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    Summary
    EudraCT Number:2013-004328-12
    Sponsor's Protocol Code Number:Europe
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-01-15
    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 ConcernedFrance - ANSM
    A.2EudraCT number2013-004328-12
    A.3Full title of the trial
    Prospective validation of a predictive model of response to romiplostim in patients with IPSS low or intermediate-1 risk myelodysplastic syndrome (MDS) and thrombocytopenia - the EUROPE-trial
    Validation prospective d’un modèle prédictif de réponse au Romiplostim (Nplate®) chez les patients atteints d’un syndrome myélodysplasique de score IPSS de faible risque ou intermédiaire-1 et de thrombocytopénie – Étude EUROPE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Assessment of a predictive response model for romiplostim in patients with low or intermediate-1 risk myelodysplastic syndrome (MDS) and thrombocytopenia - the EUROPE-trial
    Validation prospective d’un modèle prédictif de réponse au Romiplostim (Nplate®) chez les patients atteints d’un syndrome myélodysplasique de risque faible ou intermédiaire-1 et de thrombocytopénie – Étude EUROPE
    A.3.2Name or abbreviated title of the trial where available
    Europe
    Europe
    A.4.1Sponsor's protocol code numberEurope
    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 SponsorGMIHO Gesellschaft für Medizinische Innovation - Hämatologie und Onkologie mbH
    B.1.3.4CountryGermany
    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
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGMIHO Gesellschaft für Medizinische Innovation - Hämatologie und Onkologie mbH
    B.5.2Functional name of contact pointSponsor Office
    B.5.3 Address:
    B.5.3.1Street AddressAlte Jakobstraße 77
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code10179
    B.5.3.4CountryGermany
    B.5.4Telephone number004935125933281
    B.5.5Fax number004935125933289
    B.5.6E-mailnadine.albers@gmiho.de
    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 Nplate
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen Europe B.V.
    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 Yes
    D.2.5.1Orphan drug designation number EU/3/05/283
    D.3 Description of the IMP
    D.3.1Product nameNplate
    D.3.4Pharmaceutical form Powder for solution for injection
    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 INNN-plate
    D.3.9.1CAS number 267639-76-9
    D.3.9.2Current sponsor codeAMG531
    D.3.9.3Other descriptive nameROMIPLOSTIM
    D.3.9.4EV Substance CodeSUB27756
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number750
    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 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
    Patients with IPSS low or intermediate-1 risk myelodysplastic syndrome (MDS) and thrombocytopenia
    Patients atteints d'un syndrome myélodysplasique de risque faible ou intermédiaire-1 selon l'IPSS et de thrombocytopénie
    E.1.1.1Medical condition in easily understood language
    Patients with low or intermediate-1 risk myelodysplastic syndrome (MDS) and decrease in functional platelet cells
    Patients atteints d'un syndrome myélodysplasique de risque faible ou intermédiaire-1 et de thrombocytopénie
    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 18.1
    E.1.2Level LLT
    E.1.2Classification code 10068361
    E.1.2Term MDS
    E.1.2System Organ Class 100000004864
    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 is to investigate prospectively whether the current TPO level based response model can predict response to romiplostim in thrombocytopenic patients with IPPS low/int-1 MDS
    Étudier de manière prospective l’existence d’un modèle prédictif de réponse au Romiplostim en fonction du dosage initial de TPO et de l’historique transfusionnel chez les patients thrombopéniques SMD de risque faible ou int-1 selon l’IPSS
    E.2.2Secondary objectives of the trial
    Secondary objectives are Safety, bleeding events, AML evolution, peripheral blasts during therapy,
    identification of molecular parameters associated with response and progression
    Évaluer:
    - La toxicité et la tolérance au produit
    - Les évènements hémorragiques
    - Le taux de transformation en LAM
    - L’évolution du pourcentage de blastes circulants pendant la période de traitement
    - Identifier les paramètres moléculaires associés à une réponse ou à la progression
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet the following inclusion criteria tobe eligible for the study,

    1. Must understand and voluntarily sign an informed consent form
    2. Age > 18 years at the time of signing the informed consent form
    3. Must be able to adhere to the study visit schedule and other protocol requirements
    4. Diagnosis of MDS using the WHO classification for myeloid neoplasms (Vardiman et al, 2002) as assessed during the screening period
    5. Per MDS IPSS, low or intermediate-1 risk MDS as assessed during the screening period
    6. The mean of the 2 platelet counts taken within 4 weeks prior to stratification must be:
    • ≤ 30 x 109/L (with no individual count > 30 x 109/L during the screening period), with or without a history of bleeding associated with the diagnosis of MDS, OR
    • < 50 x 109/L (with no individual count >60 x 109/L during the screening period), with a history of bleeding associated with the diagnosis of MDS
    (A standard of care platelet count taken prior to Informed consent may be used as 1 of the 2 counts taken within 4 weeks prior to randomization)
    7. Adequate liver function, as evidenced by ALT ≤ 3 times the laboratory normal range, AST ≤ 3 times the laboratory normal range and total bilirubin ≤ 2 times the laboratory normal range
    8. Bone marrow aspirate (central diagnostics) with cytogenetics (local) within 8 weeks of starting first dose of investigational product
    9. Female subjects of childbearing potential† must:
    o Agree to use, and be able to comply with, effective contraception without interruption, 4 weeks before starting study drug, throughout study drug therapy and for 4 weeks after the end of study drug therapy, even if she has amenorrhoea
    Les patients doivent remplir tous les critères suivants pour participer à l’étude :
    - Les patients doivent comprendre et signer le consentement éclairé
    - Les patients doivent être capables de se rendre aux visites médicales nécessaires à cette étude et adhérer au suivi protocolaire
    - Âge 18 ans et plus
    - SMD (OMS 2008), risque faible ou int-1 avec blastes médullaires < 5%
    - 2 bilans plaquettaires disponibles dans les 4 semaines avant la stratification et à distance de 7 jours minimum depuis la dernière transfusion de plaquettes: Un des 2 bilans peut être antérieur à la date de signature du consentement.
    Avec taux de plaquettes ≤ 30 G/L, sans aucun résultat supérieur à 30 G/L pendant la période de screening associé ou non à un antécédent hémorragique lié au SMD
    OU
    Avec un taux de plaquettes < 50 G/L, sans aucun résultat supérieur à 60 G/L pendant la période de screening ET associé à un antécédent hémorragique lié au SMD
    - Une fonction hépatique adéquate définie par : ALAT & ASAT ≤ 3 x LSN et Bilirubine totale ≤ 2 x LSN
    - Un myélogramme et un caryotype dans les 8 semaines avant la première administration du Romiplostim (sous réserve de disponibilité du matériel nécessaire à la relecture centralisée)
    - Les femmes en âge de procréer doivent:
    Utiliser une contraception efficace sans interruption (4 semaines minimum avant et 2 mois après la période de traitement par Romiplostim)
    Accepter de réaliser un test de grossesse (sensibilité minimal à 25mUI/ml) dans les 3 jours minimum avant le début de traitement
    E.4Principal exclusion criteria
    1. Pregnant or lactating females
    2. IPSS intermediate-2 or high-risk
    3. ≥ 5% blasts in the bone marrow as determined by central morphology during screening
    4. Previous treatment with any thrombopoietic growth factor
    5. Prior history of hematopoietic stem cell transplantation
    6. Active or uncontrolled disease including infections or cancer
    7. Unstable angina, congestive heart failure (NYHA > class II), uncontrolled hypertension
    8. History of arterial thrombosis (eg, stroke or transient ischemic attack) within the past year
    9. History of venous thrombosis that currently requires anti-coagulation therapy
    10. Received IL-11 within 4 weeks of the first dose of investigational product
    11. Receipt of G-CSF, peg-G-CSF, or GM-CSF within 4 weeks of the first dose of investigational product
    12. Planned receipt of peg-G-CSF or GM-CSF after the first dose of investigational product
    13. Subjects of reproductive potential who are not using adequate contraceptive precautions, in the judgment of the investigator. The sponsor recommends double barrier contraception is used for all applicable patients enrolled on this study. A double barrier method is defined as 2 methods of contraception, for example 2 actual barrier methods, or 1 actual barrier method and 1 hormonal method.
    14. Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
    15. Inability to comply with study procedures.
    16. Subject currently is enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(s)
    17. Any serious medical condition or psychiatric illness that will prevent the subject from signing the informed consent form or will place the subject at unacceptable risk if he/she participates in the study.

    - Femmes enceintes ou allaitantes
    - SMD de score IPSS int-1 avec blastose médullaire ≥ 5%, int-2 ou haut risque
    - Taux de blastes médullaires au screening ≥ 5% sur la relecture centralisée
    - Traitement antérieur par un facteur de croissance de la thrombopoïétine (TPO) ou autre agoniste TPO
    - Antécédents de greffe de cellules souches hématopoïétiques, de leucémie, d’anémie aplasique ou de pathologie de la moelle osseuse autre que le SMD
    - Maladie active ou non contrôlée (incluant infections ou cancer)
    - Angine de poitrine instable, insuffisance cardiaque congestive (NYHA > II), hypertension non contrôlée
    - Antécédent de thrombose artérielle dans l’année précédant le screening
    - Antécédent de thrombose veineuse nécessitant un traitement par anticoagulant
    - Antécédent de traitement par VIDAZA (IV, SC)
    - Traitement par REVLIMID, agent stimulant l’érythropoïèse, ou facteurs de croissance (Peg-GSCF, GCSF, GM-CSF, IL-11), sans wash out de 4 semaines avant la 1ère administration de Romiplostim
    - Hypersensibilité ou allergie à un produit dérivé de la bactérie E. coli (Nplate, Infergen, Neupogen, Somatropin, Actimmune)
    - Patient sans wash-out de 30 jours au minimum depuis la dernière administration d’un traitement à l’étude
    - Condition médicale ou psychiatrique empêchant le suivi de l’essai
    E.5 End points
    E.5.1Primary end point(s)
    • Hematologic improvement of platelets (HI-P) after 4 months on therapy
    Amélioration plaquettaire après 4 mois de traitement
    E.5.1.1Timepoint(s) of evaluation of this end point
    after 4 months on therapy
    après 4 mois de traitement
    E.5.2Secondary end point(s)
    • Cumulative hematologic improvement of platelets (HI-P), erythrocytes (HI-E) and neutrophils (HI-N)
    • The incidence of disease progression to higher stage MDS or AML
    • Increase of peripheral blasts during therapy
    • Association of the presence of certain mutations with disease progression in a retrospective analysis
    • Incidence of bleeding events
    • Type, incidence and severity of all adverse events including clinically significant changes in laboratory values
    - Les améliorations plaquettaires, érythroïdes ou neutrophiles
    - L’incidence de progression en LAM ou SMD de haut risque
    - L’augmentation de la blastose sanguine
    - L’association entre la présence de certaine mutation et la progression de la maladie (Analyse rétrospective)
    - L’incidence des évènements hémorragiques
    - Le type, l’incidence et la sévérité des évènements indésirables
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of study for all patients
    sortie d'étude de tous les patients
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    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) 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.8.1.7.1Other trial design description
    tous les patients recevront le traitement par Romiplostim, stratification en 3 groupes
    each patient receives IMP (Romiplostim), patients are stratified into 3 groups
    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.8.3 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 concerned48
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 90
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 90
    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 state90
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 90
    F.4.2.2In the whole clinical trial 90
    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)
    After conclusion of the clinical trial, patients will receive further standard medical care as usual for this kind of disease.
    Après la fin de l'essai clinique, les patients recevront des soins médicaux standards habituels pour ce genre de maladie
    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 Decision2016-01-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-14
    P. End of Trial
    P.End of Trial StatusOngoing
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