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    Summary
    EudraCT Number:2014-000363-40
    Sponsor's Protocol Code Number:8409032
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-09-07
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2014-000363-40
    A.3Full title of the trial
    A prospective Randomized multicenter study comparing horse Antithymocyte globuline (hATG) + Cyclosporine A (CsA) with or without Eltrombopag as front-line therapy for severe aplastic anemia patients.
    Ensayo clínico, prospectivo, multicéntrico para la
    comparación entre Inmunoglobulina antilinfocítica (caballo) –
    (hATG), Cyclosporina A (CsA) con o sin Eltrombopag como
    tratamiento de primera línea en pacientes con anemia aplástica
    severa (AA).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A trial in which will be studied if the addition of a medicine that is used for improving platelet counts (eltrombopag) to the regular treatment for aplastic anemia (hATG + CsA) also improves the numbers of other cell lines and therefore the overall bone marrow function.
    ensayo para estudiar si la adición de un medicamento utilizado para mejorar el recuento de plaquetas (eltrombopag) para el tratamiento regular para la anemia aplásica (HATG + CsA) también mejora el número de otras líneas celulares y, por tanto, la función general de la médula ósea .
    A.3.2Name or abbreviated title of the trial where available
    RACE
    RACE
    A.4.1Sponsor's protocol code number8409032
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02099747
    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 SponsorEuropean Society for Blood and Marrow Transplantation
    B.1.3.4CountryNetherlands
    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 supportGlaxoSmithKline
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportPfizer
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEBMT
    B.5.2Functional name of contact pointAstrid Hoeppener
    B.5.3 Address:
    B.5.3.1Street AddressRijnsburgerweg 10
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333 AA
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31715261183
    B.5.5Fax number+31715266185
    B.5.6E-maila.e.l.m.hoeppener@lumc.nl
    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 Revolade
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxoSmithKline Trading Services Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 Film-coated 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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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
    Idiopathic (Severe) Aplastic Anemia, a bone marrow failure syndrome which results in the normal hematopoietic tissue completely missing from the bone marrow, accounting for the subsequent pancytopenia.
    Anemia Idiopática aplástica severa, un síndrome de insuficiencia de la médula ósea que se traduce en el tejido hematopoyético normal, completamente ausente de la médula ósea, lo que representa la pancitopenia subsiguiente.
    E.1.1.1Medical condition in easily understood language
    Severe aplastic anemia is an hematological disease of the bone marrow resulting in pancytopenia, a medical condition where there is a reduction in numbers of red-, white blood cells and platelets.
    Anemia aplástica severa es una enf. hematológica de la médula ósea que resulta en pancitopenia, una condición médica donde hay una reducción del número de células blancas en sangre, red y plaquetas
    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 18.0
    E.1.2Level PT
    E.1.2Classification code 10002967
    E.1.2Term Aplastic anaemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate whether Eltrombopag added to standard immunosuppressive treatment increases the rate of early (at three months) complete response in untreated AA patient.
    Investigar si Eltrombopag añadió al tratamiento inmunosupresor estándar aumenta la tasa de temprana (a los tres meses) respuesta completa en AA paciente no tratado.
    E.2.2Secondary objectives of the trial
    To investigate the impact of Eltrombopag added to standard treatment on all heamatology outcome measures, Quality of Life and safety/tolerability in untreated AA patients.
    Investigar el impacto de eltrombopag añadido al tratamiento estándar en todas las medidas de resultado heamatology, calidad de vida y la seguridad / tolerabilidad en pacientes no tratados AA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Diagnosis of severe or very severe aplastic anemia, defined by
    ? At least two of the following:
    ? Absolute neutrophil counts <0.5 x 10(9)/L (severe) or
    <0.2 x 10(9)/L (very severe)
    ? Platelet counts <20 x 10(9)/L
    ? Reticulocyte counts <60 x 10(9)/L
    ? Hypocellular bone marrow (<30% cellularity), without evidences of fibrosis or malignant cells
    2.Age ? 15 years;
    3.Written informed consent
    1.Diagnosis de la anemia aplástica grave o muy grave, definida por:
    Al menos dos de los siguientes:
    Recuento absoluto de neutrófilos <0,5 x 10 (9) / L (grave) o
    <0,2 x 10 (9) / L (muy grave)
    Los recuentos de plaquetas <20 x 10 (9) / L
    El recuento de reticulocitos <60 x 10 (9) / L
    Hipocelular médula ósea (<30% celularidad), sin evidencias de fibrosis o células malignas
    2.Age: 15 años;
    3.Written consentimiento informado
    E.4Principal exclusion criteria
    1.Prior immunosuppressive therapy with ATG (horse of rabbit) or any other lymphocyte depleting agent (i.e., alemtuzumab)

    2.Eligibility to a sibling allogeneic stem cell transplantation

    3.Evidence of a myelodysplastic syndrome, defined by the presence of myelodysplastic features, excess of blasts or karyotypic abnormalities typical of MDS (according to revised WHO 2008
    criteria) , as well as other primitive marrow disease. Patients with diagnosis of AA with cytogenetic abnormalities which are recurrent in MDS (according to revised WHO 2008 criteria) should be included in this category, and are not eligible for the study; patients with del(20q), +8 and ?Y are not included in this category, and thus are eligible for this study. The list of karyotypic abnormalities which qualifies for the diagnosis of MDS are listed in Appendix 1 of the protocol

    4.History or clinical suspect of constitutional aplastic anemia (i.e. Fanconi Anemia with positive DEB/MMC test or Dyskeratosis Congenita)

    5.History of malignant tumors with active disease within 5 years from enrollment and/or previous chemo-rediotherapy

    6.Previous history of stem cell transplantation

    7.Treatment with cyclosporin A <2 weeks before enrollment

    8.CMV viremia, as defined by positive PCR or pp65 test

    9.WHO performance status ?3

    10.Pregnant or breast feeding patients

    11.Patients with hepatic, renal or cardiac failure, or any other life-
    threatening concurrent disease

    12.Patients with HIV infection

    13.Patients without social health care assistance

    14.Participation in another clinical trial within 1 month before the start of this trial

    15.Subjects with known hypersensitivity to any of the component medications
    1.Terapia inmunosupresora antes con ATG (caballo de conejo) o cualquier agente que agotan otra linfocitos (es decir, el alemtuzumab)
    2.Eligibility a un hermano el trasplante alogénico de células madre
    3.Evidence de un síndrome mielodisplásico, definido por la presencia de características mielodisplásicos, exceso de blastos o anormalidades cariotípicas típicos de MDS (de acuerdo con la OMS revisada 2008
    criterios), así como otra enfermedad ósea primitiva. Los pacientes con diagnóstico de AA con anormalidades citogenéticas que son recurrentes en MDS (según criterios revisados ​​de la OMS 2008) deben incluirse en esta categoría, y no son elegibles para el estudio; pacientes con del (20q), 8 y? Y no se incluyen en esta categoría, y por lo tanto son elegibles para este estudio. La lista de las anomalías del cariotipo que califica para el diagnóstico de MDS se enumeran en el Apéndice 1 del protocolo
    4.History o sospecha clínica de la anemia aplásica constitucional (es decir, la Anemia de Fanconi con la prueba de MMC positivo DEB / o disqueratosis congénita)
    5.History de tumores malignos con enfermedad activa dentro de los 5 años a partir de la matrícula y / o el anterior quimio-rediotherapy
    Historia 6.Previous de trasplante de células madre
    7.Treatment con ciclosporina A <2 semanas antes de la inscripción
    Viremia 8.CMV, según lo definido por PCR positiva o prueba de pp65
    Rendimiento 9.WHO estado? 3
    10.Pregnant o pacientes en periodo de lactancia
    11.Patients con insuficiencia hepática, renal o insuficiencia cardíaca o cualquier otra vida
    enfermedades concurrentes mortal
    12.Patients con la infección por VIH
    13.Patients sin asistencia atención sociosanitaria
    14.Participation en otro ensayo clínico en 1 mes antes del inicio de este ensayo
    15.Subjects con hipersensibilidad conocida a cualquiera de los medicamentos de componentes
    E.5 End points
    E.5.1Primary end point(s)
    Rate of Complete response (defined as Hb >10 g/dL, ANC > 1,000/?L and Plt >100,000 ?L) at 3 months since start of treatment in untreated severe AA patients.
    Tasa de respuesta completa (definida como Hb> 10 g / dl, RAN> 1.000 L /? Y Plt> 100 000? L) a los 3 meses desde el inicio del tratamiento en pacientes AA graves no tratados.
    E.5.1.1Timepoint(s) of evaluation of this end point
    For the main endpoint a valid response has to be measured between month 3 plus or minus 10 days; in case of more than one measurement available in this time period, the best response will be taken.
    Para el punto final principal de una respuesta válida tiene que medir entre 3 meses más o menos de 10 días; en el caso de más de una medición disponible en este período de tiempo, se tomará la mejor respuesta.
    E.5.2Secondary end point(s)
    1.Time to first hematological response (complete or partial) described by a cumulative incidence curve

    2.Time to best hematological response, described by a cumulative incidence curve

    3.Time to complete response

    4.Rates of hematological response (overall, complete, partial) at 6, 12, 18 and 24 months

    5.Overall survival (OS) probability; OS is defined as time from treatment starting (day 1) to death, or last follow-up for patients alive

    6.Event-free survival (EFS) probability;

    7.Cumulative incidence of relapse, from first hematological response (complete or partial)

    8.Cumulative incidence of clonal evolution : AML, MDS or karyotypic abnormalities

    9.Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence

    10.Cumulative incidence of discontinuation of immunosuppressive therapy

    11.Rate of CsA-independent hematological response at 24 months

    12.Need for transfusions (packed red cell units and platelet units) and number of transfusions required from treatment.

    13.Need for any supportive care, including hospitalization

    14.Quality of life (as assessed by the validated EORTC QLQ-C30 questionnaire)(changes over time and differences between treatment arms)

    15.Safety and tolerability of the investigational treatment, including SAE
    E.5.2.1Timepoint(s) of evaluation of this end point
    3, 6, 12, 18 and 24 months
    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 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) Yes
    E.8.2.2Placebo No
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    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 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
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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: 15
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 160
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 25
    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
    This trial includes minors (15-17 years of age) because a peak in AA occurence has been observed around this age. For these patients, the signature of a legal representative is required.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state19
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 200
    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
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-10-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-10-08
    P. End of Trial
    P.End of Trial StatusOngoing
    The status of studies in GB is no longer updated from 1.1.2021
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