Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43839   clinical trials with a EudraCT protocol, of which   7280   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial with an Open-label Extension Phase to Evaluate the Efficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults with Chronic Immune Thrombocytopenia (Idiopathic Thrombocytopenic Purpura)

    Summary
    EudraCT number
    2011-000830-12
    Trial protocol
    NL   SK   BE   CZ   PL   BG   GR  
    Global end of trial date
    01 Mar 2014

    Results information
    Results version number
    v1(current)
    This version publication date
    08 May 2016
    First version publication date
    08 May 2016
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    E5501-G000-302
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01438840
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Eisai Inc.
    Sponsor organisation address
    155 Tice Boulevard, Woodcliff Lake, United States, 07677
    Public contact
    Medical Information, Eisai Limited, +44 08456761400, LmedInfo@eisai.net
    Scientific contact
    Medical Information, Eisai Limited, +44 08456761400, LmedInfo@eisai.net
    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
    01 Mar 2014
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    28 Nov 2013
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Mar 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Core Study To demonstrate that the efficacy of E5501 (in addition to standard of care) is superior to placebo (in addition to standard of care) for the treatment of adult participants with chronic immune thrombocytopenia (idiopathic thrombocytopenic purpura) (ITP) as measured by cumulative number of weeks of platelet response over 6 months of once daily treatment in adults participants who received at least 1 prior ITP therapy. Extension Phase To evaluate the safety and tolerability of long-term therapy with E5501 in participants with chronic ITP (cITP)
    Protection of trial subjects
    This study was conducted in accordance with standard operating procedures (SOPs) of the sponsor (or designee), which are designed to ensure adherence to Good Clinical Practice (GCP) guidelines as required by the following: • Principles of the World Medical Association Declaration of Helsinki, October 2008 • ICH E6 Guideline for GCP (CPMP/ICH/135/95) of the European Agency for the Evaluation of Medicinal Products, Committee for Proprietary Medicinal Products, International Conference on Harmonisation of Pharmaceuticals for Human Use • Title 21 of the United States Code of Federal Regulations (US 21 CFR) regarding clinical studies, including Part 50 and Part 56 concerning informed participant consent and IRB regulations and applicable sections of US 21 CFR Part 312 • A waiver from the IRB(s)/IEC(s) was obtained before study initiation for non-US studies conducted under an Investigational New Drug (IND) application. • European Good Clinical Practice Directive 2005/28/EC and Clinical Trial Directive 2001/20/EC for studies conducted within any European Union (EU) country. All suspected unexpected serious adverse reactions (SUSARs) were reported, as required, to the Competent Authorities of all involved EU member states.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    16 Feb 2012
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Poland: 22
    Country: Number of subjects enrolled
    Slovakia: 2
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Czech Republic: 7
    Country: Number of subjects enrolled
    Australia: 2
    Country: Number of subjects enrolled
    New Zealand: 1
    Country: Number of subjects enrolled
    Singapore: 2
    Country: Number of subjects enrolled
    Ukraine: 8
    Country: Number of subjects enrolled
    South Africa: 3
    Worldwide total number of subjects
    49
    EEA total number of subjects
    33
    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)
    45
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    Out of 39 participants who entered the extension phase, 29 participants completed and 10 participants discontinued the extension phase. Reason for discontinuation of participants is as follows: adverse event (3); lack of efficacy (2); not specified (1); consent withdrawn by subject (3); and lost to follow-up (1).

    Pre-assignment
    Screening details
    A total of 100 participants were screened in study. Of these 100 participants, 51 were screen failures and 49 were randomized into the study.

    Period 1
    Period 1 title
    Core Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Carer

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo (Core study)
    Arm description
    Placebo was administered as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Placebo was administered orally at a starting dose of 20 mg, once daily. Afterwards the dose could be titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on the participant's response to the study drug; placebo titration was used to maintain the blind.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo 5, 10, 20, 30 and 40 mg were administered orally, once-daily, to match avotrombopag for 26 weeks.

    Arm title
    Avatrombopag (Core study)
    Arm description
    Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg, or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, one daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.
    Arm type
    Active comparator

    Investigational medicinal product name
    Avatrombopag
    Investigational medicinal product code
    E5501
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    E5501 (avatrombopag) was administered orally as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg E5501, once daily and they were allowed to have their dose titrated up (maximum dose of 40 mg E5501) or down (minimum dose of 5 mg E5501) depending on their response to study drug.

    Number of subjects in period 1
    Placebo (Core study) Avatrombopag (Core study)
    Started
    17
    32
    Completed
    1
    22
    Not completed
    16
    10
         Consent withdrawn by subject
    1
    -
         Adverse event
    -
    3
         Lack of efficacy
    15
    7

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Placebo (Core study)
    Reporting group description
    Placebo was administered as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Placebo was administered orally at a starting dose of 20 mg, once daily. Afterwards the dose could be titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on the participant's response to the study drug; placebo titration was used to maintain the blind.

    Reporting group title
    Avatrombopag (Core study)
    Reporting group description
    Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg, or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, one daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.

    Reporting group values
    Placebo (Core study) Avatrombopag (Core study) Total
    Number of subjects
    17 32 49
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    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)
    0
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    41.2 ( 14.7 ) 46.4 ( 14.2 ) -
    Gender categorical
    Units: Subjects
        Female
    8 23 31
        Male
    9 9 18

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Placebo (Core study)
    Reporting group description
    Placebo was administered as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Placebo was administered orally at a starting dose of 20 mg, once daily. Afterwards the dose could be titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on the participant's response to the study drug; placebo titration was used to maintain the blind.

    Reporting group title
    Avatrombopag (Core study)
    Reporting group description
    Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg, or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, one daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.

    Primary: Number of Weeks with Platelet Count Greater Than or Equal to 50 x 10^9/L During 6-Month Treatment Period

    Close Top of page
    End point title
    Number of Weeks with Platelet Count Greater Than or Equal to 50 x 10^9/L During 6-Month Treatment Period
    End point description
    The cumulative number of weeks of platelet response is defined as the total numbers of weeks in which the platelet count is greater than or equal to 50 x 10^ 9/L during 6 months of treatment of core study in the absence of rescue therapy.
    End point type
    Primary
    End point timeframe
    Week 1 to Week 26
    End point values
    Placebo (Core study) Avatrombopag (Core study)
    Number of subjects analysed
    17
    32
    Units: Weeks
        median (full range (min-max))
    0 (0 to 2)
    12.4 (0 to 25)
    Statistical analysis title
    Wilcoxon (P-value)
    Comparison groups
    Placebo (Core study) v Avatrombopag (Core study)
    Number of subjects included in analysis
    49
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001
    Method
    Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Number of Participants with Platelet Count Greater Than or Equal to 50 x 10^9/L at Day 8

    Close Top of page
    End point title
    Number of Participants with Platelet Count Greater Than or Equal to 50 x 10^9/L at Day 8
    End point description
    Participants with platelet response at Day 8 are defined as those who had a platelet count greater than or equal to 50 x 10^9/L at day 8 in the absence of rescue therapy on or before Day 8.
    End point type
    Secondary
    End point timeframe
    Week 1 (Day 8)
    End point values
    Placebo (Core study) Avatrombopag (Core study)
    Number of subjects analysed
    17
    32
    Units: Participants
    number (not applicable)
        Yes
    0
    21
        No
    17
    11
    No statistical analyses for this end point

    Secondary: Number of Participants with a Reduction in Use of Concomitant Immune/idiopathic Thrombocytopenic Purpura (ITP) Medication

    Close Top of page
    End point title
    Number of Participants with a Reduction in Use of Concomitant Immune/idiopathic Thrombocytopenic Purpura (ITP) Medication
    End point description
    Only participants on concomitant ITP medications at baseline were included.
    End point type
    Secondary
    End point timeframe
    Week 1 through Week 26
    End point values
    Placebo (Core study) Avatrombopag (Core study)
    Number of subjects analysed
    17
    15
    Units: Participants
    number (not applicable)
        Yes
    0
    5
        No
    7
    10
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Core Study: up to 39 Weeks (including Screening, Titration, Treatment, Dose Taper, and Follow-up for those who did not enter the Extension Phase). Extension Phase: up to 104 weeks (including Conversion, Maintenance Period, Dose Taper, and Follow-up).
    Adverse event reporting additional description
    Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. Extension Phase SAS: All participants who received at least 1 dose of avatrombopag (either Core or Extension Phase) and had a postdose safety assessment.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.1
    Reporting groups
    Reporting group title
    Avatrombopag (Extension Phase)
    Reporting group description
    Participants who met all eligibility criteria requirements of extension phase and who discontinued the core study because of lack of treatment effect continued into the extension phase. Avatrombopag was administered to participants who entered extension phase, with a starting dose of 20 mg avatrombopag, once daily for 76 weeks and underwent dose titration.

    Reporting group title
    Placebo (Core Study)
    Reporting group description
    Placebo was administered as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Placebo was administered orally at a starting dose of 20 mg, once daily. Afterwards the dose could be titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on the participant's response to the study drug; placebo titration was used to maintain the blind.

    Reporting group title
    Avatrombopag (Core Study)
    Reporting group description
    Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg, or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, one daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.

    Serious adverse events
    Avatrombopag (Extension Phase) Placebo (Core Study) Avatrombopag (Core Study)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    15 / 47 (31.91%)
    1 / 17 (5.88%)
    9 / 32 (28.13%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Platelet count decreased
         subjects affected / exposed
    2 / 47 (4.26%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Chronic myelomonocytic leukaemia
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Jugular vein thrombosis
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Cerebrovascular accident
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Dizziness
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Headache
         subjects affected / exposed
    2 / 47 (4.26%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences causally related to treatment / all
    3 / 3
    0 / 0
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    3 / 47 (6.38%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Idiopathic thrombocytopenic purpura
         subjects affected / exposed
    0 / 47 (0.00%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Polyserositis
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Erosive duodenitis
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Food poisoning
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastritis haemorrhagic
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gingival bleeding
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Mouth haemorrhage
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    2 / 47 (4.26%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Uterine haemorrhage
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Petechiae
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Intervertebral disc disorder
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    1 / 47 (2.13%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Avatrombopag (Extension Phase) Placebo (Core Study) Avatrombopag (Core Study)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    45 / 47 (95.74%)
    10 / 17 (58.82%)
    31 / 32 (96.88%)
    Investigations
    Blood gastrin increased
         subjects affected / exposed
    2 / 47 (4.26%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    2
    0
    2
    Blood urine present
         subjects affected / exposed
    0 / 47 (0.00%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences all number
    0
    1
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    19 / 47 (40.43%)
    4 / 17 (23.53%)
    10 / 32 (31.25%)
         occurrences all number
    46
    20
    20
    Joint injury
         subjects affected / exposed
    0 / 47 (0.00%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences all number
    0
    1
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    5 / 47 (10.64%)
    1 / 17 (5.88%)
    2 / 32 (6.25%)
         occurrences all number
    5
    1
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    14 / 47 (29.79%)
    2 / 17 (11.76%)
    12 / 32 (37.50%)
         occurrences all number
    24
    5
    20
    Dizziness
         subjects affected / exposed
    2 / 47 (4.26%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences all number
    4
    1
    0
    Somnolence
         subjects affected / exposed
    0 / 47 (0.00%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences all number
    0
    3
    0
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    9 / 47 (19.15%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    11
    0
    2
    Anaemia
         subjects affected / exposed
    2 / 47 (4.26%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    3
    0
    3
    Idiopathic thrombocytopenic purpura
         subjects affected / exposed
    0 / 47 (0.00%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences all number
    0
    1
    0
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 47 (0.00%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences all number
    0
    1
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    7 / 47 (14.89%)
    1 / 17 (5.88%)
    4 / 32 (12.50%)
         occurrences all number
    8
    1
    4
    Gastrointestinal disorders
    Gingival bleeding
         subjects affected / exposed
    8 / 47 (17.02%)
    0 / 17 (0.00%)
    4 / 32 (12.50%)
         occurrences all number
    9
    0
    5
    Mouth haemorrhage
         subjects affected / exposed
    4 / 47 (8.51%)
    0 / 17 (0.00%)
    3 / 32 (9.38%)
         occurrences all number
    5
    0
    4
    Nausea
         subjects affected / exposed
    3 / 47 (6.38%)
    0 / 17 (0.00%)
    3 / 32 (9.38%)
         occurrences all number
    4
    0
    4
    Dyspepsia
         subjects affected / exposed
    1 / 47 (2.13%)
    1 / 17 (5.88%)
    1 / 32 (3.13%)
         occurrences all number
    1
    1
    1
    Vomiting
         subjects affected / exposed
    2 / 47 (4.26%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    2
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 47 (6.38%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    3
    0
    2
    Epistaxis
         subjects affected / exposed
    8 / 47 (17.02%)
    3 / 17 (17.65%)
    4 / 32 (12.50%)
         occurrences all number
    40
    5
    16
    Skin and subcutaneous tissue disorders
    Petechiae
         subjects affected / exposed
    7 / 47 (14.89%)
    1 / 17 (5.88%)
    4 / 32 (12.50%)
         occurrences all number
    15
    2
    11
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    3 / 47 (6.38%)
    1 / 17 (5.88%)
    3 / 32 (9.38%)
         occurrences all number
    4
    1
    4
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    5 / 47 (10.64%)
    0 / 17 (0.00%)
    4 / 32 (12.50%)
         occurrences all number
    9
    0
    8
    Back pain
         subjects affected / exposed
    4 / 47 (8.51%)
    0 / 17 (0.00%)
    3 / 32 (9.38%)
         occurrences all number
    4
    0
    3
    Pain in extremity
         subjects affected / exposed
    3 / 47 (6.38%)
    1 / 17 (5.88%)
    1 / 32 (3.13%)
         occurrences all number
    3
    1
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    4 / 47 (8.51%)
    0 / 17 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    4
    0
    2
    Nasopharyngitis
         subjects affected / exposed
    5 / 47 (10.64%)
    0 / 17 (0.00%)
    3 / 32 (9.38%)
         occurrences all number
    7
    0
    3
    Pharyngitis
         subjects affected / exposed
    6 / 47 (12.77%)
    1 / 17 (5.88%)
    0 / 32 (0.00%)
         occurrences all number
    6
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    11 / 47 (23.40%)
    1 / 17 (5.88%)
    6 / 32 (18.75%)
         occurrences all number
    20
    1
    7
    Urinary tract infection
         subjects affected / exposed
    3 / 47 (6.38%)
    0 / 17 (0.00%)
    1 / 32 (3.13%)
         occurrences all number
    3
    0
    1

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Jun 2012
    The ability of participants to be permanently discontinued at the discretion of the investigator after 7 days of therapy at the maximum dose if they had dangerously low platelet counts. • P-gP wording was revised to include strong P-gP inhibitor was to be added to avatrombopag therapy or if the dose of a concomitantly administered strong P-gP inhibitor was altered, platelet counts would be monitored weekly for the next 3 weeks in the event a dose adjustment of avatrombopag was required. • Wording was added to the inclusion criteria that patients with neutrophil counts above the reference range may be enrolled upon review and discussion with the Eisai medical monitor. • The fasting gastrin-17 exclusion requirement was increased to 1.5 times ULN for those participants on PPIs or H2 antagonists. Eisai Confidential Page 91 of 2178 Clinical Study Report E5501-G000-302 • Time-to-first bleeding event and time-to-first bleeding event with WHO Bleeding Scale score endpoints were included to align the current study with analyses planned in the E5501-G000-305 study. • Repeat screening laboratory evaluations due to potential laboratory error or a transient and/or reversible condition were to be made available prior to Randomization. • The ability to remove participants based on gastric biomarkers was added. • Lack of treatment effect was defined to allow participants with very low platelet counts to discontinue earlier due to lack of treatment effect and continue into the Extension Phase. • A follow-up endoscopy was requested if there was a significant abnormal endoscopy during the study.
    25 Mar 2013
    • A secondary objective was made an exploratory objective. • The secondary objective for the Extension Phase to assess the reduction in the use of steroids and concomitant ITP medication in participants receiving avatrombopag was removed. • The effectiveness assessments for the Extension Phase were revised. • The key secondary endpoint was redefined as exploratory. • The target sample size was changed to 45 participants. • The criterion that 35% of splenectomized participants will be enrolled in the study was removed. • The inclusion criterion for participants enrolling in the Extension Phase to align with study completion was clarified. • The population PK/PD analysis was revised • Study completion was defined

    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.
    Results were ready but could not be released before 21 July 2015 due to EudraCT System issues.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA