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    Summary
    EudraCT Number:2014-002277-11
    Sponsor's Protocol Code Number:20120123
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-06-12
    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 ConcernedItaly - Italian Medicines Agency
    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)
    Studio multicentrico, randomizzato, in doppio cieco, a gruppi paralleli, controllato verso placebo volto a caratterizzare l’efficacia, la sicurezza e la tollerabilità di 24 settimane di trattamento con evolocumab per la riduzione del colesterolo legato alle lipoproteine a bassa densità (C-LDL) in aggiunta alla dieta e alla terapia ipolipemizzante in soggetti pediatrici dai 10 ai 17 anni di età affetti da ipercolesterolemia familiare eterozigote (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
    Studio volto a caratterizzare l’efficacia, la sicurezza e la tollerabilità a lungo termine in soggetti pediatrici dai 10 ai 17 anni di età affetti da ipercolesterolemia familiare eterozigote (HeFH).
    A.4.1Sponsor's protocol code number20120123
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    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
    Ipercolesterolemia familiare eterozigote
    E.1.1.1Medical condition in easily understood language
    Increased cholesterol in the blood
    Aumento del colesterolo nel sangue
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    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).
    valutare l’effetto di 24 settimane di trattamento con evolocumab per via sottocutanea (sc) rispetto al placebo, in aggiunta alla terapia standard, sulla variazione percentuale dal basale dei livelli di colesterolo legato alle lipoproteine a bassa densità (C-LDL) in soggetti pediatrici dai 10 ai 17 anni di età con 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 PK exposure
    - valutare la sicurezza di evolocumab sc rispetto al placebo, in aggiunta alla terapia standard, in soggetti pediatrici dai 10 ai 17 anni di età con HeFH
    - valutare gli effetti di evolocumab sc rispetto al placebo, in aggiunta alla terapia standard, sulla variazione percentuale media dal basale alle settimane 22 e 24 e sulla variazione dal basale alla settimana 24 dei livelli di C-LDL, nonché sulla variazione percentuale dal basale alla settimana 24 dei livelli di colesterolo legato alle lipoproteine non ad alta densità (C-non HDL), apolipoproteina B (ApoB), rapporto colesterolo
    totale/C-HDL, e rapporto apoB/apolipoproteina A-1 (ApoA1), in soggetti pediatrici dai 10 ai 17 anni di età con HeFH
    - caratterizzare l’esposizione farmacocinetica
    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.
    sono eleggibili per questo studio soggetti di sesso maschile e femminile, di età compresa tra i 10 e i 17 anni, con diagnosi di HeFH e in trattamento con terapia ipolipemizzante standard ottimizzata conformemente alle linee guida applicabili a livello locale.
    I soggetti dovranno avere firmato il consenso informato o l’assenso per lo studio e dovrà essere disponibile l’apposito modulo di consenso dei genitori/del tutore. I soggetti devono seguire una dieta a basso contenuto di grassi e devono essere in trattamento con una terapia ipolipemizzante di base ottimizzata, che comprenda una statina alla dose ottimale determinata dal medico curante. La terapia ipolipemizzante deve essere stabile da ≥4 settimane prima dello screening. I livelli di C-LDL a digiuno devono essere ≥130 mg/dL (3,4 mmol/L) e i trigliceridi a digiuno ≤400 mg/dL (4,5 mmol/L) come determinato dal laboratorio centrale allo screening.
    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.
    E.4 Criteri di esclusione principali(elencare i più importanti):
    diabete di tipo 1 o diabete di tipo 2 diagnosticato di recente (nei 3 mesi precedenti la randomizzazione) o scarsamente controllato (HbA1c >8,5%) o alterata tolleranza al glucosio di nuova diagnosi; livelli di ormone tireostimolante (TSH) < il limite inferiore della norma (LLN) o TSH >1,5 volte il limite superiore della norma (ULN) e livelli di tiroxina (T4) libera al di fuori dell’intervallo normale, tasso di filtrazione glomerulare stimato (eGFR) <30 mL/min/1,73 m2, aspartato aminotransferasi (AST) o alanina
    aminotransferasi (ALT) >2x ULN, creatininchinasi (CK) >3x ULN (tutte le valutazioni di screening sono eseguite dal laboratorio centrale); infezione in atto nota o disfunzione maggiore di natura ematologica, renale, metabolica, gastrointestinale o endocrina. Sono esclusi i soggetti che hanno assunto inibitori della proteina di trasferimento degli esteri del colesterolo (CEPT) negli ultimi 12 mesi, mipomersen o lomitapide negli ultimi 5 mesi, o precedentemente sottoposti al trattamento con evolocumab o con qualsiasi altra terapia sperimentale volta a inibire la proproteina convertasi subtilisina/kexina di tipo 9 (PCSK9). I soggetti non potranno essere arruolati in studi su altri dispositivi o farmaci sperimentali, sottoporsi a trattamento con altri farmaci o procedure sperimentali, o aver concluso uno studio su altri farmaci o dispositivi sperimentali da meno di 30 giorni. I soggetti di sesso femminile potenzialmente fertili non potranno essere in stato di gravidanza, pianificare una gravidanza, essere in allattamento o pianificare di allattare e devono essere disposte a utilizzare uno o più metodi efficaci di contraccezione giudicati accettabili durante il trattamento con il prodotto sperimentale
    (evolocumab o placebo) e per altre 15 settimane dopo il termine del trattamento con il prodotto sperimentale (evolocumab o placebo).
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline to week 24 in LDL-C
    variazione percentuale dei livelli di C-LDL dal basale alla settimana 24
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and week 24
    Al basale ed alla settimana 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
    • variazione percentuale media dei livelli di C-LDL dal basale alle settimane 22 e 24
    • variazione dei livelli di C-LDL dal basale alla settimana 24
    • variazione percentuale dal basale alla settimana 24 di:
    – colesterolo non HDL
    – ApoB
    – rapporto colesterolo totale/C-HDL
    – rapporto ApoB/ApoA1
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline, week 22 and week 24
    Al basale, alla settimana 22 ed alla settimana 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 No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    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
    LVS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    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 state8
    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
    nessuno
    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 Decision2015-06-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-01-25
    P. End of Trial
    P.End of Trial StatusOngoing
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