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    Clinical Trial Results:
    A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

    Summary
    EudraCT number
    		 2017-003652-22
    Trial protocol
    		 FR   AT   IT  
    Global end of trial date
    02 Apr 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Jan 2025
    First version publication date
    03 Jan 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    INCB 50465-206
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Incyte Corporation
    Sponsor organisation address
    1801 Augustine Cutoff Drive, Wilmington, United States, 19803
    Public contact
    Study Director, Incyte Corporation, 1 8554633463, medinfo@incyte.com
    Scientific contact
    Study Director, Incyte Corporation, 1 8554633463, medinfo@incyte.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
    02 Apr 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Apr 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The purpose of this study was to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who had decreased hemoglobin and evidence of ongoing hemolysis that required treatment intervention.
    Protection of trial subjects
    This study was performed in accordance with ethical principles that have their origin in the Declaration of Helsinki and conducted in adherence to the study Protocol, Good Clinical Practices as defined in Title 21 of the United States Code of Federal Regulations Parts 11, 50, 54, 56, and 312, as well as International Conference on Harmonisation Good Clinical Practice consolidated guidelines (E6) and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 Nov 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Austria: 5
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Italy: 14
    Country: Number of subjects enrolled
    United States: 4
    Worldwide total number of subjects
    25
    EEA total number of subjects
    21
    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)
    14
    From 65 to 84 years
    10
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study was conducted in 25 participants enrolled in 8 sites in Austria, France, Italy, and the United States.

    Period 1
    Period 1 title
    12-Week Treatment Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Parsaclisib 1 mg QD
    Arm description
    		 Participants received oral parsaclisib once daily (QD) for 12 weeks at an initial dose of parsaclisib 1 milligram (mg). At Week 6, participants who continued to require transfusions or did not attain at least a stabilization of a ≥ 2 grams per deciliter (g/dL) increase in hemoglobin from Baseline to Week 6 may have had their dose increased to parsaclisib 2.5 mg QD until Week 12. If there were tolerability issues, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period (TP) could have entered into an Extension Period (EP), during which they continued to receive parsaclisib 1 mg or 2.5 mg QD until the drug was available through another clinical study, was commercially available, or the sponsor stopped the study or study drug development. Following the last dose of parsaclisib, in either the TP (for participants not entering the EP) or the EP, participants were eligible for a 12-week (3-month) follow-up period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    parsaclisib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 mg tablets taken orally

    Arm title
    		 Parsaclisib 2.5 mg QD
    Arm description
    		 Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    parsaclisib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    2.5 mg tablets taken orally

    Number of subjects in period 1
    		Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Started
    10
    15
    Completed
    9
    13
    Not completed
    1
    2
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    1
    1
    Period 2
    Period 2 title
    Treatment Extension Period
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Parsaclisib 1 mg QD
    Arm description
    		 Participants received oral parsaclisib once daily (QD) for 12 weeks at an initial dose of parsaclisib 1 milligram (mg). At Week 6, participants who continued to require transfusions or did not attain at least a stabilization of a ≥ 2 grams per deciliter (g/dL) increase in hemoglobin from Baseline to Week 6 may have had their dose increased to parsaclisib 2.5 mg QD until Week 12. If there were tolerability issues, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period (TP) could have entered into an Extension Period (EP), during which they continued to receive parsaclisib 1 mg or 2.5 mg QD until the drug was available through another clinical study, was commercially available, or the sponsor stopped the study or study drug development. Following the last dose of parsaclisib, in either the TP (for participants not entering the EP) or the EP, participants were eligible for a 12-week (3-month) follow-up period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    parsaclisib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 mg tablets taken orally

    Arm title
    		 Parsaclisib 2.5 mg QD
    Arm description
    		 Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    parsaclisib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    2.5 mg tablets taken orally

    Number of subjects in period 2 [1]
    		Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Started
    7
    11
    Completed
    0
    0
    Not completed
    7
    11
         Adverse event, serious fatal
    -
    1
         Physician decision
    1
    1
         Adverse event, non-fatal
    4
    1
         Transitioned to Rollover Study
    1
    4
         Lack of efficacy
    1
    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: Not all participants completing the 12-week Treatment Period opted to enter the Treatment Extension Period.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Parsaclisib 1 mg QD
    Reporting group description
    		 Participants received oral parsaclisib once daily (QD) for 12 weeks at an initial dose of parsaclisib 1 milligram (mg). At Week 6, participants who continued to require transfusions or did not attain at least a stabilization of a ≥ 2 grams per deciliter (g/dL) increase in hemoglobin from Baseline to Week 6 may have had their dose increased to parsaclisib 2.5 mg QD until Week 12. If there were tolerability issues, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period (TP) could have entered into an Extension Period (EP), during which they continued to receive parsaclisib 1 mg or 2.5 mg QD until the drug was available through another clinical study, was commercially available, or the sponsor stopped the study or study drug development. Following the last dose of parsaclisib, in either the TP (for participants not entering the EP) or the EP, participants were eligible for a 12-week (3-month) follow-up period.

    Reporting group title
    Parsaclisib 2.5 mg QD
    Reporting group description
    		 Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period.

    Reporting group values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD Total
    Number of subjects
    		 10 15 25
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 6 8 14
        From 65-84 years
    		 4 6 10
        85 years and over
    		 0 1 1
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 63.5 ( 9.63 ) 60.3 ( 20.74 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 6 8 14
        Male
    		 4 7 11
    Race, Customized
    Units: Subjects
        White/Caucasian
    		 9 14 23
        Black/African-American
    		 1 0 1
        Captured as "Other"
    		 0 1 1
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 1 0 1
        Not Hispanic or Latino
    		 9 14 23
        Unknown or Not Reported
    		 0 1 1
    Hemoglobin
    Units: grams per deciliter (g/dL)
        arithmetic mean (standard deviation)
    		 9.1 ( 0.80 ) 8.7 ( 0.85 ) -

    End points

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    End points reporting groups
    Reporting group title
    Parsaclisib 1 mg QD
    Reporting group description
    		 Participants received oral parsaclisib once daily (QD) for 12 weeks at an initial dose of parsaclisib 1 milligram (mg). At Week 6, participants who continued to require transfusions or did not attain at least a stabilization of a ≥ 2 grams per deciliter (g/dL) increase in hemoglobin from Baseline to Week 6 may have had their dose increased to parsaclisib 2.5 mg QD until Week 12. If there were tolerability issues, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period (TP) could have entered into an Extension Period (EP), during which they continued to receive parsaclisib 1 mg or 2.5 mg QD until the drug was available through another clinical study, was commercially available, or the sponsor stopped the study or study drug development. Following the last dose of parsaclisib, in either the TP (for participants not entering the EP) or the EP, participants were eligible for a 12-week (3-month) follow-up period.

    Reporting group title
    Parsaclisib 2.5 mg QD
    Reporting group description
    		 Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period.
    Reporting group title
    Parsaclisib 1 mg QD
    Reporting group description
    		 Participants received oral parsaclisib once daily (QD) for 12 weeks at an initial dose of parsaclisib 1 milligram (mg). At Week 6, participants who continued to require transfusions or did not attain at least a stabilization of a ≥ 2 grams per deciliter (g/dL) increase in hemoglobin from Baseline to Week 6 may have had their dose increased to parsaclisib 2.5 mg QD until Week 12. If there were tolerability issues, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period (TP) could have entered into an Extension Period (EP), during which they continued to receive parsaclisib 1 mg or 2.5 mg QD until the drug was available through another clinical study, was commercially available, or the sponsor stopped the study or study drug development. Following the last dose of parsaclisib, in either the TP (for participants not entering the EP) or the EP, participants were eligible for a 12-week (3-month) follow-up period.

    Reporting group title
    Parsaclisib 2.5 mg QD
    Reporting group description
    		 Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period.

    Subject analysis set title
    Cohort 1: parsaclisib 1 mg QD
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants who received oral parsaclisib 1 mg QD for 12 weeks and did not qualify for a dose increase to 2.5 mg QD starting at Week 6 (they did not continue to require transfusions or they attained a meaningful clinical response [at least a stabilization of a ≥ 2 g/dL increase in hemoglobin from Baseline to Week 6]) were analyzed for pharmacokinetics (PK).

    Subject analysis set title
    Cohort 1: parsaclisib 2.5 mg QD
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants who initially received oral parsaclisib 1 mg QD, but had their dose increased, with sponsor preapproval, to 2.5 mg QD starting at Week 6 (up to Week 12) because they continued to require transfusions or did not attain a meaningful clinical response (at least a stabilization of a ≥ 2 g/dL increase in hemoglobin from Baseline to Week 6) were analyzed for PK.

    Subject analysis set title
    Cohort 2: parsaclisib 2.5 mg QD
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants who received oral parsaclisib 2.5 mg QD for 12 weeks were analyzed for PK.

    Primary: Percentage of participants attaining a complete response at any visit from Week 6 to Week 12

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    End point title
    		 Percentage of participants attaining a complete response at any visit from Week 6 to Week 12 [1]
    End point description
    A complete response was defined as hemoglobin >12 grams per deciliter (g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. Analysis was conducted in members of the Full Analysis Set, defined as all participants who enrolled in the study who received at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    Week 6 to Week 12
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Statistical analysis was not conducted for this endpoint.
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [2]
    15 [3]
    Units: percentage of participants
        number (not applicable)
    20.0
    40.0
    Notes
    [2] - Full Analysis Set
    [3] - Full Analysis Set
    No statistical analyses for this end point

    Primary: Percentage of participants attaining a partial response at any visit from Week 6 to Week 12

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    End point title
    		 Percentage of participants attaining a partial response at any visit from Week 6 to Week 12 [4]
    End point description
    A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion.
    End point type
    Primary
    End point timeframe
    Week 6 to Week 12
    Notes
    [4] - 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: Statistical analysis was not conducted for this endpoint.
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [5]
    15 [6]
    Units: percentage of participants
        number (not applicable)
    60.0
    66.7
    Notes
    [5] - Full Analysis Set
    [6] - Full Analysis Set
    No statistical analyses for this end point

    Primary: Number of participants with any treatment-emergent adverse event (TEAE)

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    End point title
    		 Number of participants with any treatment-emergent adverse event (TEAE) [7]
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy, or required changes in the study drug. Anemia and transfusions should not have been reported as AEs unless they represented a clinically meaningful decrease from Baseline in hemoglobin. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.
    End point type
    Primary
    End point timeframe
    up to 1638 days
    Notes
    [7] - 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: Statistical analysis was not conducted for this endpoint.
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [8]
    15 [9]
    Units: participants
    10
    15
    Notes
    [8] - Full Analysis Set
    [9] - Full Analysis Set
    No statistical analyses for this end point

    Secondary: Percentage of participants attaining a complete response during post-Baseline visits

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    End point title
    		 Percentage of participants attaining a complete response during post-Baseline visits
    End point description
    A complete response was defined as hemoglobin >12 g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion.
    End point type
    Secondary
    End point timeframe
    up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [10]
    15 [11]
    Units: percentage of participants
    number (not applicable)
        Week 1, n=10, 15
    0.0
    6.7
        Week 2, n=10, 15
    10.0
    6.7
        Week 4, n=9, 14
    22.2
    21.4
        Week 6, n=9, 14
    22.2
    28.6
        Week 8, n=9, 13
    22.2
    23.1
        Week 10, n=9, 13
    22.2
    23.1
        Week 12, n=9, 14
    22.2
    42.9
        Follow-up Month 1, n=2, 3
    0.0
    0.0
        Follow-up Month 2, n=2, 2
    0.0
    0.0
        Follow-up Month 3, n=2, 2
    0.0
    0.0
        Extension End of Treatment, n=6, 10
    50.0
    50.0
    Notes
    [10] - Full Analysis Set. Only participants with available data were analyzed.
    [11] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage of participants attaining a partial response during post-Baseline visits

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    End point title
    		 Percentage of participants attaining a partial response during post-Baseline visits
    End point description
    A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion.
    End point type
    Secondary
    End point timeframe
    up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [12]
    15 [13]
    Units: percentage of participants
    number (not applicable)
        Week 1, n=10, 15
    50.0
    53.3
        Week 2, n=10, 15
    50.0
    66.7
        Week 4, n=9, 14
    44.4
    64.3
        Week 6, n=9, 14
    44.4
    50.0
        Week 8, n=9, 13
    55.6
    61.5
        Week 10, n=9, 13
    55.6
    61.5
        Week 12, n=9, 14
    55.6
    64.3
        Follow-up Month 1, n=2, 3
    50.0
    0.0
        Follow-up Month 2, n=2, 2
    0.0
    0.0
        Follow-up Month 3, n=2, 2
    0.0
    50.0
        Extension End of Treatment, n=6, 10
    83.3
    50.0
    Notes
    [12] - Full Analysis Set. Only participants with available data were analyzed.
    [13] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage of participants attaining a ≥ 2 g/dL increase in hemoglobin from Baseline

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    End point title
    		 Percentage of participants attaining a ≥ 2 g/dL increase in hemoglobin from Baseline
    End point description
    Hemoglobin levels were assessed throughout the study.
    End point type
    Secondary
    End point timeframe
    up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [14]
    15 [15]
    Units: percentage of participants
    number (not applicable)
        Week 1, n=10, 15
    0.0
    26.7
        Week 2, n=10, 15
    10.0
    46.7
        Week 4, n=9, 14
    22.2
    42.9
        Week 6, n=9, 14
    22.2
    35.7
        Week 8, n=9, 13
    33.3
    38.5
        Week 10, n=9, 13
    22.2
    46.2
        Week 12, n=9, 14
    33.3
    50.0
        Follow-up Month 1, n=2, 3
    0.0
    0.0
        Follow-up Month 2, n=2, 2
    0.0
    0.0
        Follow-up Month 3, n=2, 2
    0.0
    0.0
        Extension End of Treatment, n=6, 10
    66.7
    50.0
    Notes
    [14] - Full Analysis Set. Only participants with available data were analyzed.
    [15] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in hemoglobin

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    End point title
    		 Change from Baseline in hemoglobin
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. CFB=Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [16]
    15 [17]
    Units: grams per deciliter (g/dL)
    arithmetic mean (standard deviation)
        Baseline, n=10, 15
    9.1 ( 0.80 )
    8.7 ( 0.85 )
        CFB at Week 1, n=10, 15
    0.9 ( 0.84 )
    1.2 ( 1.30 )
        CFB at Week 2, n=10, 15
    0.6 ( 0.96 )
    1.5 ( 1.75 )
        CFB at Week 4, n=9, 14
    0.9 ( 1.06 )
    1.6 ( 1.79 )
        CFB at Week 6, n=9, 14
    1.2 ( 0.80 )
    1.7 ( 1.96 )
        CFB at Week 8, n=9, 13
    1.2 ( 1.14 )
    2.0 ( 2.07 )
        CFB at Week 10, n=9, 13
    1.3 ( 1.14 )
    2.1 ( 2.22 )
        CFB at Week 12, n=9, 14
    1.3 ( 1.46 )
    2.5 ( 2.67 )
        CFB at Follow-up Month 1. n=2. 3
    0.1 ( 1.06 )
    -0.4 ( 0.72 )
        CFB at Follow-up Month 2, n=2, 2
    -0.1 ( 0.28 )
    -0.2 ( 0.28 )
        CFB at Follow-up Month 3, n=2, 2
    0.5 ( 0.92 )
    0.2 ( 1.63 )
        CFB at Extension End of Treatment, n=6, 10
    2.8 ( 2.70 )
    2.6 ( 2.73 )
    Notes
    [16] - Full Analysis Set. Only participants with available data were analyzed.
    [17] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in hemoglobin

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    End point title
    		 Percentage change from Baseline in hemoglobin
    End point description
    Percentage change from Baseline was calculated as: ([post-Baseline value minus the Baseline value] / Baseline value) x 100.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [18]
    15 [19]
    Units: percent change
    arithmetic mean (standard deviation)
        Week 1, n=10, 15
    10.1 ( 9.23 )
    14.4 ( 16.31 )
        Week 2, n=10, 15
    6.0 ( 10.26 )
    18.5 ( 20.71 )
        Week 4, n=9, 14
    9.9 ( 10.83 )
    19.2 ( 20.65 )
        Week 6, n=9, 14
    13.3 ( 8.35 )
    19.5 ( 22.35 )
        Week 8, n=9, 13
    12.7 ( 12.16 )
    23.4 ( 24.57 )
        Week 10, n=9, 13
    14.1 ( 11.54 )
    25.1 ( 26.30 )
        Week 12, n=9, 14
    13.9 ( 15.72 )
    30.2 ( 33.95 )
        Follow-up Month 1, n=2, 3
    0.0 ( 12.26 )
    -4.1 ( 7.92 )
        Follow-up Month 2, n=2, 2
    -1.0 ( 3.18 )
    -2.0 ( 2.89 )
        Follow-up Month 3, n=2, 2
    5.7 ( 11.14 )
    2.0 ( 17.32 )
        Extension End of Treatment, n=6, 10
    31.6 ( 33.86 )
    30.9 ( 33.52 )
    Notes
    [18] - Full Analysis Set. Only participants with available data were analyzed.
    [19] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage of participants requiring transfusions

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    End point title
    		 Percentage of participants requiring transfusions
    End point description
    A participant was defined to have required a transfusion if his or her last transfusion was within 7 days of the visit date.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [20]
    15 [21]
    Units: percentage of participants
    number (not applicable)
        Week 1, n=10, 15
    0.0
    6.7
        Week 2, n=10, 15
    0.0
    0.0
        Week 4, n=9, 14
    22.2
    0.0
        Week 6, n=9, 14
    0.0
    0.0
        Week 8, n=9, 13
    0.0
    15.4
        Week 10, n=9, 13
    0.0
    7.7
        Week 12, n=9, 14
    0.0
    0.0
        Follow-up Month 1, n=2, 3
    0.0
    0.0
        Follow-up Month 2, n=2, 2
    0.0
    0.0
        Follow-up Month 3, n=2, 2
    0.0
    0.0
        Extension End of Treatment, n=6, 10
    0.0
    0.0
    Notes
    [20] - Full Analysis Set. Only participants with available data were analyzed.
    [21] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieved normalization of hemoglobin, haptoglobin, lactate dehydrogenase (LDH), reticulocyte count, total bilirubin, direct bilirubin, and indirect bilirubin

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    End point title
    		 Percentage of participants who achieved normalization of hemoglobin, haptoglobin, lactate dehydrogenase (LDH), reticulocyte count, total bilirubin, direct bilirubin, and indirect bilirubin
    End point description
    Normalization was determined by the Investigator based on normal ranges for the clinical reference laboratory. 8888=No participants were analyzed at this time point.
    End point type
    Secondary
    End point timeframe
    up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [22]
    15 [23]
    Units: percentage of participants
    number (not applicable)
        Hemoglobin, Week 1, n=10, 15
    0.0
    6.7
        Hemoglobin, Week 2, n=10, 15
    0.0
    6.7
        Hemoglobin, Week 4, n=9, 14
    0.0
    14.3
        Hemoglobin, Week 6, n=9, 14
    0.0
    21.4
        Hemoglobin, Week 8, n=9, 13
    0.0
    15.4
        Hemoglobin, Week 10, n=9, 13
    0.0
    15.4
        Hemoglobin, Week 12, n=9, 14
    11.1
    35.7
        Hemoglobin, Follow-up Month 1, n=2, 3
    0.0
    0.0
        Hemoglobin, Follow-up Month 2, n=2, 2
    0.0
    0.0
        Hemoglobin, Follow-up Month 3, n=2, 2
    0.0
    0.0
        Haptoglobin, Week 1, n=8, 9
    12.5
    22.2
        Haptoglobin, Week 2, n=6, 11
    0.0
    18.2
        Haptoglobin, Week 4, n=7, 13
    14.3
    15.4
        Haptoglobin, Week 6, n=7, 13
    14.3
    23.1
        Haptoglobin, Week 8, n=7, 9
    0.0
    11.1
        Haptoglobin, Week 10, n=8, 7
    12.5
    14.3
        Haptoglobin, Week 12, n=7, 12
    14.3
    25.0
        Haptoglobin, Follow-up Month 2, n=1, 0
    0.0
    0.0
        LDH, Week 1, n=8, 10
    12.5
    10.0
        LDH, Week 2, n=9, 12
    11.1
    16.7
        LDH, Week 4, n=8, 14
    12.5
    21.4
        LDH, Week 6, n=7, 14
    14.3
    14.3
        LDH, Week 8, n=8, 11
    25.0
    9.1
        LDH, Week 10, n=9, 11
    33.3
    9.1
        LDH, Week 12, n=8, 13
    25.0
    15.4
        LDH, Follow-up Month 2, n=1, 0
    0.0
    8888
        Reticulocytes, Week 1, n=10, 14
    20.0
    14.3
        Reticulocytes, Week 2, n=7, 15
    28.6
    20.0
        Reticulocytes, Week 4, n=8, 14
    25.0
    28.6
        Reticulocytes, Week 6, n=9, 14
    22.2
    28.6
        Reticulocytes, Week 8, n=8, 12
    25.0
    25.0
        Reticulocytes, Week 10, n=6, 12
    33.3
    25.0
        Reticulocytes, Week 12, n=8, 13
    37.5
    23.1
        Reticulocytes, Follow-up Month 1, n=2, 3
    50.0
    0.0
        Reticulocytes, Follow-up Month 2, n=2, 2
    50.0
    0.0
        Reticulocytes, Follow-up Month 3, n=2, 1
    50.0
    8888
        Total bilirubin, Week 1, n=10, 15
    30.0
    40.0
        Total bilirubin, Week 2, n=10, 15
    20.0
    40.0
        Total bilirubin, Week 4, n=9, 14
    33.3
    42.9
        Total bilirubin, Week 6, n=9, 14
    33.3
    42.9
        Total bilirubin, Week 8, n=9, 12
    44.4
    50.0
        Total bilirubin, Week 10, n=9, 11
    44.4
    36.4
        Total bilirubin, Week 12, n=9, 14
    44.4
    50.0
        Total bilirubin, Follow-up Month 2, n=1, 0
    100.0
    8888
        Direct bilirubin, Week 1, n=6, 8
    0.0
    25.0
        Direct bilirubin, Week 2, n=8, 9
    12.5
    22.2
        Direct bilirubin, Week 4, n=6, 8
    0.0
    12.5
        Direct bilirubin, Week 6, n=6, 8
    0.0
    25.0
        Direct bilirubin, Week 8, n=5, 5
    0.0
    20.0
        Direct bilirubin, Week 10, n=5, 7
    0.0
    14.3
        Direct bilirubin, Week 12, n=5, 7
    0.0
    28.6
        Indirect bilirubin, Week 1, n=6, 8
    0.0
    12.5
        Indirect bilirubin, Week 2, n=8, 9
    0.0
    0.0
        Indirect bilirubin, Week 4, n=6, 8
    0.0
    0.0
        Indirect bilirubin, Week 6, n=6, 8
    0.0
    0.0
        Indirect bilirubin, Week 8, n=5, 5
    0.0
    0.0
        Indirect bilirubin, Week 10, n=5, 7
    0.0
    0.0
        Indirect bilirubin, Week 12, n=5, 7
    0.0
    0.0
    Notes
    [22] - Full Analysis Set. Only participants with available data were analyzed.
    [23] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage of participants requiring a prednisone dose change (increase or decrease)

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    End point title
    		 Percentage of participants requiring a prednisone dose change (increase or decrease)
    End point description
    Prednisone use was monitored throughout the study.
    End point type
    Secondary
    End point timeframe
    up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [24]
    15 [25]
    Units: percentage of participants
    number (not applicable)
        Week 1, increased
    0.0
    6.7
        Week 1, decreased
    20.0
    6.7
        Week 2, increased
    0.0
    0.0
        Week 2, decreased
    20.0
    13.3
        Week 4, increased
    0.0
    0.0
        Week 4, decreased
    10.0
    6.7
        Week 6, increased
    0.0
    0.0
        Week 6, decreased
    10.0
    6.7
        Week 8, increased
    0.0
    0.0
        Week 8, decreased
    10.0
    6.7
        Week 10, increased
    0.0
    0.0
        Week 10, decreased
    10.0
    6.7
        Week 12, increased
    0.0
    0.0
        Week 12, decreased
    10.0
    20.0
        Extension End of Treatment, increased
    10.0
    6.7
        Extension End of Treatment, decreased
    10.0
    6.7
    Notes
    [24] - Full Analysis Set
    [25] - Full Analysis Set
    No statistical analyses for this end point

    Secondary: Change from Baseline in the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) subscale scores

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    End point title
    		 Change from Baseline in the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) subscale scores
    End point description
    The FACIT-F subscale is a 13-item instrument designed to assess fatigue/tiredness and its impact on daily activities and functioning in a number of chronic diseases. Participants were asked to respond to 13 statements that people with the illness have said are important on the following scale: 0, not at all; 1, a little bit; 2, somewhat; 3, quite a bit; 4, very much. Participants were asked to indicate the response as it applied to the last 7 days. The total fatigue subscale score ranges from 0 to 52; a higher score indicates more severe impact on daily activities and functioning. CFB=Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [26]
    15 [27]
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline, n=10, 15
    32.6 ( 12.80 )
    30.1 ( 13.88 )
        CFB at Week 6, n=9, 14
    6.7 ( 12.44 )
    8.2 ( 9.28 )
        CFB at Week 12, n=9, 14
    6.2 ( 16.08 )
    5.4 ( 15.49 )
        CFB at Follow-up Month 1, n=2, 3
    2.0 ( 2.83 )
    2.3 ( 10.69 )
        CFB at Follow-up Month 2, n=2, 2
    1.5 ( 7.78 )
    7.0 ( 15.56 )
        CFB at Follow-up Month 3, n=2, 2
    -2.0 ( 21.21 )
    13.0 ( 14.14 )
    Notes
    [26] - Full Analysis Set. Only participants with available data were analyzed.
    [27] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Mean Cmax of parsaclisib

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    End point title
    		 Mean Cmax of parsaclisib
    End point description
    Cmax was defined as the maximum observed concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. 8888=No participants were analyzed at this time point at this dose. Analysis was conducted in members of the Pharmacokinetic (PK)/Pharmacodynamic (PD) Evaluable Population, comprised of all participants who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK or biomarker assessment.
    End point type
    Secondary
    End point timeframe
    predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
    End point values
    		 Cohort 1: parsaclisib 1 mg QD Cohort 1: parsaclisib 2.5 mg QD Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [28]
    6 [29]
    15 [30]
    Units: nanomoles per Liter (nmol/L)
    geometric mean (geometric coefficient of variation)
        Week 2
    94.9 ( 33.5 )
    8888 ( 8888 )
    191 ( 56.2 )
        Week 8
    107 ( 17.8 )
    219 ( 45.2 )
    199 ( 39.7 )
    Notes
    [28] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [29] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [30] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    Statistical analysis title
    		 Cmax
    Statistical analysis description
    Week 2
    Comparison groups
    Cohort 1: parsaclisib 1 mg QD v Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.2815
    Method
    		 ANOVA
    Confidence interval

    Secondary: Mean tmax of parsaclisib

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    End point title
    		 Mean tmax of parsaclisib
    End point description
    tmax was defined as the time to the maximum concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. -8888, 8888=No participants were analyzed at this time point at this dose.
    End point type
    Secondary
    End point timeframe
    predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
    End point values
    		 Cohort 1: parsaclisib 1 mg QD Cohort 1: parsaclisib 2.5 mg QD Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [31]
    6 [32]
    15 [33]
    Units: hours
    median (full range (min-max))
        Week 2
    1.00 (0.00 to 2.07)
    8888 (-8888 to 8888)
    1.00 (0.00 to 3.33)
        Week 8
    1.00 (1.00 to 1.05)
    1.04 (0.917 to 2.00)
    1.02 (0.833 to 4.00)
    Notes
    [31] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [32] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [33] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    Statistical analysis title
    		 tmax
    Statistical analysis description
    Week 2
    Comparison groups
    Cohort 1: parsaclisib 1 mg QD v Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.2921
    Method
    		 Kruskal-wallis
    Confidence interval

    Secondary: Mean Cmin of parsaclisib

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    End point title
    		 Mean Cmin of parsaclisib
    End point description
    Cmin was defined as the minimum observed concentration over the dose interval. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. 8888=No participants were analyzed at this time point at this dose.
    End point type
    Secondary
    End point timeframe
    predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
    End point values
    		 Cohort 1: parsaclisib 1 mg QD Cohort 1: parsaclisib 2.5 mg QD Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [34]
    6 [35]
    15 [36]
    Units: nmol/L
    geometric mean (geometric coefficient of variation)
        Week 2
    20.0 ( 88.7 )
    8888 ( 8888 )
    32.3 ( 55.4 )
        Week 8
    14.2 ( 80.9 )
    39.4 ( 26.1 )
    25.5 ( 91.4 )
    Notes
    [34] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [35] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [36] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    Statistical analysis title
    		 Cmin
    Statistical analysis description
    Week 2
    Comparison groups
    Cohort 1: parsaclisib 1 mg QD v Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.1267
    Method
    		 ANOVA
    Confidence interval

    Secondary: Mean AUC0-4 of parsaclisib

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    End point title
    		 Mean AUC0-4 of parsaclisib
    End point description
    AUC0-4 was defined as the area under the concentration-time curve from time = 0 to 4 hours postdose. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. 8888=No participants were analyzed at this time point at this dose.
    End point type
    Secondary
    End point timeframe
    predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
    End point values
    		 Cohort 1: parsaclisib 1 mg QD Cohort 1: parsaclisib 2.5 mg QD Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [37]
    6 [38]
    15 [39]
    Units: hours*nmol/L
    geometric mean (geometric coefficient of variation)
        Week 2
    260 ( 24.0 )
    8888 ( 8888 )
    542 ( 46.7 )
        Week 8
    287 ( 5.73 )
    631 ( 41.9 )
    524 ( 50.8 )
    Notes
    [37] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [38] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [39] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    Statistical analysis title
    		 AUC0-4
    Statistical analysis description
    Week 2
    Comparison groups
    Cohort 1: parsaclisib 1 mg QD v Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.2676
    Method
    		 ANOVA
    Confidence interval

    Secondary: Mean AUC0-t of parsaclisib

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    End point title
    		 Mean AUC0-t of parsaclisib
    End point description
    AUC0-t was defined as the area under the concentration-time curve from time = 0 to the last measureable concentration at time = t. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. 8888=No participants were analyzed at this time point at this dose.
    End point type
    Secondary
    End point timeframe
    predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
    End point values
    		 Cohort 1: parsaclisib 1 mg QD Cohort 1: parsaclisib 2.5 mg QD Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [40]
    6 [41]
    15 [42]
    Units: hours*nmol/L
    geometric mean (geometric coefficient of variation)
        Week 2
    260 ( 24.0 )
    8888 ( 8888 )
    553 ( 41.3 )
        Week 8
    287 ( 5.73 )
    633 ( 42.4 )
    524 ( 50.8 )
    Notes
    [40] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [41] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [42] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Mean Clast of parsaclisib

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    End point title
    		 Mean Clast of parsaclisib
    End point description
    Clast was defined as the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. 8888=No participants were analyzed at this time point at this dose.
    End point type
    Secondary
    End point timeframe
    predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
    End point values
    		 Cohort 1: parsaclisib 1 mg QD Cohort 1: parsaclisib 2.5 mg QD Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [43]
    6 [44]
    15 [45]
    Units: nmol/L
    geometric mean (geometric coefficient of variation)
        Week 2
    59.6 ( 21.3 )
    8888 ( 8888 )
    128 ( 53.3 )
        Week 8
    62.4 ( 7.31 )
    134 ( 54.2 )
    138 ( 42.3 )
    Notes
    [43] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [44] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [45] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Mean Tlast of parsaclisib

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    End point title
    		 Mean Tlast of parsaclisib
    End point description
    Tlast was defined as the time of the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. 8888=No participants were analyzed at this time point at this dose.
    End point type
    Secondary
    End point timeframe
    predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
    End point values
    		 Cohort 1: parsaclisib 1 mg QD Cohort 1: parsaclisib 2.5 mg QD Cohort 2: parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [46]
    6 [47]
    15 [48]
    Units: hours
    geometric mean (geometric coefficient of variation)
        Week 2
    3.85 ( 4.77 )
    8888 ( 8888 )
    3.98 ( 9.67 )
        Week 8
    3.83 ( 5.84 )
    3.92 ( 3.71 )
    3.84 ( 6.56 )
    Notes
    [46] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [47] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    [48] - PK/PD Evaluable Population. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in reticulocyte count

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    End point title
    		 Change from Baseline in reticulocyte count
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=The standard deviation cannot be calculated for a single participant. CFB=Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [49]
    15 [50]
    Units: 10^9 cells per Liter (10^9 cells/L)
    arithmetic mean (standard deviation)
        Baseline in the Treatment Period (TP), n=10, 14
    240.7460 ( 110.0788 )
    268.1429 ( 128.3266 )
        CFB at Week 1 of the TP, n=10, 13
    -6.8244 ( 75.4853 )
    -37.9910 ( 90.4026 )
        CFB at Week 2 of the TP, n=7, 14
    -17.2571 ( 87.7742 )
    -55.4934 ( 88.8296 )
        CFB at Week 4 of the TP, n=8, 13
    -24.8375 ( 52.5381 )
    -67.8155 ( 86.6172 )
        CFB at Week 6 of the TP, n=9, 13
    -45.4739 ( 61.9611 )
    -46.3599 ( 106.7383 )
        CFB at Week 8 of the TP, n=8, 11
    -43.0819 ( 87.7012 )
    -74.8763 ( 97.2582 )
        CFB at Week 10 of the TP, n=6, 11
    -24.3667 ( 55.3112 )
    -50.4759 ( 104.4132 )
        CFB at Week 12 of the TP, n=8, 12
    -27.1250 ( 63.3308 )
    -80.7566 ( 112.2789 )
        CFB at Follow-up Month 1 of the TP, n=2, 3
    0.0000 ( 26.1630 )
    2.6297 ( 16.8763 )
        CFB Follow-up Month 2 of the TP, n=2, 2
    -18.2000 ( 17.6777 )
    -16.4500 ( 23.2638 )
        CFB at Follow-up Month 3 of the TP, n=2, 1
    -31.1500 ( 11.2430 )
    -131.900 ( 9999 )
        Baseline of the Extension Period (EP), n=7, 10
    233.3086 ( 121.4996 )
    243.9526 ( 104.4420 )
        CFB at EP End of Treatment, n=5, 9
    184.4096 ( 102.9469 )
    191.7013 ( 131.3629 )
    Notes
    [49] - Full Analysis Set. Only participants with available data were analyzed.
    [50] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in cardiolipin immunoglobulin G (IgG) antibody and cardiolipin immunoglobulin M (IgM) antibody

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    End point title
    		 Change from Baseline in cardiolipin immunoglobulin G (IgG) antibody and cardiolipin immunoglobulin M (IgM) antibody
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=No participants were analyzed at this time point.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 12
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    9 [51]
    12 [52]
    Units: micrograms per milliliter (µg/mL)
    arithmetic mean (standard deviation)
        IgG at Baseline, n=9, 12
    0.0 ( 0.00 )
    5.8 ( 20.21 )
        Change from Baseline in IgG at Week 12, n=0, 0
    9999 ( 9999 )
    9999 ( 9999 )
        IgM at Baseline, n=9, 12
    0.0 ( 0.0 )
    1.7 ( 3.24 )
        Change from Baseline in IgM at Week 12, n=0, 0
    9999 ( 9999 )
    9999 ( 9999 )
    Notes
    [51] - Full Analysis Set. Only participants with available data were analyzed.
    [52] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in cold hemagglutinin levels

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    End point title
    		 Change from Baseline in cold hemagglutinin levels
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 12
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    2 [53]
    6 [54]
    Units: titer
    arithmetic mean (standard deviation)
        Baseline
    3200 ( 2715.29 )
    11206.67 ( 10309.03 )
        Change from Baseline at Week 12
    -1760 ( 1131.371 )
    960 ( 1357.645 )
    Notes
    [53] - Full Analysis Set. Only participants with available data were analyzed.
    [54] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in haptoglobin, total bilirubin, direct bilirubin, and indirect bilirubin

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    End point title
    		 Change from Baseline in haptoglobin, total bilirubin, direct bilirubin, and indirect bilirubin
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=The standard deviation cannot be calculated for a single participant. 8888=No participants were analyzed at this time point. CFB=Change from Baseline. FU=Follow-up. Bili=Bilirubin.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [55]
    15 [56]
    Units: micromoles (µmol)/L
    arithmetic mean (standard deviation)
        Haptoglobin, Baseline of the TP, n=10, 14
    0.00 ( 0.000 )
    0.81 ( 3.020 )
        Haptoglobin, CFB at Week 1 of the TP, n=8, 9
    0.65 ( 1.838 )
    1.20 ( 1.879 )
        Haptoglobin, CFB at Week 2 of the TP, n=6, 11
    0.00 ( 0.000 )
    0.90 ( 1.640 )
        Haptoglobin, CFB at Week 4 of the TP, n=7, 13
    0.73 ( 1.928 )
    0.75 ( 2.066 )
        Haptoglobin, CFB at Week 6 of the TP, n=7, 13
    1.09 ( 2.873 )
    2.48 ( 5.069 )
        Haptoglobin, CFB at Week 8 of the TP, n=7, 9
    0.00 ( 0.000 )
    1.36 ( 3.293 )
        Haptoglobin, CFB at Week 10 of the TP, n=8, 7
    1.55 ( 3.579 )
    1.04 ( 2.759 )
        Haptoglobin, CFB at Week 12 of the TP, n=7, 12
    0.73 ( 1.928 )
    1.34 ( 2.654 )
        Haptoglobin, CFB at FU Month 2 of the TP, n=1, 0
    0.00 ( 9999 )
    8888 ( 8888 )
        Haptoglobin, Baseline of the EP, n=7, 11
    0.0000 ( 0.0000 )
    0.0000 ( 0.0000 )
        Haptoglobin, CFB at Extension EOT, n=5, 7
    2.9200 ( 5.4906 )
    4.0000 ( 5.2386 )
        Indirect bili, Baseline of the TP, n=6, 14
    32.7 ( 13.29 )
    36.1 ( 22.77 )
        Indirect bili, CFB at Week 1 of the TP, n=5, 8
    -6.2 ( 7.66 )
    -11.9 ( 18.73 )
        Indirect bili, CFB at Week 2 of the TP, n=6, 9
    -2.3 ( 4.59 )
    -11.6 ( 17.67 )
        Indirect bili, CFB at Week 4 of the TP, n=5, 8
    -7.6 ( 9.63 )
    -11.1 ( 21.63 )
        Indirect bili, CFB at Week 6 of the TP, n=5, 8
    -11.0 ( 14.20 )
    -13.6 ( 22.39 )
        Indirect bili, CFB at Week 8 of the TP, n=4, 5
    -3.5 ( 15.63 )
    -16.8 ( 34.58 )
        Indirect bili, CFB at Week 10 of the TP, n=4, 7
    -6.5 ( 9.95 )
    -14.7 ( 25.44 )
        Indirect bili, CFB at Week 12 of the TP, n=4, 7
    -2.5 ( 13.03 )
    -17.0 ( 24.39 )
        Indirect bili, Baseline of the EP, n=4, 10
    35.2500 ( 15.7348 )
    33.4000 ( 24.5185 )
        Indirect bili, CFB at Extension EOT, n=1, 3
    -34.3610 ( 9999 )
    -0.7057 ( 8.3763 )
        Total bili, Baseline of the TP, n=10, 15
    34.7 ( 19.58 )
    54.3 ( 51.65 )
        Total bili, CFB at Week 1 of the TP, n=10, 15
    -2.8 ( 14.65 )
    -18.3 ( 34.92 )
        Total bili, CFB at Week 2 of the TP, n=10, 15
    1.7 ( 11.94 )
    -18.7 ( 34.92 )
        Total bili, CFB at Week 4 of the TP, n=9, 14
    -2.2 ( 16.53 )
    -19.5 ( 35.14 )
        Total bili, CFB at Week 6 of the TP, n=9, 14
    -3.6 ( 22.82 )
    -21.6 ( 31.27 )
        Total bili, CFB at Week 8 of the TP, n=9, 12
    -0.9 ( 17.24 )
    -22.3 ( 41.89 )
        Total bili, CFB at Week 10 of the TP, n=9, 11
    -2.7 ( 17.59 )
    -23.6 ( 29.83 )
        Total bili, CFB at Week 12 of the TP, n=9, 14
    -2.0 ( 14.86 )
    -21.3 ( 24.03 )
        Total bili, CFB at FU Month 2 of the TP, n=1, 0
    2.0 ( 9999 )
    8888 ( 8888 )
        Total bili, CFB at FU Month 2 of the TP, n=7, 11
    34.7143 ( 22.2165 )
    40.0000 ( 27.6586 )
        Total bili, CFB at Extension EOT, n=6, 9
    -11.7758 ( 31.5724 )
    -10.6219 ( 24.1217 )
        Direct bili, Baseline of the TP, n=6, 14
    14.7 ( 2.16 )
    22.9 ( 39.88 )
        Direct bili, CFB at Week 1 of the TP, n=5, 8
    -1.6 ( 4.72 )
    -12.1 ( 33.14 )
        Direct bili, CFB at Week 2 of the TP, n=6, 9
    -0.5 ( 1.97 )
    -12.0 ( 33.84 )
        Direct bili, CFB at Week 4 of the TP, n=5, 8
    -1.2 ( 4.27 )
    -14.1 ( 38.46 )
        Direct bili, CFB at Week 6 of the TP, n=5, 8
    -1.2 ( 5.89 )
    -14.5 ( 35.89 )
        Direct bili, CFB at Week 8 of the TP, n=4, 5
    0.0 ( 4.24 )
    -22.0 ( 49.79 )
        Direct bili, CFB at Week 10 of the TP, n=4, 7
    -0.3 ( 2.99 )
    -13.7 ( 32.36 )
        Direct bili, CFB at Week 12 of the TP, n=4, 7
    1.0 ( 4.76 )
    -12.4 ( 28.98 )
        Direct bili, Baseline of the EP, n=4, 10
    14.2500 ( 2.6300 )
    11.70000 ( 2.9458 )
        Direct bili, CFB at Extension EOT, n=2, 7
    -7.1322 ( 3.4977 )
    0.1213 ( 6.2545 )
    Notes
    [55] - Full Analysis Set. Only participants with available data were analyzed.
    [56] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in lactate dehydrogenase (LDH)

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    End point title
    		 Change from Baseline in lactate dehydrogenase (LDH)
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=The standard deviation cannot be calculated for a single participant. 8888=No participants were analyzed at this time point. CFB=Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [57]
    15 [58]
    Units: Units (U)/L
    arithmetic mean (standard deviation)
        Baseline of the TP, n=10, 14
    328.9 ( 78.34 )
    469.0 ( 208.96 )
        CFB at Week 1 of the TP, n=8, 10
    11.8 ( 70.89 )
    -85.1 ( 128.31 )
        CFB at Week 2 of the TP, n=9, 11
    32.2 ( 167.98 )
    -82.8 ( 129.32 )
        CFB at Week 4 of the TP, n=8, 13
    65.8 ( 190.35 )
    -61.8 ( 152.80 )
        CFB at Week 6 of the TP, n=7, 13
    23.9 ( 133.94 )
    -71.4 ( 137.51 )
        CFB at Week 8 of the TP, n=8, 10
    59.4 ( 144.20 )
    -66.8 ( 144.36 )
        CFB at Week 10 of the TP, n=9, 10
    36.6 ( 118.68 )
    -73.5 ( 153.74 )
        CFB at Week 12 of the TP, n=8, 13
    33.8 ( 117.39 )
    -66.8 ( 137.28 )
        CFB at Follow-up Month 2 of the TP, n=1, 0
    44.0 ( 9999 )
    8888 ( 8888 )
        Baseline of the EP, n=7, 10
    316.9 ( 86.74 )
    439.8 ( 178.39 )
        CFB at Extension End of Treatment, n=5, 8
    0.0 ( 173.26 )
    105.0 ( 444.64 )
    Notes
    [57] - Full Analysis Set. Only participants with available data were analyzed.
    [58] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in CH50

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    End point title
    		 Change from Baseline in CH50
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=The standard deviation cannot be calculated for a single participant. CFB=Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [59]
    15 [60]
    Units: microgram equivalents per Liter (µgEq/L)
    arithmetic mean (standard deviation)
        Baseline of the TP, n=9, 14
    84.3 ( 53.91 )
    69.5 ( 53.53 )
        CFB at Week 12 of the TP, n=8, 13
    5.4 ( 21.66 )
    17.2 ( 31.74 )
        Baseline of the EP, n=6, 10
    91.5 ( 52.20 )
    67.6 ( 56.61 )
        CFB at Extension End of Treatment, n=1, 1
    27.0 ( 9999 )
    12.0 ( 9999 )
    Notes
    [59] - Full Analysis Set. Only participants with available data were analyzed.
    [60] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Complement C3 and Complement C4

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    End point title
    		 Change from Baseline in Complement C3 and Complement C4
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=The standard deviation cannot be calculated for a single participant. 8888=No participants were analyzed at this time point. CFB=Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 1638 days
    End point values
    		 Parsaclisib 1 mg QD Parsaclisib 2.5 mg QD
    Number of subjects analysed
    10 [61]
    15 [62]
    Units: grams per Liter (g/L)
    arithmetic mean (standard deviation)
        Complement C3, Baseline of the TP, n=8, 14
    0.96 ( 0.288 )
    0.91 ( 0.183 )
        Complement C3, CFB at Week 2 of the TP, n=1, 0
    0.20 ( 9999 )
    8888 ( 8888 )
        Complement C3, CFB at Week 6 of the TP, n=1, 0
    0.20 ( 9999 )
    8888 ( 8888 )
        Complement C3, CFB at Week 12 of the TP, n=7, 13
    0.04 ( 0.098 )
    0.12 ( 0.199 )
        Complement C3, Baseline of the EP, n=5, 10
    0.9400 ( 0.1949 )
    0.9400 ( 0.1897 )
        Complement C3, CFB at EOT in the EP, n=1, 1
    0.1000 ( 9999 )
    0.1400 ( 9999 )
        Complement C4, Baseline of the TP, n=8, 14
    0.1736 ( 0.0974 )
    0.1242 ( 0.1046 )
        Complement C4, CFB at Week 2 of the TP, n=1, 0
    0.0410 ( 9999 )
    8888 ( 8888 )
        Complement C4, CFB at Week 6 of the TP, n=1, 0
    0.0330 ( 9999 )
    8888 ( 8888 )
        Complement C4, CFB at Week 12 of the TP, n=7, 13
    0.0136 ( 0.0283 )
    0.0274 ( 0.0366 )
        Complement C4, Baseline of the EP, n=5, 10
    0.1628 ( 0.0407 )
    0.1160 ( 0.0842 )
        Complement C4, CFB at Extension EOT, n=1, 1
    0.0170 ( 9999 )
    0.1100 ( 9999 )
    Notes
    [61] - Full Analysis Set. Only participants with available data were analyzed.
    [62] - Full Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    up to 1638 days
    Adverse event reporting additional description
    As dose increases and decreases, based on clinical benefit and tolerability issues, respectively, were allowed during the course of the study for participants randomized to receive both 1 mg and 2.5 mg, AEs are reported per the treatment participants were randomized to rather than the dose level that was administered at the time the AE occurred.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    Parsaclisib 2.5 mg QD
    Reporting group description
    		 Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period.

    Reporting group title
    Parsaclisib 1 mg QD
    Reporting group description
    		 Participants received oral parsaclisib once daily (QD) for 12 weeks at an initial dose of parsaclisib 1 milligram (mg). At Week 6, participants who continued to require transfusions or did not attain at least a stabilization of a ≥ 2 grams per deciliter (g/dL) increase in hemoglobin from Baseline to Week 6 may have had their dose increased to parsaclisib 2.5 mg QD until Week 12. If there were tolerability issues, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period (TP) could have entered into an Extension Period (EP), during which they continued to receive parsaclisib 1 mg or 2.5 mg QD until the drug was available through another clinical study, was commercially available, or the sponsor stopped the study or study drug development. Following the last dose of parsaclisib, in either the TP (for participants not entering the EP) or the EP, participants were eligible for a 12-week (3-month) follow-up period.

    Serious adverse events
    		 Parsaclisib 2.5 mg QD Parsaclisib 1 mg QD
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 15 (33.33%)
    6 / 10 (60.00%)
         number of deaths (all causes)
    1
    0
         number of deaths resulting from adverse events
    1
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Invasive ductal breast carcinoma
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Femur fracture
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Fall
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tibia fracture
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Aortic thrombosis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Haemolytic anaemia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Autoimmune haemolytic anaemia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Pulmonary haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary fibrosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Interstitial lung disease
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory failure
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Psoriasis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Endocrine disorders
    Diabetes insipidus
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Amoebic dysentery
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Enterococcal infection
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cytomegalovirus infection reactivation
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Campylobacter colitis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19 pneumonia
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bacterial sepsis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Atypical mycobacterial infection
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pseudomonas infection
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Staphylococcal sepsis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypernatraemia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Parsaclisib 2.5 mg QD Parsaclisib 1 mg QD
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 15 (100.00%)
    10 / 10 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lipoma
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Non-Hodgkin's lymphoma
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Vascular disorders
    Flushing
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Hypertension
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Hypotension
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    2
    Chest discomfort
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Influenza like illness
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Oedema peripheral
         subjects affected / exposed
    4 / 15 (26.67%)
    1 / 10 (10.00%)
         occurrences all number
    4
    1
    Injection site pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    6 / 15 (40.00%)
    4 / 10 (40.00%)
         occurrences all number
    6
    7
    Reproductive system and breast disorders
    Oedema genital
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 10 (20.00%)
         occurrences all number
    2
    2
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    2
    Dyspnoea
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 10 (10.00%)
         occurrences all number
    2
    2
    Hypoxia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Tachypnoea
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Pleural effusion
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Pneumonitis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Pneumothorax
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Productive cough
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Psychiatric disorders
    Agitation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Delirium
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Disorientation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Insomnia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Investigations
    Blood bilirubin increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Blood creatinine increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Complement factor C4 decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Breath sounds abnormal
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Blood potassium decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Lipids increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Lymphocyte count decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Neutrophil count decreased
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Total complement activity decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    SARS-CoV-2 test positive
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    Injury, poisoning and procedural complications
    Humerus fracture
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Arrhythmia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Cardiac discomfort
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Mitral valve incompetence
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Palpitations
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Supraventricular extrasystoles
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Disturbance in attention
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Dizziness
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    Headache
         subjects affected / exposed
    2 / 15 (13.33%)
    3 / 10 (30.00%)
         occurrences all number
    4
    4
    Tremor
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Sciatica
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Blood and lymphatic system disorders
    Autoimmune haemolytic anaemia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Anaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Eosinophilia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Haemolysis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Leukopenia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Neutropenia
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 10 (20.00%)
         occurrences all number
    2
    4
    Thrombocytopenia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Macular oedema
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Vitreous haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Abdominal distension
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Diarrhoea
         subjects affected / exposed
    5 / 15 (33.33%)
    4 / 10 (40.00%)
         occurrences all number
    5
    5
    Dry mouth
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Crohn's disease
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Constipation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Dysphagia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Eosinophilic colitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Ileus
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Irritable bowel syndrome
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Nausea
         subjects affected / exposed
    1 / 15 (6.67%)
    3 / 10 (30.00%)
         occurrences all number
    1
    4
    Vomiting
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 10 (20.00%)
         occurrences all number
    2
    2
    Hepatobiliary disorders
    Hepatotoxicity
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Jaundice
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Blister
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Dermatitis contact
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Drug eruption
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Dry skin
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Lichen planus
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Palmoplantar keratoderma
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Pruritus
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Psoriasis
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Purpura
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    3 / 15 (20.00%)
    2 / 10 (20.00%)
         occurrences all number
    5
    3
    Rash erythematous
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    2
    Rash macular
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Rash pruritic
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 10 (20.00%)
         occurrences all number
    0
    3
    Skin ulcer
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Chromaturia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Dysuria
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Urinary tract obstruction
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Endocrine disorders
    Hyperthyroidism
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 10 (0.00%)
         occurrences all number
    4
    0
    Bone pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Haemarthrosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Pain in extremity
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    Osteoporosis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Osteoarthritis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Muscle spasms
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    Muscular weakness
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Myalgia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Plantar fasciitis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Infections and infestations
    Bacteraemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Cystitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Cytomegalovirus infection reactivation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Conjunctivitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    COVID-19
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 10 (0.00%)
         occurrences all number
    6
    0
    Fungal infection
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Gastroenteritis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Gastroenteritis viral
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Influenza
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Oral candidiasis
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Oropharyngeal candidiasis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 10 (10.00%)
         occurrences all number
    1
    2
    Pneumonia
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Fluid overload
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Fluid retention
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Hyperuricaemia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Hypokalaemia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    Hypomagnesaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Hyponatraemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Jan 2018
    The primary purpose of this amendment was to limit the autoimmune hemolytic anemia (AIHA) population to participants who had disease progression after treatment with standard therapies and to add a stopping rule for futility.
    20 Jun 2018
    The primary purpose of this amendment was to clarify that the AIHA population enrolled in this study did not include treatment-naive participants and to provide further information on sample size determination.
    01 Feb 2019
    The primary purpose of this amendment was to add an extension period for participants who were receiving clinical benefit from the study treatment and to provide guidance for hematology panel collection.
    31 Oct 2019
    The primary purpose of this amendment was to increase the number of participants in Cohort 2 (from 10 to approximately 15) and change the proportion of cold agglutinin disease (CAD) and warm AIHA patients. These changes were based on the recommendations from the internal Safety Review Committee meeting.
    02 Nov 2020
    The primary purpose of this amendment was to allow participants in the open-label extension period to continue to receive treatment until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. In addition, due to the impact of the COVID-19 pandemic on study enrollment, and the rare occurrence of patients with CAD, Cohort 2 changed the enrollment of CAD AIHA to approximately 5 CAD AIHA participants.
    07 Sep 2022
    The primary purpose of this amendment was to describe risks associated with COVID-19.

    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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