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    Clinical Trial Results:
    An Open-label Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura (ITP)

    Summary
    EudraCT number
    2009-016203-32
    Trial protocol
    ES   Outside EU/EEA  
    Global end of trial date
    12 Jan 2017

    Results information
    Results version number
    v1(current)
    This version publication date
    16 Jul 2017
    First version publication date
    16 Jul 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    20090340
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01071954
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Amgen Inc.
    Sponsor organisation address
    One Amgen Center Drive, Thousand Oaks, CA, United States, 91320
    Public contact
    IHQ Medical Info-Clinical Trials, Amgen (EUROPE) GmbH, MedInfoInternational@amgen.com
    Scientific contact
    IHQ Medical Info-Clinical Trials, Amgen (EUROPE) GmbH, MedInfoInternational@amgen.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-000653-PIP01-09
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    12 Jan 2017
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Jan 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to evaluate the safety of romiplostim as a long-term treatment in pediatric thrombocytopenic subjects with ITP.
    Protection of trial subjects
    This study was conducted in accordance with International Council on Harmonisation (ICH) Good Clinical Practice (GCP) regulations/guidelines, United States Food and Drug Administration (FDA) GCP regulations/guidelines, and the GCP regulations/guidelines to all regions where the study was conducted. The investigator submitted the protocol, informed consent form, investigators brochure, and any other relevant supporting information to the appropriate IEC or IRB for review and approval before study initiation. Amendments to the protocol were approved by the IEC/IRB and the local regulatory agencies, as appropriate, before the implementation of changes to the study. The investigator or his/her designee informed the subject of all aspects pertaining to the subject’s participation in the study before any screening procedures or investigational drug was administered.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    30 Dec 2009
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 7
    Country: Number of subjects enrolled
    Canada: 6
    Country: Number of subjects enrolled
    Spain: 2
    Country: Number of subjects enrolled
    United States: 51
    Worldwide total number of subjects
    66
    EEA total number of subjects
    2
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    37
    Adolescents (12-17 years)
    28
    Adults (18-64 years)
    1
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    This study was conducted at 28 centers in Australia, Canada, Spain, and the United States. Participants were enrolled from 30 December 2009 until 19 February 2015.

    Pre-assignment
    Screening details
    Pediatric subjects who completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP were eligible to participate in this study.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Romiplostim
    Arm description
    Participants received romiplostim administered by subcutaneous injection once a week. The starting dose of romiplostim was 1 μg/kg; weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of between 50 x 10^9/L and 200 x 10^9/L.
    Arm type
    Experimental

    Investigational medicinal product name
    Romiplostim
    Investigational medicinal product code
    AMG 531
    Other name
    Nplate
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Administered by subcutaneous injection with a starting dose of 1 μg/kg/week up to a maximum dose of 10 μg/kg.

    Number of subjects in period 1
    Romiplostim
    Started
    66
    Received Treatment
    65
    Completed
    39
    Not completed
    27
         Requirement for alternative therapy
    5
         Adverse event
    1
         Noncompliance
    4
         Administrative decision
    5
         Consent withdrawn by subject
    11
         Protocol Specified Criteria
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    -

    Reporting group values
    Overall Study Total
    Number of subjects
    66 66
    Age Categorical
    Units: Subjects
        In utero
    0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0
        Newborns (0-27 days)
    0 0
        Infants and toddlers (28 days-23 months)
    0 0
        Children (2-11 years)
    37 37
        Adolescents (12-17 years)
    28 28
        Adults (18-64 years)
    1 1
        From 65-84 years
    0 0
        85 years and over
    0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    10.3 ± 4.2 -
    Gender Categorical
    Units: Subjects
        Female
    37 37
        Male
    29 29
    Race
    Units: Subjects
        White or Caucasian
    40 40
        Black or African American
    9 9
        Hispanic or Latino
    9 9
        Asian
    6 6
        Japanese
    0 0
        American Indian or Alaska Native
    0 0
        Native Hawaiian or Other Pacific Islander
    0 0
        Aborigine
    0 0
        Other
    2 2

    End points

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    End points reporting groups
    Reporting group title
    Romiplostim
    Reporting group description
    Participants received romiplostim administered by subcutaneous injection once a week. The starting dose of romiplostim was 1 μg/kg; weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of between 50 x 10^9/L and 200 x 10^9/L.

    Primary: Number of Participants with Adverse Events

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    End point title
    Number of Participants with Adverse Events [1]
    End point description
    The adverse event severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: • fatal • life threatening (places the subject at immediate risk of death) • required in-patient hospitalization or prolongation of existing hospitalization • resulted in persistent or significant disability/incapacity • congenital anomaly/birth defect • other medically important serious event. The investigator assessed whether each adverse event was possibly related to the investigational product.
    End point type
    Primary
    End point timeframe
    From first dose of study drug until 1 week after last dose. The median (Q1, Q3) duration of treatment was 135.0 weeks (95.0 weeks, 184.0 weeks).
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No hypotheses were tested. The statistical analysis in this open-label extension study were descriptive in nature.
    End point values
    Romiplostim
    Number of subjects analysed
    65 [2]
    Units: participants
        All adverse events (AEs)
    64
        Serious adverse events
    19
        AEs leading to discontinuation of study drug
    2
        AEs leading to withdrawal from study
    1
        Grade 3 adverse events
    21
        Grade 4 adverse events
    5
        Grade 5 adverse events
    0
        Treatment-related adverse events (TRAEs)
    17
        Treatment-related serious adverse events
    1
        TRAEs leading to discontinuation of study drug
    0
        TRAEs leading to withdrawal from study
    0
        Grade 3 treatment-related adverse events
    3
        Grade 4 treatment-related adverse events
    1
        Grade 5 treatment-related adverse events
    0
    Notes
    [2] - Enrolled participants who received at least one dose of romiplostim.
    No statistical analyses for this end point

    Primary: Duration Adjusted Rate of Treatment Emergent Adverse Events

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    End point title
    Duration Adjusted Rate of Treatment Emergent Adverse Events [3]
    End point description
    Exposure adjusted rate was defined as total number of events divided by duration of time when subjects were under observation.
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 1 week after the last dose. The median (Q1, Q3) duration of treatment was 135.0 weeks (95.0 weeks, 184.0 weeks).
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No hypotheses were tested. The statistical analysis in this open-label extension study were descriptive in nature.
    End point values
    Romiplostim
    Number of subjects analysed
    65 [4]
    Units: events per 100 subject-years
    number (not applicable)
        All adverse events (AEs)
    1397.2
        Serious adverse events
    29.7
        AEs leading to discontinuation of study drug
    2.8
        AEs leading to withdrawal from study
    0.6
        Grade 3 adverse events
    38.5
        Grade 4 adverse events
    6.6
        Grade 5 adverse events
    0
        Treatment-related adverse events (TRAEs)
    24.2
        Serious treatment-related adverse events
    1.7
        TRAEs leading to discontinuation of study drug
    0
        TRAEs leading to withdrawal from study
    0
        Grade 3 treatment-related adverse events
    2.8
        Grade 4 treatment-related adverse events
    0.6
        Grade 5 treatment-related adverse events
    0
    Notes
    [4] - Total subject years on study = 181.8
    No statistical analyses for this end point

    Primary: Number of Participants who Developed Antibodies to Romiplostim

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    End point title
    Number of Participants who Developed Antibodies to Romiplostim [5]
    End point description
    Two validated assays were used to test for antibodies to romiplostim / the thrombopoietin-mimetic peptide component of romiplostim (TMP). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.
    End point type
    Primary
    End point timeframe
    Once a year until the end of treatment
    Notes
    [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No hypotheses were tested. The statistical analysis in this open-label extension study were descriptive in nature.
    End point values
    Romiplostim
    Number of subjects analysed
    64 [6]
    Units: participants
        Binding antibodies to romiplostim
    5
        Transient binding antibodies to romiplostim
    4
        Persistent binding antibodies to romiplostim
    1
        Neutralizing antibodies to romiplostim
    1
        Transient neutralizing antibodies to romiplostim
    0
        Persistent neutralizing antibodies to romiplostim
    1
    Notes
    [6] - Subjects with a post-baseline result
    No statistical analyses for this end point

    Primary: Number of Participants who Developed Antibodies to Endogenous Thrombopoietin

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    End point title
    Number of Participants who Developed Antibodies to Endogenous Thrombopoietin [7]
    End point description
    Two validated assays were used to test for antibodies to endogenous thrombopoietin (TPO). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.
    End point type
    Primary
    End point timeframe
    Once a year until the end of treatment
    Notes
    [7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No hypotheses were tested. The statistical analysis in this open-label extension study were descriptive in nature.
    End point values
    Romiplostim
    Number of subjects analysed
    64 [8]
    Units: participants
        Binding antibodies to TPO
    2
        Transient binding antibodies to TPO
    2
        Persistent binding antibodies to TPO
    0
        Neutralizing antibodies to TPO
    0
        Transient neutralizing antibodies to TPO
    0
        Persistent neutralizing antibodies to TPO
    0
    Notes
    [8] - Subjects with a post-baseline result
    No statistical analyses for this end point

    Secondary: Percentage of Participants with a Platelet Response

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    End point title
    Percentage of Participants with a Platelet Response
    End point description
    Platelet response was defined as any platelet counts ≥ 50 x 10^9/L in the absence of rescue medication
    End point type
    Secondary
    End point timeframe
    Every 4 weeks for the duration of treatment
    End point values
    Romiplostim
    Number of subjects analysed
    65 [9]
    Units: percentage of participants
        number (confidence interval 95%)
    93.8 (85 to 98.3)
    Notes
    [9] - Enrolled participants who received at least one dose of romiplostim.
    No statistical analyses for this end point

    Secondary: Percentage of Participants who used Concomitant ITP Therapy

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    End point title
    Percentage of Participants who used Concomitant ITP Therapy
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to the end of treatment
    End point values
    Romiplostim
    Number of subjects analysed
    65 [10]
    Units: percentage of participants
        number (confidence interval 95%)
    47.7 (35.1 to 60.5)
    Notes
    [10] - Enrolled participants who received at least one dose of romiplostim.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study drug until 1 week after last dose. The median (Q1, Q3) duration of treatment was 135.0 weeks (95.0 weeks, 184.0 weeks).
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    19.1
    Reporting groups
    Reporting group title
    Romiplostim
    Reporting group description
    Participants received romiplostim administered by subcutaneous injection once a week. The starting dose of romiplostim was 1 μg/kg; weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of between 50 x 10^9/L and 200 x 10^9/L.

    Serious adverse events
    Romiplostim
    Total subjects affected by serious adverse events
         subjects affected / exposed
    19 / 65 (29.23%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Haemangioma
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pyrexia
         subjects affected / exposed
    3 / 65 (4.62%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Ulcer haemorrhage
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Suicidal ideation
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Head injury
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Post procedural haemorrhage
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Transfusion reaction
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Platelet count decreased
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 6
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Febrile neutropenia
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Leukopenia
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune thrombocytopenic purpura
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences causally related to treatment / all
    1 / 6
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    2 / 65 (3.08%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Asthma
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 65 (3.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Mouth haemorrhage
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vomiting
         subjects affected / exposed
    2 / 65 (3.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Biliary dyskinesia
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal infection
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Clostridium difficile infection
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infection
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gingivitis
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Meningitis viral
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metapneumovirus infection
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Pneumonia mycoplasmal
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Viral infection
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Subcutaneous abscess
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory syncytial virus infection
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Romiplostim
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    64 / 65 (98.46%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    13
    Haemorrhage
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    5
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    8 / 65 (12.31%)
         occurrences all number
    8
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Fatigue
         subjects affected / exposed
    15 / 65 (23.08%)
         occurrences all number
    32
    Injection site bruising
         subjects affected / exposed
    6 / 65 (9.23%)
         occurrences all number
    9
    Pyrexia
         subjects affected / exposed
    28 / 65 (43.08%)
         occurrences all number
    76
    Pain
         subjects affected / exposed
    8 / 65 (12.31%)
         occurrences all number
    10
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    6
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    7 / 65 (10.77%)
         occurrences all number
    15
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    32 / 65 (49.23%)
         occurrences all number
    434
    Arthropod bite
         subjects affected / exposed
    7 / 65 (10.77%)
         occurrences all number
    8
    Fall
         subjects affected / exposed
    5 / 65 (7.69%)
         occurrences all number
    13
    Laceration
         subjects affected / exposed
    10 / 65 (15.38%)
         occurrences all number
    11
    Ligament sprain
         subjects affected / exposed
    7 / 65 (10.77%)
         occurrences all number
    13
    Head injury
         subjects affected / exposed
    5 / 65 (7.69%)
         occurrences all number
    6
    Limb injury
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Procedural pain
         subjects affected / exposed
    6 / 65 (9.23%)
         occurrences all number
    8
    Scratch
         subjects affected / exposed
    7 / 65 (10.77%)
         occurrences all number
    10
    Skin abrasion
         subjects affected / exposed
    15 / 65 (23.08%)
         occurrences all number
    29
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    5
    Thrombocytopenia
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Epistaxis
         subjects affected / exposed
    31 / 65 (47.69%)
         occurrences all number
    101
    Cough
         subjects affected / exposed
    30 / 65 (46.15%)
         occurrences all number
    63
    Nasal congestion
         subjects affected / exposed
    22 / 65 (33.85%)
         occurrences all number
    33
    Rhinitis allergic
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Rhinorrhoea
         subjects affected / exposed
    19 / 65 (29.23%)
         occurrences all number
    29
    Oropharyngeal pain
         subjects affected / exposed
    30 / 65 (46.15%)
         occurrences all number
    80
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    11 / 65 (16.92%)
         occurrences all number
    14
    Headache
         subjects affected / exposed
    38 / 65 (58.46%)
         occurrences all number
    149
    Paraesthesia
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    8
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    9 / 65 (13.85%)
         occurrences all number
    14
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    12 / 65 (18.46%)
         occurrences all number
    19
    Constipation
         subjects affected / exposed
    11 / 65 (16.92%)
         occurrences all number
    19
    Abdominal pain upper
         subjects affected / exposed
    20 / 65 (30.77%)
         occurrences all number
    32
    Diarrhoea
         subjects affected / exposed
    20 / 65 (30.77%)
         occurrences all number
    32
    Gingival bleeding
         subjects affected / exposed
    13 / 65 (20.00%)
         occurrences all number
    21
    Gingival pain
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    8
    Mouth haemorrhage
         subjects affected / exposed
    9 / 65 (13.85%)
         occurrences all number
    22
    Nausea
         subjects affected / exposed
    24 / 65 (36.92%)
         occurrences all number
    36
    Vomiting
         subjects affected / exposed
    32 / 65 (49.23%)
         occurrences all number
    60
    Skin and subcutaneous tissue disorders
    Ecchymosis
         subjects affected / exposed
    8 / 65 (12.31%)
         occurrences all number
    13
    Acne
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    5
    Petechiae
         subjects affected / exposed
    20 / 65 (30.77%)
         occurrences all number
    117
    Skin lesion
         subjects affected / exposed
    5 / 65 (7.69%)
         occurrences all number
    5
    Purpura
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    7
    Rash
         subjects affected / exposed
    14 / 65 (21.54%)
         occurrences all number
    24
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    15 / 65 (23.08%)
         occurrences all number
    27
    Musculoskeletal pain
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Back pain
         subjects affected / exposed
    11 / 65 (16.92%)
         occurrences all number
    15
    Joint swelling
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    4
    Myalgia
         subjects affected / exposed
    6 / 65 (9.23%)
         occurrences all number
    6
    Neck pain
         subjects affected / exposed
    7 / 65 (10.77%)
         occurrences all number
    7
    Pain in extremity
         subjects affected / exposed
    14 / 65 (21.54%)
         occurrences all number
    22
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    6 / 65 (9.23%)
         occurrences all number
    17
    Infections and infestations
    Ear infection
         subjects affected / exposed
    7 / 65 (10.77%)
         occurrences all number
    8
    Bronchitis
         subjects affected / exposed
    5 / 65 (7.69%)
         occurrences all number
    5
    Conjunctivitis
         subjects affected / exposed
    7 / 65 (10.77%)
         occurrences all number
    7
    Gastroenteritis viral
         subjects affected / exposed
    6 / 65 (9.23%)
         occurrences all number
    6
    Influenza
         subjects affected / exposed
    10 / 65 (15.38%)
         occurrences all number
    11
    Nasopharyngitis
         subjects affected / exposed
    22 / 65 (33.85%)
         occurrences all number
    67
    Tonsillitis
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    5
    Sinusitis
         subjects affected / exposed
    5 / 65 (7.69%)
         occurrences all number
    5
    Pharyngitis streptococcal
         subjects affected / exposed
    13 / 65 (20.00%)
         occurrences all number
    19
    Urinary tract infection
         subjects affected / exposed
    4 / 65 (6.15%)
         occurrences all number
    6
    Viral infection
         subjects affected / exposed
    5 / 65 (7.69%)
         occurrences all number
    8
    Upper respiratory tract infection
         subjects affected / exposed
    32 / 65 (49.23%)
         occurrences all number
    101

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    10 Dec 2011
    • The sample size was increased from 20 subjects to approximately 100 subjects. • The sample size was increased from 20 subjects to approximately 100 subjects. • 36 months treatment duration was added. • Inclusion/exclusion criteria were updated. • An end of follow-up visit was added. • Information about consenting to participate in an optional PK portion of the study was added. • The number of study centers was increased from 10 to 50 centers. • The study completion date was changed from December 2012 to December 2014. • The dosage (and adjustments), administration, and schedule were clarified. • Medications were added to the list of permitted rescue medications. • Medications were added to the list of prohibited medications. • The laboratory sample collection and shipping instructions were changed to include the additional collections of blood smears, biopsies, or aspirate, if necessary. • The collection of antibody samples was added. • The activities occurring at the study were clarified. • Reporting procedures for adverse events were clarified.
    20 Mar 2012
    • The pharmacy guide in Appendix E was removed and replaced with the Investigational Product Information Manual to accommodate study center-specific drug distribution options and to ensure that the most current investigational product details were accurately communicated. • The serious adverse event guidance was updated to reflect a 24-hour rather than 1 work day timeline for reporting serious adverse events.
    25 Sep 2012
    • Study contacts and other minor updates were made. • The definition of the study duration for subjects was updated. • The serious adverse event reporting language was updated per current Amgen safety guidelines. • The adverse event reporting language and grading criteria (National Cancer Institute - Common Terminology Criteria for Adverse Events [CTCAE] version 3.0) were changed for consistency within the romiplostim pediatric program.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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