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    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo-Controlled Study of the PI3Kδ Inhibitor Parsaclisib Plus Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib

    Summary
    EudraCT number
    		 2020-003415-98
    Trial protocol
    		 HU   FR   BE   DE   NO   FI   IT  
    Global end of trial date
    21 Aug 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Aug 2025
    First version publication date
    23 Aug 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    INCB 50465-304/LIMBER-304
    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, 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
    21 Aug 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Aug 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    This study was conducted to evaluate and compare the efficacy of parsaclisib plus ruxolitinib versus placebo plus ruxolitinib on spleen volume at Week 24.
    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, applicable Good Clinical Practices, and applicable laws and country-specific regulations in which the study was being conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    26 May 2021
    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
    China: 41
    Country: Number of subjects enrolled
    Finland: 3
    Country: Number of subjects enrolled
    France: 8
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Italy: 45
    Country: Number of subjects enrolled
    Japan: 19
    Country: Number of subjects enrolled
    Korea, Republic of: 3
    Country: Number of subjects enrolled
    Norway: 7
    Country: Number of subjects enrolled
    Poland: 3
    Country: Number of subjects enrolled
    Spain: 16
    Country: Number of subjects enrolled
    Türkiye: 6
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 20
    Country: Number of subjects enrolled
    Hungary: 1
    Worldwide total number of subjects
    177
    EEA total number of subjects
    86
    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)
    90
    From 65 to 84 years
    87
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted across sites in Austria, China, Finland, France, Germany, Hungary, Italy, Japan, South Korea, Norway, Poland, Spain, Turkey, the United Kingdom, and the United States.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Parsaclisib plus ruxolitinib
    Arm description
    		 Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ruxolitinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    5-mg to 25-mg tablets (all tablet strengths may not be available in all countries), administered orally per labelling instructions

    Investigational medicinal product name
    parsaclisib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    5-mg tablets, 2.5-mg tablets, and 1-mg tablets, administered orally

    Arm title
    		 Placebo plus ruxolitinib
    Arm description
    		 Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ruxolitinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    5-mg to 25-mg tablets (all tablet strengths may not be available in all countries), administered orally per labelling instructions

    Investigational medicinal product name
    placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    matching placebo tablets, administered orally

    Number of subjects in period 1
    		Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Started
    90
    87
    Completed
    0
    0
    Not completed
    90
    87
         Consent withdrawn by subject
    12
    5
         Adverse event, non-fatal
    2
    -
         Death
    10
    9
         Medical Decision
    1
    1
         Study Terminated by Sponsor
    57
    65
         Splenapendectomi
    1
    -
         Lost to follow-up
    -
    2
         Lack of efficacy
    2
    1
         Transitioned to Rollover per Protocol
    5
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Parsaclisib plus ruxolitinib
    Reporting group description
    		 Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.

    Reporting group title
    Placebo plus ruxolitinib
    Reporting group description
    		 Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.

    Reporting group values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib Total
    Number of subjects
    		 90 87 177
    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)
    		 43 47 90
        From 65-84 years
    		 47 40 87
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 64.0 ( 9.88 ) 62.3 ( 9.92 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 37 38 75
        Male
    		 53 49 102
    Race, Customized
    Units: Subjects
        White or Caucasian
    		 45 52 97
        Black or African American
    		 1 1 2
        Asian
    		 37 26 63
        Not Reported
    		 7 8 15
    Ethnicity, Customized
    Units: Subjects
        Hispanic or Latino
    		 2 10 12
        Not Hispanic or Latino
    		 70 57 127
        Not Reported
    		 7 4 11
        Unknown
    		 2 4 6
        Captured as "Other" in Database
    		 9 12 21

    End points

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    End points reporting groups
    Reporting group title
    Parsaclisib plus ruxolitinib
    Reporting group description
    		 Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.

    Reporting group title
    Placebo plus ruxolitinib
    Reporting group description
    		 Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.

    Subject analysis set title
    Placebo switch to parsaclisib
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.

    Primary: Percentage of participants achieving ≥25% reduction in spleen volume from Baseline to Week 24 as measured by magnetic resonance imaging (MRI) (or computed tomography [CT] scan in applicable participants)

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    End point title
    		 Percentage of participants achieving ≥25% reduction in spleen volume from Baseline to Week 24 as measured by magnetic resonance imaging (MRI) (or computed tomography [CT] scan in applicable participants)
    End point description
    Participants had an MRI of the upper and lower abdomen and pelvis to determine the spleen volume. A CT scan was substituted for participants who were not candidates for MRI or when MRI was not readily available. Intent-to-Treat (ITT) Population: all randomized participants. Participants were analyzed if they had both Baseline and Week 24 measurements, or discontinued treatment before 03MAR2023 or switched treatment before Week 24, or reached Week 24 before 03MAR2023 but were missing Week 24 assessments. For participants who switched to parsaclisib plus ruxolitinib treatment early, data was truncated at the time of switch.
    End point type
    Primary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Number of subjects analysed
    72 [1]
    72 [2]
    Units: percentage of participants
        number (not applicable)
    16.7
    9.7
    Notes
    [1] - ITT Population
    [2] - ITT Population
    Statistical analysis title
    		 Cochran Mantel-Haenszel (CMH) test
    Comparison groups
    Parsaclisib plus ruxolitinib v Placebo plus ruxolitinib
    Number of subjects included in analysis
    144
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [3]
    P-value
    		 = 0.2567 [4]
    Method
    		 Cochran-Mantel-Haenszel
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.65
         upper limit
    		 5.02
    Notes
    [3] - p-value for superiority is half of the CMH p-value.
    [4] - CMH test for un-equality stratified by Dynamic International Prognostic Scoring System (DIPSS) category (intermediate 1 versus intermediate 2 and high) and Baseline platelet count (≥100 × 10^9/Liters [L] versus 50 to <100 × 10^9/L inclusive)

    Secondary: Percentage of participants who have a ≥50% reduction in Total Symptom Score (TSS) from Baseline to Week 24 as measured by the Myelofibrosis Symptom Assessment Form v.4.0 (MFSAF v4.0) diary

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    End point title
    		 Percentage of participants who have a ≥50% reduction in Total Symptom Score (TSS) from Baseline to Week 24 as measured by the Myelofibrosis Symptom Assessment Form v.4.0 (MFSAF v4.0) diary
    End point description
    Symptoms of myelofibrosis were assessed using the MFSAF v4.0 diary. The MFSAF v4.0 is composed of 7 individual symptom scores (fatigue, night sweats, itchiness, abdominal discomfort, pain under left ribs, early satiety, bone pain), each collected daily using a 0- (no symptoms) to 10-point (worst imaginable symptoms) scale. The daily TSS (0 to 70) is the sum of the 7 individual symptom scores. A higher TSS corresponds to more severe symptoms. The Baseline/Week 24 total score was defined as the average of the daily total scores from the last 7 days before the first dose of parsaclisib, placebo, or ruxolitinib/the Week 24 visit. Participants were analyzed if they had both Baseline and Week 24 measurements, or discontinued treatment before 03MAR2023 or switched treatment before Week 24, or reached Week 24 before 03MAR2023 but were missing Week 24 assessments. For participants who switched to parsaclisib plus ruxolitinib treatment early, data was truncated at the time of switch.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Number of subjects analysed
    70 [5]
    71 [6]
    Units: percentage of participants
        number (not applicable)
    17.1
    14.1
    Notes
    [5] - ITT Population
    [6] - ITT Population
    Statistical analysis title
    		 Reduction in TSS score
    Comparison groups
    Parsaclisib plus ruxolitinib v Placebo plus ruxolitinib
    Number of subjects included in analysis
    141
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [7]
    P-value
    		 = 0.5349 [8]
    Method
    		 Cochran-Mantel-Haenszel
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.34
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.53
         upper limit
    		 3.39
    Notes
    [7] - p-value for superiority is half of the CMH p-value.
    [8] - calculated from Cochran Mantel-Haenszel test for un-equality stratified by DIPSS category (intermediate 1 versus intermediate 2 and high) and Baseline platelet count (≥100 × 10^9/L versus 50 to <100 × 10^9/L inclusive)

    Secondary: Change in TSS from Baseline to Week 24 as measured by the MFSAF v4.0 diary

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    End point title
    		 Change in TSS from Baseline to Week 24 as measured by the MFSAF v4.0 diary
    End point description
    Symptoms of myelofibrosis were assessed using the MFSAF v4.0 diary. The MFSAF v4.0 is composed of 7 individual symptom scores (fatigue, night sweats, itchiness, abdominal discomfort, pain under left ribs, early satiety, bone pain), each collected daily using a 0- (no symptoms) to 10-point (worst imaginable symptoms) scale. The daily TSS (0 to 70) is the sum of the 7 individual symptom scores collected in a day. A higher TSS corresponds to more severe symptoms. The TSS was marked as missing if there were any missing individual scores. Observations with missing dates were excluded from the analysis. The Baseline/Week 24 total score was defined as the average of the daily total scores from the last 7 days before the first dose of parsaclisib, placebo, or ruxolitinib/the Week 24 visit. The Baseline/Week 24 total score was marked as missing if there were ≥4 out of the 7 daily TSSs missing. Change from Baseline was calculated as the Week 24 value minus the Baseline value.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Number of subjects analysed
    88 [9]
    82 [10]
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline, n=88, 82
    17.2 ( 11.61 )
    21.6 ( 14.28 )
        Change from Baseline at Week 24, n=57, 61
    -2.7 ( 8.67 )
    -2.7 ( 9.98 )
    Notes
    [9] - ITT Population. Only participants with data available were analyzed.
    [10] - ITT Population. Only participants with data available were analyzed.
    No statistical analyses for this end point

    Secondary: Time to the first ≥50% reduction in TSS as measured by the MFSAF v4.0 diary

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    End point title
    		 Time to the first ≥50% reduction in TSS as measured by the MFSAF v4.0 diary
    End point description
    Symptoms were assessed using the MFSAF v4.0 diary. The MFSAF v4.0 is composed of 7 symptom scores (fatigue, night sweats, itchiness, abdominal discomfort, pain under left ribs, early satiety, bone pain), each collected daily using a 0- (no symptoms) to 10-point (worst imaginable symptoms) scale. The daily TSS (0 to 70) is the sum of the 7 individual symptom scores. A higher TSS corresponds to more severe symptoms. The TSS was marked as missing if there were any missing individual scores. Observations with missing dates were excluded from the analysis. The Baseline/Week 24 total score was defined as the average of the daily total scores from the last 7 days before the first dose of parsaclisib, placebo, or ruxolitinib/the Week 24 visit. The Baseline/Week 24 total scores was marked as missing if there were ≥4 out of the 7 daily TSSs missing. -9999, 9999=Values were not estimable because there were too few participants with a ≥50% reduction in TSS at the time of study termination.
    End point type
    Secondary
    End point timeframe
    Baseline; up to Week 24
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Number of subjects analysed
    90 [11]
    87 [12]
    Units: days
        median (confidence interval 95%)
    9999 (160.0 to 9999)
    9999 (-9999 to 9999)
    Notes
    [11] - ITT Population
    [12] - ITT Population
    Statistical analysis title
    		 Time to the first ≥50% reduction in TSS
    Comparison groups
    Parsaclisib plus ruxolitinib v Placebo plus ruxolitinib
    Number of subjects included in analysis
    177
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.2224 [13]
    Method
    		 Logrank
    Confidence interval
    Notes
    [13] - calculated from log-rank test stratified by DIPSS category (intermediate 1 versus intermediate 2 and high) and Baseline platelet count (≥100 x 10^9/L versus 50 to <100 x 10^9/L inclusive)

    Secondary: Overall survival

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    End point title
    		 Overall survival
    End point description
    Overall survival was defined as the interval between the randomization date and the date of death due to any cause. -9999, 9999=Due to study termination, the follow-up time was not long enough to estimate the median and the upper and lower limits of the confidence interval.
    End point type
    Secondary
    End point timeframe
    up to 917 days
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Number of subjects analysed
    90 [14]
    87 [15]
    Units: days
        median (confidence interval 95%)
    9999 (-9999 to 9999)
    9999 (-9999 to 9999)
    Notes
    [14] - ITT Population
    [15] - ITT Population
    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) is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is 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 treatment. A TEAE is defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of study treatment. Safety Population: all randomized participants who received at least 1 dose of parsaclisib, placebo, or ruxolitinib. Treatment groups for this population were determined according to the actual treatment the participant received regardless of assigned study drug treatment.
    End point type
    Secondary
    End point timeframe
    up to 917 days
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib Placebo switch to parsaclisib
    Number of subjects analysed
    90 [16]
    87 [17]
    41 [18]
    Units: participants
    81
    76
    33
    Notes
    [16] - Safety Population
    [17] - Safety Population
    [18] - Safety Population
    No statistical analyses for this end point

    Secondary: Time to the first ≥25% reduction in spleen volume

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    End point title
    		 Time to the first ≥25% reduction in spleen volume
    End point description
    The time to the first ≥25% reduction in spleen volume is defined as the time from randomization to the first time participants had ≥25% reduction in spleen volume. Participants with a Baseline and post-Baseline MRI or CT scan who did not have ≥25% reduction in spleen volume at the time of analysis were censored at the time of the last MRI or CT scan. If the participants had no Baseline or post-Baseline MRI or CT scan, they were censored at the date of randomization. -9999, 9999=The median and the upper and lower limits of the confidence interval were not estimable because too few participants had a ≥25% reduction in spleen volume.
    End point type
    Secondary
    End point timeframe
    up to 898 days
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Number of subjects analysed
    90 [19]
    87 [20]
    Units: days
        median (confidence interval 95%)
    9999 (-9999 to 9999)
    9999 (-9999 to 9999)
    Notes
    [19] - ITT Population
    [20] - ITT Population
    No statistical analyses for this end point

    Secondary: Number of participants with any Grade 3 or higher TEAE

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    End point title
    		 Number of participants with any Grade 3 or higher TEAE
    End point description
    An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. A TEAE is defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of study treatment. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events (CTCAE) v5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: moderate; minimal, local or noninvasive intervention indicated; limiting instrumental activities of daily living (ADL). Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4: life-threatening urgent intervention indicated. Grade 5: death related to AE.
    End point type
    Secondary
    End point timeframe
    up to 917 days
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib Placebo switch to parsaclisib
    Number of subjects analysed
    90 [21]
    87 [22]
    41 [23]
    Units: participants
    54
    37
    18
    Notes
    [21] - Safety Population
    [22] - Safety Population
    [23] - Safety Population
    No statistical analyses for this end point

    Secondary: Duration of maintenance of a ≥25% reduction in spleen volume

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    End point title
    		 Duration of maintenance of a ≥25% reduction in spleen volume
    End point description
    The duration of ≥25% reduction from Baseline in spleen volume was defined as the interval between the first spleen volume measurement that was a ≥25% reduction from Baseline and the date of the first measurement that was no longer a ≥25% reduction from Baseline. If the end date was not observed before the database cutoff, the duration was censored at the last assessment. Only those participants who had at least 1 measurement of ≥25% reduction from Baseline were analyzed. -9999, 9999=The median and the upper and lower limits of the confidence interval were not estimable because too few participants had a ≥25% reduction in spleen volume.
    End point type
    Secondary
    End point timeframe
    up to 898 days
    End point values
    		 Parsaclisib plus ruxolitinib Placebo plus ruxolitinib
    Number of subjects analysed
    12 [24]
    7 [25]
    Units: days
        median (confidence interval 95%)
    9999 (-9999 to 9999)
    9999 (-9999 to 9999)
    Notes
    [24] - ITT Population
    [25] - ITT Population
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    up to 917 days
    Adverse event reporting additional description
    Treatment-emergent adverse events (TEAEs), defined as adverse events reported for the first time or the worsening of pre-existing events after the first dose of study treatment, have been reported for members of the Safety Population.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    Parsaclisib plus ruxolitinib
    Reporting group description
    		 Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.

    Reporting group title
    Placebo swith to parsaclisib
    Reporting group description
    		 After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.

    Reporting group title
    Placebo plus ruxolitinib
    Reporting group description
    		 Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.

    Serious adverse events
    		 Parsaclisib plus ruxolitinib Placebo swith to parsaclisib Placebo plus ruxolitinib
    Total subjects affected by serious adverse events
         subjects affected / exposed
    33 / 90 (36.67%)
    8 / 41 (19.51%)
    15 / 87 (17.24%)
         number of deaths (all causes)
    10
    2
    7
         number of deaths resulting from adverse events
    6
    2
    3
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Acute myeloid leukaemia
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Breast neoplasm
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Basal cell carcinoma
         subjects affected / exposed
    1 / 90 (1.11%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Papillary renal cell carcinoma
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin squamous cell carcinoma metastatic
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Transformation to acute myeloid leukaemia
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Multiple organ dysfunction syndrome
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Malaise
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    2 / 90 (2.22%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Interstitial lung disease
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Haemoptysis
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lung disorder
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Investigations
    Platelet count decreased
         subjects affected / exposed
    2 / 90 (2.22%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Ankle fracture
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Arterial injury
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Spinal compression fracture
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Subdural haematoma
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    Cardiac disorders
    Atrioventricular block second degree
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Syncope
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    2 / 90 (2.22%)
    0 / 41 (0.00%)
    2 / 87 (2.30%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Febrile neutropenia
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Leukocytosis
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pancytopenia
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Splenic haemorrhage
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Subcapsular splenic haematoma
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ear and labyrinth disorders
    Vertigo positional
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Faeces discoloured
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Necrotising colitis
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ulcerative gastritis
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Pyoderma gangrenosum
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Anal abscess
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Abscess
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Bronchitis
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Bronchopulmonary aspergillosis
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    COVID-19 pneumonia
         subjects affected / exposed
    5 / 90 (5.56%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    1 / 5
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 3
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    3 / 90 (3.33%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Diarrhoea infectious
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Enterocolitis bacterial
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Enterocolitis infectious
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Herpes zoster
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia influenzal
         subjects affected / exposed
    2 / 90 (2.22%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Pneumonia escherichia
         subjects affected / exposed
    0 / 90 (0.00%)
    0 / 41 (0.00%)
    1 / 87 (1.15%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia cytomegaloviral
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    8 / 90 (8.89%)
    2 / 41 (4.88%)
    2 / 87 (2.30%)
         occurrences causally related to treatment / all
    5 / 11
    1 / 2
    2 / 2
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Pulmonary tuberculosis
         subjects affected / exposed
    1 / 90 (1.11%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    0 / 90 (0.00%)
    1 / 41 (2.44%)
    0 / 87 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Parsaclisib plus ruxolitinib Placebo swith to parsaclisib Placebo plus ruxolitinib
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    71 / 90 (78.89%)
    25 / 41 (60.98%)
    58 / 87 (66.67%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    8 / 90 (8.89%)
    4 / 41 (9.76%)
    3 / 87 (3.45%)
         occurrences all number
    9
    4
    4
    Cytomegalovirus test positive
         subjects affected / exposed
    4 / 90 (4.44%)
    3 / 41 (7.32%)
    0 / 87 (0.00%)
         occurrences all number
    4
    3
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    7 / 90 (7.78%)
    4 / 41 (9.76%)
    4 / 87 (4.60%)
         occurrences all number
    8
    5
    5
    Neutrophil count decreased
         subjects affected / exposed
    5 / 90 (5.56%)
    1 / 41 (2.44%)
    3 / 87 (3.45%)
         occurrences all number
    7
    1
    3
    Platelet count decreased
         subjects affected / exposed
    20 / 90 (22.22%)
    5 / 41 (12.20%)
    14 / 87 (16.09%)
         occurrences all number
    33
    5
    23
    White blood cell count decreased
         subjects affected / exposed
    8 / 90 (8.89%)
    0 / 41 (0.00%)
    7 / 87 (8.05%)
         occurrences all number
    12
    0
    17
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    7 / 90 (7.78%)
    1 / 41 (2.44%)
    2 / 87 (2.30%)
         occurrences all number
    9
    1
    2
    Headache
         subjects affected / exposed
    8 / 90 (8.89%)
    1 / 41 (2.44%)
    4 / 87 (4.60%)
         occurrences all number
    8
    1
    4
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    26 / 90 (28.89%)
    6 / 41 (14.63%)
    20 / 87 (22.99%)
         occurrences all number
    38
    6
    36
    Neutropenia
         subjects affected / exposed
    0 / 90 (0.00%)
    3 / 41 (7.32%)
    1 / 87 (1.15%)
         occurrences all number
    0
    3
    3
    Thrombocytopenia
         subjects affected / exposed
    10 / 90 (11.11%)
    8 / 41 (19.51%)
    8 / 87 (9.20%)
         occurrences all number
    11
    14
    10
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    7 / 90 (7.78%)
    3 / 41 (7.32%)
    4 / 87 (4.60%)
         occurrences all number
    7
    3
    4
    Fatigue
         subjects affected / exposed
    5 / 90 (5.56%)
    0 / 41 (0.00%)
    4 / 87 (4.60%)
         occurrences all number
    6
    0
    4
    Pyrexia
         subjects affected / exposed
    13 / 90 (14.44%)
    1 / 41 (2.44%)
    8 / 87 (9.20%)
         occurrences all number
    20
    2
    14
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    8 / 90 (8.89%)
    0 / 41 (0.00%)
    2 / 87 (2.30%)
         occurrences all number
    9
    0
    2
    Constipation
         subjects affected / exposed
    6 / 90 (6.67%)
    0 / 41 (0.00%)
    0 / 87 (0.00%)
         occurrences all number
    7
    0
    0
    Diarrhoea
         subjects affected / exposed
    10 / 90 (11.11%)
    1 / 41 (2.44%)
    9 / 87 (10.34%)
         occurrences all number
    10
    4
    10
    Nausea
         subjects affected / exposed
    7 / 90 (7.78%)
    2 / 41 (4.88%)
    7 / 87 (8.05%)
         occurrences all number
    8
    2
    8
    Stomatitis
         subjects affected / exposed
    6 / 90 (6.67%)
    1 / 41 (2.44%)
    2 / 87 (2.30%)
         occurrences all number
    8
    1
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    8 / 90 (8.89%)
    2 / 41 (4.88%)
    4 / 87 (4.60%)
         occurrences all number
    8
    2
    4
    Dyspnoea
         subjects affected / exposed
    6 / 90 (6.67%)
    0 / 41 (0.00%)
    3 / 87 (3.45%)
         occurrences all number
    6
    0
    3
    Epistaxis
         subjects affected / exposed
    5 / 90 (5.56%)
    0 / 41 (0.00%)
    3 / 87 (3.45%)
         occurrences all number
    6
    0
    5
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    5 / 90 (5.56%)
    0 / 41 (0.00%)
    6 / 87 (6.90%)
         occurrences all number
    5
    0
    10
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    4 / 90 (4.44%)
    0 / 41 (0.00%)
    5 / 87 (5.75%)
         occurrences all number
    4
    0
    5
    Arthralgia
         subjects affected / exposed
    4 / 90 (4.44%)
    3 / 41 (7.32%)
    3 / 87 (3.45%)
         occurrences all number
    4
    3
    3
    Pain in extremity
         subjects affected / exposed
    5 / 90 (5.56%)
    1 / 41 (2.44%)
    3 / 87 (3.45%)
         occurrences all number
    5
    1
    3
    Infections and infestations
    COVID-19
         subjects affected / exposed
    16 / 90 (17.78%)
    6 / 41 (14.63%)
    10 / 87 (11.49%)
         occurrences all number
    16
    6
    10
    Cytomegalovirus infection
         subjects affected / exposed
    10 / 90 (11.11%)
    2 / 41 (4.88%)
    0 / 87 (0.00%)
         occurrences all number
    10
    2
    0
    Metabolism and nutrition disorders
    Hyperuricaemia
         subjects affected / exposed
    7 / 90 (7.78%)
    0 / 41 (0.00%)
    4 / 87 (4.60%)
         occurrences all number
    7
    0
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Oct 2021
    The primary purpose of this amendment was to address changes requested by different national regulatory agencies and ethics committees.
    20 Oct 2022
    The primary purpose of this amendment was to update safety information for parsaclisib, including information regarding coronavirus disease 2019 (COVID-19), and the potential impact of parsaclisib therapy on infection risk, vaccine effectiveness, and severity of disease.

    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.
    Following an interim analysis by an independent Data Monitoring Committee (DMC), the study was terminated early due to futility.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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