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    Summary
    EudraCT Number:2017-003641-14
    Sponsor's Protocol Code Number:D5136C00010
    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:2018-01-02
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2017-003641-14
    A.3Full title of the trial
    A Multi-centre, Phase I, Open-label, Single-dose Study to Investigate Pharmacokinetics (PK) of Ticagrelor in Infants and Toddlers, Aged 0 to less than 24 Months, with Sickle Cell Disease
    Estudio de fase I multicéntrico, abierto y de dosis única para investigar la farmacocinética (FC) de ticagrelor en lactantes y bebés de entre 0 y menos de 24 meses de edad con anemia drepanocítica.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study of the level of drug (Ticagrelor) in blood in infants and toddlers, aged 0 to less than 24 months with Sickle Cell Disease
    Ensayo clínico sobre el nivel de fármaco (Ticagrelor) en sangre en lactantes y bebés de entre 0 y menos de 24 meses de edad con enfermedad con anemia drepanocítica.
    A.3.2Name or abbreviated title of the trial where available
    HESTIA4
    A.4.1Sponsor's protocol code numberD5136C00010
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/170/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAstraZeneca AB
    B.1.3.4CountrySweden
    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 supportAstraZeneca AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAstraZeneca AB
    B.5.2Functional name of contact pointInformation Centre
    B.5.3 Address:
    B.5.3.1Street AddressN/A
    B.5.3.2Town/ citySödertälje
    B.5.3.3Post code151 85
    B.5.3.4CountrySweden
    B.5.4Telephone number+34913908282
    B.5.6E-mailinformation.centre@astrazeneca.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 nameTicagrelor
    D.3.4Pharmaceutical form Granules for oral suspension
    D.3.4.1Specific paediatric formulation Yes
    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 INNTicagrelor
    D.3.9.1CAS number 274693-27-5
    D.3.9.2Current sponsor codeAZD6140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Sickle Cell Disease
    Anemia drepanocítica
    E.1.1.1Medical condition in easily understood language
    Sickle Cell Disease
    Anemia drepanocítica
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the PK properties of ticagrelor after a single oral dose
    Determinar las propiedades FC de ticagrelor tras una dosis única por vía oral.
    E.2.2Secondary objectives of the trial
    - To determine the PK properties of the active metabolite (AR-C124910XX) after a single oral dose
    - To assess the acceptability and the palatability of a single oral dose of ticagrelor
    - To assess safety and tolerability of a single oral dose of ticagrelor
    - Determinar las propiedades FC del metabolito activo (AR-C124910XX) tras una dosis única por vía oral.
    -Evaluar la aceptabilidad y la palatabilidad de una única dosis por vía oral de ticagrelor.
    -Evaluar la seguridad y la tolerabilidad de una única dosis por vía oral de ticagrelor
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Paediatric patients aged <24 months, diagnosed with homozygous sickle cell (HbSS) or sickle beta-zero-thalassemia (HbS/β0), as confirmed by high performance liquid chromatography or haemoglobin electrophoresis.
    2.Body weight ≥5 kg at the time of screening.
    3.If treated with an anti-sickling agent such as hydroxyurea, the weight-adjusted dose must be stable for 3 months before screening/enrolment.
    4.Provision of signed and dated written informed consent from parents/legal guardians prior to any study specific procedures not part of standard medical care.
    1. Pacientes pediátricos <24 meses de edad, diagnosticados con anemia drepanocítica homocigota (HbSS) o beta-0 talasemia (HbS//β0), mediante confirmación por cromatografía líquida de alto rendimiento o electroforesis de hemoglobina.
    2. Peso corporal ≥5 kg en el momento de la selección.
    3. Si se les ha tratado con algún fármaco contra la anemia drepanocítica como la hidroxiurea, la dosis ajustada por peso debe ser estable durante 3 meses antes de la selección/inscripción.
    4. Suministro del consentimiento informado por escrito firmado y fechado de parte de los padres/tutores legales antes de realizar cualquier procedimiento específico del estudio que no sea parte de la atención médica estándar.
    E.4Principal exclusion criteria
    1. History of transient ischaemic attack or cerebrovascular event/accident (ischaemic or haemorrhagic), severe head trauma, intracranial haemorrhage, intracranial neoplasm, arteriovenous malformation, aneurysm, or proliferative retinopathy.
    2. Significantly underdeveloped with regards to height, weight or head circumference for age, as judged by the Investigator.
    3. Severe developmental delay (eg, cerebral palsy or mental retardation).
    4. Receiving chronic treatment (>3 days/week) with non-steroidal anti-inflammatory drugs (NSAIDs).
    5. Receiving chronic treatment with anticoagulants or antiplatelet drugs that cannot be discontinued.
    6. Moderate or severe hepatic impairment, defined as laboratory values of alanine aminotransferase (ALT) >2 × upper limit of normal (ULN), total bilirubin >2 × ULN (unless judged by the Investigator to be caused by haemolysis), albumin <35 g/L and international normalised ratio (INR) >1.4, or symptoms of liver disease (eg, ascites).
    7. Renal failure requiring dialysis.
    8. Active pathological bleeding or increased risk of bleeding complications according to the Investigator.
    9. Haemoglobin <6 g/dL from test performed at Screening (Visit 1).
    10. Platelets <100 × 10E9/L from test performed at Screening (Visit 1).
    11. Patient considered to be at risk of bradycardic events (eg, known sick sinus syndrome or second or third degree atrioventricular block).
    12. Concomitant oral or intravenous therapy with moderate or strong CYP3A4 inhibitors, CYP3A4 substrates with narrow therapeutic indices, or strong CYP3A4 inducers, that have not been stopped at least 5 half-lives before dose administration.
    13. Patient breastfed by mother who is under treatment of strong CYP3A4 inhibitors, as defined in Appendix E of the protocol.
    14. Active untreated malaria. Patients with suspected malaria at Screening (Visit 1) will be tested.
    15. Surgical procedure planned to occur during the study including 5 days after ticagrelor administration.
    16. Known hypersensitivity or contraindication to ticagrelor.
    17. Concern for the inability of the patient or parents to comply with study procedures and/or follow-up.
    18. Any condition which, in the opinion of the Investigator, would make it unsafe or unsuitable for the patient to participate in this study.
    19. Previously administered ticagrelor in the present study.
    20. Participation in another clinical study with an investigational medicinal product (IMP) or device during the last 30 days preceding screening/enrolment.
    21. Involvement of member of patient’s family in planning and/or conduct of the study (applies to both AstraZeneca personnel and personnel at study centre).
    1. Antecedentes de accidente isquémico transitorio o acontecimiento/accidente cerebrovascular (isquémico o hemorrágico), traumatismo craneoencefálico intenso, hemorragia intracraneal, neoplasma intracraneal, malformación arteriovenosa, aneurisma o retinopatía proliferativa.
    2. Significativamente poco desarrollado respecto a peso, estatura o circunferencia craneal para la edad, a juicio del investigador.
    3. Retraso de desarrollo intenso (por ejemplo, parálisis cerebral infantil o retraso mental).
    4. Recibir tratamiento crónico (>3 días/semana) con antiinflamatorios no esteroideos (AINE).
    5. Recibir tratamiento crónico con anticoagulantes o antiagregantes plaquetarios que no puede interrumpirse.
    6. Insuficiencia hepática moderada o intensa, definida como valores analíticos de alanina aminotransferasa (ALT) >2 × límite superior de la normalidad (LSN), bilirrubina total >2 × LSN (a menos que el investigador juzgue que está provocado por hemólisis), albúmina <35 g/l e índice internacional normalizado (INR) >1,4, o síntomas de enfermedad hepática (por ejemplo, ascitis).
    7. Insuficiencia renal que requiere diálisis.
    8. Hemorragia activa patológica o mayor riesgo de complicaciones hemorrágicas de acuerdo con el investigador.
    9. Hemoglobina <6 g/dl según la prueba realizada en la selección (visita 1).
    10. Plaquetas <100 × 109/l según la prueba realizada en la selección (visita 1).
    11. Pacientes considerados en riesgo de padecer episodios de bradicardia (por ejemplo, síndrome de disfunción sinusal o bloqueo auriculoventricular de segundo o tercer grado).
    12. Tratamiento concomitante por vía oral o intravenosa con inhibidores moderados o potentes del CYP3A4, sustratos de CYP3A4 con índices terapéuticos estrechos o inductores potentes del CYP3A4 (consulte el Anexo E), que no se han interrumpido al menos 5 semividas antes de la administración de la dosis.
    13. Paciente al que amamanta una madre que está en tratamiento con inhibidores potentes del CYP3A4, tal y como se define en el Anexo E.
    14. Paludismo activo sin tratar. A los pacientes con sospecha de paludismo se les realizará la prueba en la selección (visita 1).
    15. Procedimiento quirúrgico programado durante el estudio que incluye los 5 días posteriores a la administración de ticagrelor.
    16. Hipersensibilidad o contraindicación conocidas a ticagrelor.
    17. Preocupación por la incapacidad del paciente o de los padres para cumplir con los procedimientos y/o el seguimiento del estudio.
    18. Cualquier afección que, en opinión del investigador, podría hacer que el hecho de participar en este estudio no sea seguro o adecuado para el paciente.
    19. Ticagrelor administrado previamente en el presente estudio.
    20. Participación en otro estudio clínico con un producto en investigación (PEI) o dispositivo durante los 30 días previos a la selección/inscripción.
    21. Implicación de un miembro de la familia del paciente en la programación y/o realización del estudio (se aplica tanto al personal de AstraZeneca como al personal del centro del estudio).
    E.5 End points
    E.5.1Primary end point(s)
    Observed ticagrelor plasma concentrations as well as PK parameters obtained using a population PK analysis approach, eg, CL/F (oral clearance), Cmax, and AUC
    Concentraciones plasmáticas observadas así como parámetros FC obtenidos utilizando un enfoque de análisis de FC de la población, por ejemplo, CL/F (aclaramiento oral), Cmáx y ABC.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1, 2, 4 and 6 hours postdose
    1, 2, 4 y 6 horas postdosis.
    E.5.2Secondary end point(s)
    a- Observed plasma concentrations of the active metabolite (AR-C124910XX) as well as PK parameters obtained using a population PK analysis approach, eg, Cmax, and AUC
    b- Observer assessment of acceptability and palatability
    a.- Concentraciones plasmáticas observadas del metabolito activo (R-C124910XX) sí como parámetros FC obtenidos utilizando un enfoque de análisis de FC de la población, por ejemplo, Cmáx y ABC
    b.- Evaluación de la aceptabilidad y de la palatabilidad por parte del observador
    E.5.2.1Timepoint(s) of evaluation of this end point
    a - 1, 2, 4 and 6 hours postdose
    b - during administration of dose
    a- 1,2,4 y 6 horas postdosis.
    b- durante la administación de la dosis.
    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 No
    E.6.6Pharmacokinetic Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Pharmacokinetic
    E.7.2Therapeutic exploratory (Phase II) No
    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 No
    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 trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    Belgium
    Egypt
    Ghana
    Italy
    Kenya
    Lebanon
    Spain
    United Kingdom
    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
    LPLV
    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 months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months9
    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: 20
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 1
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 20
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children aged 0-24 months need ICF from the parent/both parents//legal guardian (according to local regulations)
    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 7
    F.4.2.2In the whole clinical trial 20
    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
    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 Decision2018-05-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-09-03
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