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    Summary
    EudraCT Number:2011-005019-96
    Sponsor's Protocol Code Number:20101221
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-08-21
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2011-005019-96
    A.3Full title of the trial
    A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and Safety Study of Romiplostim in Paediatrics with Immune Thrombocytopenia
    A.4.1Sponsor's protocol code number20101221
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/114/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmgen Inc
    B.1.3.4CountryUnited Kingdom
    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 Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmgen (EUROPE) GmbH
    B.5.2Functional name of contact pointIHQ Medical Information - Clinical
    B.5.3 Address:
    B.5.3.1Street AddressDammstrasse 23, P.O. Box 1557
    B.5.3.2Town/ cityZug
    B.5.3.3Post codeCH 6300
    B.5.3.4CountrySwitzerland
    B.5.6E-mailMedinfoInternational@amgen.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 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 numberEMEA/OD/008/05
    D.3 Description of the IMP
    D.3.1Product nameNplate
    D.3.2Product code AMG 531
    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 INNNplate
    D.3.9.1CAS number 267639-76-9
    D.3.9.2Current sponsor codeAMG 531
    D.3.9.3Other descriptive nameROMIPLOSTIM
    D.3.9.4EV Substance CodeSUB27756
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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
    Immune Thrombocytopenia (ITP) in Paediatric Subjects
    E.1.1.1Medical condition in easily understood language
    Bleeding Disorder - ITP is a condition that may cause easy bruising or bleeding due to an
    abnormally low number of platelets in the blood.
    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 10043558
    E.1.2Term Thrombocytopenia purpura
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to describe the percentage of time that pediatric subjects with immune thrombocytopenia (ITP) have a platelet response in the first 6 months from the start of treatment with romiplostim.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    • To describe the percentage of time that pediatric subjects with ITP have a platelet response over the study duration
    • To describe the percentage of time that pediatric subjects with ITP have an increase in platelet count ≥ 20 x 10 (to the ) 9/L above baseline over the study duration
    • To describe the use of rescue ITP medications
    • To describe the incidence of antibody formation
    • To describe the safety of romiplostim as a long-term treatment in pediatric thrombocytopenic subjects with ITP
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    A supplement to the protocol for European Union (EU) Switzerland, and Turkey. These changes are being made to fulfill the binding elements of the European Medicines Agency Pediatric Investigation Plan (PIP) and include the addition of mandatory bone marrow biopsies and aspirates for evaluation of collagen, reticulin and cytogenetics at baseline and after Year 1 and Year 2 (in separate cohorts) to assess the long term safety of romiplostim in pediatric patients with immune thrombocytopenia purpura (ITP).

    Title: A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)

    Date: 15 July 2014 (Version 2)

    A subset of at least 60 subjects will be enrolled sequentially into the following cohorts:
    • Bone marrow biopsy and aspirate at baseline and Year 1
    • Bone marrow biopsy and aspirate at baseline and Year 2
    All subjects in these 2 cohorts will receive romiplostim for 3 years, unless withdrawn from the study early. They will complete an End of Treatment (EOT) visit at the conclusion of their treatment period and will then return an End of Study (EOS) visit.

    Primary Objective: same as main study with addition of:
    To evaluate incidence of changes in bone marrow findings at Year 1 and Year 2 after initial exposure to romiplostim

    Secondary objectives: same as main study with addition of:

    • To evaluate the incidence of increased reticulin as evidenced by silver staining at Year 1 or Year 2, after exposure to romiplostim
    E.3Principal inclusion criteria
    • Diagnosis of primary ITP according to The American Society of Hematology (ASH) Guidelines (Neunert et al, 2011) at least 6 months before screening, regardless of splenectomy status
    • Age ≥ 1 year and < 18 years at the time of providing informed consent
    • Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or be ineligible for other ITP therapies
    • Examples of prior therapy include but are not limited to: corticosteroids, IVIG, anti-D immunoglobulin, and platelet transfusions. Subjects who have failed a splenectomy are eligible for study participation
    • Subject has a documented platelet count ≤ 30 x10 (to the)9/L or is experiencing bleeding that is uncontrolled with conventional therapies
    • Subject’s legally acceptable representative (or subject, if applicable) has provided informed consent before any study-specific procedure; and subject has proved assent, where required by the IRB/IEC
    • Adequate hematologic, renal, and liver function during the screening period:
    -Hemoglobin > 10.0 g/dL
    -Serum creatinine ≤ 1.5 times the upper limit of normal
    -Total serum bilirubin ≤ 1.5 times the upper limit of normal
    -AST and ALT ≤ 3.0 times the upper limit of normal
    E.4Principal exclusion criteria
    Exclusion Criteria
    • Known history of a bone marrow stem cell disorder (Any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study)
    • Prior bone marrow transplant or peripheral blood progenitor cell transplant
    • Known active or prior malignancy except non-melanoma skin cancers within the last 5 years
    • Known history of myelodysplastic syndrome
    • Known history of bleeding diathesis
    • Known history of congenital thrombocytopenia
    • Known history of hepatitis B, hepatitis C or human immunodeficiency virus
    • Known history of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia
    • Known history of antiphospholipid antibody syndrome or known positive for lupus anticoagulant
    • Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
    • History of venous thromboembolism or thrombotic events
    • Previous use of romiplostim. Previous use of eltrombopag within 4 weeks of enrollment.
    • Previous use of PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO) or any other platelet producing agent
    • Rituximab (for any indication) or 6-mercaptopurine within 8 weeks of enrollment, or anticipated use at any time during the study
    • Splenectomy within 4 weeks of the screening visit

    • Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
    • Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
    • Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s)
    • Subject will have investigational procedures performed while enrolled in this clinical study
    • Female subject of child bearing potential (defined as having first menses) is not willing to use, in combination with her partner highly effective methods of birth control during treatment and for 1 month after the end of treatment
    • Subject is pregnant or breast feeding, or might become pregnant within 1 month after the end of treatment
    • Subject has known hypersensitivity to any recombinant Escherichia coli derived product (eg, Infergen, Neupogen, somatropin, and Actimmune®)
    • Subject has previously enrolled into this study
    • Subject will not be available for protocol-required study visits or procedures, to the best of the subject’s and investigator’s knowledge
    • Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures
    E.5 End points
    E.5.1Primary end point(s)
    The percentage of time with a platelet count of ≥ 50 x 10 (to the) 9/L starting from week 2 in the first 6 months of the treatment period without rescue medication use within the past 4 weeks

    In addition, for protocol supplement:

    • Evaluation of bone marrow changes after Year 1 and Year 2 for the following:
    - Incidence of collagen as evidenced by trichrome staining (using the modified Bauermeister grading scale) after romiplostim exposure
    - Incidence of bone marrow reticulin increases in severity ≥ 2 grades (ie, grade 0 to 2-4, 1 to 3-4, 2 to 4), compared to baseline, or an increase to grade 3 or grade 4 as
    evidenced by reticulin silver staining using the modified Bauermeister grading scale after romiplostim exposure
    - Incidence of bone marrow abnormalities (eg, myelodysplastic syndrome, monosomy 7) as evidenced by cytogenetics and fluorescence in situ hybridization
    E.5.1.1Timepoint(s) of evaluation of this end point
    From week 2
    E.5.2Secondary end point(s)
    The percentage of time with a platelet count of ≥ 50 x 10 (to the) 9/L starting from week 2 until the end of the treatment period without rescue medication use within the past 4 weeks
    • The percentage of time with an increase in platelet count ≥ 20 x 10 (to the) 9/L above baseline starting from week 2 until the end of the treatment period without rescue medication use in the past 4 weeks.
    • Subject incidence of rescue ITP medications used
    • The incidence of anti-romiplostim neutralizing antibodies and cross reactive antibodies to TPO at any time during the study
    • The incidence of adverse events, including clinically significant changes in laboratory values

    In addition, for protocol supplement:

    • The incidence of increased reticulin as evidenced by silver staining at Year 1 or Year 2, after exposure to romiplostim
    E.5.2.1Timepoint(s) of evaluation of this end point
    From week 2
    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 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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA27
    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
    Australia
    Belgium
    Brazil
    Canada
    Czech Republic
    France
    Hungary
    Israel
    Mexico
    Peru
    Poland
    Russian Federation
    South Africa
    Spain
    Switzerland
    Turkey
    United Kingdom
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    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: 200
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 4
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 118
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 78
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Trial includes pediatric subjects (from 1 year old).
    All subjects and/or their legally acceptable representatives (eg, parents, legal guardian) will sign & date the ICF before subjects can undergo any study-specific procedures.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    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)
    Subjects may be eligible for continued treatment as provided for by the local country's regulatory mechanism.
    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 Decision2014-12-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-11-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-08-15
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