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    EudraCT Number:2010-018426-39
    Sponsor's Protocol Code Number:20080279
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2014-09-09
    Trial results View results
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    A. Protocol Information
    A.2EudraCT number2010-018426-39
    A.3Full title of the trial
    A Phase 3 Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy 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 number20080279
    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 States
    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
    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. name Nplate
    D. 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 and solution 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 INNromiplostim
    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 typerange
    D.3.10.3Concentration number0.125 to 0.5
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    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 17.0
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric subjects with immune thrombocytopenia (ITP) as
    measured by durable platelet response.
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    • To evaluate overall and weekly platelet response
    • To evaluate the use of rescue ITP medications
    • To evaluate a combined bleeding event and rescue medication use endpoint (composite bleeding episodes)
    • To evaluate the overall safety of romiplostim
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Diagnosis of primary ITP according to the ASH Guidelines at least 6 months prior to screening, regardless of splenectomy status
    • Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies
    -Prior therapy includes first-line therapies
    •Age ≥ 1 year and < 18 years at the time of providing informed consent
    • The mean of 2 platelet counts taken during the screening period must be
    ≤ 30 x 109/L with neither count > 35 x 109/L
    • A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period
    • Adequate liver function;
    -serum bilirubin ≤ 1.5 times the laboratory normal range during the
    screening period
    -AST and ALT ≤ 3.0 times the laboratory normal range during the
    screening period
    •Hemoglobin > 10.0 g/dL during the screening period
    • Subject and/or subject’s legally acceptable representative has provided informed consent prior to any study-specific procedure; subject has provided assent, where required
    E.4Principal 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
    •Known active or prior malignancy except adequately treated basal cell carcinoma
    •Known history of congenital thrombocytopenia
    •Known history of hepatitis B, hepatitis C, or HIV
    •Known history of H. pylori by urea breath test or stool antigen test within 6 months of enrollment or successfully treated with no evidence of infection
    •Known history of systemic lupus erythematosus, evans syndrome, or autoimmune neutropenia
    •Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant
    •Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
    •Previous history of venous thromboembolism or thrombotic events
    •Previous use of romiplostim, PEG-rHuMGDF, Eltrombopag, rHuTPO or any platelet producing agent
    •Rituximab (for any indication) or 6-MP within 14 weeks before the screening visit, or anticipated use during the time of the proposed study
    •Splenectomy within 4 weeks of the screening visit
    •All hematopoietic growth factors including IL-11 (oprelvekin) within 4 weeks before 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
    •Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
    •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 any other investigational procedures performed while enrolled in this clinical study
    •Subject is pregnant or breast feeding, or planning to become pregnant within
    1 month after the end of treatment
    •Female subject of child bearing potential is not willing to use, in combination with her partner, 2 forms highly effective contraception during treatment and for 1 month after the end of treatment
    •Subject has known sensitivity to any of the products to be administered during dosing
    •Subject has previously enrolled into a prior Amgen romiplostim study
    •Subject will not be available for protocol-required study visits, 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 primary endpoint is the incidence of durable platelet response. A subject with durable platelet response is defined as achieving at least 6 weekly platelet counts of ≥ 50 x 109/L during weeks 18 through 25 of treatment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From week 18
    E.5.2Secondary end point(s)
    • Subject incidence of overall platelet response. Overall responders are defined as subjects who achieve a platelet count ≥ 50 x 109/L at a minimum of 4 times during weeks 2 to 25 of the treatment period
    • Number of weekly platelet counts ≥ 50 x 109/L during weeks 2 to 25 of the
    treatment period
    • Subject incidence of rescue ITP medications used
    • The number of composite bleeding episodes. The composite bleeding episode is defined as clinically significant bleeding events or the use of a rescue medication to prevent a clinical significant bleeding event during weeks 2 to 25 of the
    treatment period. A clinically significant event is defined as a CTCAE version 3.0 grade ≥ 2 bleeding event
    • Incidence of adverse events, including thromboembolic events and hematologic malignancies, clinically significant changes in laboratory values and the incidence of antibody formation
    E.5.2.1Timepoint(s) of evaluation of this end point
    From week 0
    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. 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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    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
    The study will end once the last subject participating in the study has completed either the EOS visit (and is eligible for the 20090340 study) or requires continued platelet monitoring and subsequently completes the EOF visit and all data through that respective subject visit is recorded, verified and cleaned.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    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: 60
    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) Yes
    F. of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 34
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 20
    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 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. of subjects incapable of giving consent
    Subjects or a legally acceptable representative will provide informed consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 60
    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)
    All subjects will be offered enrollment on a roll over trial 20090340 to receive open label romiplostim and be followed on that protocol.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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