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    The EU Clinical Trials Register currently displays   38147   clinical trials with a EudraCT protocol, of which   6265   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2015-003766-85
    Sponsor's Protocol Code Number:DFI14223
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-06-02
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2015-003766-85
    A.3Full title of the trial
    An 8-Week Open-Label, Sequential, Repeated Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Heterozygous Familial Hypercholesterolemia Followed by an Extension Phase
    Estudio abierto de ocho semanas de duración, de búsqueda de dosis repetidas, secuencial, para evaluar la eficacia y seguridad de alirocumab en niños y adolescentes con hipercolesterolemia Familiar heterocigótica, seguido de una fase de extensión
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Heterozygous Familial Hypercholesterolemia
    Estudio de ocho semanas de duración, de búsqueda de dosis para evaluar la eficacia y seguridad de alirocumab en niños y adolescentes con hipercolesterolemia Familiar heterocigótica
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberDFI14223
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1178-4764
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/297/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsorsanofi-aventis recherche&developpement
    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 supportsanofi-aventis recherche & developpement
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationsanofi-aventis, s.a.
    B.5.2Functional name of contact pointUnidad de Estudios Clínicos
    B.5.3 Address:
    B.5.3.1Street Addressc/ Josep Pla nº2, 4ª planta
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08019
    B.5.4Telephone number93 485 94 00
    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 namealirocumab
    D.3.2Product code SAR236553
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    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 INNalirocumab
    D.3.9.1CAS number 1245916-14-6
    D.3.9.2Current sponsor codeSAR236553 (RGN727)
    D.3.9.3Other descriptive nameALIROCUMAB
    D.3.9.4EV Substance CodeSUB170596
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    E.1.1.1Medical condition in easily understood language
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10020603
    E.1.2Term Hypercholesterolaemia
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    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 alirocumab on low-density lipoprotein cholesterol (LDL-C) levels after 8 weeks of treatment in heterozygous familial hypercholesterolemia (heFH) patients age of 8 to 17 years, with LDL-C ≥130 mg/dL (3.37 mmol/L) on optimal stable daily dose of statin therapy +/- other lipid modifying therapies (LMTs) or a stable dose of non-statin LMTs in case of intolerance to statins for at least 4 weeks prior to the screening period.
    Evaluar el efecto de alirocumab administrado cada 2 semanas (C2S) o cada 4 semanas (C4S) sobre los niveles de colesterol de lipoproteínas de baja densidad (C LDL) tras 8 semanas de tratamiento en pacientes con hipercolesterolemia familiar heterocigótica (HFhe) con edades comprendidas entre 8 y 17 años, con niveles de C LDL ≥130 mg/dl (3,37 mmol/l) que estén recibiendo una dosis diaria estable óptima de tratamiento con estatinas ± otros tratamientos modificadores de lípidos (TML) o una dosis estable de TML sin estatinas en caso de intolerancia a las estatinas, durante al menos 4 semanas antes del periodo de selección.
    E.2.2Secondary objectives of the trial
    -To evaluate the safety and tolerability of alirocumab.
    -To evaluate the pharmacokinetics profile of alirocumab.
    -To evaluate the effects of alirocumab on other lipid parameters.
    -Evaluar la seguridad y la tolerabilidad de alirocumab.
    -Evaluar el perfil farmacocinético de alirocumab.
    -Evaluar los efectos de alirocumab sobre los niveles de otros parámetros lipídicos
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Children and adolescent male and female patients age of 8 to 17 years at the time of signed informed consent. For Russia only: Male and female patients aged ≥12 and ≤17 years at the time of signed informed consent.
    -Patients with diagnosis of heterozygous familial hypercholesterolemia (heFH) through genotyping or clinical criteria.
    -Patients treated with optimal dose of statin +/- other LMT(s) or non-statin LMT(s) if statin intolerant at stable dose for at least 4 weeks prior to screening visit (Week -2).
    -Patients with calculated LDL-C greater than or equal to 130 mg/dL (≥3.37 mmol/L) at the screening visit (Week -2).
    -Patients with body weight greater than or equal to 25kg.
    -Patients age of 8 to 9 years to be at Tanner stage 1 and patients age of 10 to 17 years to be at least at Tanner stage 2 in their development.
    -A signed informed consent indicating parental permission with or without patient assent.
    I 01. Niños y adolescentes de ambos sexos con edades comprendidas entre 8 y 17 años en el momento de la firma del consentimiento informado.
    I 02. Pacientes con un diagnóstico de hipercolesterolemia familiar heterocigótica (HFhe) por genotipado o criterios clínicos.
    I 03. Pacientes tratados con una dosis óptima de estatinas con o sin otro(s) TML o TML sin estatinas en caso de intolerancia a las estatinas a una dosis estable durante al menos 4 semanas antes de la visita de selección (Semana -2).
    I 04. Pacientes con C LDL calculado mayor o igual a 130 mg/dl (≥3,37 mmol/l) en la visita de selección (Semana -2).
    I 05. Pacientes con peso corporal superior o igual a 25 kg.
    I 06. Pacientes con edades comprendidas entre 8 y 9 años con estadio 1 de Tanner y pacientes con edades comprendidas entre 10 y 17 años al menos con estadio 2 de Tanner en su desarrollo.
    I 07. Firma de consentimiento informado indicando el permiso paterno con o sin el asentimiento del paciente, dependiendo de la capacidad de comprensión basada en el grado de madurez.
    E.4Principal exclusion criteria
    -Patient with secondary hyperlipidemia.
    -Diagnosis of homozygous familial hypercholesterolemia.
    -Patient who has received lipid apheresis treatment within 2 months prior to the screening period, or has plans to receive it during the study.
    -Known history of type 1 or type 2 diabetes mellitus.
    -Known history of thyroid disease.
    -Known history of hypertension.
    -Fasting triglycerides >350 mg/dL (3.95 mmol/L).
    -Severe renal impairment (ie, estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m^2.
    -Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 x upper limit of normal (ULN).
    -Creatinine phosphokinase (CPK) >3 x ULN.
    • Paciente con hiperlipidemia secundaria.
    • Diagnóstico de hipercolesterolemia familiar homocigótica.
    • Paciente que ha recibido tratamiento de aféresis de lípidos en los 2 meses previos al periodo de selección, o que tiene previsto recibirlo durante el estudio.
    • Antecedentes conocidos de diabetes mellitus tipo 1 o tipo 2.
    • Antecedentes conocidos de enfermedad tiroidea.
    • Antecedentes conocidos de hipertensión.
    • Triglicéridos en ayunas >350 mg/dl (3,95 mmol/l) en la visita de selección (Semana -2).
    • Insuficiencia renal grave (es decir, TFGe <30 ml/min/1,73 m2 en la visita de selección [Semana -2]).
    • ALT o AST >2 x LSN (se permite 1 repetición de la analítica).
    E.5 End points
    E.5.1Primary end point(s)
    Percent change in calculated LDL-C
    Cambio porcentual en el C LDL calculado
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to Week 8
    Desde el momento basal hasta la Semana 8.
    E.5.2Secondary end point(s)
    a) Absolute change in calculated LDL-C
    b) Percentage of participants achieving a calculated LDL-C level lower than 130 mg/dL (3.37 mmol/L)
    c) Percentage of participants achieving a calculated LDL-C level lower than 110 mg/dL (2.84 mmol/L)
    d) Percent change in Apolipoprotein B (Apo B)
    e) Percent change in non-high density lipoprotein cholesterol (non HDL-C)
    f) Percent change in Total-C
    g) Percent change in Lipoprotein (a) (Lp[a])
    h) Percent change in triglycerides (TG)
    I) Percent change in HDL-C
    l) Percent change in Apo A-1
    m) Absolute change in Apo B
    n) Absolute change in non-HDL-C
    o) Absolute change in Total-C
    p) Absolute change in Lp(a)
    q) Absolute change in TG
    r) Absolute change in HDL-C
    s) Absolute change in Apo A-1
    t) Absolute change in ratio Apo B/Apo A-1
    a) Cambio absoluto en el C-LDL calculado
    b) Porcentaje de participantes que alcanzaron un nivel de LDL-C por debajo de 130 mg / dl (3,37 mmol / L)
    c) Porcentaje de participantes que alcanzaron un nivel de LDL-C inferior a 110 mg / dl (2,84 mmol / L)
    d) El porcentaje de cambio en la apolipoproteína B (Apo B)
    e) El porcentaje de cambio en las lipoproteínas de colesterol de no-alta densidad (no HDL-C)
    f) Cambio porcentual en el total-C
    g) Cambio porcentual en el lipoproteína (a) (Lp [a])
    h) El porcentaje de cambio en los triglicéridos (TG)
    I) El porcentaje de cambio en el HDL-C
    E.5.2.1Timepoint(s) of evaluation of this end point
    a) From baseline to Week 8
    b), c) : At week 8
    d), e), f), g) h), I), l), m), n), o), p), q), r), s), t) : From baseline to Week 8
    a) Desde el inicio hasta la semana 8
    b), c): En la semana 8
    d), e), f), g), h), I), l), m), n), o), p), q), r), s), t): Desde el inicio hasta la semana 8
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    ascending dose
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial6
    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 EEA11
    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
    Russian Federation
    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
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    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 months6
    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: 80
    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. of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) No
    F. of subjects for this age range: 60
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 Yes
    F. of other specific vulnerable populations
    children age 8 from 17 years
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 13
    F.4.2.2In the whole clinical trial 30
    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)
    Patients participating in the open label extension period will have the possibility to participate in the paediatric Phase 3 study
    Los pacientes que participen en el período de extensión abierta tendrán la posibilidad de participar en el estudio de fase 3 pediátrica
    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 Decision2016-07-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-06-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-02-22
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