυχαιοποιημένη, πολυκεντρική, ελεγχόμενη με εικονικό
    φάρμακο μελέτη παράλληλων ομάδων για τον προσδιορισμό της αποτελεσματικότητας, της ασφάλειας και της ανοχής της θεραπείας 24 εβδομάδων με evolocumab για τη μείωση της χοληστερόλης χαμηλής πυκνότητας λιποπρωτεϊνών (LDL-C), επιπροσθέτως της δίαιτας και της υπολιπιδαιμικής θεραπείας, σε παιδιατρικούς ασθενείς ηλικίας 10 έως 17 ετών με ετερόζυγο
    οικογενή υπερχοληστερολαιμία (HeFH)»
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to assess the long term safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia
    « Μελέτη για την Αξιολόγηση της Μακροχρόνιας Ασφάλειας και Αποτελεσματικότητας του Evolocumab σε παιδιατρικούς ασθενείς ηλικίας 10 έως 17 ετών με ετερόζυγο οικογενή υπερχοληστερολαιμία»
    A.4.1Sponsor's protocol code number20120123
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/071/2015
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmgen Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAmgen Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmgen (EUROPE) GmbH
    B.5.2Functional name of contact pointIHQ Medical Info - Clinical Trials
    B.5.3 Address:
    B.5.3.1Street AddressDammstrasse 23, P.O. Box 1557
    B.5.3.2Town/ cityZug
    B.5.3.3Post code(CH-)6300
    B.5.3.4CountrySwitzerland
    B.5.6E-mailMedinfoInternational@amgen.com
    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 No
    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.3.1Product nameEvolocumab
    D.3.2Product code AMG 145
    D.3.4Pharmaceutical form Solution for injection in pre-filled pen
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEvolocumab
    D.3.9.3Other descriptive nameAMG145
    D.3.9.4EV Substance CodeSUB32500
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number140
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 placeboSolution for injection in pre-filled pen
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Heterozygous familial hypercholesterolaemia
    Eτερόζυγη Οικογενής Υπερχοληστερολαιμία
    E.1.1.1Medical condition in easily understood language
    Increased cholesterol in the blood
    Αυξημένα επίπεδα χοληστερόλης στο αίμα
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level LLT
    E.1.2Classification code 10057079
    E.1.2Term Heterozygous familial hypercholesterolemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with placebo, when added to standard of care, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with heterozygous familial hypercholesterolemia (HeFH).
    Η εκτίμηση της επίδρασης της υποδόριας (SC) χορήγησης evolocumab επί 24 εβδομάδες στην ποσοστιαία μεταβολή της χοληστερόλης χαμηλής πυκνότητας λιποπρωτεϊνών (LDL-C) από την έναρξη, σε σύγκριση με το εικονικό φάρμακο, κατά την προσθήκη του στη συνήθη θεραπεία παιδιατρικών ασθενών ηλικίας 10 έως 17 ετών με ετερόζυγο οικογενή υπερχοληστερολαιμία (HeFH).
    E.2.2Secondary objectives of the trial
    • to evaluate the safety of SC evolocumab compared with placebo, when added to standard of care, in pediatric subjects 10 to 17 years of age with HeFH
    • to assess the effects of SC evolocumab compared with placebo, when added to standard of care, on mean percent change from baseline to weeks 22 and 24 and change from baseline to week 24 in LDL-C, and on percent change from baseline to week 24 in other lipid parameters in
    pediatric subjects 10 to 17 years of age with HeFH
    • To characterize pharmacokinetic (PK) exposure
    Due to space limitations please see above.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Subject has provided informed consent or subject assent prior to initiation of any study-specific activities/procedures.
    and/or
    - Subject’s legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written subject assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
    - Male or female, ≥ 10 to ≤ 17 years of age at time of randomization (includes the year after the subject completes the 17th year after birth but not the day of completing the 18th year after birth).
    -Diagnosis of heterozygous familial hypercholesterolemia by local applicable diagnostic criteria for HeFH (ie, criteria outlined by the Simon Broome Register Group [Scientific Steering Committee, 1991] or the Dutch Lipid Clinic Network [World Health Organization, 1999]) or by genetic testing.
    - On an approved statin with stable dose for ≥ 4 weeks before LDL-C screening and, in the opinion of the investigator, not requiring up-titration.
    - Παροχή γραπτής συγκατάθεσης κατόπιν ενημέρωσης ή συναίνεσης από τον
    ασθενή πριν από την έναρξη οποιασδήποτε σχετιζόμενης με τη μελέτη
    δραστηριότητας/διαδικασίας
    ή/και
    - Παροχή συγκατάθεσης κατόπιν ενημέρωσης από τον νόμιμο εκπρόσωπο του
    ασθενή, όταν ο τελευταίος είναι δικαιοπρακτικά ανίκανος λόγω ανηλικότητας να
    παρέχει συγκατάθεση κατόπιν ενημέρωσης και παροχή γραπτής συναίνεσης από
    τον ασθενή βάσει των τοπικών κανονισμών ή/και κατευθυντήριων γραμμών πριν
    από την έναρξη οποιασδήποτε σχετιζόμενης με τη μελέτη δραστηριότητας/
    διαδικασίας.
    - Αγόρια ή κορίτσια ηλικίας ≥ 10 έως ≤ 17 ετών κατά το χρόνο τυχαιοποίησης
    (περιλαμβάνεται ο χρόνος μετά τη συμπλήρωση του 17ου έτους μετά τη γέννηση
    του ασθενή, αλλά όχι η ημέρα συμπλήρωσης του 18ου έτος μετά τη γέννηση).
    - Διάγνωση ετερόζυγου οικογενούς υπερχοληστερολαιμίας σύμφωνα με τα κατά
    τόπους ισχύοντα διαγνωστικά κριτήρια για την HeFH (δηλ., τα κριτήρια που
    περιγράφονται συνοπτικά από τις επιστημονικές ομάδες Simon Broome Register
    Group [Επιστημονική Συντονιστική Επιτροπή, 1991], Dutch Lipid Clinic Network
    [Παγκόσμιος Οργανισμός Υγείας, 1999] και MEDPED [Williams et al, 1993]) ή
    κατόπιν γενετικού ελέγχου.
    - Ο ασθενής λαμβάνει στατίνη που κυκλοφορεί ήδη στο εμπόριο σε σταθερή δόση
    για ≥ 4 εβδομάδες πριν τον προληπτικό έλεγχο της LDL-C και, κατά την κρίση του
    ερευνητή, δεν απαιτείται αύξηση της δόσης.
    E.4Principal exclusion criteria
    - Type 1 diabetes or newly diagnosed (within 3 months of randomization) type 2 diabetes, or poorly controlled type 2 diabetes (HbA1c > 8.5%) or newly diagnosed impaired glucose tolerance (within 3 months of randomization).
    - Untreated or inadequately treated hyperthyroidism or hypothyroidism as defined by thyroid stimulating hormone (TSH) < lower limit of normal (LLN) or > 1.5 times the upper limit of normal (ULN), respectively, and free thyroxine (T4) levels that are outside normal range at screening.
    - Moderate to severe renal dysfunction, defined as an estimated glomerular filtration rate (eGFR) < 30 ml/min/1.73m2 at screening, confirmed by a repeat measurement at least 1 week apart.
    - Persistent active liver disease or hepatic dysfunction, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 2 times the ULN as determined by central laboratory analysis at screening, confirmed by a repeat measurement at least 1 week apart.
    -Ομόζυγος οικογενής υπερχοληστερολαιμία.
    -Λιπιδαφαίρεση εντός των τελευταίων 12 εβδομάδων πριν τον προκαταρκτικό έλεγχο.
    -Διαβήτης τύπου 1 ή νεοδιαγνωσθείς (μέσα σε διάστημα 3 μηνών από την τυχαιοποίηση) διαβήτης τύπου 2 ή ανεπαρκώς ελεγχόμενος διαβήτης τύπου 2(HbA1c > 8,5%) ή νεοδιαγνωσθείσα διαταραγμένη ανοχή στη γλυκόζη (μέσα σε διάστημα 3 μηνών από την τυχαιοποίηση).
    -Υπερθυρεοειδισμός ή υποθυρεοειδισμός που δεν αντιμετωπίστηκε ή δεν αντιμετωπίστηκε επαρκώς και ορίζεται ως θυρεοειδοτρόπος ορμόνη (TSH) <κατώτερα φυσιολογικά όρια (LLN) ή > 1,5 φορές πάνω από τα ανώτερα φυσιολογικά όρια (ULN), αντίστοιχα, και επίπεδα ελεύθερης θυροξίνης (T4) εκτός φυσιολογικών ορίων κατά τον προκαταρκτικό έλεγχο.
    -Μέτρια έως σοβαρή νεφρική δυσλειτουργία οριζόμενη ως εκτιμώμενος ρυθμός σπειραματικής διήθησης (eGFR) < 30 ml/λεπτό/1,73m2 κατά τον προκαταρκτικό έλεγχο, που επιβεβαιώθηκε με επαναληπτική μέτρηση μετά από τουλάχιστον 1 εβδομάδα.
    -Εμμένουσα, ενεργός ηπατική νόσος ή ηπατική δυσλειτουργία οριζόμενη ως ασπαρτική αμινοτρανσφεράση (AST) ή αμινοτρανσφεράση της αλανίνης (ALT) > 2x ULN, όπως προέκυψε από την ανάλυση του κεντρικού εργαστηρίου κατά τον προκαταρκτικό έλεγχο, η οποία επιβεβαιώθηκε με επαναληπτική μέτρηση μετά από τουλάχιστον 1 εβδομάδα.
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline to week 24 in LDL-C
    Ποσοστιαία μεταβολή της LDL-C από την έναρξη έως την εβδομάδα 24.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and week 24
    Αξιολόγηση αναφοράς και Εβδομάδα 24.
    E.5.2Secondary end point(s)
    -Mean percent change from baseline to weeks 22 and 24 in LDL-C
    -Change from baseline to week 24 in LDL-C
    -Percent change from baseline to week 24 in the following:
    − non-HDL-C
    − ApoB
    − total cholesterol/HDL-C ratio
    − ApoB/ApoA1 ratio
    Μέση ποσοστιαία μεταβολή της LDL-C από την έναρξη έως τις εβδομάδες 22 και 24
    • Μεταβολή της LDL-C από την έναρξη έως την εβδομάδα 24
    • Ποσοστιαία μεταβολή των ακολούθων από την έναρξη έως την εβδομάδα 24:
    • μη-HDL-C
    • ApoB
    • λόγος ολικής χοληστερόλης/HDL-C
    • λόγος ApoB/ApoA1
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline, week 22 and week 24
    Αξιολόγηση αναφοράς και Εβδομάδες 22 και 24.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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 Information not present in EudraCT
    E.8.4 The trial involves multiple sites in the Member State concerned Information not present in EudraCT
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Brazil
    Canada
    New Zealand
    South Africa
    Turkey
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days29
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days0
    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: 150
    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: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 135
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 150
    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)
    None
    Κανένα.
    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 Decision2016-04-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-03-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-11-25
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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