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    Clinical Trial Results:
    A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia followed by an Open-Label Treatment Period

    Summary
    EudraCT number
    		 2016-003038-26
    Trial protocol
    		 GB   HU   CZ   BE   DE   AT   ES  
    Global end of trial date
    09 Apr 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 May 2020
    First version publication date
    03 May 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    ARGX-113-1603
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03102593
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    argenx BVBA
    Sponsor organisation address
    Industriepark-Zwijnaarde 7, Zwijnaarde, Belgium, 9052
    Public contact
    Regulatory Manager, argenx BVBA, +32 93103400, regulatory@argenx.com
    Scientific contact
    Regulatory Manager, argenx BVBA, +32 93103400, regulatory@argenx.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
    09 Apr 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Apr 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of ARGX-113.
    Protection of trial subjects
    This study was conducted, and the informed consent was obtained according to the ethical principles stated in the Declaration of Helsinki (latest version), the applicable guidelines for Good Clinical Practice, or the applicable drug and data protection laws and regulations of the countries where the study was conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Mar 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Poland: 2
    Country: Number of subjects enrolled
    Spain: 6
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Czech Republic: 3
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Hungary: 14
    Country: Number of subjects enrolled
    Ukraine: 8
    Worldwide total number of subjects
    38
    EEA total number of subjects
    30
    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)
    34
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 From 30 March 2017, 38 patients with Primary Immune Thrombocytopenia (ITP) were randomized in 19 study centers in Ukraine and Europe into a double-blind main study. Eligible patients continued into an optional open-label treatment period. The last patient last visit was 09 April 2019.

    Pre-assignment
    Screening details
    		 Patients were randomized into the main study in a 1:1:1 ratio to receive either ARGX-113 at 5 or 10 milligram/kilogram (mg/kg) body weight or placebo, in addition to Standard of Care (SoC). During screening, average platelet counts had to be <30×10^9/Liter (L) with no single reading >35×10^9/L, measured on 2 separate occasions at least 1 day apart.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 ARGX-113 5 mg/kg
    Arm description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received ARGX-113 at a dose of 5 mg/kg in 4 intravenous (IV) infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ARGX-113
    Investigational medicinal product code
    Other name
    Efgartigimod
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    ARGX-113 was supplied as 20 mg/millilters (mL) sterile, colorless, and clear concentrate solution for IV administration, administered in total volume of 250 mL over a period of 2 hours.

    Arm title
    		 ARGX-113 10 mg/kg
    Arm description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received ARGX-113 at a dose of 10 mg/kg in 4 IV infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ARGX-113
    Investigational medicinal product code
    Other name
    Efgartigimod
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    ARGX-113 was supplied as 20 mg/mL sterile, colorless, and clear concentrate solution for IV administration, administered in total volume of 250 mL over a period of 2 hours.

    Arm title
    		 Placebo
    Arm description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received matching placebo in 4 IV infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    A matching placebo was supplied as a sterile, colorless, and clear concentrate solution for IV administration, administered in total volume of 250 mL over a period of 2 hours.

    Number of subjects in period 1
    		ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Started
    13
    13
    12
    Entered Main Study Extended Follow-up
    6 [1]
    6 [2]
    2 [3]
    Completed
    13
    11
    8
    Not completed
    0
    2
    4
         Consent withdrawn by subject
    -
    1
    1
         Unspecified
    -
    1
    -
         Lack of efficacy
    -
    -
    3
    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: Patients classed as completing the study had completed up to visit 16 (treatment period plus 8 weeks follow-up). The optional extended follow-up period began after completion of the main study.
    [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: Patients classed as completing the study had completed up to visit 16 (treatment period plus 8 weeks follow-up). The optional extended follow-up period began after completion of the main study.)
    [3] - 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: Patients classed as completing the study had completed up to visit 16 (treatment period plus 8 weeks follow-up). The optional extended follow-up period began after completion of the main study.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    ARGX-113 5 mg/kg
    Reporting group description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received ARGX-113 at a dose of 5 mg/kg in 4 intravenous (IV) infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.

    Reporting group title
    ARGX-113 10 mg/kg
    Reporting group description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received ARGX-113 at a dose of 10 mg/kg in 4 IV infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.

    Reporting group title
    Placebo
    Reporting group description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received matching placebo in 4 IV infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.

    Reporting group values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo Total
    Number of subjects
    		 13 13 12 38
    Age categorical
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0
        Newborns (0-27 days)
    		 0
        Infants and toddlers (28 days-23 months)
    		 0
        Children (2-11 years)
    		 0
        Adolescents (12-17 years)
    		 0
        Adults (18-64 years)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 45.7 ( 18.41 ) 45.5 ( 12.75 ) 41.8 ( 16.41 ) -
    Gender categorical
    Units: Subjects
        Female
    		 9 4 7 20
        Male
    		 4 9 5 18
    Race
    Units: Subjects
        White
    		 12 13 11 36
        Not Reported
    		 1 0 1 2
    Platelet Count Level at Baseline
    Units: Subjects
        Baseline Platelet Count Level <15 x 10^9/L
    		 7 7 6 20
        Baseline Platelet Count Level >=15 x 10^9/L
    		 6 6 6 18
    Patients with Previous ITP Therapy
    Patients who had received at least 1 ITP therapy, either previously received or ongoing at Baseline.
    Units: Subjects
        Patients with previous ITP therapy
    		 13 12 12 37
        Patients with no previous ITP therapy
    		 0 1 0 1
    ITP Therapy Ongoing at Baseline
    Patients who had received at least 1 ITP therapy which was ongoing at Baseline.
    Units: Subjects
        ITP therapy ongoing at Baseline
    		 11 8 8 27
        ITP therapy not ongoing at Baseline
    		 2 5 4 11
    Body Weight
    Units: kg
        arithmetic mean (standard deviation)
    		 80.32 ( 15.591 ) 86.96 ( 22.280 ) 78.73 ( 18.811 ) -
    Duration of ITP Prior to Screening
    Units: years
        median (full range (min-max))
    		 4.46 (0.1 to 34.2) 5.42 (0.7 to 28.7) 3.51 (0.3 to 47.8) -
    Number of Unique ITP Therapies
    Median number of unique ITP therapies, either previously received or ongoing at Baseline.
    Units: Unique ITP therapies
        median (full range (min-max))
    		 2.00 (1.0 to 8.0) 1.00 (0.0 to 10.0) 2.00 (1.0 to 7.0) -

    End points

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    End points reporting groups
    Reporting group title
    ARGX-113 5 mg/kg
    Reporting group description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received ARGX-113 at a dose of 5 mg/kg in 4 intravenous (IV) infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.

    Reporting group title
    ARGX-113 10 mg/kg
    Reporting group description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received ARGX-113 at a dose of 10 mg/kg in 4 IV infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.

    Reporting group title
    Placebo
    Reporting group description
    		 The main study included a 2-week screening, a 3-week treatment period (visit 1 through visit 7), and an 8-week follow-up period (visit 8 through visit 16). Patients received matching placebo in 4 IV infusions, administered 1 week apart, in addition to SoC. Patients who completed the initial 8-week follow-up and did not receive any rescue treatment were given the option of an extended follow-up period, up to maximum 13 weeks after visit 16.

    Subject analysis set title
    ARGX-113 5 mg/kg
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients received 5 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Safety analysis set included all patients who had received any portion of study drug.

    Subject analysis set title
    ARGX-113 10 mg/kg
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients received 10 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Safety analysis set included all patients who had received any portion of study drug.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients received placebo in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16 Safety analysis set included all patients who had received any portion of study drug.

    Subject analysis set title
    ARGX-113 5 mg/kg
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients received 5 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Safety analysis set included all patients who had received any portion of study drug.

    Subject analysis set title
    ARGX-113 10 mg/kg
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients received 10 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Safety analysis set included all patients who had received any portion of study drug.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Patients received placebo in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Safety analysis set included all patients who had received any portion of study drug.

    Subject analysis set title
    ARGX-113 5 mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients received 5 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Full analysis set included all randomized patients with at least 1 post-Baseline primary efficacy observation (platelet count results)

    Subject analysis set title
    ARGX-113 10 mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients received 10 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Full analysis set included all randomized patients with at least 1 post-Baseline primary efficacy observation (platelet count results).

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients received placebo in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16. Full analysis set included all randomized patients with at least 1 post-Baseline primary efficacy observation (platelet count results).

    Subject analysis set title
    ARGX-113 5 mg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Patients received 5 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16.

    Subject analysis set title
    ARGX-113 10 mg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Patients received 10 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16.

    Subject analysis set title
    Pooled ARGX-113
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients received either 5 or 10 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients received placebo in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16.

    Primary: Number of Patients With Treatment Emergent Adverse Events (TEAES) and Treatment Emergent Serious Adverse Events (SAEs)

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    End point title
    		 Number of Patients With Treatment Emergent Adverse Events (TEAES) and Treatment Emergent Serious Adverse Events (SAEs) [1]
    End point description
    TEAEs were defined as undesirable events not present prior to medical treatment, or already present events that worsened either in intensity or frequency following the treatment. A SAE was any untoward medical occurrence that: resulted in death; was life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability or incapacity; was a congenital abnormality or birth defect; or other medically significant events. All TEAEs observed were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03. Any patients experiencing a TEAE at Grade 3 or above are reported. Grade 3 = severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care. Grade 4 = life-threatening consequences; urgent intervention indicated. Grade 5 = death related to AE.
    End point type
    Primary
    End point timeframe
    Main study: visit 1 to end of extended follow-up visit
    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: As the primary endpoints were all safety analyses, no comparative statistics were planned.
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    13
    13
    12
    Units: patients
        At least 1 TEAE
    9
    11
    7
        At least 1 treatment-related TEAE
    0
    1
    2
        At least 1 SAE
    0
    1
    0
        At least 1 treatment-related SAE
    0
    0
    0
        Withdrawn from treatment with at least SAE
    0
    1
    0
        Discontinued due to at least 1 TEAE
    0
    1
    0
        CTCAE severity Grade ≥3
    0
    1
    0
        Number of Deaths
    0
    0
    0
    No statistical analyses for this end point

    Primary: Mean Change From Baseline in Vital Signs (Body Temperature)

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    End point title
    		 Mean Change From Baseline in Vital Signs (Body Temperature) [2]
    End point description
    The mean change from Baseline in body temperature at end of treatment (visit 7) and at end of study (visit 16) is summarized for the main study. Baseline was defined as the last non-missing measurement (including unscheduled assessments) taken prior to the first dose of study drug in the main study. Only patients with data available for analysis at each visit are presented.
    End point type
    Primary
    End point timeframe
    Main study: Baseline, end of treatment (visit 7), end of study (visit 16)
    Notes
    [2] - 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: As the primary endpoints were all safety analyses, no comparative statistics were planned.
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    13
    12 [3]
    11 [4]
    Units: degrees centigrade
    arithmetic mean (standard deviation)
        End of Treatment
    0.22 ( 0.347 )
    0.16 ( 0.427 )
    -0.01 ( 0.277 )
        End of Study
    0.02 ( 0.316 )
    0.02 ( 0.447 )
    -0.04 ( 0.407 )
    Notes
    [3] - End of Study: n=11
    [4] - End of Study: n=8
    No statistical analyses for this end point

    Primary: Change in Physical Examination Abnormalities

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    End point title
    		 Change in Physical Examination Abnormalities [5]
    End point description
    Physical examination of different body systems or parameters were measured and categorized as either: Normal; Abnormal, same as previous assessment; Abnormal, new or worsened or Not Done. The number of patients recorded in the 'Abnormal, new or worsened' category at end of treatment (visit 7) and at end of study (visit 16) are summarized to represent a change across the study. Only patients with data available for analysis at each visit are presented.
    End point type
    Primary
    End point timeframe
    Main study: end of treatment (visit 7), end of study (visit 16)
    Notes
    [5] - 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: As the primary endpoints were all safety analyses, no comparative statistics were planned.
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    13
    13 [6]
    12 [7]
    Units: patients
        Abdomen: End of Treatment
    0
    0
    0
        Abdomen: End of Study
    0
    0
    0
        Breast: End of Treatment
    0
    0
    0
        Breast: End of Study
    0
    0
    0
        Cardiovascular: End of Treatment
    0
    0
    0
        Cardiovascular: End of Study
    0
    0
    0
        General Appearance: End of Treatment
    0
    0
    0
        General Appearance: End of Study
    0
    0
    0
        Genital/Rectal: End of Treatment
    1
    0
    0
        Genital/Rectal: End of Study
    0
    0
    0
        Head and Neck: End of Treatment
    0
    1
    0
        Head and Neck: End of Study
    0
    0
    0
        Lymph Nodes: End of Treatment
    0
    0
    0
        Lymph Nodes: End of Study
    0
    0
    0
        Musculoskeletal/Extremities: End of Treatment
    1
    0
    0
        Musculoskeletal/ Extremities: End of Study
    0
    0
    0
        Neurological: End of Treatment
    0
    0
    0
        Neurological: End of Study
    0
    0
    0
        Respiratory: End of Treatment
    0
    0
    0
        Respiratory: End of Study
    0
    0
    0
        Skin: End of Treatment
    2
    1
    0
        Skin: End of Study
    2
    0
    0
        Thyroid: End of Treatment
    0
    0
    0
        Thyroid: End of Study
    0
    0
    0
    Notes
    [6] - General Appearance: End of Study: n=11
    [7] - General Appearance: End of Study: n = 7
    No statistical analyses for this end point

    Primary: Mean Change From Baseline in Electrocardiogram (ECG) Parameters

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    End point title
    		 Mean Change From Baseline in Electrocardiogram (ECG) Parameters [8]
    End point description
    ECG parameters were measured and the QT correction factor was based on both the Bazett and Fridericia formulae (QTcF). The mean change from Baseline values in QT interval and QTcF interval to the last observation on treatment up to visit 7 is summarized with the mean of ECG readings for each visit and parameter in the main study used for analysis. Baseline was defined as the last non-missing measurement (including unscheduled assessments) taken prior to the first dose of study drug in the main study. Only patients with data available for analysis at each visit are presented.
    End point type
    Primary
    End point timeframe
    Main study: from Baseline to end of treatment (visit 7)
    Notes
    [8] - 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: As the primary endpoints were all safety analyses, no comparative statistics were planned.
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    13
    11
    11
    Units: milliseconds (msec)
    arithmetic mean (standard deviation)
        QT Interval
    2.27 ( 34.229 )
    2.32 ( 34.113 )
    5.32 ( 33.685 )
        QTcF Interval
    2.31 ( 37.641 )
    0.50 ( 18.087 )
    -1.00 ( 22.762 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline in Laboratory Parameters (Platelets)

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    End point title
    		 Mean Change From Baseline in Laboratory Parameters (Platelets)
    End point description
    Blood platelet count was measured at baseline and at every study visit. Baseline was defined as the last non-missing measurement (including unscheduled assessments) taken prior to the first dose of study drug in the main study. Any changes in laboratory values that were classed as clinically significant, required therapy or led to treatment discontinuation are captured in the adverse events (AE) section. The mean change from baseline in platelets at end of treatment (visit 7) and at end of study (visit 16) are summarized for the main study. Only patients with data available for analysis at each visit are presented.
    End point type
    Secondary
    End point timeframe
    Main study: Baseline, end of treatment (visit 7), end of study (visit 16)
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    12
    11 [9]
    10 [10]
    Units: 10^9/L
    arithmetic mean (standard deviation)
        End of Treatment
    24.8 ( 60.43 )
    21.2 ( 33.56 )
    10.6 ( 29.40 )
        End of Study
    60.3 ( 126.67 )
    57.5 ( 108.62 )
    9.4 ( 13.33 )
    Notes
    [9] - End of Treatment: n = 10
    [10] - End of Study: n = 7
    No statistical analyses for this end point

    Secondary: Percentage of Patients With a Platelet Count Response

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    End point title
    		 Percentage of Patients With a Platelet Count Response
    End point description
    The percentage of patients reaching any of the following platelet count thresholds at any time during the main study were reported: • Initial response: platelet count ≥30 × 10^9/L, and/or at least doubling of the Baseline count and absence of bleeding at any time during the study. • Confirmed complete response: platelet count ≥100 × 10^9/L, confirmed on at least 2 separate consecutive occasions ≥7 days apart, and the absence of bleeding. • Confirmed response: platelet count ≥30 × 10^9/L and <100 × 10^9/L, and a greater than 2-fold increase in platelet count from Baseline, confirmed on at least 2 separate consecutive occasions ≥7 days apart, and the absence of bleeding. • No response: platelet count <30 × 10^9/L or less than doubling of the Baseline count or bleeding. • Platelet count ≥50 × 10^9/L: platelet count increased to at least 50 × 10^9/L at any time during the study. Only patients with data available for analysis at each visit are presented.
    End point type
    Secondary
    End point timeframe
    Main study: from Baseline to end of study (visit 16)
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    13
    13
    12
    Units: percentage of patients
    number (confidence interval 95%)
        Initial Response
    23.1 (5.04 to 53.81)
    38.5 (13.86 to 68.42)
    33.3 (9.92 to 65.11)
        Confirmed Complete Response
    15.4 (1.92 to 45.45)
    23.1 (5.04 to 53.81)
    0.0 (0.00 to 22.09)
        Confirmed Response
    23.1 (5.04 to 53.81)
    15.4 (1.92 to 45.45)
    16.7 (2.09 to 48.41)
        No Response
    46.2 (19.22 to 74.87)
    53.8 (25.13 to 80.78)
    66.7 (34.89 to 90.08)
        Platelet ≥50 × 10^9/L
    53.8 (25.13 to 80.78)
    53.8 (25.13 to 80.78)
    50.0 (21.09 to 78.91)
    No statistical analyses for this end point

    Secondary: Pharmacokinetic (PK) Parameters - Serum Concentrations of ARGX-113

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    End point title
    		 Pharmacokinetic (PK) Parameters - Serum Concentrations of ARGX-113
    End point description
    The appropriate PK parameters were calculated after single (visit 1) and multiple administrations (visits 3, 5, and 7) of ARGX-113. The mean maximum observed serum concentration (Cmax) and serum concentration at the end of the dosing interval (Ctrough) is summarized for all visits in the main study. All patients who had at least 1 serum concentration data after the start of ARGX-113 treatment without major protocol violations/deviations thought to impact PK are included in the PK analysis set. Only patients with data available for analysis are presented.
    End point type
    Secondary
    End point timeframe
    Main study: visit 1 (day 1), visit 3 (day 8), visit 5 (day 15) and end of treatment (visit 7/day 22)
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg
    Number of subjects analysed
    13 [11]
    13 [12]
    Units: nanograms (ng)/mL
    geometric mean (geometric coefficient of variation)
        Cmax: Visit 1
    120500 ( 35.2 )
    190800 ( 21.2 )
        Cmax: Visit 3
    114000 ( 40.2 )
    173700 ( 14.7 )
        Cmax: Visit 5
    92100 ( 29.0 )
    152200 ( 19.0 )
        Cmax: Visit 7
    116600 ( 31.0 )
    190000 ( 23.1 )
        Ctrough: Visit 1
    4870 ( 40.4 )
    7906 ( 25.8 )
        Ctrough: Visit 3
    5518 ( 37.1 )
    8748 ( 8.7 )
        Ctrough: Visit 5
    6587 ( 39.2 )
    12060 ( 41.4 )
        Ctrough: Visit 7
    5498 ( 62.0 )
    9851 ( 48.0 )
    Notes
    [11] - Cmax: Visit 1: N = 12
    [12] - Cmax visits 1,3,5, Ctrough visit 3: n =12 Cmax visit 7, Ctrough visit 5, 7: n= 11
    No statistical analyses for this end point

    Secondary: Number of Patients With an Anti-drug Antibodies (ADA) Response

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    End point title
    		 Number of Patients With an Anti-drug Antibodies (ADA) Response
    End point description
    Blood samples to assess ADA were collected pre-dose on all study drug infusion days. The number of patients with a positive and negative ADA response at Baseline, end of treatment (visit 7), end of study (visit 16) in the main study are summarized. Baseline was defined as the last non-missing measurement (including unscheduled assessments) taken prior to the first dose of study drug in the main study. All patients who had received at least 1 dose of study drug without major protocol violations/deviations thought to impact PD were included in the PD analysis set. Only patients with data available for analysis are presented.
    End point type
    Secondary
    End point timeframe
    Main study: Baseline, end of treatment (visit 7), end of study (visit 16)
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    13
    13 [13]
    12 [14]
    Units: patients
        Positive: Baseline
    1
    3
    2
        Negative: Baseline
    12
    10
    10
        Positive: End of Treatment
    0
    0
    1
        Negative: End of Treatment
    13
    11
    10
        Positive: End of Study
    3
    4
    2
        Negative: End of Study
    10
    9
    10
    Notes
    [13] - End of treatment: n = 11
    [14] - End of Treatment: n = 11
    No statistical analyses for this end point

    Post-hoc: Percentage of Patients With Clinically Meaningful Platelet Count Response

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    End point title
    		 Percentage of Patients With Clinically Meaningful Platelet Count Response
    End point description
    A platelet count response of ≥50 x 10^9/L during two or more visits was classed as clinically meaningful. The percentage of patients with improved platelet count ≥50 x 10^9/L during two or more visits was recorded as a post hoc analysis.
    End point type
    Post-hoc
    End point timeframe
    Main study: from Baseline to end of study (visit 16)
    End point values
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Number of subjects analysed
    13
    13
    12
    Units: percentage of patients
        number (confidence interval 95%)
    46.2 (19.22 to 74.87)
    46.2 (19.22 to 74.87)
    25.0 (5.49 to 57.19)
    Statistical analysis title
    		 Pooled ARGX-113 Treatment Groups Vs Placebo
    Statistical analysis description
    Comparison of proportion between pooled ARGX-113 treatment groups and Placebo group based on exact logistic regression model adjusted for the platelet count category at Baseline.
    Comparison groups
    ARGX-113 5 mg/kg v ARGX-113 10 mg/kg v Placebo
    Number of subjects included in analysis
    38
    Analysis specification
    Post-hoc
    Analysis type
    		 other
    P-value
    		 = 0.3721
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    2.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.48
         upper limit
    		 17.51

    Post-hoc: Cumulative Duration of Clinically Meaningful Platelet Count Response

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    End point title
    		 Cumulative Duration of Clinically Meaningful Platelet Count Response
    End point description
    A platelet count response of ≥50 x 10^9/L during two or more visits was classed as clinically meaningful. The mean cumulative duration of platelet count response in days was recorded for pooled ARGX-113 treatment groups and Placebo group and analysed based on a conservative approach where adjacent visits were considered for calculation of duration. If visits were not adjacent, only one day was considered as duration. This approach corresponds to treating missing platelet count levels as <50 x 10^9/L.
    End point type
    Post-hoc
    End point timeframe
    Main study: from Baseline to end of study (visit 16)
    End point values
    		 Pooled ARGX-113 Placebo
    Number of subjects analysed
    26
    12
    Units: days
        arithmetic mean (standard deviation)
    24.6 ( 20.64 )
    7.7 ( 3.21 )
    No statistical analyses for this end point

    Post-hoc: Percentage of Patients With Cumulative Duration of Clinically Meaningful Platelet Count Response Greater Than 10 Days

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    End point title
    		 Percentage of Patients With Cumulative Duration of Clinically Meaningful Platelet Count Response Greater Than 10 Days
    End point description
    A platelet count response of ≥50 x 10^9/L during two or more visits was classed as clinically meaningful. Patients with a clinically meaningful platelet count response for at least 10 cumulative days was recorded for pooled ARGX-113 treatment groups and Placebo group and analysed based on a conservative approach where adjacent visits were considered for calculation of duration. If visits were not adjacent, only one day was considered as duration. This approach corresponds to treating missing platelet count levels as <50 x 10^9/L.
    End point type
    Post-hoc
    End point timeframe
    Main study: from Baseline to end of study (visit 16)
    End point values
    		 Pooled ARGX-113 Placebo
    Number of subjects analysed
    26
    12
    Units: Percentage of patients
        number (not applicable)
    38.46
    0.00
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Main study: visit 1 to end of extended follow up visit (maximum of 176 days).
    Adverse event reporting additional description
    TEAEs were monitored continuously from visit 1 until last study-related activity. In case of early discontinuation, any TEAEs/SAEs were assessed for 30 days following the patient's last visit or until satisfactory resolution or stabilization.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.1
    Reporting groups
    Reporting group title
    ARGX-113 5 mg/kg
    Reporting group description
    		 Patients received 5 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16.

    Reporting group title
    ARGX-113 10 mg/kg
    Reporting group description
    		 Patients received 10 mg/kg ARGX-113 in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients received placebo in 4 IV infusions 1 week apart (up to visit 16) and SoC. Patients who completed the initial 8-week follow-up period (visit 16) and did not receive any rescue treatment were given the option to participate in an extended follow-up period, up to a maximum 13 weeks after visit 16.

    Serious adverse events
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 ARGX-113 5 mg/kg ARGX-113 10 mg/kg Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 13 (69.23%)
    11 / 13 (84.62%)
    7 / 12 (58.33%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    3 / 13 (23.08%)
    2 / 13 (15.38%)
    0 / 12 (0.00%)
         occurrences all number
    6
    4
    0
    Hypertension
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 13 (15.38%)
    1 / 12 (8.33%)
         occurrences all number
    0
    2
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Oedema periphera
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    Pyrexia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Reproductive system and breast disorders
    Amenorrhoea
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    2
    Menorrhagia
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Metrorrhagia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Vaginal discharge
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Catarrh
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Productive cough
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    1
    Psychiatric disorders
    Depressed mood
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Investigations
    Body temperature increased
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    2
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 13 (7.69%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    1
    Scratch
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Subcutaneous haematoma
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    Sinus bradycardia
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    1
    0
    2
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Ear congestion
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Angina bullosa haemorrhagica
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Constipation
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Diarrhoea
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Dyspepsia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Mouth haemorrhage
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Nausea
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    Oral mucosal blistering
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Toothache
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 13 (15.38%)
    0 / 12 (0.00%)
         occurrences all number
    0
    2
    0
    Hepatobiliary disorders
    Hepatomegaly
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Ecchymosis
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    7
    1
    0
    Petechiae
         subjects affected / exposed
    2 / 13 (15.38%)
    2 / 13 (15.38%)
    1 / 12 (8.33%)
         occurrences all number
    2
    2
    1
    Pruritus
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    3
    0
    Purpura
         subjects affected / exposed
    2 / 13 (15.38%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    2
    1
    0
    Rash
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    Renal and urinary disorders
    Glycosuria
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Fibromyalgia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Joint swelling
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Muscle oedema
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    Pubic pain
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    2
    Tendonitis
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Cystitis
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    2
    0
    Oral herpes
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Tonsillitis
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Hypokalaemia
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 13 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Jun 2017
    The protocol was updated as follows: • to integrate the new toxicological data available on ARGX-113. • to include “wait and see” SoC treatment approach if it was according to local practice, and there was at least 1 prior line of therapy. • to include clarification on eligibility criteria. • to specify that during the follow-up period SoC of the patients were tapered by level of 25% at the discretion of the investigator when deemed medically indicated and only in patients who achieved CR (CR = platelet count >100 × 10^9/L confirmed on at least 2 separate occasions).
    07 Nov 2017
    The protocol was updated as follows: • the title of study was modified to include open-label treatment period. • the objectives, study design, study duration, inclusion and exclusion criteria, end points, and statistical sections were revised to reflect the inclusion of extended follow-up period and open-label treatment period. • for all randomized patients, platelet counts from the local laboratory was collected retrospectively/prospectively for all time points, from the patient’s medical file after patient re-consent.

    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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