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    Clinical Trial Results:
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Persistent/Chronic Primary Immune Thrombocytopenia

    Summary
    EudraCT number
    		 2019-004103-12
    Trial protocol
    		 DE   SI   BG   HR   IT   ES   GR  
    Global end of trial date
    29 Apr 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 May 2025
    First version publication date
    09 May 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    TAK-079-1004
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04278924
    WHO universal trial number (UTN)
    		 U1111-1245-3760
    Sponsors
    Sponsor organisation name
    Takeda
    Sponsor organisation address
    95 Hayden Avenue, Lexington, MA, United States, 02421
    Public contact
    Study Director, Takeda, TrialDisclosures@takeda.com
    Scientific contact
    Study Director, Takeda, TrialDisclosures@takeda.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
    29 Apr 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    29 Apr 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this trial was to evaluate the safety and tolerability of TAK-079 in participants with persistent/chronic primary immune thrombocytopenia (ITP).
    Protection of trial subjects
    All study participants were required to read and sign an informed consent form (ICF).
    Background therapy
    		 NA
    Evidence for comparator
    		 NA
    Actual start date of recruitment
    09 Nov 2020
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 4 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Bulgaria: 12
    Country: Number of subjects enrolled
    China: 2
    Country: Number of subjects enrolled
    Croatia: 3
    Country: Number of subjects enrolled
    Greece: 4
    Country: Number of subjects enrolled
    Italy: 8
    Country: Number of subjects enrolled
    Japan: 1
    Country: Number of subjects enrolled
    Slovenia: 3
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    Ukraine: 4
    Worldwide total number of subjects
    41
    EEA total number of subjects
    34
    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)
    36
    From 65 to 84 years
    4
    85 years and over
    1

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Participants took part in the study at 24 investigative sites globally from 09 November 2020 to 29 April 2024.

    Pre-assignment
    Screening details
    		 Participants who had persistent/chronic primary immune thrombocytopenia (ITP) were randomized to receive either mezagitamab (TAK-079) or matching placebo in Part A or Part B of this study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part A & B: Double Blind Period: Placebo
    Arm description
    		 Participants received TAK-079 placebo-matching injection SC, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Placebo-assigned participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received TAK-079 placebo-matching injection SC, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Placebo-assigned participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Arm title
    		 Part A: Double Blind Period: TAK-079 100 mg
    Arm description
    		 Participants received TAK-079 100 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.
    Arm type
    		 Experimental

    Investigational medicinal product name
    TAK-079
    Investigational medicinal product code
    TAK-079
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received TAK-079 100 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Arm title
    		 Part A: Double Blind Period: TAK-079 300 mg
    Arm description
    		 Participants received TAK-079 300 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.
    Arm type
    		 Experimental

    Investigational medicinal product name
    TAK-079
    Investigational medicinal product code
    TAK-079
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received TAK-079 300 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Arm title
    		 Part B: Double Blind Period: TAK-079 600 mg
    Arm description
    		 Participants received TAK-079 600 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.
    Arm type
    		 Experimental

    Investigational medicinal product name
    TAK-079
    Investigational medicinal product code
    TAK-079
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received TAK-079 600 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Number of subjects in period 1
    		Part A & B: Double Blind Period: Placebo Part A: Double Blind Period: TAK-079 100 mg Part A: Double Blind Period: TAK-079 300 mg Part B: Double Blind Period: TAK-079 600 mg
    Started
    13
    9
    8
    11
    Completed
    12
    7
    8
    9
    Not completed
    1
    2
    0
    2
         Consent withdrawn by subject
    1
    1
    -
    2
         Reason Not Specified
    -
    1
    -
    -
    Period 2
    Period 2 title
    Open-label Extension Period
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part A: Open-label Extension (OLE) Period: TAK-079 100 mg
    Arm description
    		 Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 100 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    TAK-079
    Investigational medicinal product code
    TAK-079
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 100 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Arm title
    		 Part A: OLE Period: TAK-079 300 mg
    Arm description
    		 Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 300 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    TAK-079
    Investigational medicinal product code
    TAK-079
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 300 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Arm title
    		 Part B: OLE Period: TAK-079 600 mg
    Arm description
    		 Participants who received placebo in double-blind Part B and opted to receive treatment with TAK-079 received TAK-079 600 mg, SC injection, QW for 8 weeks in OLE Period of Part B. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    TAK-079
    Investigational medicinal product code
    TAK-079
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants who received placebo in double-blind Part B and opted to receive treatment with TAK-079 received TAK-079 600 mg, SC injection, QW for 8 weeks in OLE Period of Part B. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Number of subjects in period 2 [1]
    		Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part A: OLE Period: TAK-079 300 mg Part B: OLE Period: TAK-079 600 mg
    Started
    4
    4
    4
    Completed
    4
    4
    4
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Only participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 in Open-label Extension (OLE) Period.

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Part A & B: Double Blind Period: Placebo
    Reporting group description
    		 Participants received TAK-079 placebo-matching injection SC, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Placebo-assigned participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part A: Double Blind Period: TAK-079 100 mg
    Reporting group description
    		 Participants received TAK-079 100 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part A: Double Blind Period: TAK-079 300 mg
    Reporting group description
    		 Participants received TAK-079 300 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part B: Double Blind Period: TAK-079 600 mg
    Reporting group description
    		 Participants received TAK-079 600 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group values
    		 Part A & B: Double Blind Period: Placebo Part A: Double Blind Period: TAK-079 100 mg Part A: Double Blind Period: TAK-079 300 mg Part B: Double Blind Period: TAK-079 600 mg Total
    Number of subjects
    		 13 9 8 11
    Age Categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 38.8 ( 15.86 ) 49.0 ( 14.45 ) 52.3 ( 16.59 ) 48.4 ( 19.68 ) -
    Gender categorical
    Units: Subjects
        Female
    		 9 5 5 9 28
        Male
    		 4 4 3 2 13
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 1 0 0 0 1
        Not Hispanic or Latino
    		 12 9 8 9 38
        Unknown or Not Reported
    		 0 0 0 2 2
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0
        Asian
    		 1 0 0 2 3
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0
        Black or African American
    		 0 0 0 0 0
        White
    		 11 9 8 9 37
        More than one race
    		 0 0 0 0 0
        Unknown or Not Reported
    		 1 0 0 0 1

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Part A & B: Double Blind Period: Placebo
    Reporting group description
    		 Participants received TAK-079 placebo-matching injection SC, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Placebo-assigned participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part A: Double Blind Period: TAK-079 100 mg
    Reporting group description
    		 Participants received TAK-079 100 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part A: Double Blind Period: TAK-079 300 mg
    Reporting group description
    		 Participants received TAK-079 300 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part B: Double Blind Period: TAK-079 600 mg
    Reporting group description
    		 Participants received TAK-079 600 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.
    Reporting group title
    Part A: Open-label Extension (OLE) Period: TAK-079 100 mg
    Reporting group description
    		 Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 100 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Reporting group title
    Part A: OLE Period: TAK-079 300 mg
    Reporting group description
    		 Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 300 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Reporting group title
    Part B: OLE Period: TAK-079 600 mg
    Reporting group description
    		 Participants who received placebo in double-blind Part B and opted to receive treatment with TAK-079 received TAK-079 600 mg, SC injection, QW for 8 weeks in OLE Period of Part B. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Primary: Percentage of Participants with at Least One Grade 3 or Higher Treatment Emergent Adverse Event (TEAE), Treatment Emergent Serious Adverse Event (SAE), and TEAEs Leading to TAK-079 Discontinuation

    		 Close Top of page
    End point title
    		 Percentage of Participants with at Least One Grade 3 or Higher Treatment Emergent Adverse Event (TEAE), Treatment Emergent Serious Adverse Event (SAE), and TEAEs Leading to TAK-079 Discontinuation [1]
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with the treatment. SAE means any untoward medical occurrence that at any dose: a) results in death; b) is life-threatening; c) requires inpatient hospitalization or prolongation of an existing hospitalization; d) results in persistent or significant disability or incapacity; e) is a congenital anomaly/birth defect; f) is a medically important event. TEAEs were defined as an AE having a start date and time equal to or later than the start date and time of the first dose of investigational medicinal product (IMP). Percentages were rounded off to the nearest single decimal place.
    End point type
    Primary
    End point timeframe
    Up to Week 32 in each Period of the study
    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: Only descriptive analyses were planned for this endpoint.
    End point values
    		 Part A & B: Double Blind Period: Placebo Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part A: Double Blind Period: TAK-079 100 mg Part A: OLE Period: TAK-079 300 mg Part A: Double Blind Period: TAK-079 300 mg Part B: OLE Period: TAK-079 600 mg Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects analysed
    13
    4
    9
    4
    8
    4
    11
    Units: percentage of participants
    number (not applicable)
        Grade 3 or Higher TEAE
    23.1
    0
    22.2
    0
    0
    25.0
    27.3
        Treatment Emergent SAE
    7.7
    25.0
    22.2
    0
    0
    0
    18.2
        TEAEs Leading to TAK-079 Discontinuation
    0
    25.0
    22.2
    0
    0
    0
    18.2
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Platelet Response at Weeks 16 and 32

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    End point title
    		 Percentage of Participants with Platelet Response at Weeks 16 and 32
    End point description
    Platelet response is defined as a platelet count ≥50,000/microliter (μL) and ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
    End point type
    Secondary
    End point timeframe
    At Weeks 16 and 32
    End point values
    		 Part A & B: Double Blind Period: Placebo Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part A: Double Blind Period: TAK-079 100 mg Part A: OLE Period: TAK-079 300 mg Part A: Double Blind Period: TAK-079 300 mg Part B: OLE Period: TAK-079 600 mg Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects analysed
    13
    4
    9
    4
    8
    4
    11
    Units: percentage of participants
    number (confidence interval 95%)
        Week 16 (n=13, 4, 9, 4, 8, 4, 11)
    23.08 (5.04 to 53.81)
    50.00 (6.76 to 93.24)
    66.67 (29.93 to 92.51)
    50.00 (6.76 to 93.24)
    62.50 (24.49 to 91.48)
    25.00 (0.63 to 80.59)
    90.91 (58.72 to 99.77)
        Week 32 (n=0, 4, 9, 4, 8, 4, 11)
    999 (999 to 999)
    50.00 (6.76 to 93.24)
    66.67 (29.93 to 92.51)
    50.00 (6.76 to 93.24)
    62.50 (24.49 to 91.48)
    25.00 (0.63 to 80.59)
    90.91 (58.72 to 99.77)
    Statistical analysis title
    		 Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 100 mg
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.056
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference
    Point estimate
    43.59
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.81
         upper limit
    		 73.35
    Statistical analysis title
    		 Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.001
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    67.83
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 29.21
         upper limit
    		 87.29
    Statistical analysis title
    		 Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 300 mg
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.088
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    39.42
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -4.24
         upper limit
    		 71.38

    Secondary: Percentage of Participants with Complete Platelet Response at Weeks 16 and 32

    		 Close Top of page
    End point title
    		 Percentage of Participants with Complete Platelet Response at Weeks 16 and 32
    End point description
    Complete platelet response is defined as a platelet count ≥100,000/μL on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
    End point type
    Secondary
    End point timeframe
    At Weeks 16 and 32
    End point values
    		 Part A & B: Double Blind Period: Placebo Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part A: Double Blind Period: TAK-079 100 mg Part A: OLE Period: TAK-079 300 mg Part A: Double Blind Period: TAK-079 300 mg Part B: OLE Period: TAK-079 600 mg Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects analysed
    13
    4
    9
    4
    8
    4
    11
    Units: percentage of participants
    number (confidence interval 95%)
        Week 16 (n=13, 4, 9, 4, 8, 4, 11)
    0 (0.00 to 24.71)
    0 (0.00 to 60.24)
    55.56 (21.20 to 86.30)
    25.00 (0.63 to 80.59)
    50.00 (15.70 to 84.30)
    25.00 (0.63 to 80.59)
    81.82 (48.22 to 97.72)
        Week 32 (n=0, 4, 9, 4, 8, 4, 11)
    999 (999 to 999)
    25.00 (0.63 to 80.59)
    55.56 (21.20 to 86.30)
    25.00 (0.63 to 80.59)
    50.00 (15.70 to 84.30)
    25.00 (0.63 to 80.59)
    81.82 (48.22 to 97.72)
    Statistical analysis title
    		 Complete Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 100 mg
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.001
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    55.56
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 25.11
         upper limit
    		 81.51
    Statistical analysis title
    		 Complete Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    81.82
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 51.24
         upper limit
    		 94.99
    Statistical analysis title
    		 Complete Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 300 mg
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.004
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    50
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 19.63
         upper limit
    		 78.95

    Secondary: Percentage of Participants with Clinically Meaningful Platelet Response at Weeks 16 and 32

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    End point title
    		 Percentage of Participants with Clinically Meaningful Platelet Response at Weeks 16 and 32
    End point description
    A clinically meaningful platelet response is defined as a platelet count ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
    End point type
    Secondary
    End point timeframe
    At Weeks 16 and 32
    End point values
    		 Part A & B: Double Blind Period: Placebo Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part A: Double Blind Period: TAK-079 100 mg Part A: OLE Period: TAK-079 300 mg Part A: Double Blind Period: TAK-079 300 mg Part B: OLE Period: TAK-079 600 mg Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects analysed
    13
    4
    9
    4
    8
    4
    11
    Units: percentage of participants
    number (confidence interval 95%)
        Week 16 (n=13, 4, 9, 4, 8, 4, 11)
    30.77 (9.09 to 61.43)
    75.00 (19.41 to 99.37)
    66.67 (29.93 to 92.51)
    50.00 (6.76 to 93.24)
    75.00 (34.91 to 96.81)
    25.00 (0.63 to 80.59)
    90.91 (58.72 to 99.77)
        Week 32 (n=0, 4, 9, 4, 8, 4, 11)
    999 (999 to 999)
    75.00 (19.41 to 99.37)
    66.67 (29.93 to 92.51)
    50.00 (6.76 to 93.24)
    75.00 (34.91 to 96.81)
    25.00 (0.63 to 80.59)
    90.91 (58.72 to 99.77)
    Statistical analysis title
    		 Clinically Meaningful Platelet Response
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 100 mg
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.12
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    35.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.22
         upper limit
    		 67.75
    Statistical analysis title
    		 Clinically Meaningful Platelet Response
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.003
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    60.14
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 21.33
         upper limit
    		 82.42
    Statistical analysis title
    		 Clinically Meaningful Platelet Response
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 300 mg
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.06
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    44.23
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.83
         upper limit
    		 73.77

    Secondary: Percentage of Participants with Hemostatic Platelet Response at Weeks 16 and 32

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    End point title
    		 Percentage of Participants with Hemostatic Platelet Response at Weeks 16 and 32
    End point description
    A hemostatic platelet response is defined for participants with a baseline platelet count of <15,000/μL who achieved a platelet count of ≥30,000/μL and ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
    End point type
    Secondary
    End point timeframe
    At Weeks 16 and 32
    End point values
    		 Part A & B: Double Blind Period: Placebo Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part A: Double Blind Period: TAK-079 100 mg Part A: OLE Period: TAK-079 300 mg Part A: Double Blind Period: TAK-079 300 mg Part B: OLE Period: TAK-079 600 mg Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects analysed
    5
    2
    5
    1
    4
    3
    4
    Units: percentage of participants
    number (confidence interval 95%)
        Week 16 (n=5, 2, 5, 1, 4, 3, 4)
    0 (0.00 to 52.18)
    100.00 (15.81 to 100.00)
    40.00 (5.27 to 85.34)
    100.00 (2.50 to 100.00)
    25.00 (0.63 to 80.59)
    0 (0.00 to 70.76)
    100.00 (39.76 to 100.00)
        Week 32 (n=0, 2, 5, 1, 4, 3, 4)
    999 (999 to 999)
    100.00 (15.81 to 100.00)
    40.00 (5.27 to 85.34)
    100.00 (2.50 to 100.00)
    25.00 (0.63 to 80.59)
    0 (0.00 to 70.76)
    100.00 (39.76 to 100.00)
    Statistical analysis title
    		 Hemostatic Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 100 mg
    Number of subjects included in analysis
    10
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.187
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    40
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -16.28
         upper limit
    		 78.17
    Statistical analysis title
    		 Hemostatic Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part B: Double Blind Period: TAK-079 600 mg
    Number of subjects included in analysis
    9
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.004
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    100
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 35.12
         upper limit
    		 100
    Statistical analysis title
    		 Hemostatic Platelet Response at Weeks 16 and 32
    Comparison groups
    Part A & B: Double Blind Period: Placebo v Part A: Double Blind Period: TAK-079 300 mg
    Number of subjects included in analysis
    9
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.315
    Method
    		 Barnard's test
    Parameter type
    		 Risk difference (RD)
    Point estimate
    25
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -28.85
         upper limit
    		 71.78

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to Week 32 in each Period of the study
    Adverse event reporting additional description
    The Safety Analysis Set included all participants who received at least 1 dose of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    Part A: Double Blind Period: TAK-079 300 mg
    Reporting group description
    		 Participants received TAK-079 300 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part A: Double Blind Period: TAK-079 100 mg
    Reporting group description
    		 Participants received TAK-079 100 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part A & B: Double Blind Period: Placebo
    Reporting group description
    		 Participants received TAK-079 placebo-matching injection SC, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Placebo-assigned participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Reporting group title
    Part A: OLE Period: TAK-079 300 mg
    Reporting group description
    		 Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 300 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Reporting group title
    Part A: Open-label Extension (OLE) Period: TAK-079 100 mg
    Reporting group description
    		 Participants who received placebo in double-blind Part A and SFP and opted to receive treatment with TAK-079 were randomized to receive TAK-079 100 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Reporting group title
    Part B: OLE Period: TAK-079 600 mg
    Reporting group description
    		 Participants who received placebo in double-blind Part B and opted to receive treatment with TAK-079 received TAK-079 600 mg, SC injection, QW for 8 weeks in OLE Period of Part B. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.

    Reporting group title
    Part B: Double Blind Period: TAK-079 600 mg
    Reporting group description
    		 Participants received TAK-079 600 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32.

    Serious adverse events
    		 Part A: Double Blind Period: TAK-079 300 mg Part A: Double Blind Period: TAK-079 100 mg Part A & B: Double Blind Period: Placebo Part A: OLE Period: TAK-079 300 mg Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part B: OLE Period: TAK-079 600 mg Part B: Double Blind Period: TAK-079 600 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 9 (22.22%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    2 / 11 (18.18%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    0
    Blood and lymphatic system disorders
    Immune thrombocytopenia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Conjunctivitis allergic
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Haemorrhagic ovarian cyst
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pyelonephritis chronic
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Staphylococcal bacteraemia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Part A: Double Blind Period: TAK-079 300 mg Part A: Double Blind Period: TAK-079 100 mg Part A & B: Double Blind Period: Placebo Part A: OLE Period: TAK-079 300 mg Part A: Open-label Extension (OLE) Period: TAK-079 100 mg Part B: OLE Period: TAK-079 600 mg Part B: Double Blind Period: TAK-079 600 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 8 (62.50%)
    7 / 9 (77.78%)
    9 / 13 (69.23%)
    4 / 4 (100.00%)
    2 / 4 (50.00%)
    2 / 4 (50.00%)
    6 / 11 (54.55%)
    Vascular disorders
    Aortic aneurysm
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Essential hypertension
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    3
    0
    0
    0
    0
    Thrombophlebitis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Hypertension
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Hypotension
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Haematoma
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    0
    General disorders and administration site conditions
    Administration site haematoma
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Asthenia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    2 / 13 (15.38%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    1
    0
    Chills
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Feeling cold
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Influenza like illness
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    2
    Injection site reaction
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Injection site bruising
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    5
    0
    0
    0
    0
    Injection site haematoma
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 9 (0.00%)
    3 / 13 (23.08%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    3
    0
    0
    0
    0
    Reproductive system and breast disorders
    Heavy menstrual bleeding
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Productive cough
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    Pharyngeal inflammation
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    1
    Epistaxis
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    0
    Dyspnoea
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Confusional state
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Investigations
    Electrocardiogram abnormal
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Blood urine present
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Blood bilirubin increased
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Contusion
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    2 / 13 (15.38%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    1
    0
    Traumatic haematoma
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Post procedural haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    Joint injury
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Injection related reaction
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Nervous system disorders
    Vocal cord paralysis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Post-traumatic headache
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Nystagmus
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Headache
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Dizziness
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    0
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Anaemia
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    0
    Thrombocytopenia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    2
    Leukocytosis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    3
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Eye disorders
    Conjunctival haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    2 / 13 (15.38%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    2
    0
    1
    0
    0
    Myopia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    1
    0
    0
    0
    Optic neuropathy
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Gingival bleeding
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    0
    Anal fissure
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Abdominal pain upper
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    0
    0
    0
    0
    0
    Abdominal pain lower
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Abdominal pain
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    0
    Glossitis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    1
    Vomiting
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    0
    Haematochezia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Mouth haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Ecchymosis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    1
    0
    0
    0
    Eczema
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Urticaria
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Pruritus
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Petechiae
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    2 / 13 (15.38%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    5
    0
    0
    0
    0
    Renal and urinary disorders
    Urinary retention
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Osteoarthritis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    0
    Myalgia
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
    1 / 13 (7.69%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    1
    1
    1
    0
    0
    0
    Exostosis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Polyarthritis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Injection site cellulitis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Influenza
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    COVID-19
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 9 (22.22%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    2
    1
    0
    1
    0
    1
    Bacteriuria
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Hyperuricaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Diabetes mellitus
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    0
    Iron deficiency
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Oct 2020
    The following changes were made as per Amendment 02: 1. Allowed participants to receive predefined rescue therapies during the dosing period without automatic discontinuation from IMP dosing and advancement to the SFP. 2. Enhanced access to the OLE for placebo participants who receive rescue therapies during the study. 3. Add contingency plans for the coronavirus disease 2019 (COVID-19) pandemic by incorporating flexibility for study participants, investigators, and study-site monitors while continuing to maintain participant safety and study integrity as per local site regulations.
    18 Dec 2020
    The following changes were made as per Amendment 03: 1. Revised timing of sample collection for the ITP Bleeding Scores to decrease the routine frequency of administrations of the test for participant comfort. 2. Revised the procedure for occult blood samples to provide more detailed description of the ITP Bleeding Score, including the collection of samples as part of the assessment.
    05 May 2021
    The following change was made as per Amendment 04: 1. Changed the legal entity name for the sponsor to Takeda Development Center Americas, Inc.
    28 Apr 2022
    The following changes were made as per Amendment 05: 1. Updated the number of participants enrolled in each study part to expedite the safety review of Part A. 2. Updated exclusion criteria to also exclude participants with significant ocular medical conditions and participants in vaccine studies. 3. Added urticaria, fever, and blurred vision to the list of hypersensitivity symptoms to ensure that these symptoms were examined by the investigator in determining any potential hypersensitivity reaction. 4. Modified the prophylactic coadministration regimens for each dose of mezagitamab, to prevent possible infusion related reactions and provide an option for updating guidance to sites based on emerging data. 5. Added that an equivalent of tocilizumab may be used for symptomatic treatment of cytokine release syndrome (CRS) to allow investigators flexibility in treatment and support of study participants.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

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