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    Clinical Trial Results:
    An Open-Label Phase 1b Study of the Safety, Tolerability, and Preliminary Antitumor Activity of INCB059872 in Participants With Relapsed or Refractory Ewing Sarcoma

    Summary
    EudraCT number
    		 2018-000062-11
    Trial protocol
    		 GB   ES   IT  
    Global end of trial date
    06 Jul 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    08 Jan 2021
    First version publication date
    08 Jan 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    INCB 59872-103
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03514407
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Incyte Corporation
    Sponsor organisation address
    1801 Augustine Cut-Off, Wilmington, United States, 19803
    Public contact
    Clinical Trials Information, Incyte Corporation, RA@incyte.com
    Scientific contact
    Clinical Trials Information, Incyte Corporation, RA@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
    06 Jul 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Jul 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study to evaluate the safety and tolerability of INCB059872 in subjects with relapsed/refractory Ewing sarcoma.
    Protection of trial subjects
    All study subjects were required to read and sign an Informed Consent Form.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    27 Jun 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 5
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    Italy: 6
    Country: Number of subjects enrolled
    United States: 12
    Worldwide total number of subjects
    24
    EEA total number of subjects
    12
    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)
    5
    Adults (18-64 years)
    19
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Subjects were enrolled at 9 investigative sites in Spain, United Kingdom, Italy, and the United States from 27 June 2018 to 06 Jul 2020.

    Pre-assignment
    Screening details
    		 Subjects with histologically or cytologically confirmed diagnosis of Ewing sarcoma and have progressed on or after standard therapies were enrolled.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 INCB059872 2 mg QOD
    Arm description
    		 Subjects received INCB059872 2 mg, tablets, orally, once every other day (QOD) in consecutive 28-day cycles as long as participants are receiving benefit and have not met any criteria for study withdrawal. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    INCB059872
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    INCB059872 1 mg tablets self-administered in the morning either once every other day.

    Arm title
    		 INCB059872 3 mg QOD
    Arm description
    		 Subjects received INCB059872 3 mg, tablets, orally, once every other day (QOD) in consecutive 28-day cycles as long as participants are receiving benefit and have not met any criteria for study withdrawal. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    INCB059872
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    INCB059872 1 mg tablets self-administered in the morning either once every other day.

    Number of subjects in period 1
    		INCB059872 2 mg QOD INCB059872 3 mg QOD
    Started
    5
    19
    Completed
    0
    0
    Not completed
    5
    19
         Physician decision
    -
    1
         Consent withdrawn by subject
    -
    2
         Death
    3
    9
         Study Terminated by Sponsor
    1
    5
         Global Pandemic
    -
    1
         Reason not Specified
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    INCB059872 2 mg QOD
    Reporting group description
    		 Subjects received INCB059872 2 mg, tablets, orally, once every other day (QOD) in consecutive 28-day cycles as long as participants are receiving benefit and have not met any criteria for study withdrawal. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.

    Reporting group title
    INCB059872 3 mg QOD
    Reporting group description
    		 Subjects received INCB059872 3 mg, tablets, orally, once every other day (QOD) in consecutive 28-day cycles as long as participants are receiving benefit and have not met any criteria for study withdrawal. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.

    Reporting group values
    		 INCB059872 2 mg QOD INCB059872 3 mg QOD Total
    Number of subjects
    		 5 19 24
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 0 5 5
        Adults (18-64 years)
    		 5 14 19
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 32.6 ± 9.79 24.5 ± 9.44 -
    Gender categorical
    Units: Subjects
        Female
    		 2 8 10
        Male
    		 3 11 14

    End points

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    End points reporting groups
    Reporting group title
    INCB059872 2 mg QOD
    Reporting group description
    		 Subjects received INCB059872 2 mg, tablets, orally, once every other day (QOD) in consecutive 28-day cycles as long as participants are receiving benefit and have not met any criteria for study withdrawal. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.

    Reporting group title
    INCB059872 3 mg QOD
    Reporting group description
    		 Subjects received INCB059872 3 mg, tablets, orally, once every other day (QOD) in consecutive 28-day cycles as long as participants are receiving benefit and have not met any criteria for study withdrawal. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.

    Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Subjects With Treatment Emergent Adverse Events (TEAEs) [1]
    End point description
    A TEAE is any untoward medical occurrence in a clinical investigation administered a drug; it does not necessarily have a causal relationship with the treatment. A serious TEAE is defined as any untoward medical occurrence that resulted in death, was life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability or incapacity, led to a birth defect, or was an important medical event that may have required intervention to prevent any of items above. Data for severity was presented as Grade 3 or higher. Grade 3 AEs are defined as any severe or medically significant but not immediately life-threatening events; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living, and Grade 4 AEs are considered events with life-threatening consequences; urgent intervention indicated. Safety set included all subjects enrolled in the study who received at least 1 dose of the study drug.
    End point type
    Primary
    End point timeframe
    From signing the informed consent form up to 30 days after the last dose of study treatment or until the start of new anticancer therapy (approximately up to 6 months)
    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: No statistical analyses for this end point.
    End point values
    		 INCB059872 2 mg QOD INCB059872 3 mg QOD
    Number of subjects analysed
    5
    19
    Units: subjects
        Subject With TEAEs
    4
    17
        Subjects with Serious TEAEs
    2
    3
        Subjects with Grade 3 or 4 TEAEs
    3
    10
    No statistical analyses for this end point

    Secondary: Objective Response Rate (ORR)

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    End point title
    		 Objective Response Rate (ORR)
    End point description
    ORR is defined as the percentage of subjects who have a Complete Response (CR) or Partial Response (PR) as determined by investigator assessment of response in accordance with Response Evaluation Criteria in Solid Tumors (RECIST version1.1). CR is the disappearance of all target lesions. PR is at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters. Full analysis set all included all subjects enrolled in the study who received at least 1 dose of the study drug.
    End point type
    Secondary
    End point timeframe
    Every 8 weeks from Cycle 1 Day 1 (each cycle of 28 days) up to follow up (approximately up to 6 months)
    End point values
    		 INCB059872 2 mg QOD INCB059872 3 mg QOD
    Number of subjects analysed
    5
    19
    Units: percentage of subjects
        number (confidence interval 95%)
    20 (0.51 to 71.64)
    0 (0.00 to 17.65)
    No statistical analyses for this end point

    Secondary: Maximum Observed Plasma Concentration (Cmax) in INCB059872 2 mg Treatment Arm

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    End point title
    		 Maximum Observed Plasma Concentration (Cmax) in INCB059872 2 mg Treatment Arm [2]
    End point description
    Pharmacokinetic/Pharmacodynamic (PK/PD) evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s).
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 2 mg QOD
    Number of subjects analysed
    5
    Units: nanomoles (nM)
    arithmetic mean (standard deviation)
        Day 1 (n=5)
    46.9 ± 18.4
        Day 15 (n=3)
    40.6 ± 6.09
    No statistical analyses for this end point

    Secondary: Cmax in INCB059872 3 mg Treatment Arm

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    End point title
    		 Cmax in INCB059872 3 mg Treatment Arm [3]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s). Here, 99999 indicates that standard deviation was not calculated as only 1 subject was evaluated.
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 3 mg QOD
    Number of subjects analysed
    19
    Units: nM
    arithmetic mean (standard deviation)
        Day 1 Adolescent (n=4)
    174.0 ± 89.8
        Day 1 Adult (n=10)
    81.7 ± 35.9
        Day 15 Adolescent (n=1)
    61.5 ± 99999
        Day 15 Adult (n=8)
    96.3 ± 66.6
    No statistical analyses for this end point

    Secondary: Time to Maximum Concentration (Tmax) in INCB059872 2 mg Treatment Arm

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    End point title
    		 Time to Maximum Concentration (Tmax) in INCB059872 2 mg Treatment Arm [4]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s).
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 2 mg QOD
    Number of subjects analysed
    5
    Units: hour (hr)
    median (full range (min-max))
        Day 1 (n=5)
    1.0 (0.5 to 2.0)
        Day 15 (n=3)
    1.0 (0.5 to 2.0)
    No statistical analyses for this end point

    Secondary: Tmax in INCB059872 3 mg Treatment Arm

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    End point title
    		 Tmax in INCB059872 3 mg Treatment Arm [5]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s). Here, 99999 indicates that the lower and upper limit for median was not calculated as only 1 subject was evaluated.
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 3 mg QOD
    Number of subjects analysed
    19
    Units: hr
    median (full range (min-max))
        Day 1 Adolescent (n=4)
    1.0 (0.5 to 1.0)
        Day 1 Adult (n=10)
    1.0 (0.5 to 2.0)
        Day 15 Adolescent (n=1)
    1.0 (-99999 to 99999)
        Day 15 Adult (n=8)
    1.0 (0.5 to 2.0)
    No statistical analyses for this end point

    Secondary: Elimination Half-life (t½) in INCB059872 2 mg Treatment Arm

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    End point title
    		 Elimination Half-life (t½) in INCB059872 2 mg Treatment Arm [6]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s).
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 2 mg QOD
    Number of subjects analysed
    5
    Units: hr
    arithmetic mean (standard deviation)
        Day 1 (n=5)
    3.65 ± 0.0388
        Day 15 (n=3)
    4.21 ± 0.901
    No statistical analyses for this end point

    Secondary: t½ in INCB059872 3 mg Treatment Arm

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    End point title
    		 t½ in INCB059872 3 mg Treatment Arm [7]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s). Here, 99999 indicates that the values were not estimable.
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 3 mg QOD
    Number of subjects analysed
    19
    Units: hr
    arithmetic mean (standard deviation)
        Day 1 Adolescent (n=4)
    2.29 ± 0.509
        Day 1 Adult (n=10)
    2.79 ± 0.816
        Day 15 Adolescent (n=1)
    99999 ± 99999
        Day 15 Adult (n=8)
    4.03 ± 0.903
    No statistical analyses for this end point

    Secondary: Minimum Observed Plasma Concentration (Cmin) in INCB059872 2 mg Treatment Arm

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    End point title
    		 Minimum Observed Plasma Concentration (Cmin) in INCB059872 2 mg Treatment Arm [8]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s). Here, 99999 indicates that data was not estimable for this endpoint because of the short half-life and use of QOD dose regimes.
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 2 mg QOD
    Number of subjects analysed
    5
    Units: nM
    arithmetic mean (standard deviation)
        Day 1 (n=5)
    99999 ± 99999
        Day 15 (n=3)
    99999 ± 99999
    No statistical analyses for this end point

    Secondary: Cmin in INCB059872 3 mg Treatment Arm

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    End point title
    		 Cmin in INCB059872 3 mg Treatment Arm [9]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s). Here, 99999 indicates that data was not estimable for this endpoint because of the short half-life and use of QOD dose regimes.
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 3 mg QOD
    Number of subjects analysed
    19
    Units: nM
    arithmetic mean (standard deviation)
        Day 1 Adolescent (n=4)
    99999 ± 99999
        Day 1 Adult (n=10)
    99999 ± 99999
        Day 15 Adolescent (n=1)
    99999 ± 99999
        Day 15 Adult (n=8)
    99999 ± 99999
    No statistical analyses for this end point

    Secondary: Area Under the Plasma or Serum Concentration From Time Zero to Time of Last Measurable Concentration (AUClast) of INCB059872 2 mg Treatment Arm

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    End point title
    		 Area Under the Plasma or Serum Concentration From Time Zero to Time of Last Measurable Concentration (AUClast) of INCB059872 2 mg Treatment Arm [10]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s).
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 2 mg QOD
    Number of subjects analysed
    5
    Units: nanomoles*hour (nM*hr)
    arithmetic mean (standard deviation)
        Day 1 (n=5)
    166.0 ± 71.4
        Day 15 (n=3)
    261.0 ± 137.0
    No statistical analyses for this end point

    Secondary: AUClast of INCB059872 3 mg Treatment Arm

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    End point title
    		 AUClast of INCB059872 3 mg Treatment Arm [11]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s). Here, 99999 indicates that standard deviation was not calculated as only 1 subject was evaluated.
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 3 mg QOD
    Number of subjects analysed
    19
    Units: nM*hr
    arithmetic mean (standard deviation)
        Day 1 Adolescent (n=4)
    468.0 ± 199.0
        Day 1 Adult (n=10)
    290.0 ± 81.8
        Day 15 Adolescent (n=1)
    253 ± 99999
        Day 15 Adult (n=8)
    368.0 ± 168.0
    No statistical analyses for this end point

    Secondary: Apparent Clearance (Cl/F) in INCB059872 2 mg Treatment Arm

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    End point title
    		 Apparent Clearance (Cl/F) in INCB059872 2 mg Treatment Arm [12]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s).
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [12] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 2 mg QOD
    Number of subjects analysed
    5
    Units: liters per hour (L/hr)
    arithmetic mean (standard deviation)
        Day 1 (n=5)
    10.0 ± 3.92
        Day 15 (n=3)
    9.66 ± 2.89
    No statistical analyses for this end point

    Secondary: Cl/F in INCB059872 3 mg Treatment Arm

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    End point title
    		 Cl/F in INCB059872 3 mg Treatment Arm [13]
    End point description
    PK/PD evaluable set included all subjects who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK/PD assessment(s). Here, 99999 indicates that the values were not estimable.
    End point type
    Secondary
    End point timeframe
    Pre-dose, and post-dose 0.5, 1, 2, 4, 6, and 24 hours on Days 1 and 15, and pre-dose on Day 8 of Cycle 1
    Notes
    [13] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analyses for this end point.
    End point values
    		 INCB059872 3 mg QOD
    Number of subjects analysed
    19
    Units: L/h
    arithmetic mean (standard deviation)
        Day 1 Adolescent (n=4)
    8.51 ± 4.35
        Day 1 Adult (n=10)
    12.2 ± 3.20
        Day 15 Adolescent (n=1)
    99999 ± 99999
        Day 15 Adult (n=8)
    7.97 ± 2.03
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From signing the informed consent form up to 30 days after the last dose of study treatment or until the start of new anticancer therapy (approximately up to 6 months
    Adverse event reporting additional description
    AE additional description
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21
    Reporting groups
    Reporting group title
    INCB059872 2 mg
    Reporting group description
    		 Subjects received INCB059872 2 mg, tablets, orally, once every other day (QOD) for Cycle 1 (each cycle of 28 days) for a maximum of up to 6 months. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.

    Reporting group title
    INCB059872 3 mg
    Reporting group description
    		 Subjects received INCB059872 3 mg, tablets, orally, QOD for Cycle 1 (each cycle of 28 days) for a maximum of up to 6 months. Subjects had an option to continue Part 2 (expansion phase) of the study based on the investigator’s discretion.

    Reporting group title
    Total
    Reporting group description
    		 Total

    Serious adverse events
    		 INCB059872 2 mg INCB059872 3 mg Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 5 (40.00%)
    3 / 19 (15.79%)
    5 / 24 (20.83%)
         number of deaths (all causes)
    3
    10
    13
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Platelet count decreased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Kidney infection
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hyperuricaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hyponatraemia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         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
    		 INCB059872 2 mg INCB059872 3 mg Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 5 (60.00%)
    17 / 19 (89.47%)
    20 / 24 (83.33%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    4
    2
    6
    Pallor
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Fatigue
         subjects affected / exposed
    0 / 5 (0.00%)
    5 / 19 (26.32%)
    5 / 24 (20.83%)
         occurrences all number
    0
    7
    7
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Oedema peripheral
         subjects affected / exposed
    1 / 5 (20.00%)
    3 / 19 (15.79%)
    4 / 24 (16.67%)
         occurrences all number
    1
    3
    4
    Pyrexia
         subjects affected / exposed
    0 / 5 (0.00%)
    5 / 19 (26.32%)
    5 / 24 (20.83%)
         occurrences all number
    0
    7
    7
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 5 (40.00%)
    1 / 19 (5.26%)
    3 / 24 (12.50%)
         occurrences all number
    2
    2
    4
    Dyspnoea
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Epistaxis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    2
    2
    Hypocapnia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    2
    2
    Oropharyngeal pain
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Rhinorrhoea
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Sinus congestion
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Throat irritation
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Investigations
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    1 / 5 (20.00%)
    2 / 19 (10.53%)
    3 / 24 (12.50%)
         occurrences all number
    1
    2
    3
    Alanine aminotransferase decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 5 (20.00%)
    2 / 19 (10.53%)
    3 / 24 (12.50%)
         occurrences all number
    1
    2
    3
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 5 (20.00%)
    4 / 19 (21.05%)
    5 / 24 (20.83%)
         occurrences all number
    1
    5
    6
    Blood bicarbonate increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Blood bilirubin increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Blood creatinine increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Haematocrit decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    4
    4
    International normalised ratio increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    3
    0
    3
    Lymphocyte count increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Neutrophil count decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Platelet count decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    5 / 19 (26.32%)
    5 / 24 (20.83%)
         occurrences all number
    0
    5
    5
    Prothrombin level increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Red blood cell count decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Weight decreased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    White blood cell count decreased
         subjects affected / exposed
    1 / 5 (20.00%)
    2 / 19 (10.53%)
    3 / 24 (12.50%)
         occurrences all number
    2
    3
    5
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 5 (0.00%)
    4 / 19 (21.05%)
    4 / 24 (16.67%)
         occurrences all number
    0
    4
    4
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Headache
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    2
    Hypoaesthesia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    8 / 19 (42.11%)
    8 / 24 (33.33%)
         occurrences all number
    0
    8
    8
    Leukopenia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Lymphopenia
         subjects affected / exposed
    1 / 5 (20.00%)
    2 / 19 (10.53%)
    3 / 24 (12.50%)
         occurrences all number
    1
    3
    4
    Neutropenia
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 19 (10.53%)
    2 / 24 (8.33%)
         occurrences all number
    0
    2
    2
    Thrombocytopenia
         subjects affected / exposed
    1 / 5 (20.00%)
    5 / 19 (26.32%)
    6 / 24 (25.00%)
         occurrences all number
    2
    9
    11
    Eye disorders
    Vision blurred
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Abdominal pain
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    2
    Constipation
         subjects affected / exposed
    1 / 5 (20.00%)
    3 / 19 (15.79%)
    4 / 24 (16.67%)
         occurrences all number
    1
    3
    4
    Diarrhoea
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    2
    Dry mouth
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    2
    Dyspepsia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Dysphagia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Nausea
         subjects affected / exposed
    0 / 5 (0.00%)
    5 / 19 (26.32%)
    5 / 24 (20.83%)
         occurrences all number
    0
    5
    5
    Vomiting
         subjects affected / exposed
    1 / 5 (20.00%)
    3 / 19 (15.79%)
    4 / 24 (16.67%)
         occurrences all number
    1
    3
    4
    Skin and subcutaneous tissue disorders
    Dry skin
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Hyperhidrosis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Night sweats
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Rash
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Skin exfoliation
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Haematuria
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Musculoskeletal pain
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Myalgia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Infections and infestations
    Lung infection
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Respiratory tract infection
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Systemic infection
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    2
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    2 / 5 (40.00%)
    3 / 19 (15.79%)
    5 / 24 (20.83%)
         occurrences all number
    2
    3
    5
    Hyperglycaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Hypertriglyceridaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 19 (5.26%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Hypoalbuminaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 19 (10.53%)
    2 / 24 (8.33%)
         occurrences all number
    0
    3
    3
    Hypochloraemia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 19 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    3
    0
    3
    Hypokalaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 19 (10.53%)
    2 / 24 (8.33%)
         occurrences all number
    0
    3
    3
    Hyponatraemia
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    2
    Hypophosphataemia
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 19 (5.26%)
    2 / 24 (8.33%)
         occurrences all number
    5
    1
    6

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Feb 2018
    The primary purpose of this amendment was to revise and/or clarify treatment group assignments, the definition of dose-limiting toxicities, and the criteria for intraparticipant dose escalation in addition to providing a conversion table to ECOG performance status for Karnofsky and Lansky performance measures.
    20 May 2018
    The primary purpose of this amendment was to remove the INCB057643 treatment group, add a PK timepoint, and add a pharmacodynamic sample collection.
    22 Jul 2018
    The primary purpose of this amendment was to include additional safety monitoring.
    24 Jun 2019
    The primary purpose of this amendment was to include additional safety monitoring.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    24 Jun 2020
    This study was terminated due to strategic business decision.
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    The last patient was not able to finish the study due to the Covid-19 pandemic.
    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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