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    Summary
    EudraCT Number:2017-004489-88
    Sponsor's Protocol Code Number:RC31/16-8913
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-12-11
    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 ConcernedFrance - ANSM
    A.2EudraCT number2017-004489-88
    A.3Full title of the trial
    Evaluation of perioperative eltrombopag for the management of elective surgery and invasive acts in patients with inherited thrombocytopenia
    Evaluation de l’ELtrombopag en Peri-Opératoire lors de chirurgies et actes invasifs programmés chez les patients ayant une Thrombopénie constitutionnelle
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of perioperative eltrombopag in patients with inherited thrombocytopenia
    Evaluation de l’ELtrombopag en Peri-Opératoire chez les patients ayant une Thrombopénie constitutionnelle
    A.3.2Name or abbreviated title of the trial where available
    ELPOT
    ELPOT
    A.4.1Sponsor's protocol code numberRC31/16-8913
    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 SponsorCHU Toulouse
    B.1.3.4CountryFrance
    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 supportGIRCI
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCHU Toulouse
    B.5.2Functional name of contact pointCLINICAL RESEARCH ASSISTANT
    B.5.3 Address:
    B.5.3.1Street AddressHôtel-Dieu 2 rue Viguerie TSA 80035
    B.5.3.2Town/ cityToulouse
    B.5.3.3Post code31059
    B.5.3.4CountryFrance
    B.5.4Telephone number05 61 77 84 90
    B.5.5Fax number05 61 77 84 11
    B.5.6E-maildaguzan.c@chu-toulouse.fr
    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.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameEltrombopag
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.11 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
    Inherited thrombocytopenias
    Thrombopénie constitutionnelle
    E.1.1.1Medical condition in easily understood language
    Inherited thrombocytopenias
    Thrombopénie constitutionnelle
    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 HLT
    E.1.2Classification code 10043555
    E.1.2Term Thrombocytopenias
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to estimate the response to eltrombopag based on platelet count increase and lack of requirement for PC transfusion for performing invasive acts at mild or high bleeding risk
    L’objectif principal de l’étude est d’estimer la réponse à l’eltrombopag, sur la base de la correction de la numération plaquettaire , combinée à la possibilité de réaliser des actes invasifs à risque hémorragique modéré ou élevé sans recourir aux transfusions de CP.
    E.2.2Secondary objectives of the trial
    The secondary objective is to document in this particular setting the safety profile of eltrombopag, the kinetics of platelet recovery and the predictive value Inherited thrombocytopenia (IT) type, serum thrombopoietin (TPO) level and baseline platelet count on the response to eltrombopag.
    L’objectif secondaire est de documenter dans ce contexte particulier le profil de tolérance de l’eltrombopag, la cinétique de correction de la thrombopénie et la valeur prédictive du taux basal de thrombopoïétine endogène sérique sur la réponse plaquettaire.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Related to patients: Symptomatic inherited thrombocytopenia (IT) with family history and/or molecular identification with an average platelet count in the previous year below the safety level required for the procedure. Written informed consent of the patient or relatives.
    Related to the procedure: scheduled (≥4 weeks) invasive procedure with anticipated risk and limited possibility of mechanical control of bleeding, which would have systematically required prophylactic and therapeutic PC transfusions, according to current international recommendations (standard care).
    Relatifs aux patients: IT symptomatique avec antécédent familiaux et/ou confirmation moléculaire, avec un taux moyen de plaquettes au cours de la dernière année inférieur au seuil de sécurité requis pour l’acte invasif. L’information du patient ou des titulaires de l’autorité parentale et le recueil du consentement écrit sont impératifs. Le patient devra être affilié à un régime de sécurité sociale.
    Relatifs à l’acte invasif: acte programmé (≥4 semaines) avec un risqué anticipé de saignement excessif et une possibilité réduite de contrôle mécanique d’une hémorragie éventuelle, ce qui justifie l’administration systématique de CP à titre préventif ou curatif selon les recommandations actuelles internationales (prise en charge standard).
    E.4Principal exclusion criteria
    • age <6 and >75 yrs
    • personal history of arterial or venous thromboembolic events
    • association with another acquired or constitutional hemorrhagic diathesis
    • chronic hepatitis, cirrhosis, with moderate to severe liver failure (Child-Pugh score ≥5)
    • previous or concurrent myeloid malignancy, including myelodysplastic syndrome
    • alanine aminotransferase (ALT) or bilirubin levels 2 times the upper limit of normal
    • severe altered renal function (creatinin clearance <30 ml/min)
    • pregnancy (negative test required before inclusion in fertile women) or lactating women
    • refusal of safe contraception
    • ocular lenses opacity
    • hypersensitivity to eltrombopag or one of excipients
    • previous participation to the present study
    • current treatment with antiplatelet drugs, anticoagulants or direct acting antiviral agents approved for treatment of chronic hepatitis C infection
    • psychiatric, social or behavioral condition judged to be non-compatible with the respect of the protocol, including good observance of treatment and compliance to follow-up.
    • adult protected by the law.
    • age <6 et >75 ans
    • antécédent personnel de thrombose artérielle ou veineuse
    • association à une diathèse hémorragique d’autre nature (constitutionnelle ou acquise)
    • hépatite chronique ou cirrhose, avec insuffisance hépatique modérée ou sévère (score Child-Pugh ≥5)
    • Antécédent d’hémopathie myéloïde (incluant les syndromes myélodysplasiques)
    • Taux d’alanine aminotransferase (ALT) ou de bilirubine >2 fois la limite normale supérieure du laboratoire
    • Insuffisance rénale sévère (clearance de la créatinine <30 ml/min)
    • Grossesse en cours (test négatif requis au moment de l’inclusion chez les femmes en âge de procréer).
    • Refus d’une contraception efficace pendant la durée de l’essai chez les femmes en âge de procréer.
    • Cataracte oculaire.
    • Intolérance à l’eltrombopag ou à l’un de ses excipients.
    • Participation antérieure à ce même essai
    • Traitement en cours par un antiagrégant plaquettaire, un anticoagulant ou un antiviral direct utilisé pour le traitement de l’infection par le virus de l’hépatite C.
    • Etat psychiatrique, social ou comportemental jugée non compatible avec le respect du protocole de l’essai, incluant la bonne observance, et l’acceptation du suivi médical.
    • Adulte protégé par la loi.
    E.5 End points
    E.5.1Primary end point(s)
    Full response (“success”) is defined as an increase in platelet count above the safety level and a procedure performed without PC transfusion,
    Une réponse complète (synonyme de succès) est définie par une numération plaquettaire atteignant le seuil de sécurité et l’absence de recours à l’administration périopératoire de CP.
    E.5.1.1Timepoint(s) of evaluation of this end point
    4 weeks
    4 semaines
    E.5.2Secondary end point(s)
    Adverse effects recorded during treatment and the 4-week follow-up after discontinuation, including excessive bleeding or any complication related to bleeding or thrombotic events.
    Serial platelet counts and change in platelet size
    Tout effet indésirable enregistré pendant et après le traitement pendant la durée du suivi (4 semaines), incluant un saignement jugé excessif et toute complication liée à celui-ci, et/ou un évènement thrombotique vasculaire.
    Cinétique de la numération plaquettaire et des variations de la taille des plaquettes.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/week during 8 weeks
    1/semaine durant 8 semaines
    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 No
    E.6.5Efficacy No
    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) 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 Yes
    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 concerned25
    E.8.5The trial involves multiple Member States No
    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 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
    LVLS
    Dernière visite du dernier patient
    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 months36
    E.8.9.1In the Member State concerned days
    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: 25
    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: 25
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 25
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    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 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 No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    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)
    When the subject has ended the participation in the trial, he is follow
    as usual practice
    A la fin de la recherche, les patients sont pris en charge conformément
    à la pratique courante
    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 Decision2018-02-01
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
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