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    Clinical Trial Results:
    A Phase 2, Interventional, Single Arm Study Describing Platelet Responses and ITP Remission Rates in Adult Subjects with Immune Thrombocytopenia Purpura Receiving Romiplostim

    Summary
    EudraCT number
    2010-019987-35
    Trial protocol
    IT   DE   GB   ES   FR   CZ  
    Global end of trial date
    26 Dec 2013

    Results information
    Results version number
    v1(current)
    This version publication date
    20 Jun 2016
    First version publication date
    30 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    20080435
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01143038
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Amgen Inc.
    Sponsor organisation address
    One Amgen Center Drive, Thousand Oaks, 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)
    No
    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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    26 Dec 2013
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Dec 2013
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To describe the number of months with a subject platelet response over a 12 month treatment period
    Protection of trial subjects
    This study was conducted in accordance with applicable FDA and International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) regulations/guidelines. A copy of the protocol, the proposed informed consent, and all other subject information and/or recruitment materials were submitted to the IEC or IRB of each study center for approval. The investigator or a designee obtained written informed consent from their subjects or legally acceptable representatives after adequate explanation of the aims, methods, anticipated benefits, and potential hazards of the study and before any protocol-specific screening procedures were conducted or investigational product was administered.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    30 Nov 2010
    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
    Poland: 15
    Country: Number of subjects enrolled
    Spain: 8
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Czech Republic: 12
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Germany: 11
    Country: Number of subjects enrolled
    Italy: 11
    Country: Number of subjects enrolled
    Australia: 3
    Country: Number of subjects enrolled
    United States: 7
    Worldwide total number of subjects
    75
    EEA total number of subjects
    65
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    61
    From 65 to 84 years
    12
    85 years and over
    2

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Ninety-eight adult subjects with ITP were screened for the study; 23 subjects were considered screen failures. Seventy-five subjects were enrolled and received at least 1 dose of romiplostim.

    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 weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with 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 ≥ 50 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
    Romiplostim was administered weekly by subcutaneous injection. 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 ≥ 50 x 10^9/L. Dose adjustments were allowed during the treatment period to maintain a platelet count between 50 x 10^9/L and 200 x 10^9/L.

    Number of subjects in period 1
    Romiplostim
    Started
    75
    Completed
    59
    Not completed
    16
         Death
    1
         Protocol deviation
    1
         Other
    1
         Requirement for alternative therapy
    4
         Adverse event
    3
         Consent withdrawn by subject
    4
         Lost to follow-up
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Romiplostim
    Reporting group description
    Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with 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 ≥ 50 x 10^9/L.

    Reporting group values
    Romiplostim Total
    Number of subjects
    75 75
    Age categorical
    Units: Subjects
        < 65 years
    61 61
        ≥ 65 years
    14 14
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    44.5 ± 18.17 -
    Gender categorical
    Units: Subjects
        Female
    44 44
        Male
    31 31
    Race
    Units: Subjects
        American Indian or Alaska Native
    0 0
        Asian
    1 1
        Black (or African American)
    1 1
        Native Hawaiian or Other Pacific Islander
    0 0
        White
    72 72
        Other
    0 0
        Unknown
    1 1
    Ethnicity
    Units: Subjects
        Hispanic/Latino
    6 6
        Not Hispanic/Latino
    69 69
    Time since ITP diagnosis
    Units: months
        median (full range (min-max))
    2.2 (0.1 to 6.6) -
    Platelet count at screening
    Units: x10^9/L
        arithmetic mean (standard deviation)
    19.78 ± 15.8 -

    End points

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    End points reporting groups
    Reporting group title
    Romiplostim
    Reporting group description
    Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with 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 ≥ 50 x 10^9/L.

    Primary: Number of Months with Platelet Response During the 12-Month Treatment period

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    End point title
    Number of Months with Platelet Response During the 12-Month Treatment period [1]
    End point description
    The primary endpoint was the number of months a subject achieved a platelet response during the 12-month treatment period. A platelet response for any 1 month was defined as the median of platelet counts measured in the month ≥ 50 x 10^9/L. Platelet counts within 4 weeks following a rescue medication use or following splenectomy were considered non-response. Months without any platelet count measurement and months following splenectomy were considered as months with no platelet response. Safety Analysis Set includes all subjects who have received at least 1 dose of romiplostim.
    End point type
    Primary
    End point timeframe
    12 months
    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: A formal hypothesis was not tested in this study. The primary analysis of the primary and secondary endpoints was descriptive.
    End point values
    Romiplostim
    Number of subjects analysed
    75 [2]
    Units: months
        arithmetic mean (standard error)
    9.2 ± 0.4
    Notes
    [2] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Percentage of subjects with ITP remisssion

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    End point title
    Percentage of subjects with ITP remisssion
    End point description
    ITP remission was defined as all platelet counts ≥ 50 x 10^9/L in the absence of romiplostim and all other therapies dosed with the intent to treat ITP for at least 6 months.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    Romiplostim
    Number of subjects analysed
    75 [3]
    Units: percentage of subjects
        number (confidence interval 95%)
    32 (21.7 to 43.8)
    Notes
    [3] - Safety analysis set
    No statistical analyses for this end point

    Secondary: Percentage of subjects with splenectomy during the 12-month treatment period

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    End point title
    Percentage of subjects with splenectomy during the 12-month treatment period
    End point description
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    Romiplostim
    Number of subjects analysed
    75 [4]
    Units: percentage of subjects
        number (confidence interval 95%)
    1.3 (0 to 7.2)
    Notes
    [4] - Safety analysis set
    No statistical analyses for this end point

    Secondary: Number of subjects who developed antibodies to romiplostim

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    End point title
    Number of subjects who developed antibodies to romiplostim
    End point description
    The number of subjects developing antibody formation (defined as negative at baseline and positive at post-baseline, transient or persistent) to romiplostim, endogenous thrombopoietin (eTPO), and thrombopoietin mimetic peptide (TMP, the peptide component of romiplostim) was summarized
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    Romiplostim
    Number of subjects analysed
    69 [5]
    Units: subjects
        Antibodies to romiplostim
    2
        Antibodies to TPO
    1
        Antibodies to TMP
    2
        Neutralizing antibodies to romiplostim
    1
        Neutralizing antibodies to TPO
    0
        Neutralizing antibodies to TMP
    0
    Notes
    [5] - Safety analysis set participants with available results
    No statistical analyses for this end point

    Secondary: Number of subjects with adverse events

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    End point title
    Number of subjects with adverse events
    End point description
    An adverse event is defined as any untoward medical occurrence in a clinical trial subject. The event does not necessarily have a causal relationship with study treatment. A serious adverse event is defined as an adverse event that meets at least one of the following serious criteria: • fatal • life threatening (places the subject at immediate risk of death) • requires in-patient hospitalization or prolongation of existing hospitalization • results in persistent or significant disability/incapacity • congenital anomaly/birth defect • other significant medical hazard. Whether an adverse event was treatment related (TRAE) or not was determined by investigator.
    End point type
    Secondary
    End point timeframe
    From first dose date of romiplostim to end of study (up to 24 months).
    End point values
    Romiplostim
    Number of subjects analysed
    75 [6]
    Units: subjects
        All treatment-emergent adverse events
    63
        Serious adverse events
    17
        Leading to discontinuation of romiplostim
    4
        Leading to discontinuation from study
    3
        Fatal adverse events
    0
        Treatment-related treatment-emergent adverse event
    21
        Treatment-related serious adverse events
    3
        TRAEs leading to discontinuation of romiplostim
    2
        TRAEs leading to discontinuation from study
    2
        Treatment-related fatal adverse events
    0
    Notes
    [6] - Safety analysis set
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose date of romiplostim to end of study (up to 24 months)
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.1
    Reporting groups
    Reporting group title
    Romiplostim
    Reporting group description
    Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with 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 ≥ 50 x 10^9/L.

    Serious adverse events
    Romiplostim
    Total subjects affected by serious adverse events
         subjects affected / exposed
    17 / 75 (22.67%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    Vascular disorders
    Peripheral vascular disorder
         subjects affected / exposed
    1 / 75 (1.33%)
         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)
    Non-Hodgkin's lymphoma
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Papillary thyroid cancer
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Delirium tremens
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Tendon rupture
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Transaminases increased
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Idiopathic thrombocytopenic purpura
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pleuritic pain
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Reversible ischaemic neurological deficit
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Faecaloma
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastritis
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Renal failure acute
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Dapsone syndrome
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Erysipelas
         subjects affected / exposed
    1 / 75 (1.33%)
         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
    50 / 75 (66.67%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    8 / 75 (10.67%)
         occurrences all number
    9
    Hypertension
         subjects affected / exposed
    6 / 75 (8.00%)
         occurrences all number
    7
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    7 / 75 (9.33%)
         occurrences all number
    7
    Epistaxis
         subjects affected / exposed
    6 / 75 (8.00%)
         occurrences all number
    8
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    5
    Headache
         subjects affected / exposed
    13 / 75 (17.33%)
         occurrences all number
    18
    Eye disorders
    Conjunctivitis
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    4
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    5 / 75 (6.67%)
         occurrences all number
    6
    Fatigue
         subjects affected / exposed
    6 / 75 (8.00%)
         occurrences all number
    7
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    7
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    4
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    5
    Petechiae
         subjects affected / exposed
    7 / 75 (9.33%)
         occurrences all number
    13
    Rash
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    6
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    11 / 75 (14.67%)
         occurrences all number
    14
    Myalgia
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    4
    Infections and infestations
    Influenza
         subjects affected / exposed
    7 / 75 (9.33%)
         occurrences all number
    10
    Nasopharyngitis
         subjects affected / exposed
    10 / 75 (13.33%)
         occurrences all number
    11
    Rhinitis
         subjects affected / exposed
    4 / 75 (5.33%)
         occurrences all number
    5
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 75 (8.00%)
         occurrences all number
    9

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Jan 2013
    - The study schema was updated to illustrate and clarify the various treatment scenarios. - Clarification was made to describe the various treatment periods and their definitions. - Clarification was made in the language throughout the protocol for medications for ITP given either concomitantly or as rescue medications. - Updates to pregnancy, antibody testing parameters, and contraception requirements were updated per the current Amgen requirements. - Updates were made to the serious adverse event reporting language in Section 9.2 per the current Amgen safety requirements.

    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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