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    Clinical Trial Results:
    A phase II, open-label, prospective, single-arm, study to assess ability of eltrombopag to induce sustained remission in subjects with ITP who are refractory or relapsed after first-line steroids

    Summary
    EudraCT number
    		 2018-000452-18
    Trial protocol
    		 AT   GR   ES   GB   IT  
    Global end of trial date
    03 Oct 2022

    Results information
    Results version number
    		 v2(current)
    This version publication date
    11 Feb 2024
    First version publication date
    13 Oct 2023
    Other versions
    		 v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    CETB115J2411
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03524612
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    Novartis Campus, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.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
    03 Oct 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    03 Oct 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to assess ability of eltrombopag to induce sustained response off treatment by Month 12 in Immune Thrombocytopenia (ITP) patients who relapsed or failed to respond to first-line steroid treatment.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Nov 2018
    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
    Austria: 4
    Country: Number of subjects enrolled
    Brazil: 6
    Country: Number of subjects enrolled
    Chile: 11
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Greece: 8
    Country: Number of subjects enrolled
    Italy: 14
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    Mexico: 3
    Country: Number of subjects enrolled
    Oman: 4
    Country: Number of subjects enrolled
    Russian Federation: 9
    Country: Number of subjects enrolled
    Spain: 21
    Country: Number of subjects enrolled
    Switzerland: 2
    Country: Number of subjects enrolled
    Türkiye: 12
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    United States: 3
    Worldwide total number of subjects
    105
    EEA total number of subjects
    50
    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)
    78
    From 65 to 84 years
    24
    85 years and over
    3

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in 32 investigative sites in 15 countries:1 (Austria), 3 (Brazil), 2 (Chile), 2 (France), 2 (Greece), 2 (Italy), 1 (Japan), 2 (Mexico), 1 (Oman), 2 (Russia), 7 (Spain), 1 (Switzerland), 3 (Turkey), 1 (UK), 2 (USA).

    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
    		 Eltrombopag
    Arm description
    		 Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of >=100×10^9/L (CR), after 1st line steroids had failed.
    Arm type
    		 Tablet for oral use

    Investigational medicinal product name
    Eltrombopag
    Investigational medicinal product code
    Other name
    ETB115
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    12.5, 25, 50 and 75 mg tablets for oral use once daily

    Number of subjects in period 1
    		Eltrombopag
    Started
    105
    Completed
    63
    Not completed
    42
         Adverse event, serious fatal
    3
         Consent withdrawn by subject
    7
         Adverse event, non-fatal
    10
         Physician Decision (Patient non-compliant)
    1
         Patient Decision (Patient Changed Her Address)
    1
         Patient Decision (Personal Reasons)
    1
         Lost to follow-up
    2
         New therapy for study indication
    6
         Lack of efficacy
    10
         Protocol deviation
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Eltrombopag
    Reporting group description
    		 Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of >=100×10^9/L (CR), after 1st line steroids had failed.

    Reporting group values
    		 Eltrombopag Total
    Number of subjects
    		 105 105
    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)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 78 78
        From 65-84 years
    		 24 24
        85 years and over
    		 3 3
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 48.1 ( 19.39 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 64 64
        Male
    		 41 41
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 5 5
        Asian
    		 2 2
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 2 2
        White
    		 95 95
        More than one race
    		 0 0
        Unknown or Not Reported
    		 1 1
    Time since initial diagnosis
    Time since initial diagnosis is the difference in days between the date of initial diagnosis and the date of first dose of eltrombopag.
    Units: Days
        arithmetic mean (standard deviation)
    		 366.37 ( 911.420 ) -

    End points

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    End points reporting groups
    Reporting group title
    Eltrombopag
    Reporting group description
    		 Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of >=100×10^9/L (CR), after 1st line steroids had failed.

    Primary: Percentage of participants with sustained response off treatment (SRoT) by 12 months

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    End point title
    		 Percentage of participants with sustained response off treatment (SRoT) by 12 months [1]
    End point description
    Sustained response off treatment (SRoT) was defined as: reaching platelet count >= 100×10^9/L (complete response [CR]) and then maintaining platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count >= 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.
    End point type
    Primary
    End point timeframe
    Month 12
    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: Due to EudraCT system limitations, there must be at least two comparison groups selected for statistical analysis to be entered in the EudraCT system.
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Percentage of participants
        number (confidence interval 95%)
    30.5 (21.9 to 40.2)
    No statistical analyses for this end point

    Secondary: Median duration of sustained response off treatment (SRoT) after treatment discontinuation for participants with sustained response off treatment

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    End point title
    		 Median duration of sustained response off treatment (SRoT) after treatment discontinuation for participants with sustained response off treatment
    End point description
    Sustained response off treatment (SRoT) was defined as: reaching platelet count >= 100×10^9/L (complete response [CR]) and then maintaining platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count >= 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.
    End point type
    Secondary
    End point timeframe
    From last dose of eltrombopag to month 12
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Weeks
        median (full range (min-max))
    33.3 (4 to 51)
    No statistical analyses for this end point

    Secondary: Estimated median duration of sustained response off treatment (SRoT) for participants with sustained response off treatment at month 12 and who enter 12 months follow-up period

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    End point title
    		 Estimated median duration of sustained response off treatment (SRoT) for participants with sustained response off treatment at month 12 and who enter 12 months follow-up period
    End point description
    Sustained response off treatment (SRoT) was defined as: reaching platelet count >= 100×10^9/L (complete response [CR]) and then maintaining platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count >= 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy. Patients with SRoT until month 12 who entered follow-up and did not relapse by cut-off date/Month 24 were censored at the earliest of discontinuation date/death date/Month 24 platelet assessment date/cutoff date. Patients with SRoT until Month 12 patients who did not enter or do not yet have data in follow-up phase are censored at their Month 12 platelet assessment date.
    End point type
    Secondary
    End point timeframe
    From last dose of eltrombopag to relapse, assessed up to month 24
    End point values
    		 Eltrombopag
    Number of subjects analysed
    0 [2]
    Units: Weeks
        median (full range (min-max))
    ( to )
    Notes
    [2] - Not estimable due to number of patients who are censored
    No statistical analyses for this end point

    Secondary: Estimated median duration of sustained response off treatment (SRot) for all patients

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    End point title
    		 Estimated median duration of sustained response off treatment (SRot) for all patients
    End point description
    Patients who tapered and discontinued successfully, or did not relapse/die by cutoff date /month 24 were censored at the earliest of discontinuation date/death date/month 24 platelet assessment date/cutoff date.
    End point type
    Secondary
    End point timeframe
    From last dose of eltrombopag to month 24
    End point values
    		 Eltrombopag
    Number of subjects analysed
    0 [3]
    Units: Weeks
        median (full range (min-max))
    ( to )
    Notes
    [3] - Not estimable due to number of patients who are censored
    No statistical analyses for this end point

    Secondary: Percentage of participants with sustained response off treatment until month 24

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    End point title
    		 Percentage of participants with sustained response off treatment until month 24
    End point description
    Sustained response off treatment is defined as reach platelet count ≥ 100×10^9/L (complete response [CR]) and then maintain platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L) AND then taper off the drug until treatment discontinuation while, maintain platelet count ≥ 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy
    End point type
    Secondary
    End point timeframe
    Month 15, 18, 21 and 24
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Participants
        Month 15
    21
        Month 18
    18
        Month 21
    14
        Month 24
    15
    No statistical analyses for this end point

    Secondary: Percentage of participants with early response within first month

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    End point title
    		 Percentage of participants with early response within first month
    End point description
    Early response is defined as reaching a platelet count >= 50×10^9/L at least once within the first month (month 1) without bleeding events and no rescue therapy.
    End point type
    Secondary
    End point timeframe
    By 1 month
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Percentage of participants
        number (confidence interval 95%)
    76.2 (66.9 to 84.0)
    No statistical analyses for this end point

    Secondary: Relative change from baseline in platelet count over time

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    End point title
    		 Relative change from baseline in platelet count over time
    End point description
    Relative change (%) is the absolute change divided by the platelet counts at baseline and multiplied by 100.
    End point type
    Secondary
    End point timeframe
    Baseline, month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    104
    Units: Percentage of change
    arithmetic mean (standard deviation)
        3 Months
    1384.29 ( 3383.702 )
        6 Months
    1313.10 ( 3459.616 )
        9 Months
    1396.68 ( 3238.680 )
        12 Months
    1464.95 ( 3509.964 )
        15 Months
    1622.19 ( 3712.156 )
        18 Months
    1664.90 ( 3718.191 )
        21 Months
    2527.20 ( 5098.612 )
        24 Months
    1691.68 ( 3611.977 )
        end of treatment visit
    1213.91 ( 2952.719 )
    No statistical analyses for this end point

    Secondary: Percentage of participants with recovery response in case of loss of response during or after tapering of eltrombopag until month 24

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    End point title
    		 Percentage of participants with recovery response in case of loss of response during or after tapering of eltrombopag until month 24
    End point description
    Recovery response is defined as platelet count >=30×10^9/L after eltrombopag is re-introduced, in case of loss of response (< 30×10^9/L and/or bleeding event) without bleeding events and no rescue therapy.
    End point type
    Secondary
    End point timeframe
    Up to month 24
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Percentage of participants
    number (confidence interval 95%)
        Participants with loss of response
    11.4 (6.0 to 19.1)
        Participants with recovery response
    6.7 (2.7 to 13.3)
    No statistical analyses for this end point

    Secondary: Percentage of participants who maintain platelet counts level within 12 months and every 3 months until month 24

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    End point title
    		 Percentage of participants who maintain platelet counts level within 12 months and every 3 months until month 24
    End point description
    Platelet counts level is defined as having platelet counts >=30×10^9/L without bleeding events and no rescue therapy.
    End point type
    Secondary
    End point timeframe
    From first time of reaching the level to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Percentage of participants
    number (confidence interval 95%)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 3
    49.5 (39.6 to 59.5)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 6
    38.1 (28.8 to 48.1)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 9
    28.6 (20.2 to 38.2)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 12
    28.6 (20.2 to 38.2)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 15
    20.0 (12.8 to 28.9)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 18
    17.1 (10.5 to 25.7)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 21
    13.3 (7.5 to 21.4)
        Mnt PLT lvl f/1st time of reach. that lvl to mo 24
    14.3 (8.2 to 22.5)
        Mnt PLT lvl f/1st time of reach. that lvl to EOT
    31.4 (22.7 to 41.2)
    No statistical analyses for this end point

    Secondary: Change from baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) questionaire

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    End point title
    		 Change from baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) questionaire
    End point description
    The Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue©) is a 13- item questionnaire that assesses self-reported fatigue and its impact upon daily activities over the past 7 days. FACIT-fatigue is scored using a 4-point Likert scale. Items are scored as follows: 4 = Not At All; 3 = A Little Bit; 2 = Somewhat; 1 = Quite A Bit; 0 = Very Much, EXCEPT items #7 and #8 which are reversed scored. Score range from 0-52. A score of less than 30 indicates severe fatigue. The higher the score, the better the quality of life (less fatigue).
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    5.42 ( 9.700 )
        6 Months
    6.15 ( 8.779 )
        9 Months
    6.89 ( 9.291 )
        12 Months
    7.90 ( 9.064 )
        15 Months
    7.12 ( 11.720 )
        18 Months
    8.87 ( 11.644 )
        21 Months
    8.80 ( 11.112 )
        24 Months
    8.69 ( 10.635 )
        end of treatment visit
    5.40 ( 10.116 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) questionnaire

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    End point title
    		 Change from baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) questionnaire
    End point description
    FACT-Th6 instrument is used to measure worry/concern about bleeding and bruising, and the impact of this worry/concern on physical and social activity (Cella 2006). FACT-Th6 is a 6-item subset of the more detailed FACT-Th, which is an 18-item subscale of the validated FACT that specifically measures concerns related to thrombocytopenia in the past 7 days. The FACT-Th6 is scored using a 5-level Likert scale (0=not at all to 4=very much) and is calculated by summing scores for the 6-items; therefore, scores can range from 0–24, with higher scores representing better HRQoL
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    3.80 ( 5.130 )
        6 Months
    4.35 ( 6.312 )
        9 Months
    5.60 ( 6.114 )
        12 Months
    5.62 ( 6.505 )
        15 Months
    5.44 ( 8.949 )
        18 Months
    7.04 ( 7.654 )
        21 Months
    7.32 ( 7.351 )
        24 Months
    7.08 ( 7.048 )
        end of treatment visit
    4.15 ( 6.631 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Bodily Pain (BP) Score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Bodily Pain (BP) Score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    7.55 ( 24.199 )
        6 Months
    2.75 ( 24.636 )
        9 Months
    3.82 ( 21.507 )
        12 Months
    7.53 ( 25.547 )
        15 Months
    1.96 ( 36.697 )
        18 Months
    13.61 ( 26.662 )
        21 Months
    3.00 ( 20.967 )
        24 Months
    9.27 ( 29.090 )
        end of treatment visit
    5.19 ( 29.460 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: General Health (GH) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: General Health (GH) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    4.12 ( 18.894 )
        6 Months
    5.39 ( 20.646 )
        9 Months
    7.40 ( 17.990 )
        12 Months
    7.62 ( 18.420 )
        15 Months
    11.17 ( 20.470 )
        18 Months
    14.78 ( 26.801 )
        21 Months
    14.36 ( 25.541 )
        24 Months
    13.92 ( 19.577 )
        end of treatment visit
    3.80 ( 18.122 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Mental Health (MH) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Mental Health (MH) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    6.74 ( 15.775 )
        6 Months
    6.97 ( 18.149 )
        9 Months
    9.78 ( 15.074 )
        12 Months
    10.26 ( 16.125 )
        15 Months
    7.08 ( 22.259 )
        18 Months
    15.22 ( 22.334 )
        21 Months
    14.32 ( 20.948 )
        24 Months
    12.50 ( 17.103 )
        end of treatment visit
    6.82 ( 17.058 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Physical Functioning (PF) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Physical Functioning (PF) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    8.48 ( 23.842 )
        6 Months
    10.90 ( 26.069 )
        9 Months
    12.67 ( 28.074 )
        12 Months
    14.31 ( 28.767 )
        15 Months
    11.87 ( 37.469 )
        18 Months
    19.35 ( 33.449 )
        21 Months
    24.09 ( 31.983 )
        24 Months
    16.54 ( 35.882 )
        end of treatment visit
    9.00 ( 29.376 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Role Emotional (RE) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Role Emotional (RE) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    4.42 ( 22.371 )
        6 Months
    6.15 ( 23.270 )
        9 Months
    8.89 ( 22.361 )
        12 Months
    10.20 ( 23.260 )
        15 Months
    4.86 ( 27.022 )
        18 Months
    13.04 ( 26.211 )
        21 Months
    16.29 ( 25.520 )
        24 Months
    16.03 ( 20.128 )
        end of treatment visit
    7.25 ( 22.078 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Role Physical (RP) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Role Physical (RP) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    13.26 ( 22.958 )
        6 Months
    9.43 ( 27.066 )
        9 Months
    17.36 ( 24.165 )
        12 Months
    19.94 ( 29.232 )
        15 Months
    15.89 ( 37.454 )
        18 Months
    22.55 ( 32.680 )
        21 Months
    27.27 ( 33.604 )
        24 Months
    29.81 ( 32.849 )
        end of treatment visit
    15.88 ( 33.332 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Social Functioning (SF) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Social Functioning (SF) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    6.06 ( 24.143 )
        6 Months
    7.17 ( 26.951 )
        9 Months
    10.00 ( 22.863 )
        12 Months
    16.38 ( 26.408 )
        15 Months
    14.58 ( 30.986 )
        18 Months
    26.63 ( 24.514 )
        21 Months
    20.45 ( 26.318 )
        24 Months
    25.96 ( 22.617 )
        end of treatment visit
    11.18 ( 26.236 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Vitality (VT) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Vitality (VT) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    7.48 ( 18.418 )
        6 Months
    6.45 ( 20.696 )
        9 Months
    12.78 ( 20.982 )
        12 Months
    14.33 ( 22.398 )
        15 Months
    8.33 ( 26.237 )
        18 Months
    12.23 ( 27.918 )
        21 Months
    17.33 ( 26.087 )
        24 Months
    11.06 ( 24.705 )
        end of treatment visit
    8.97 ( 22.503 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Physical Component Summary (PCS) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Physical Component Summary (PCS) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    3.46 ( 7.445 )
        6 Months
    2.72 ( 8.165 )
        9 Months
    3.92 ( 7.608 )
        12 Months
    4.85 ( 8.946 )
        15 Months
    4.37 ( 11.754 )
        18 Months
    6.77 ( 9.950 )
        21 Months
    6.65 ( 8.017 )
        24 Months
    6.63 ( 9.627 )
        end of treatment visit
    3.30 ( 9.728 )
    No statistical analyses for this end point

    Secondary: Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Mental Component Summary (MCS) score

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    End point title
    		 Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire: Mental Component Summary (MCS) score
    End point description
    The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    85
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        3 Months
    2.34 ( 8.443 )
        6 Months
    2.85 ( 9.451 )
        9 Months
    4.52 ( 7.815 )
        12 Months
    5.33 ( 8.719 )
        15 Months
    3.28 ( 11.083 )
        18 Months
    7.12 ( 11.312 )
        21 Months
    7.18 ( 11.143 )
        24 Months
    6.87 ( 9.081 )
        end of treatment visit
    3.53 ( 9.002 )
    No statistical analyses for this end point

    Secondary: Percentage of participants with worst post-baseline value in Functional Assessment of Cancer Therapy-G (GP5)

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    End point title
    		 Percentage of participants with worst post-baseline value in Functional Assessment of Cancer Therapy-G (GP5)
    End point description
    The GP5 is a single question used to assess the overall bothersomeness of treatment side effects. The GP5 is scored using a 5-point rating scale (0 = not at all; 1 = a little bit; 2 = somewhat; 3 = quite a bit; and 4 = very much), where lower scores reflect less bothersomeness from treatment side effects.
    End point type
    Secondary
    End point timeframe
    Baseline to end of treatment visit, assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Participants
        Rating 0
    51
        Rating 1
    15
        Rating 2
    10
        Rating 3
    2
        Rating 4
    5
        Missing
    22
    No statistical analyses for this end point

    Secondary: Overall change of treatment satisfaction using Treatment Satisfaction Questionnaire (TSQM-9)

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    End point title
    		 Overall change of treatment satisfaction using Treatment Satisfaction Questionnaire (TSQM-9)
    End point description
    The Treatment Satisfaction Questionnaire for Medication (TSQM-9) is a psychometric measure of a patient's satisfaction with medication. It consists of 3 subscales: effectiveness, convenience and global satisfaction. The scores were computed by adding items for each domain, i.e. 1 to 3 for effectiveness, 4 - 6 for convenience and 7 to 9 for global satisfaction. The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range. The greatest range was (7-1) X 3 items = 18 for the effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction. This provided a transformed score between 0 and 1 that was then multiplied by 100. A positive change from baseline indicates improvement.
    End point type
    Secondary
    End point timeframe
    Baseline to month 12 (End of Treatment Visit for non-responders) and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
    End point values
    		 Eltrombopag
    Number of subjects analysed
    66
    Units: Unit on a scale
    arithmetic mean (standard deviation)
        Global Satisfaction change from BL at 12 months
    17.78 ( 29.201 )
        Global Satisfaction change from BL at 24 months
    17.69 ( 26.110 )
        Global Satisfaction change from BL at EOT
    9.96 ( 33.966 )
        Effectiveness change from BL at 12 months
    18.82 ( 33.809 )
        Effectiveness change from BL at 24 months
    17.99 ( 29.913 )
        Effectiveness change from BL at EOT
    14.14 ( 36.673 )
        Convenience change from BL at 12 months
    17.69 ( 30.758 )
        Convenience change from BL at 24 months
    19.05 ( 25.189 )
        Convenience change from BL at EOT
    15.07 ( 30.253 )
        Total score change from BL at 12 months
    54.29 ( 78.218 )
        Total score change from BL at 24 months
    54.72 ( 75.463 )
        Total score change from BL at EOT
    39.17 ( 86.449 )
    No statistical analyses for this end point

    Post-hoc: All collected deaths

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    End point title
    		 All collected deaths
    End point description
    On-treatment deaths were reported from the date of first dose of eltrombopag to 30 days after the date of the last non-zero dose of eltrombopag for patients who discontinued on or before month 12 (i.e., non-responders). For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021; approximately 35 months), as a patient could restart treatment at any time during study. Post-treatment deaths were collected in the post treatment period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021; approximately 35 months). These are not considered Adverse Events.
    End point type
    Post-hoc
    End point timeframe
    On-treatment deaths: Up to approximately 35 months. Post-treatment deaths: Up to approximately 35 months
    End point values
    		 Eltrombopag
    Number of subjects analysed
    105
    Units: Participants
        On-treatment deaths
    3
        Post-treatment deaths
    1
        All deaths
    4
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 35 months
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    All patients
    Reporting group description
    		 All patients

    Serious adverse events
    		 All patients
    Total subjects affected by serious adverse events
         subjects affected / exposed
    21 / 105 (20.00%)
         number of deaths (all causes)
    3
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Metastases to peritoneum
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Basal cell carcinoma
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Platelet count decreased
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Intentional product misuse
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences causally related to treatment / all
    1 / 3
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Superficial vein thrombosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Central nervous system haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Carotid artery stenosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cerebral haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemorrhage intracranial
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Upper gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholecystitis acute
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Epistaxis
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Petechiae
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Tuberculosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cavernous sinus thrombosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diarrhoea infectious
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Peritonsillar abscess
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 All patients
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    89 / 105 (84.76%)
    Vascular disorders
    Peripheral venous disease
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Deep vein thrombosis
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Extravasation blood
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hot flush
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hypertension
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    4
    Hypotension
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    3
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    8 / 105 (7.62%)
         occurrences all number
    10
    Fatigue
         subjects affected / exposed
    6 / 105 (5.71%)
         occurrences all number
    7
    Discomfort
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Injection site swelling
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Malaise
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Mucosal disorder
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Mucosal haemorrhage
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    10
    Oedema peripheral
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    4
    Pyrexia
         subjects affected / exposed
    5 / 105 (4.76%)
         occurrences all number
    7
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Reproductive system and breast disorders
    Breast mass
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Dysmenorrhoea
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Heavy menstrual bleeding
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    4
    Intermenstrual bleeding
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Uterine polyp
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Vaginal haemorrhage
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    4
    Respiratory, thoracic and mediastinal disorders
    Bronchitis chronic
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Cough
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    6
    Dysphonia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Dyspnoea
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Epistaxis
         subjects affected / exposed
    5 / 105 (4.76%)
         occurrences all number
    11
    Lung infiltration
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Oropharyngeal discomfort
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Oropharyngeal pain
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    3
    Productive cough
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Rhinitis allergic
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Confusional state
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Depression
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Insomnia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Irritability
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Sleep disorder
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Anxiety disorder
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    9 / 105 (8.57%)
         occurrences all number
    10
    Aspartate aminotransferase increased
         subjects affected / exposed
    6 / 105 (5.71%)
         occurrences all number
    6
    Blood alkaline phosphatase increased
         subjects affected / exposed
    7 / 105 (6.67%)
         occurrences all number
    14
    Amylase increased
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Blood bilirubin increased
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Blood creatine phosphokinase increased
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    4
    Blood creatinine increased
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Blood glucose increased
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    3
    Blood phosphorus decreased
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Blood uric acid increased
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Gamma-glutamyltransferase abnormal
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    3
    Platelet count increased
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    SARS-CoV-2 test positive
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Serum ferritin decreased
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Transaminases increased
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Contusion
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    6
    Head injury
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Muscle strain
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Neck injury
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Periorbital haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Procedural dizziness
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Procedural pain
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Wrong technique in product usage process
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Congenital, familial and genetic disorders
    Gilbert's syndrome
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    6
    Tachycardia
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    23 / 105 (21.90%)
         occurrences all number
    45
    Vocal cord paralysis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Anosmia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Autonomic nervous system imbalance
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Cerebrovascular accident
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Cervical radiculopathy
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Dizziness
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    5
    Dizziness postural
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Dysarthria
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hypotonia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Migraine
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Neuralgia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Neuropathy peripheral
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    2
    Paraesthesia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Presyncope
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Syncope
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    7 / 105 (6.67%)
         occurrences all number
    8
    Haemorrhagic diathesis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Bone marrow reticulin fibrosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Haemoglobinaemia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Thrombocytopenia
         subjects affected / exposed
    16 / 105 (15.24%)
         occurrences all number
    31
    Hyperchromasia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Leukocytosis
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Lymphadenopathy
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Neutropenia
         subjects affected / exposed
    5 / 105 (4.76%)
         occurrences all number
    7
    Neutrophilia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Platelet disorder
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Thrombocytosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    2
    Iron deficiency anaemia
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    3
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    3
    Eye disorders
    Cataract
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Central serous chorioretinopathy
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Conjunctival haemorrhage
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Eye haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Optic nerve cupping
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Periorbital oedema
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Photopsia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Retinal degeneration
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Retinal haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Scleral haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Visual impairment
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    7 / 105 (6.67%)
         occurrences all number
    10
    Diarrhoea
         subjects affected / exposed
    10 / 105 (9.52%)
         occurrences all number
    13
    Gingival bleeding
         subjects affected / exposed
    6 / 105 (5.71%)
         occurrences all number
    6
    Nausea
         subjects affected / exposed
    8 / 105 (7.62%)
         occurrences all number
    10
    Vomiting
         subjects affected / exposed
    6 / 105 (5.71%)
         occurrences all number
    9
    Mouth haemorrhage
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    3
    Abdominal discomfort
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Abdominal pain upper
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Constipation
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Dry mouth
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Dyspepsia
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    3
    Haematochezia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Lip blister
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Odynophagia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Stomatitis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Oral pain
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hepatobiliary disorders
    Cholestasis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hepatic failure
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hepatic function abnormal
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hyperbilirubinaemia
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    5
    Hypertransaminasaemia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Petechiae
         subjects affected / exposed
    11 / 105 (10.48%)
         occurrences all number
    18
    Night sweats
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Acne
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Alopecia
         subjects affected / exposed
    5 / 105 (4.76%)
         occurrences all number
    5
    Dermatitis allergic
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    2
    Dry skin
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Ecchymosis
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    3
    Eczema
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Erythema
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Onychoclasis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Pigmentation disorder
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Pruritus
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    5
    Purpura
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    2
    Rash
         subjects affected / exposed
    5 / 105 (4.76%)
         occurrences all number
    7
    Rash maculo-papular
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Skin reaction
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Skin haemorrhage
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Renal and urinary disorders
    Chromaturia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Haematuria
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Pollakiuria
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    7 / 105 (6.67%)
         occurrences all number
    10
    Back pain
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    5
    Bone pain
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Joint lock
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Joint swelling
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Limb discomfort
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Lumbar spinal stenosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Muscle spasms
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    4
    Myalgia
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Pain in extremity
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Infections and infestations
    Oral herpes
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Abscess limb
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Acute sinusitis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Adenovirus infection
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Bronchitis
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    COVID-19
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Conjunctivitis
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Coronavirus infection
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Escherichia urinary tract infection
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Eye infection viral
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Furuncle
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    6
    Gastroenteritis
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Gastrointestinal viral infection
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Helicobacter infection
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    7
    Nasopharyngitis
         subjects affected / exposed
    5 / 105 (4.76%)
         occurrences all number
    6
    Onychomycosis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Papilloma viral infection
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Periodontitis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Rhinitis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    4
    Subcutaneous abscess
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Suspected COVID-19
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Tooth abscess
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Tooth infection
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    4
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Urinary tract infection
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    4
    Viral infection
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Vulvovaginal candidiasis
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Dyslipidaemia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    2
    Hypercholesterolaemia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hyperglycaemia
         subjects affected / exposed
    3 / 105 (2.86%)
         occurrences all number
    4
    Hyperkalaemia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hypertriglyceridaemia
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    2
    Hyperuricaemia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Hypokalaemia
         subjects affected / exposed
    2 / 105 (1.90%)
         occurrences all number
    3
    Hypomagnesaemia
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1
    Iron deficiency
         subjects affected / exposed
    4 / 105 (3.81%)
         occurrences all number
    6
    Diabetes mellitus inadequate control
         subjects affected / exposed
    1 / 105 (0.95%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Mar 2019
    Amendment 01: The purpose was to update the pregnancy prevention language to align with CTFG recommendations, update the biomarker sample collection schedule, add a follow-up period of 12 Months, update the DILI guidance, and align with current eltrombopag program risk language.

    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
    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.

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