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    Clinical Trial Results:
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants with Primary Warm Autoimmune Hemolytic Anemia (PATHWAY)

    Summary
    EudraCT number
    		 2021-002844-66
    Trial protocol
    		 DE   AT   IT   ES   NL   HU   FR  
    Global end of trial date
    29 Apr 2024

    Results information
    Results version number
    		 v1
    This version publication date
    25 Oct 2024
    First version publication date
    25 Oct 2024
    Other versions
    		 v2

    Trial information

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    Trial identification
    Sponsor protocol code
    INCB 50465-309
    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
    29 Apr 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    29 Apr 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The purpose of this study was to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA).
    Protection of trial subjects
    This study was to be performed in accordance with ethical principles that have their origin in the Declaration of Helsinki (Brazil 2013) and conducted in adherence to the study Protocol, applicable Good Clinical Practices, and applicable laws and country-specific regulations, including WMO (Medical Research Involving Human Participants Act) and Clinical Trials Regulation (European Union) No. 536/2014, in which the study was being conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    15 Mar 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Austria: 2
    Country: Number of subjects enrolled
    Spain: 2
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    Poland: 5
    Country: Number of subjects enrolled
    United States: 1
    Worldwide total number of subjects
    13
    EEA total number of subjects
    10
    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)
    7
    From 65 to 84 years
    6
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was designed to evaluate parsaclisib 2.5 mg QD compared with placebo over a 24-week double-blind treatment period followed by a 24-week open-label treatment period with parsaclisib. Participants could then continue to receive parsaclisib in a long-term extension period.

    Period 1
    Period 1 title
    24-week Double-blind Treatment Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Parsaclisib
    Arm description
    		 Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.
    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 QD

    Arm title
    		 Placebo followed by parsaclisib
    Arm description
    		 Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24.
    Arm type
    		 Experimental

    Investigational medicinal product name
    placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    2.5 mg QD

    Number of subjects in period 1
    		Parsaclisib Placebo followed by parsaclisib
    Started
    7
    6
    Completed
    4
    4
    Not completed
    3
    2
         Physician decision
    1
    -
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    1
    -
         Study Terminated by Sponsor
    1
    1
    Period 2
    Period 2 title
    24-week Open-label Period
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Parsaclisib
    Arm description
    		 Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.
    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 QD

    Arm title
    		 Placebo followed by parsaclisib
    Arm description
    		 Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24.
    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 QD

    Number of subjects in period 2
    		Parsaclisib Placebo followed by parsaclisib
    Started
    4
    4
    Completed
    2
    2
    Not completed
    2
    2
         Consent withdrawn by subject
    1
    -
         Adverse event, non-fatal
    1
    2
    Period 3
    Period 3 title
    Long-term Extension Period (~2 years)
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Parsaclisib
    Arm description
    		 Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.
    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 QD

    Arm title
    		 Placebo followed by parsaclisib
    Arm description
    		 Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24.
    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 QD

    Number of subjects in period 3
    		Parsaclisib Placebo followed by parsaclisib
    Started
    2
    2
    Completed
    2
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Parsaclisib
    Reporting group description
    		 Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.

    Reporting group title
    Placebo followed by parsaclisib
    Reporting group description
    		 Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24.

    Reporting group values
    		 Parsaclisib Placebo followed by parsaclisib Total
    Number of subjects
    		 7 6 13
    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)
    		 3 4 7
        From 65-84 years
    		 4 2 6
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 62.3 ( 14.28 ) 57.8 ( 15.54 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 4 6 10
        Male
    		 3 0 3
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 1 1
        Not Hispanic or Latino
    		 7 4 11
        Unknown or Not Reported
    		 0 1 1
    Race/Ethnicity, Customized
    Units: Subjects
        White/Caucasian
    		 6 4 10
        Asian
    		 1 1 2
        Missing
    		 0 1 1
    Subject analysis sets

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received matching placebo QD for 24 weeks during the double-blind treatment period.

    Subject analysis sets values
    		 Placebo
    Number of subjects
    6
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    0
        Children (2-11 years)
    0
        Adolescents (12-17 years)
    0
        Adults (18-64 years)
    4
        From 65-84 years
    2
        85 years and over
    0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    ( )
    Sex: Female, Male
    Units: participants
        Female
        Male
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
        Not Hispanic or Latino
        Unknown or Not Reported
    Race/Ethnicity, Customized
    Units: Subjects
        White/Caucasian
        Asian
        Missing

    End points

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    End points reporting groups
    Reporting group title
    Parsaclisib
    Reporting group description
    		 Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.

    Reporting group title
    Placebo followed by parsaclisib
    Reporting group description
    		 Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24.
    Reporting group title
    Parsaclisib
    Reporting group description
    		 Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.

    Reporting group title
    Placebo followed by parsaclisib
    Reporting group description
    		 Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24.
    Reporting group title
    Parsaclisib
    Reporting group description
    		 Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.

    Reporting group title
    Placebo followed by parsaclisib
    Reporting group description
    		 Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received matching placebo QD for 24 weeks during the double-blind treatment period.

    Primary: Percentage of participants attaining a durable hemoglobin response

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    End point title
    		 Percentage of participants attaining a durable hemoglobin response [1]
    End point description
    A durable hemoglobin response was defined as hemoglobin ≥10 grams per deciliter (g/dL) with an increase from Baseline of ≥2 g/dL not attributed to rescue therapy at ≥3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period. Analysis was conducted in members of the Safety Analysis Set, comprised of all randomized participants who received at least 1 dose of study drug. Treatment groups were determined according to the actual treatment the participant received on Day 1 regardless of assigned study treatment.
    End point type
    Primary
    End point timeframe
    up to Week 24
    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 Placebo followed by parsaclisib
    Number of subjects analysed
    3 [2]
    4 [3]
    Units: percentage of participants
        number (not applicable)
    33.3
    25.0
    Notes
    [2] - Safety Analysis Set. Only participants with available data were analyzed.
    [3] - Safety Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage of participants with a ≥3-point increase from Baseline in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) score at Week 24

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    End point title
    		 Percentage of participants with a ≥3-point increase from Baseline in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) score at Week 24
    End point description
    The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (“not at all”) to 4 (“very much”), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    3 [4]
    4 [5]
    Units: percentage of participants
        number (not applicable)
    66.7
    50.0
    Notes
    [4] - Safety Analysis Set. Only participants with available data were analyzed.
    [5] - Safety Analysis Set. Only participants with available data were analyzed.
    No statistical analyses for this end point

    Secondary: Percentage of participants with a 50 meter increase from Baseline to Week 24 in a 6-minute walk test (6MWT)

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    End point title
    		 Percentage of participants with a 50 meter increase from Baseline to Week 24 in a 6-minute walk test (6MWT)
    End point description
    The 6MWT is used to evaluate submaximal exercise capacity. It is a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [6]
    0 [7]
    Units: percentage of participants
        number (not applicable)
    Notes
    [6] - Data analysis was not performed for this outcome measure.
    [7] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Change from Baseline in the FACIT-F score at each post-Baseline visit

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    End point title
    		 Change from Baseline in the FACIT-F score at each post-Baseline visit
    End point description
    The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (“not at all”) to 4 (“very much”), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [8]
    0 [9]
    Units: scores on a scale
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [8] - Data analysis was not performed for this outcome measure.
    [9] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in the FACIT-F score at each post-Baseline visit

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    End point title
    		 Percentage change from Baseline in the FACIT-F score at each post-Baseline visit
    End point description
    The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (“not at all”) to 4 (“very much”), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [10]
    0 [11]
    Units: percent change
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [10] - Data analysis was not performed for this outcome measure.
    [11] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Change from Baseline in hemoglobin at each post-Baseline visit

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    End point title
    		 Change from Baseline in hemoglobin at each post-Baseline visit
    End point description
    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [12]
    0 [13]
    Units: grams per liter
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [12] - Data analysis was not performed for this outcome measure.
    [13] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in hemoglobin at each post-Baseline visit

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    End point title
    		 Percentage change from Baseline in hemoglobin at each post-Baseline visit
    End point description
    Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [14]
    0 [15]
    Units: percent change
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [14] - Data analysis was not performed for this outcome measure.
    [15] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Change from Baseline in daily corticosteroid dose at Week 24

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    End point title
    		 Change from Baseline in daily corticosteroid dose at Week 24
    End point description
    A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [16]
    0 [17]
    Units: milligrams
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [16] - Data analysis was not performed for this outcome measure.
    [17] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Percentage of participants who received transfusions from Week 6 to Week 24 and from Week 24 to Week 48

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    End point title
    		 Percentage of participants who received transfusions from Week 6 to Week 24 and from Week 24 to Week 48
    End point description
    Transfusion was permitted as a rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of warm autoimmune hemolytic anemia (wAIHA). As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Week 6 to Week 24; Week 24 to Week 48
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [18]
    0 [19]
    Units: percentage of participants
        number (not applicable)
    Notes
    [18] - Data analysis was not performed for this outcome measure.
    [19] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in daily corticosteroid dose at Week 24

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    End point title
    		 Percentage change from Baseline in daily corticosteroid dose at Week 24
    End point description
    A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [20]
    0 [21]
    Units: percent change
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [20] - Data analysis was not performed for this outcome measure.
    [21] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: 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)
    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 it was considered drug-related. An AE could therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug.
    End point type
    Secondary
    End point timeframe
    up to 446 days
    End point values
    		 Parsaclisib Placebo followed by parsaclisib Placebo
    Number of subjects analysed
    7 [22]
    4 [23]
    6 [24]
    Units: participants
    7
    4
    4
    Notes
    [22] - Safety Analysis Set
    [23] - Safety Analysis Set
    [24] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Percentage of participants who required rescue therapy at any visit from Week 6 through Week 24, and from Week 24 to Week 48

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    End point title
    		 Percentage of participants who required rescue therapy at any visit from Week 6 through Week 24, and from Week 24 to Week 48
    End point description
    Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. As pre-specified in the Statistical Analysis Plan, with limited participants enrolled in each treatment group, as well as the option for early transition to open-label parsaclisib, the statistical analyses, as related to efficacy and defined by the Protocol, are not applicable. Thus, data analysis was not performed for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Week 6 to Week 24; Week 24 to Week 48
    End point values
    		 Parsaclisib Placebo followed by parsaclisib
    Number of subjects analysed
    0 [25]
    0 [26]
    Units: percentage of participants
        number (not applicable)
    Notes
    [25] - Data analysis was not performed for this outcome measure.
    [26] - Data analysis was not performed for this outcome measure.
    No statistical analyses for this end point

    Secondary: Number of participants with any ≥Grade 3 TEAE

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    End point title
    		 Number of participants with any ≥Grade 3 TEAE
    End point description
    An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. The severity of AEs were assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.
    End point type
    Secondary
    End point timeframe
    up to 446 days
    End point values
    		 Parsaclisib Placebo followed by parsaclisib Placebo
    Number of subjects analysed
    7 [27]
    4 [28]
    6 [29]
    Units: participants
    6
    3
    1
    Notes
    [27] - Safety Analysis Set
    [28] - Safety Analysis Set
    [29] - Safety Analysis Set
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    up to 446 days
    Adverse event reporting additional description
    For safety analysis, participants who transitioned from treatment with placebo to treatment with parsaclisib 2.5 milligrams (mg) once daily (QD) in the the open-label treatment period and the long-term extension period have been counted both in the placebo arm and the parsaclisib arm.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27
    Reporting groups
    Reporting group title
    Parsaclisib
    Reporting group description
    		 Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment continued into the open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received matching placebo once daily (QD) for 24 weeks during the double-blind treatment period.

    Serious adverse events
    		 Parsaclisib Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 11 (54.55%)
    0 / 6 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Haemoglobin decreased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Colitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Diarrhoea
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune-mediated enterocolitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Parsaclisib Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 11 (90.91%)
    4 / 6 (66.67%)
    Vascular disorders
    Embolism
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Hot flush
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    0
    3
    Oedema peripheral
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Influenza like illness
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Oedema
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 6 (16.67%)
         occurrences all number
    1
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Catarrh
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Mood altered
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Insomnia
         subjects affected / exposed
    2 / 11 (18.18%)
    1 / 6 (16.67%)
         occurrences all number
    2
    1
    Investigations
    C-reactive protein increased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Blood potassium decreased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Blood alkaline phosphatase increased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    Neutrophil count decreased
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 6 (0.00%)
         occurrences all number
    2
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Skin abrasion
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Amnesia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Headache
         subjects affected / exposed
    2 / 11 (18.18%)
    2 / 6 (33.33%)
         occurrences all number
    3
    2
    Blood and lymphatic system disorders
    Lymphopenia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Diarrhoea
         subjects affected / exposed
    3 / 11 (27.27%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    Constipation
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Abdominal pain lower
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Flatulence
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 6 (0.00%)
         occurrences all number
    2
    0
    Faeces soft
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Haemorrhoidal haemorrhage
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Gastritis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Alopecia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Pruritus
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Rash erythematous
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Choluria
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Micturition disorder
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    2
    Myalgia
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 6 (16.67%)
         occurrences all number
    1
    1
    Infections and infestations
    Cytomegalovirus infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    COVID-19
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Pneumonia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Respiratory tract infection
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    Iron overload
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    Hyperglycaemia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 6 (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
    18 Aug 2022
    The primary purpose of the amendment was to incorporate feedback from advisory board members, which included the addition of a long-term extension for continued access to parscalisib, and to include prior Protocol Administrative Changes. This amendment also included the changes from local adaptations for Germany and France.
    10 May 2023
    The primary purpose of the amendment was to reduce or eliminate protocol-required procedures and visits due to closure of study enrollment.

    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
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