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    Clinical Trial Results:
    A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1 3 Alterations (FIGHT-209)

    Summary
    EudraCT number
    		 2021-004740-24
    Trial protocol
    		 DE   ES   DK   FR   IT   NL  
    Global end of trial date
    17 Dec 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Dec 2025
    First version publication date
    25 Dec 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    INCB 54828-209
    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, 1 855-463-3463, medinfo@incyte.com
    Scientific contact
    Study Director, Incyte Corporation, 1 855-463-3463, 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
    17 Dec 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Dec 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    This study was conducted to determine the efficacy of pemigatinib in participants with recurrent glioblastoma with an activating fibroblast growth factor receptor (FGFR)1-3 mutation or fusion/rearrangement.
    Protection of trial subjects
    This study was performed in accordance with ethical principles that have their origin in the Declaration of Helsinki (Brazil 2013) and conducted in adherence to the study Protocol, applicable Good Clinical Practices, and applicable laws and country-specific regulations, including WMO (Medical Research Involving Human Participants Act) and Clinical Trials Regulation (European Union) No. 536/2014, in which the study was conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 May 2022
    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
    Denmark: 3
    Country: Number of subjects enrolled
    France: 11
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Italy: 17
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    Netherlands: 3
    Country: Number of subjects enrolled
    Spain: 15
    Country: Number of subjects enrolled
    United Kingdom: 9
    Country: Number of subjects enrolled
    United States: 21
    Worldwide total number of subjects
    83
    EEA total number of subjects
    51
    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)
    64
    From 65 to 84 years
    19
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted at 31 study centers in Denmark, France, Germany, Italy, Japan, Netherlands, Spain, the United Kingdom, and the United States.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Recurrent glioblastoma
    Arm description
    		 Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
    Arm type
    		 Experimental

    Investigational medicinal product name
    pemigatinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Unit dose strength(s)/dosage level(s): 13.5 mg, 9 mg, and 4.5 mg

    Arm title
    		 Recurrent non-glioblastoma CNS tumors
    Arm description
    		 Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.
    Arm type
    		 Experimental

    Investigational medicinal product name
    pemigatinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Unit dose strength(s)/dosage level(s): 13.5 mg, 9 mg, and 4.5 mg

    Number of subjects in period 1
    		Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Started
    74
    9
    Completed
    0
    0
    Not completed
    74
    9
         Consent withdrawn by subject
    -
    1
         Study terminated by Sponsor
    24
    6
         Death
    50
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Recurrent glioblastoma
    Reporting group description
    		 Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.

    Reporting group title
    Recurrent non-glioblastoma CNS tumors
    Reporting group description
    		 Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.

    Reporting group values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors Total
    Number of subjects
    		 74 9 83
    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)
    		 55 9 64
        From 65-84 years
    		 19 0 19
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 57.0 ( 11.45 ) 39.2 ( 12.75 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 30 3 33
        Male
    		 44 6 50
    Race/Ethnicity, Customized
    Units: Subjects
        White/Caucasian
    		 58 6 64
        Black/African-American
    		 2 0 2
        Asian
    		 2 0 2
        Not Reported
    		 7 1 8
        Unknown
    		 5 1 6
        Captured as "Other" in Database
    		 0 1 1
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 1 0 1
        Not Hispanic or Latino
    		 60 6 66
        Unknown or Not Reported
    		 13 3 16

    End points

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    End points reporting groups
    Reporting group title
    Recurrent glioblastoma
    Reporting group description
    		 Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.

    Reporting group title
    Recurrent non-glioblastoma CNS tumors
    Reporting group description
    		 Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.

    Primary: Objective response rate (ORR) in participants with recurrent glioblastoma based on Independent Central Review

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    End point title
    		 Objective response rate (ORR) in participants with recurrent glioblastoma based on Independent Central Review [1]
    End point description
    ORR=percentage of participants with a best overall response of complete response (CR) or partial response (PR) based on Response Assessment in Neuro-Oncology (RANO), determined by an independent centralized radiological review committee. CR requires: disappearance of all enhancing measurable/nonmeasurable disease for ≥4 weeks; no new lesions; stable/improved nonenhancing lesions; off corticosteroids/on physiologic replacement doses only and stable/improved clinically. PR requires: ≥50% decrease, compared with baseline (BL), in the sum of products of perpendicular diameters of measurable enhancing lesions sustained for ≥4 weeks; no progression of nonmeasurable disease; no new lesions; stable or improved nonenhancing lesions on same/lower dose of corticosteroids compared with BL scan; on a corticosteroid dose not greater than the dose at time of BL scan and stable/improved clinically. The 95% confidence interval (CI) was calculated based on the exact method for binomial distribution.
    End point type
    Primary
    End point timeframe
    up to 651 days
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Statistical analysis was not conducted for this endpoint.
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [2]
    0 [3]
    Units: percentage of participants
    number (confidence interval 95%)
        Confirmed tumor responses
    5.4 (1.49 to 13.27)
    ( to )
        Unconfirmed tumor responses
    8.1 (3.03 to 16.82)
    ( to )
    Notes
    [2] - Full Analysis Set: all enrolled participants who received ≥1 dose of pemigatinib
    [3] - Full Analysis Set: all enrolled participants who received ≥1 dose of pemigatinib
    No statistical analyses for this end point

    Secondary: ORR in participants with recurrent non-glioblastoma central nervous system tumors based on Independent Central Review

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    End point title
    		 ORR in participants with recurrent non-glioblastoma central nervous system tumors based on Independent Central Review
    End point description
    ORR was defined as the percentage of participants who achieved a best overall response of CR or PR based on RANO as determined by an independent centralized radiological review committee. CR requires all of the following: disappearance of all enhancing measurable/nonmeasurable disease sustained for ≥4 weeks; no new lesions; stable/improved nonenhancing lesions; off corticosteroids/on physiologic replacement doses only and stable/improved clinically. PR requires all of the following: ≥50% decrease, compared with baseline, in the sum of products of perpendicular diameters of all measurable enhancing lesions sustained for ≥4 weeks; no progression of nonmeasurable disease; no new lesions; stable or improved nonenhancing lesions on same/lower dose of corticosteroids compared with baseline scan; on a corticosteroid dose not greater than the dose at time of baseline scan and stable/improved clinically.
    End point type
    Secondary
    End point timeframe
    up to 784 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    0 [4]
    9 [5]
    Units: percentage of participants
    number (confidence interval 95%)
        Confirmed tumor responses
    ( to )
    22.2 (2.81 to 60.01)
        Unconfirmed tumor responses
    ( to )
    22.2 (2.81 to 60.01)
    Notes
    [4] - Full Analysis Set. The 95% CI was calculated based on the exact method for binomial distribution.
    [5] - Full Analysis Set. The 95% CI was calculated based on the exact method for binomial distribution.
    No statistical analyses for this end point

    Secondary: Duration of confirmed response based on Independent Central Review

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    End point title
    		 Duration of confirmed response based on Independent Central Review
    End point description
    Duration of response=time from first assessment of confirmed CR/PR until progressive disease (PD) (according to RANO per an independent centralized radiological review committee) or death (whichever occurred first). Progression was any of: ≥25% increase in sum of products of perpendicular diameters of enhancing lesions (compared with BL if no decrease) on stable/increasing doses of corticosteroids; a significant increase in T2/FLAIR nonenhancing lesions on stable/increasing doses of corticosteroids compared with BL scan or best response after initiation of therapy, not due to comorbid events; the appearance of any new lesions; clear progression of nonmeasurable lesions; or definite clinical deterioration not attributable to other causes apart from the tumor or to decrease in corticosteroid dose. The 95% CIs were calculated using the Brookmeyer and Crowley method. -9999, 9999=Values were not estimable because no responders had disease progression or died.
    End point type
    Secondary
    End point timeframe
    up to 784 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    4 [6]
    2 [7]
    Units: months
        median (confidence interval 95%)
    9999 (-9999 to 9999)
    9999 (-9999 to 9999)
    Notes
    [6] - Full Analysis Set. Only those participants with a confirmed CR or PR were analyzed.
    [7] - Full Analysis Set. Only those participants with a confirmed CR or PR were analyzed.
    No statistical analyses for this end point

    Secondary: Duration of unconfirmed response based on Independent Central Review

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    End point title
    		 Duration of unconfirmed response based on Independent Central Review
    End point description
    PFS was the time from the first dose until progressive disease (according to RANO per an independent centralized radiological review committee) or death (whichever occurred first). Progression was any of: ≥25% increase in sum of products of perpendicular diameters of enhancing lesions (compared with BL if no decrease) on stable/increasing doses of corticosteroids; a significant increase in T2/FLAIR nonenhancing lesions on stable/increasing doses of corticosteroids compared with BL scan or best response after initiation of therapy, not due to comorbid events; the appearance of any new lesions; clear progression of nonmeasurable lesions; or definite clinical deterioration not attributable to other causes apart from the tumor or to decrease in corticosteroid dose. The 95% CIs were calculated using the Brookmeyer and Crowley method. -9999, 9999=Values were not estimable because too few participants had disease progression or died.
    End point type
    Secondary
    End point timeframe
    up to 784 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    6 [8]
    2 [9]
    Units: months
        median (confidence interval 95%)
    9999 (1.74 to 9999)
    9999 (-9999 to 9999)
    Notes
    [8] - Full Analysis Set. Only those participants with an unconfirmed CR or PR were analyzed.
    [9] - Full Analysis Set. Only those participants with an unconfirmed CR or PR were analyzed.
    No statistical analyses for this end point

    Secondary: ORR as determined by investigator assessment

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    End point title
    		 ORR as determined by investigator assessment
    End point description
    ORR was defined as the percentage of participants who achieved an unconfirmed best overall response of CR or PR based on RANO as determined by investigator assessment. CR requires all of the following: disappearance of all enhancing measurable/nonmeasurable disease sustained for ≥4 weeks; no new lesions; stable/improved nonenhancing lesions; off corticosteroids/on physiologic replacement doses only and stable/improved clinically. PR requires all of the following: ≥50% decrease, compared with baseline, in the sum of products of perpendicular diameters of all measurable enhancing lesions sustained for ≥4 weeks; no progression of nonmeasurable disease; no new lesions; stable or improved nonenhancing lesions on same/lower dose of corticosteroids compared with baseline scan; on a corticosteroid dose not greater than the dose at time of baseline scan and stable/improved clinically. The 95% CIs were calculated based on the exact method for binomial distribution.
    End point type
    Secondary
    End point timeframe
    up to 784 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [10]
    9 [11]
    Units: percentage of participants
        number (confidence interval 95%)
    8.1 (3.03 to 16.82)
    22.2 (2.81 to 60.01)
    Notes
    [10] - Full Analysis Set
    [11] - Full Analysis Set
    No statistical analyses for this end point

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

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    End point title
    		 Number of participants with any treatment-emergent adverse event (TEAE)
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. A TEAE was defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of pemigatinib and within 30 days of the last dose of pemigatinib.
    End point type
    Secondary
    End point timeframe
    up to 814 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [12]
    9 [13]
    Units: participants
    73
    9
    Notes
    [12] - Safety Population: all enrolled participants who received at least 1 dose of pemigatinib
    [13] - Safety Population: all enrolled participants who received at least 1 dose of pemigatinib
    No statistical analyses for this end point

    Secondary: Number of participants with TEAEs leading to discontinuation of pemigatinib, pemigatinib dose interruption, and pemigatinib dose reduction

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    End point title
    		 Number of participants with TEAEs leading to discontinuation of pemigatinib, pemigatinib dose interruption, and pemigatinib dose reduction
    End point description
    An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. A TEAE was defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of pemigatinib and within 30 days of the last dose of pemigatinib.
    End point type
    Secondary
    End point timeframe
    up to 814 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [14]
    9 [15]
    Units: participants
        TEAEs leading to discontinuation of pemigatinib
    2
    0
        TEAEs leading to pemigatinib dose interruption
    22
    4
        TEAEs leading to pemigatinib dose reduction
    5
    2
    Notes
    [14] - Safety Population
    [15] - Safety Population
    No statistical analyses for this end point

    Secondary: Number of participants with any ≥Grade 3 TEAE

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    End point title
    		 Number of participants with any ≥Grade 3 TEAE
    End point description
    An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. A TEAE was defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of pemigatinib and within 30 days of the last dose of pemigatinib. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.
    End point type
    Secondary
    End point timeframe
    up to 814 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [16]
    9 [17]
    Units: participants
    27
    3
    Notes
    [16] - Safety Population
    [17] - Safety Population
    No statistical analyses for this end point

    Secondary: Disease control rate (DCR) based on Independent Central Review

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    End point title
    		 Disease control rate (DCR) based on Independent Central Review
    End point description
    DCR was defined as the percentage of participants who achieved a best overall response of CR, PR, or stable disease (SD) based on RANO as determined by an independent centralized radiological review committee. In the case of SD, measurements must have met the SD criteria after the date of the first dose at a minimum interval of 42 days. SD occurred if the participant didn’t qualify for CR, PR, or PD and required the following: stable nonenhancing (T2/FLAIR) lesions on same or lower dose of corticosteroids compared with baseline scan and clinically stable status. The 95% CIs were calculated based on the exact method for binomial distribution.
    End point type
    Secondary
    End point timeframe
    up to 784 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [18]
    9 [19]
    Units: percentage of participants
    number (confidence interval 95%)
        Confirmed tumor responses
    36.5 (25.60 to 48.49)
    66.7 (29.93 to 92.51)
        Unconfirmed tumor responses
    36.5 (25.60 to 48.49)
    66.7 (29.93 to 92.51)
    Notes
    [18] - Full Analysis Set
    [19] - Full Analysis Set
    No statistical analyses for this end point

    Secondary: Progression-free survival (PFS) based on Independent Central Review

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    End point title
    		 Progression-free survival (PFS) based on Independent Central Review
    End point description
    PFS was the time from the first dose until progressive disease (according to RANO and assessed by an independent centralized radiological review committee) or death (whichever occurred first). Progression was any of: ≥25% increase in sum of products of perpendicular diameters of enhancing lesions (compared with BL if no decrease) on stable/increasing doses of corticosteroids; a significant increase in T2/FLAIR nonenhancing lesions on stable/increasing doses of corticosteroids compared with BL scan or best response after initiation of therapy, not due to comorbid events; the appearance of any new lesions; clear progression of nonmeasurable lesions; or definite clinical deterioration not attributable to other causes apart from the tumor or to decrease in corticosteroid dose. The 95% CIs were calculated using the Brookmeyer and Crowley method. 9999=The median and the upper bound of the confidence interval were not estimable because too few participants had disease progression or died.
    End point type
    Secondary
    End point timeframe
    up to 784 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [20]
    9 [21]
    Units: months
        median (confidence interval 95%)
    2.07 (2.04 to 2.37)
    9999 (1.74 to 9999)
    Notes
    [20] - Full Analysis Set
    [21] - Full Analysis Set
    No statistical analyses for this end point

    Secondary: Overall survival

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    End point title
    		 Overall survival
    End point description
    Overall survival was defined as the time from the first dose of study drug to death due to any cause. The 95% CIs were calculated using the Brookmeyer and Crowley method. 9999=The upper bound of the confidence interval was not estimable because too few participants died.
    End point type
    Secondary
    End point timeframe
    up to 784 days
    End point values
    		 Recurrent glioblastoma Recurrent non-glioblastoma CNS tumors
    Number of subjects analysed
    74 [22]
    9 [23]
    Units: months
        median (confidence interval 95%)
    11.37 (9.23 to 14.85)
    24.08 (18.79 to 9999)
    Notes
    [22] - Full Analysis Set
    [23] - Full Analysis Set
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    up to 814 days
    Adverse event reporting additional description
    Adverse events have been reported for the Safety Population, comprised of all enrolled participants who received at least 1 dose of pemigatinib.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.1
    Reporting groups
    Reporting group title
    Recurrent glioblastoma
    Reporting group description
    		 Participants with recurrent glioblastoma with defined activating fibroblast growth factor receptor (FGFR) gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.

    Reporting group title
    Total
    Reporting group description
    		 Total

    Reporting group title
    Recurrent non-glioblastoma CNS tumors
    Reporting group description
    		 Participants with recurrent non-glioblastoma central nervous system (CNS) tumors with defined activating FGFR gene alterations received pemigatinib 13.5 milligrams (mg) once daily (QD) on a 2-week on-therapy and 1-week off-therapy schedule as long as they were receiving benefit and had not met any criteria for treatment discontinuation. Defined activating gene alterations included FGFR 1-3 fusions or rearrangements with intact FGFR kinase domain, or a defined set of FGFR 1-3 activating mutations or in-frame deletions.

    Serious adverse events
    		 Recurrent glioblastoma Total Recurrent non-glioblastoma CNS tumors
    Total subjects affected by serious adverse events
         subjects affected / exposed
    17 / 74 (22.97%)
    17 / 83 (20.48%)
    0 / 9 (0.00%)
         number of deaths (all causes)
    50
    52
    2
         number of deaths resulting from adverse events
    1
    1
    0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lumbar vertebral fracture
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Stroke-like migraine attacks after radiation therapy
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Wound dehiscence
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Cognitive disorder
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Aphasia
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Epilepsy
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hemiparesis
         subjects affected / exposed
    2 / 74 (2.70%)
    2 / 83 (2.41%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Haemorrhage intracranial
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Partial seizures with secondary generalisation
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Seizure
         subjects affected / exposed
    2 / 74 (2.70%)
    2 / 83 (2.41%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Faecaloma
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Intestinal perforation
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Confusional state
         subjects affected / exposed
    2 / 74 (2.70%)
    2 / 83 (2.41%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Herpes zoster
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 74 (2.70%)
    2 / 83 (2.41%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 83 (1.20%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    2 / 74 (2.70%)
    2 / 83 (2.41%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    1 / 2
    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
    		 Recurrent glioblastoma Total Recurrent non-glioblastoma CNS tumors
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    71 / 74 (95.95%)
    80 / 83 (96.39%)
    9 / 9 (100.00%)
    Vascular disorders
    Embolism
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Hypertension
         subjects affected / exposed
    5 / 74 (6.76%)
    5 / 83 (6.02%)
    0 / 9 (0.00%)
         occurrences all number
    6
    6
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    10 / 74 (13.51%)
    12 / 83 (14.46%)
    2 / 9 (22.22%)
         occurrences all number
    17
    20
    3
    Fatigue
         subjects affected / exposed
    21 / 74 (28.38%)
    24 / 83 (28.92%)
    3 / 9 (33.33%)
         occurrences all number
    23
    26
    3
    Influenza like illness
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Pyrexia
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    6
    7
    1
    Temperature regulation disorder
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    5
    6
    1
    Epistaxis
         subjects affected / exposed
    2 / 74 (2.70%)
    4 / 83 (4.82%)
    2 / 9 (22.22%)
         occurrences all number
    2
    4
    2
    Nasal dryness
         subjects affected / exposed
    0 / 74 (0.00%)
    2 / 83 (2.41%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    2
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Confusional state
         subjects affected / exposed
    6 / 74 (8.11%)
    6 / 83 (7.23%)
    0 / 9 (0.00%)
         occurrences all number
    7
    7
    0
    Depressed mood
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Depression
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Insomnia
         subjects affected / exposed
    3 / 74 (4.05%)
    4 / 83 (4.82%)
    1 / 9 (11.11%)
         occurrences all number
    3
    4
    1
    Investigations
    Amylase decreased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    4
    4
    Alanine aminotransferase increased
         subjects affected / exposed
    12 / 74 (16.22%)
    14 / 83 (16.87%)
    2 / 9 (22.22%)
         occurrences all number
    15
    18
    3
    Aspartate aminotransferase increased
         subjects affected / exposed
    4 / 74 (5.41%)
    6 / 83 (7.23%)
    2 / 9 (22.22%)
         occurrences all number
    5
    8
    3
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Blood creatinine increased
         subjects affected / exposed
    5 / 74 (6.76%)
    7 / 83 (8.43%)
    2 / 9 (22.22%)
         occurrences all number
    5
    7
    2
    Blood creatinine decreased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    Blood alkaline phosphatase increased
         subjects affected / exposed
    2 / 74 (2.70%)
    3 / 83 (3.61%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Blood parathyroid hormone decreased
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Blood urea decreased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Blood phosphorus increased
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    5
    6
    1
    Cortisol decreased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Haemoglobin decreased
         subjects affected / exposed
    3 / 74 (4.05%)
    4 / 83 (4.82%)
    1 / 9 (11.11%)
         occurrences all number
    3
    4
    1
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    Lipase decreased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    Lipase increased
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    7
    12
    5
    Lymphocyte count decreased
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    5
    5
    0
    Neutrophil count increased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    Transaminases increased
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    White blood cell count decreased
         subjects affected / exposed
    3 / 74 (4.05%)
    4 / 83 (4.82%)
    1 / 9 (11.11%)
         occurrences all number
    5
    6
    1
    Weight decreased
         subjects affected / exposed
    5 / 74 (6.76%)
    5 / 83 (6.02%)
    0 / 9 (0.00%)
         occurrences all number
    5
    5
    0
    Vitamin D decreased
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    4
    4
    0
    White blood cell count increased
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    2 / 74 (2.70%)
    3 / 83 (3.61%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Fall
         subjects affected / exposed
    6 / 74 (8.11%)
    6 / 83 (7.23%)
    0 / 9 (0.00%)
         occurrences all number
    8
    8
    0
    Nervous system disorders
    Aphasia
         subjects affected / exposed
    7 / 74 (9.46%)
    7 / 83 (8.43%)
    0 / 9 (0.00%)
         occurrences all number
    7
    7
    0
    Dysgeusia
         subjects affected / exposed
    8 / 74 (10.81%)
    10 / 83 (12.05%)
    2 / 9 (22.22%)
         occurrences all number
    8
    10
    2
    Headache
         subjects affected / exposed
    14 / 74 (18.92%)
    15 / 83 (18.07%)
    1 / 9 (11.11%)
         occurrences all number
    16
    20
    4
    Partial seizures
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Paraesthesia
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    5
    6
    1
    Seizure
         subjects affected / exposed
    10 / 74 (13.51%)
    11 / 83 (13.25%)
    1 / 9 (11.11%)
         occurrences all number
    13
    15
    2
    Taste disorder
         subjects affected / exposed
    2 / 74 (2.70%)
    4 / 83 (4.82%)
    2 / 9 (22.22%)
         occurrences all number
    2
    4
    2
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    2 / 74 (2.70%)
    3 / 83 (3.61%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Neutropenia
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    4
    4
    0
    Eye disorders
    Chalazion
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    Dry eye
         subjects affected / exposed
    8 / 74 (10.81%)
    11 / 83 (13.25%)
    3 / 9 (33.33%)
         occurrences all number
    9
    12
    3
    Eyelash thickening
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Eye pruritus
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Keratopathy
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Punctate keratitis
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    5
    5
    0
    Retinal exudates
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    Vision blurred
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    5
    6
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    7
    8
    1
    Abdominal discomfort
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    Anal haemorrhage
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Constipation
         subjects affected / exposed
    16 / 74 (21.62%)
    20 / 83 (24.10%)
    4 / 9 (44.44%)
         occurrences all number
    17
    21
    4
    Dyspepsia
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Diarrhoea
         subjects affected / exposed
    33 / 74 (44.59%)
    41 / 83 (49.40%)
    8 / 9 (88.89%)
         occurrences all number
    57
    69
    12
    Dry mouth
         subjects affected / exposed
    10 / 74 (13.51%)
    13 / 83 (15.66%)
    3 / 9 (33.33%)
         occurrences all number
    10
    14
    4
    Gastrooesophageal reflux disease
         subjects affected / exposed
    2 / 74 (2.70%)
    3 / 83 (3.61%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Gastritis
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Nausea
         subjects affected / exposed
    9 / 74 (12.16%)
    11 / 83 (13.25%)
    2 / 9 (22.22%)
         occurrences all number
    9
    11
    2
    Oral pain
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    4
    4
    0
    Stomatitis
         subjects affected / exposed
    14 / 74 (18.92%)
    14 / 83 (16.87%)
    0 / 9 (0.00%)
         occurrences all number
    21
    21
    0
    Vomiting
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    16 / 74 (21.62%)
    23 / 83 (27.71%)
    7 / 9 (77.78%)
         occurrences all number
    16
    25
    9
    Dry skin
         subjects affected / exposed
    11 / 74 (14.86%)
    16 / 83 (19.28%)
    5 / 9 (55.56%)
         occurrences all number
    14
    19
    5
    Dermatitis atopic
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Hair colour changes
         subjects affected / exposed
    2 / 74 (2.70%)
    3 / 83 (3.61%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Nail discolouration
         subjects affected / exposed
    3 / 74 (4.05%)
    4 / 83 (4.82%)
    1 / 9 (11.11%)
         occurrences all number
    3
    4
    1
    Madarosis
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Nail disorder
         subjects affected / exposed
    15 / 74 (20.27%)
    16 / 83 (19.28%)
    1 / 9 (11.11%)
         occurrences all number
    15
    16
    1
    Onychomadesis
         subjects affected / exposed
    4 / 74 (5.41%)
    5 / 83 (6.02%)
    1 / 9 (11.11%)
         occurrences all number
    4
    5
    1
    Onycholysis
         subjects affected / exposed
    4 / 74 (5.41%)
    8 / 83 (9.64%)
    4 / 9 (44.44%)
         occurrences all number
    4
    8
    4
    Onychoclasis
         subjects affected / exposed
    2 / 74 (2.70%)
    3 / 83 (3.61%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Onychalgia
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Palmar-plantar erythrodysaesthesia syndrome
         subjects affected / exposed
    5 / 74 (6.76%)
    5 / 83 (6.02%)
    0 / 9 (0.00%)
         occurrences all number
    8
    8
    0
    Endocrine disorders
    Gonadotrophin deficiency
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Hyperthyroidism
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Hypothalamo-pituitary disorder
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    6 / 74 (8.11%)
    7 / 83 (8.43%)
    1 / 9 (11.11%)
         occurrences all number
    7
    10
    3
    Back pain
         subjects affected / exposed
    1 / 74 (1.35%)
    3 / 83 (3.61%)
    2 / 9 (22.22%)
         occurrences all number
    2
    4
    2
    Myalgia
         subjects affected / exposed
    2 / 74 (2.70%)
    3 / 83 (3.61%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Muscular weakness
         subjects affected / exposed
    6 / 74 (8.11%)
    6 / 83 (7.23%)
    0 / 9 (0.00%)
         occurrences all number
    6
    6
    0
    Muscle spasms
         subjects affected / exposed
    5 / 74 (6.76%)
    6 / 83 (7.23%)
    1 / 9 (11.11%)
         occurrences all number
    5
    6
    1
    Pain in extremity
         subjects affected / exposed
    2 / 74 (2.70%)
    4 / 83 (4.82%)
    2 / 9 (22.22%)
         occurrences all number
    3
    6
    3
    Infections and infestations
    Conjunctivitis
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    COVID-19
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    4
    4
    0
    Ear infection
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Nail infection
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    3
    2
    Nasopharyngitis
         subjects affected / exposed
    1 / 74 (1.35%)
    2 / 83 (2.41%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Pharyngitis
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Paronychia
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 83 (1.20%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Skin infection
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    4
    4
    0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    4
    4
    0
    Decreased appetite
         subjects affected / exposed
    5 / 74 (6.76%)
    5 / 83 (6.02%)
    0 / 9 (0.00%)
         occurrences all number
    5
    5
    0
    Hypercalcaemia
         subjects affected / exposed
    2 / 74 (2.70%)
    4 / 83 (4.82%)
    2 / 9 (22.22%)
         occurrences all number
    4
    8
    4
    Hyperphosphataemia
         subjects affected / exposed
    54 / 74 (72.97%)
    61 / 83 (73.49%)
    7 / 9 (77.78%)
         occurrences all number
    89
    100
    11
    Hypomagnesaemia
         subjects affected / exposed
    3 / 74 (4.05%)
    4 / 83 (4.82%)
    1 / 9 (11.11%)
         occurrences all number
    4
    5
    1
    Hyponatraemia
         subjects affected / exposed
    5 / 74 (6.76%)
    5 / 83 (6.02%)
    0 / 9 (0.00%)
         occurrences all number
    7
    7
    0
    Hypophosphataemia
         subjects affected / exposed
    16 / 74 (21.62%)
    20 / 83 (24.10%)
    4 / 9 (44.44%)
         occurrences all number
    21
    27
    6
    Vitamin D deficiency
         subjects affected / exposed
    4 / 74 (5.41%)
    4 / 83 (4.82%)
    0 / 9 (0.00%)
         occurrences all number
    4
    4
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Nov 2021
    The primary purpose of this amendment was to incorporate updates based on regulatory agency review of the Protocol.
    19 Jan 2023
    The primary purpose of this amendment was to incorporate participants from Cohort C with activating mutations into Cohorts A and B.
    26 Jul 2024
    The main purpose of this amendment was to indicate that at the end of the study, participants who continued to receive the study drug would have continued access to it in accordance with local regulations or via Incyte.

    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.
    The study was terminated by the sponsor after the prespecified interim analysis did not meet the futility boundary.
    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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