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   44335   clinical trials with a EudraCT protocol, of which   7366   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 II, randomized (1:1) open label study to assess the efficacy and safety of eltrombopag in combination with dexamethasone compared to dexamethasone, as first-line treatment in adult patients with newly diagnosed immune thrombocytopenia (XPAG-ITP) Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd. com/CtrdWeb/home.nov for complete trial results Summary.

    Summary
    EudraCT number
    2019-002658-21
    Trial protocol
    DE  
    Global end of trial date
    22 Sep 2023

    Results information
    Results version number
    v1(current)
    This version publication date
    25 Sep 2024
    First version publication date
    25 Sep 2024
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    CETB115JDE01
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04346654
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Novartis Pharma, AG
    Sponsor organisation address
    CH-4002, 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
    22 Sep 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Sep 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To compare the ability of eltrombopag in combination with a short course of dexamethasone to induce a sustained response off treatment at 52 weeks versus a defined course of dexamethasone
    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
    09 Oct 2020
    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
    Germany: 26
    Worldwide total number of subjects
    26
    EEA total number of subjects
    26
    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)
    26
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    The study was conducted in 25 centers in Germany.

    Pre-assignment
    Screening details
    Participants had to abstain from using investigational/marketed drugs and taking herbal supplements prior to taking the first dose of study treatment.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Eltrombopag + Dexamethasone
    Arm description
    Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.
    Arm type
    Experimental

    Investigational medicinal product name
    Eltrombopag
    Investigational medicinal product code
    ETB115
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Eltrombopag 25 mg, 50 mg taken daily

    Investigational medicinal product name
    Dexamethasone
    Investigational medicinal product code
    ETB115
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Dexamethasone 40 mg taken daily

    Arm title
    Dexamethasone
    Arm description
    Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
    Arm type
    Active comparator

    Investigational medicinal product name
    Dexamethasone
    Investigational medicinal product code
    ETB115
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Dexamethasone 40 mg taken daily

    Number of subjects in period 1
    Eltrombopag + Dexamethasone Dexamethasone
    Started
    13
    13
    Randomized, not treated
    1 [1]
    0 [2]
    Completed
    8
    10
    Not completed
    5
    3
         Consent withdrawn by subject
    2
    -
         Adverse event, non-fatal
    2
    -
         Pregnancy
    -
    1
         Lost to follow-up
    -
    1
         Non-response
    1
    1
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The number of subjects is consistent. Just added this milestone for clarification to the reader.
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The number of subjects is consistent. Just added this milestone for clarification to the reader.

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Eltrombopag + Dexamethasone
    Reporting group description
    Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.

    Reporting group title
    Dexamethasone
    Reporting group description
    Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.

    Reporting group values
    Eltrombopag + Dexamethasone Dexamethasone Total
    Number of subjects
    13 13 26
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    13 13 26
        From 65-84 years
    0 0 0
        85 years and over
    0 0 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    60.6 ( 14.5 ) 45.3 ( 14.3 ) -
    Sex: Female, Male
    Units: Participants
        Female
    6 6 12
        Male
    7 7 14
    Race/Ethnicity, Customized
    Units: Subjects
        Caucasian
    12 12 24
        Other
    1 1 2

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Eltrombopag + Dexamethasone
    Reporting group description
    Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.

    Reporting group title
    Dexamethasone
    Reporting group description
    Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.

    Primary: Percentage of patients with sustained response off treatment at 52 weeks

    Close Top of page
    End point title
    Percentage of patients with sustained response off treatment at 52 weeks
    End point description
    Sustained response off treatment at 52 weeks is defined as maintenance of platelet count ≥ 30 × 10^9/L after treatment discontinuation until Week 52 in the absence of bleeding events ≥ Grade II or use of any rescue medication at all visits until Week 52
    End point type
    Primary
    End point timeframe
    Study treatment discontinuation until week 52
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Participants
    2
    2
    Statistical analysis title
    Sustained Response of treatment - Primary endpoint
    Comparison groups
    Eltrombopag + Dexamethasone v Dexamethasone
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.5133
    Method
    Regression, Logistic
    Confidence interval

    Secondary: Percentage of patients with overall response at Week 52

    Close Top of page
    End point title
    Percentage of patients with overall response at Week 52
    End point description
    Overall response after treatment at week 52 was defined as maintenance of platelet count ≥ 30 x 109/L and ≥ 2-fold increase of screening platelet count after treatment discontinuation in the absence of bleeding event ≥ Grade II and no rescue therapy at all visits until Week 52.
    End point type
    Secondary
    End point timeframe
    Study treatment discontinuation until week 52
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Participants
    2
    2
    Statistical analysis title
    ORR at week 52
    Comparison groups
    Eltrombopag + Dexamethasone v Dexamethasone
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.5133
    Method
    Regression, Logistic
    Confidence interval

    Secondary: Duration of sustained response off treatment

    Close Top of page
    End point title
    Duration of sustained response off treatment
    End point description
    Duration of sustained response off treatment is defined as time of treatment discontinuation until platelet count < 30 × 109/L or bleeding events ≥ Grade II or use of any rescue therapy. If a patient did not loose sustained response the interval was censored with the date of the last platelet assessment.
    End point type
    Secondary
    End point timeframe
    from last dose of study treatment until loss of response, approx. 52 weeks
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    2
    2
    Units: Weeks
        arithmetic mean (standard deviation)
    49.1 ( 0.0 )
    45.7 ( 2.8 )
    No statistical analyses for this end point

    Secondary: Complete response by Week 4

    Close Top of page
    End point title
    Complete response by Week 4
    End point description
    Complete Response by week 4 is defined as platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy until week 4.
    End point type
    Secondary
    End point timeframe
    By Week 4
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Participants
    8
    9
    No statistical analyses for this end point

    Secondary: Overall response by Week 4

    Close Top of page
    End point title
    Overall response by Week 4
    End point description
    Overall response by week 4 is defined as platelet count ≥ 30 × 109/L and ≥ 2 fold increase of screening platelet count and absence of bleeding and no rescue therapy within the first 4 weeks
    End point type
    Secondary
    End point timeframe
    By Week 4
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Participants
    10
    11
    No statistical analyses for this end point

    Secondary: Absolute change in platelet count from pre-treatment/screening to baseline and to various time points

    Close Top of page
    End point title
    Absolute change in platelet count from pre-treatment/screening to baseline and to various time points
    End point description
    Absolute change in platelet count from pre-treatment or screening to baseline and to 1, 2, 4, 13, 27 and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate).
    End point type
    Secondary
    End point timeframe
    Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: G/L
    arithmetic mean (standard deviation)
        Week 1 (Baseline) (n = 11, 11)
    32.0 ( 44.3 )
    23.2 ( 48.0 )
        Week 2 (n = 11, 12)
    145.2 ( 151.9 )
    80.7 ( 68.8 )
        Week 4 (n = 11, 12)
    192.5 ( 165.8 )
    92.0 ( 68.7 )
        Week 13 (n = 10, 11)
    119.4 ( 115.9 )
    143.0 ( 88.5 )
        Week 27 (n = 9, 11)
    217.8 ( 126.5 )
    141.2 ( 94.7 )
        Week 53 (n = 8, 10)
    139.0 ( 88.0 )
    141.7 ( 61.4 )
    No statistical analyses for this end point

    Secondary: Relative change in platelet count from pre-treatment/screening to baseline and to various time points

    Close Top of page
    End point title
    Relative change in platelet count from pre-treatment/screening to baseline and to various time points
    End point description
    Relative change in platelet count from pre-treatment or screening to baseline and to 1, 2, 4, 13, 27, and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate).
    End point type
    Secondary
    End point timeframe
    Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Percentage change in platelet counts
    arithmetic mean (standard deviation)
        Week 1 (Baseline) (n = 10, 11)
    347.9 ( 486.8 )
    293.4 ( 418.4 )
        Week 2 (n = 10, 12)
    5795.5 ( 9672.4 )
    2028.2 ( 4382.0 )
        Week 4 (n = 10, 12)
    11400.9 ( 16875.2 )
    2865.4 ( 3805.4 )
        Week 13 (n = 9, 11)
    8050.6 ( 12277.8 )
    4725.7 ( 8418.2 )
        Week 27 (n = 8, 11)
    13667.9 ( 15717.1 )
    4381.1 ( 8195.2 )
        Week 53 (n = 7, 10)
    9392.3 ( 11476.0 )
    5612.1 ( 8300.7 )
    No statistical analyses for this end point

    Secondary: Time to overall response (TOR)

    Close Top of page
    End point title
    Time to overall response (TOR)
    End point description
    Time to overall response is defined as time from starting study treatment to time of achievement of overall response. Overall response is defined as a platelet count ≥ 30 × 10^9/L and ≥ 2 fold increase of baseline platelet count and absence of bleeding and no rescue therapy censored with the last visit date for patients not achieving overall response. Results of TOR are reported per Kaplan-Meier estimates.
    End point type
    Secondary
    End point timeframe
    Time from starting study treatment to achievement of complete response (up to 52 weeks)
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Weeks
        median (confidence interval 95%)
    1.1 (0.0 to 4.1)
    1.0 (0.1 to 1.3)
    No statistical analyses for this end point

    Secondary: Time to complete response

    Close Top of page
    End point title
    Time to complete response
    End point description
    Time to complete response is defined as time from starting study treatment to time of achievement of complete response. Complete response is defined as a platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy. Results of time to complete response are reported per Kaplan-Meier estimates.
    End point type
    Secondary
    End point timeframe
    Time from starting study treatment to achievement of complete response (up to 52 weeks)
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Weeks
        median (confidence interval 95%)
    1.1 (0.1 to 999)
    2.1 (0.6 to 999)
    No statistical analyses for this end point

    Secondary: Duration of overall response (OR) and complete response (CR)

    Close Top of page
    End point title
    Duration of overall response (OR) and complete response (CR)
    End point description
    Duration of overall or complete response is defined as time of achievement of overall or complete response (as defined above) until loss of overall or complete response. The duration of CR was calculated from the date of onset of CR until platelet count < 100 x 109/L, or bleeding events ≥ Grade II, or use of any rescue therapy, whatever was earlier. Results of duration of overall and complete response are reported per Kaplan-Meier estimates.
    End point type
    Secondary
    End point timeframe
    Achievement of overall or complete response until loss of response (up to 52 weeks)
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Weeks
    median (confidence interval 95%)
        Median duration of overall response
    8.3 (0.3 to 999)
    3.6 (1.1 to 999)
        Median duration of complete response
    9.7 (1.3 to 999)
    1.3 (0.9 to 6.1)
    No statistical analyses for this end point

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

    Close Top of page
    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) instrument is a 13-item validated tool used to measure an individual's level of fatigue during usual daily activities over the past 7 days. Items are scored on a 0-4 response scale (4=not at all to 0=very much) where the total possible score ranges from 0-52 (all items are summed up to create the total score); A score of less than 30 indicates severe fatigue. The higher scores represent better HRQoL.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: scores on a scale
    median (standard deviation)
        Change from Baseline (BL) to Week 2 (n = 10, 11)
    -4.2 ( 9.6 )
    -4.5 ( 6.9 )
        Change from BL to Week 3 (n = 9, 10)
    1.0 ( 4.4 )
    -2.9 ( 5.1 )
        Change from BL to Week 5 (n = 10, 10)
    1.2 ( 5.0 )
    -5.1 ( 6.9 )
        Change from BL to Week 13 (n = 8, 9)
    0.3 ( 8.3 )
    0.3 ( 2.8 )
        Change from BL to Week 27 (n = 8, 10)
    -3.6 ( 11.0 )
    -3.1 ( 7.7 )
        Change from BL to Week 53 (n = 6, 9)
    2.7 ( 5.6 )
    -3.4 ( 5.4 )
    No statistical analyses for this end point

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

    Close Top of page
    End point title
    Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire
    End point description
    SF36 questionnaire is a tool to measure health-related QoL. SF36 questionnaires (physical and mental score) were answered throughout the study and is a validated instrument with 36 questions to measure general physical and mental health status via assessment of 8 domains—Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health—over the past 4 weeks. The SF36 is scored using norm-based scoring procedures and scores ranging from 0-100; higher values indicate less impairment, a higher QoL. In addition to this SAP-planned scoring score, an alternative scoring for both the physical SF36 score and the mental SF36 were performed by QualityMetric (QM) Incorporated, an IQVIA business.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: scores on a scale
    arithmetic mean (standard deviation)
        CBL to Wk 2: Physical Score (PS) (n = 10, 11)
    1.9 ( 4.6 )
    -3.3 ( 6.6 )
        CBL to Week 3: PS (n = 8, 10)
    2.5 ( 5.8 )
    -1.4 ( 8.8 )
        CBL to Week 5: PS (n = 8, 9)
    1.5 ( 6.2 )
    -2.7 ( 10.5 )
        CBL to Week 13: PS (n = 7, 10)
    3.5 ( 9.9 )
    0.3 ( 8.0 )
        CBL to Week 27: PS (n = 7, 10)
    2.6 ( 14.7 )
    -0.9 ( 10.8 )
        CBL to Week 53: PS (n = 6, 10)
    8.1 ( 11.8 )
    0.3 ( 10.0 )
        CBL to Week 2: Mental Score (MS) (n = 10, 11)
    -0.8 ( 6.1 )
    -1.2 ( 7.6 )
        CBL to Week 3: MS (n = 8, 10)
    -2.0 ( 11.0 )
    -1.6 ( 9.7 )
        CBL to Week 5: MS (n = 8, 9)
    0.1 ( 7.3 )
    -1.7 ( 11.5 )
        CBL to Week 13: MS (n = 7, 10)
    3.3 ( 7.5 )
    4.1 ( 7.4 )
        CBL to Week 27: MS (n = 7, 10)
    -1.4 ( 4.9 )
    -2.7 ( 7.4 )
        CBL to Week 53: MS (n = 6, 10)
    2.1 ( 8.8 )
    0.6 ( 5.7 )
        CBL to Week 2: PS-QM (n = 11, 12)
    2.0 ( 4.1 )
    -3.3 ( 6.0 )
        CBL to Week 3: PS-QM (n = 11, 11)
    3.4 ( 6.4 )
    -1.4 ( 7.8 )
        CBL to Week 5: PS-QM (n = 9, 12)
    2.4 ( 6.0 )
    -2.7 ( 8.5 )
        CBL to Week 13: PS-QM (n = 10, 10)
    4.1 ( 8.5 )
    0.3 ( 7.6 )
        CBL to Week 27: PS-QM (n = 9, 11)
    3.6 ( 12.4 )
    -1.0 ( 9.7 )
        CBL to Week 53: PS-QM (n = 7, 10)
    6.2 ( 10.7 )
    0.2 ( 9.4 )
    No statistical analyses for this end point

    Secondary: Incidence and severity of bleeding events

    Close Top of page
    End point title
    Incidence and severity of bleeding events
    End point description
    Incidence and severity of bleeding assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Incidence of bleeding: participants had at least one bleeding event. Severity of bleeding: bleeding event is from grade 2 and higher
    End point type
    Secondary
    End point timeframe
    Baseline up to 52 weeks
    End point values
    Eltrombopag + Dexamethasone Dexamethasone
    Number of subjects analysed
    13
    13
    Units: Participants
        Incidence of Bleeding events
    10
    12
        Severity of Bleeding events: ≥ grade 2
    6
    2
        Severity of Bleeding events: ≥ grade 3
    2
    1
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Adverse events (AEs) are collected from first dose of study treatment until end of study treatment plus 30 days post treatment. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment, approx. 3 years.
    Adverse event reporting additional description
    Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    Dexamethasone
    Reporting group description
    Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.

    Reporting group title
    Eltrombopag + Dexamethasone
    Reporting group description
    Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.

    Serious adverse events
    Dexamethasone Eltrombopag + Dexamethasone
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 13 (38.46%)
    5 / 12 (41.67%)
         number of deaths (all causes)
    1
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    B-cell lymphoma
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myelodysplastic syndrome
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Prostate cancer
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Haematoma
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Myocardial infarction
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Aplastic anaemia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune thrombocytopenia
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 12 (16.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Vascular stent occlusion
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Oesophageal stenosis
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    2 / 13 (15.38%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Renal and urinary disorders
    Ureterolithiasis
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal colic
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Herpes zoster
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Dexamethasone Eltrombopag + Dexamethasone
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    13 / 13 (100.00%)
    11 / 12 (91.67%)
    Vascular disorders
    Hot flush
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Hypertension
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Hypotension
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Generalised oedema
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    General physical health deterioration
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Fatigue
         subjects affected / exposed
    3 / 13 (23.08%)
    2 / 12 (16.67%)
         occurrences all number
    4
    2
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Oedema peripheral
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Pyrexia
         subjects affected / exposed
    4 / 13 (30.77%)
    1 / 12 (8.33%)
         occurrences all number
    4
    1
    Performance status decreased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Pain
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Erectile dysfunction
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Hiccups
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Dyspnoea
         subjects affected / exposed
    2 / 13 (15.38%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Cough
         subjects affected / exposed
    2 / 13 (15.38%)
    2 / 12 (16.67%)
         occurrences all number
    2
    2
    Psychiatric disorders
    Initial insomnia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Insomnia
         subjects affected / exposed
    2 / 13 (15.38%)
    2 / 12 (16.67%)
         occurrences all number
    2
    3
    Nervousness
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Sleep disorder
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Nocturnal fear
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Restlessness
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Lipase increased
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Weight increased
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 13 (7.69%)
    2 / 12 (16.67%)
         occurrences all number
    1
    3
    Infusion related reaction
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Ligament sprain
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Rib fracture
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Transfusion reaction
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Cardiac disorders
    Arteriosclerosis coronary artery
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Cardiovascular disorder
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    2
    Coronary artery disease
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Tachycardia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Nervous system disorders
    Sciatica
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Restless legs syndrome
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Polyneuropathy
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Paraesthesia
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Headache
         subjects affected / exposed
    4 / 13 (30.77%)
    2 / 12 (16.67%)
         occurrences all number
    8
    6
    Dizziness postural
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Dizziness
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    5
    Ageusia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Taste disorder
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Tremor
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Febrile neutropenia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Anaemia
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Thrombocytosis
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Leukopenia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Leukocytosis
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Eye disorders
    Hypermetropia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Dry eye
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Conjunctival oedema
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Vision blurred
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Photopsia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    2
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Abdominal pain
         subjects affected / exposed
    0 / 13 (0.00%)
    3 / 12 (25.00%)
         occurrences all number
    0
    3
    Constipation
         subjects affected / exposed
    0 / 13 (0.00%)
    3 / 12 (25.00%)
         occurrences all number
    0
    3
    Loose tooth
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Irritable bowel syndrome
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Hiatus hernia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    2 / 13 (15.38%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Diarrhoea
         subjects affected / exposed
    3 / 13 (23.08%)
    3 / 12 (25.00%)
         occurrences all number
    3
    3
    Nausea
         subjects affected / exposed
    1 / 13 (7.69%)
    2 / 12 (16.67%)
         occurrences all number
    1
    3
    Vomiting
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Hepatobiliary disorders
    Hepatotoxicity
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    2 / 13 (15.38%)
    2 / 12 (16.67%)
         occurrences all number
    2
    2
    Skin exfoliation
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    3 / 13 (23.08%)
    1 / 12 (8.33%)
         occurrences all number
    3
    1
    Pruritus
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    Dry skin
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Dermatitis atopic
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Endocrine disorders
    Hyperthyroidism
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Musculoskeletal pain
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Muscle spasms
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Joint swelling
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Coccydynia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Bone pain
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Pain in extremity
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Arthralgia
         subjects affected / exposed
    1 / 13 (7.69%)
    2 / 12 (16.67%)
         occurrences all number
    1
    7
    Myalgia
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Neck pain
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Back pain
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Plantar fascial fibromatosis
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Infection
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Helicobacter infection
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Gastroenteritis viral
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    COVID-19
         subjects affected / exposed
    4 / 13 (30.77%)
    3 / 12 (25.00%)
         occurrences all number
    4
    3
    Bronchitis
         subjects affected / exposed
    2 / 13 (15.38%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Rhinitis
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Sinusitis
         subjects affected / exposed
    2 / 13 (15.38%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Pneumonia fungal
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 13 (7.69%)
    2 / 12 (16.67%)
         occurrences all number
    4
    2
    Keratitis viral
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Influenza
         subjects affected / exposed
    2 / 13 (15.38%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Respiratory tract infection
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Diabetes mellitus
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Dyslipidaemia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Hypokalaemia
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Steroid diabetes
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Iron deficiency
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Jul 2020
    To address the requirement of the Ethics committee to include a more detailed information on the recruitment procedure in the protocol. Version 00 of this protocol was approved by end of March 2020 by the German health authority (Bundesamt für Arzneimittel und Medizinprodukte, BfArM) and by the Ethics Committee Halle-Wittenberg. As feedback, inclusion of recruitment procedures was requested by the Ethics Committee, which was covered by this amendment. In addition, other clarifications and administrative changes were included as needed.
    27 Nov 2020
    Due to slow enrollment, it was decided to relax inclusion criteria to one single assessment of thrombocyte count at the time of screening and omitting the second threshold check at baseline. This was due to the fact that for some patients the thrombocyte count was below the pre-specified threshold at screening, but above at baseline and after pretreatment. It was decided to also include the screening instead of baseline thrombocyte count as a cofactor into the statistical model for the primary outcome analysis, as this was regarded as the more adequate way of taking into account the initial value at the time of diagnosis and before pre-treatment for all patients. Additional sensitivity analyses were included to assess the impact of pre-treatment and baseline thrombocyte count.
    28 Jul 2022
    Due to slow enrollment the feasibility of this trial has been considered and led to the following changes: Premature termination of recruitment due to feasibility reasons, resulting in an expected sample size of ca. 24 patients at the time of approval by the EC/HA. A scenario based on 12 patients per group, assuming the above rates of 30 versus 65%, would allow for a power of 24% to analyze the primary endpoint. Consequently, all analyses were to be interpreted in a purely descriptive manner; Reduction of the follow-up for responders after Week 52 (secondary endpoint). Assuming that 65% of the patients in the combination arm were in sustained response off treatment at Week 52 and in respect to slow enrollment, the sample size for this secondary endpoint would be insufficient to detect a meaningful result; Based on the proposed changes to reduce the follow-up of the responders it was possible to share the study data 6 months earlier to increase the benefits of future patients and support recent updates in ITP guidelines to prevent an extended and recurrent use of corticosteroids that is associated with substantial toxicity.

    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.
    Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd. com/ for complete trial results Summary
    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-Fri May 09 20:36:53 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA