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    Clinical Trial Results:
    A Phase 2, Open-Label, Monotherapy, Multicenter Study to Evaluate the Efficacy and Safety of INCB054828 in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement

    Summary
    EudraCT number
    		 2016-002596-10
    Trial protocol
    		 GB   DE   AT   ES   BE   IT  
    Global end of trial date
    30 Oct 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    20 Nov 2025
    First version publication date
    20 Nov 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    INCB 54828-203
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03011372
    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
    30 Oct 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Oct 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to evaluate the efficacy of pemigatinib in participants with myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR) 1 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
    25 Apr 2017
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Efficacy
    Long term follow-up duration
    		 24 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Italy: 7
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    Spain: 2
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 18
    Worldwide total number of subjects
    47
    EEA total number of subjects
    24
    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)
    29
    From 65 to 84 years
    18
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted at 21 study centers in Belgium, Canada, France, Germany, Italy, Japan, Spain, 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
    Arm title
    		 Pemigatinib 13.5 mg
    Arm description
    		 Pemigatinib 13.5 milligrams (mg) was self-administered once daily (QD) as oral tablets on an intermittent dosing (ID) schedule or continuous dosing (CD) schedule. Participants started pemigatinib 13.5 mg on the ID schedule (i.e., 2 weeks on/1 week off) as per the initial Protocol and on the CD schedule in 21-day cycles following a Protocol amendment.
    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
    2 and 4.5 mg unit dose strength tablets

    Number of subjects in period 1
    		Pemigatinib 13.5 mg
    Started
    47
    Completed
    0
    Not completed
    47
         Consent withdrawn by subject
    7
         Moved to Commercial Supply of Investigational Drug
    2
         Received Chemotherapy for Stem Cell Transplant
    1
         Sponsor's Decision
    2
         Death
    16
         Sponsor Decision
    17
         Lost to follow-up
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pemigatinib 13.5 mg
    Reporting group description
    		 Pemigatinib 13.5 milligrams (mg) was self-administered once daily (QD) as oral tablets on an intermittent dosing (ID) schedule or continuous dosing (CD) schedule. Participants started pemigatinib 13.5 mg on the ID schedule (i.e., 2 weeks on/1 week off) as per the initial Protocol and on the CD schedule in 21-day cycles following a Protocol amendment.

    Reporting group values
    		 Pemigatinib 13.5 mg Total
    Number of subjects
    		 47 47
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 29 29
        From 65-84 years
    		 18 18
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 58.3 ( 13.25 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 25 25
        Male
    		 22 22
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 30 30
        Black or African American
    		 4 4
        Asian
    		 4 4
        Missing
    		 5 5
        Not Provided
    		 2 2
        White/Caucasian and American-Indian Alaska Native
    		 1 1
        African
    		 1 1
    Race/Ethnicity, Customized
    Units: Subjects
        Hispanic or Latino
    		 1 1
        Not Hispanic or Latino
    		 33 33
        Not Reported
    		 8 8
        Unknown
    		 1 1
        Captured as "Other" in Database
    		 4 4

    End points

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    End points reporting groups
    Reporting group title
    Pemigatinib 13.5 mg
    Reporting group description
    		 Pemigatinib 13.5 milligrams (mg) was self-administered once daily (QD) as oral tablets on an intermittent dosing (ID) schedule or continuous dosing (CD) schedule. Participants started pemigatinib 13.5 mg on the ID schedule (i.e., 2 weeks on/1 week off) as per the initial Protocol and on the CD schedule in 21-day cycles following a Protocol amendment.

    Primary: Percentage of participants who achieved complete response (CR) as determined by investigator assessment according to the response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement

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    End point title
    		 Percentage of participants who achieved complete response (CR) as determined by investigator assessment according to the response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement [1]
    End point description
    CR was defined as the presence of all of the following improvements: (1) bone marrow: ≤5% myeloblasts (including monocytic blast equivalent) and no lymphoblasts, with normal maturation of all cell lines, and return to age-adjusted normal cellularity; (2) osteomyelofibrosis absent or equal to “mild reticulin fibrosis” (Grade 1 or less fibrosis); (3) peripheral blood: white blood cells (WBC) ≤10 x 10^9 cells/Liter (L); hemoglobin (Hgb) ≥11 grams per deciliter (g/dL); platelets ≥100 x 10^9/L and ≤450 x 10^9/L; neutrophils ≥1.0 x 10^9/L; blasts = 0%; neutrophil precursors reduced to ≤2%; monocytes ≤1 x 10^9/L; eosinophils ≤0.5 x 10^9/L; (4) extramedullary disease: complete resolution of extramedullary disease present before therapy (e.g., lymphadenopathy), including palpable hepatosplenomegaly. Persistent low-level dysplasia was permitted given subjectivity of assignment of dysplasia. Response criteria by investigator assessment were the same for chronic phase (CP) and blast phase (BP).
    End point type
    Primary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    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
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [2]
    Units: percentage of participants
        number (confidence interval 95%)
    68.9 (53.35 to 81.83)
    Notes
    [2] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieved a best overall response of complete response (CR) or partial response (PR) as determined by investigator and CRC assessment according to the response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement

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    End point title
    		 Percentage of participants who achieved a best overall response of complete response (CR) or partial response (PR) as determined by investigator and CRC assessment according to the response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement
    End point description
    CR=all of the following improvements: (1) bone marrow: ≤5% myeloblasts (including monocytic blast equivalent) and no lymphoblasts, with normal maturation of all cell lines, and return to age-adjusted normal cellularity; (2) osteomyelofibrosis absent/equal to “mild reticulin fibrosis”; (3) WBC ≤10 x 10^9 cells/L; Hgb ≥11 g/dL; platelets ≥100 x 10^9/L, ≤450 x 10^9/L; neutrophils ≥1.0 x 10^9/L; blasts=0%; neutrophil precursors reduced to ≤2%; monocytes ≤1 x 10^9/L; eosinophils ≤0.5 x 10^9/L; (4) extramedullary disease: complete resolution of extramedullary disease present pre-therapy, including palpable hepatosplenomegaly. Persistent low-level dysplasia was permitted. PR=all of the following improvements: (1) reduction of bone marrow blasts/blast equivalents by 50%, but remaining >5% of cellularity (except in cases with ≤5% bone marrow blasts at baseline); (2) normalization of peripheral blood indices per CR Criterion 3; (3) extra medullary disease response of CMR/CR or PMR/PR.
    End point type
    Secondary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [3]
    Units: percentage of participants
    number (confidence interval 95%)
        Investigator assessment
    77.8 (62.91 to 88.80)
        CRC assessment
    75.6 (60.46 to 87.12)
    Notes
    [3] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieved a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation and CRC assessment

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    End point title
    		 Percentage of participants who achieved a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation and CRC assessment
    End point description
    CCyR was defined as 0% 8p11 translocated metaphases as seen on classic karyotyping with minimal of 20 metaphases, or fluorescence in situ hybridization (FISH). Loss of cytogenetic burden of disease (via FISH or classic karyotyping) was required to reach CCyR. Confidence intervals were calculated based on the exact method for binomial distribution.
    End point type
    Secondary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [4]
    Units: percentage of participants
    number (confidence interval 95%)
        Investigator assessment
    73.3 (58.06 to 85.40)
        CRC assessment
    73.3 (58.06 to 85.40)
    Notes
    [4] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieved a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation and CRC assessment

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    End point title
    		 Percentage of participants who achieved a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation and CRC assessment
    End point description
    PCyR was defined as the decrease from baseline of 50% or more 8p11 translocated metaphases as seen on classic karyotyping with minimal of 20 metaphases, or FISH.
    End point type
    Secondary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [5]
    Units: percentage of participants
    number (confidence interval 95%)
        Investigator assessment
    8.9 (2.48 to 21.22)
        CRC assessment
    15.6 (6.49 to 29.46)
    Notes
    [5] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Secondary: Duration of complete response

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    End point title
    		 Duration of complete response
    End point description
    Duration of complete response was defined as the time from the first assessment of complete response to the earlier of the date of first worsening assessment after complete response or death due to any cause. Confidence intervals were calculated using the Brookmeyer and Crowley's method. 9999=The median and the upper limit of the confidence interval were not estimable because too few participants had worsening assessment after response or death.
    End point type
    Secondary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    31 [6]
    Units: months
    median (confidence interval 95%)
        Investigator assessment
    53.29 (12.22 to 9999)
        CRC assessment
    9999 (27.86 to 9999)
    Notes
    [6] - Full Efficacy-Evaluable Population. Only participants with a complete response were analyzed.
    No statistical analyses for this end point

    Secondary: Duration of response

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    End point title
    		 Duration of response
    End point description
    Duration of response was defined as the time from the first assessment of complete response or partial response to the earlier of the date of first worsening assessment after response or death due to any cause. Confidence intervals were calculated based on the exact method for binomial distribution. 9999=The median and the upper limit of the confidence interval were not estimable because too few participants had events of loss of response (disease progression) or death.
    End point type
    Secondary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [7]
    Units: months
    median (confidence interval 95%)
        Investigator assessment, n=35
    53.29 (15.51 to 9999)
        CRC assessment, n=34
    9999 (27.86 to 9999)
    Notes
    [7] - Full Efficacy-Evaluable Population. Only participants with CR or PR were analyzed.
    No statistical analyses for this end point

    Secondary: Progression-free survival (PFS)

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    End point title
    		 Progression-free survival (PFS)
    End point description
    PFS was defined as the time from the first date of taking study drug until the date of disease progression or until death due to any cause, whichever was earlier. Disease progression was defined as the combination of 2 major criteria, 1 major and 2 minor criteria, or 3 minor criteria from the following lists. Major criteria: (1) increase in blast count; (2) evidence of cytogenetic evolution (re-appearance of a previously present or appearance of a new cytogenetic abnormality, or increase in cytogenetic burden of disease); (3) new or worsening extramedullary disease (worsening splenomegaly or extramedullary disease outside of the spleen). Minor criteria: (1) transfusion dependence; (2) significant loss of maximal response on cytopenias ≥50% decrement from maximum remission/response in granulocytes or platelets; (3) reduction in Hgb by ≥1.5g/dL from best response or from baseline as noted on complete blood count; (4) evidence of clonal evolution (molecular). 9999=not estimable.
    End point type
    Secondary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [8]
    Units: months
    median (confidence interval 95%)
        Investigator assessment
    73.89 (54.74 to 9999)
        CRC assessment
    73.89 (29.17 to 9999)
    Notes
    [8] - Full Efficacy-Evaluable Population
    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 as the time from the first day of taking study drug until death due to any cause. Confidence intervals were calculated using the Brookmeyer and Crowley's method. Participants without death observed at the time of the analysis were censored at the last date known to be alive. 9999=The median and the upper limit of the confidence interval were not estimable because too few participants died.
    End point type
    Secondary
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [9]
    Units: months
        median (confidence interval 95%)
    9999 (54.74 to 9999)
    Notes
    [9] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

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

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    End point title
    		 Number of participants with any treatment-emergent adverse event (TEAE)
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurs after a participant provides informed consent. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.
    End point type
    Secondary
    End point timeframe
    up to 2543 days
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    47 [10]
    Units: participants
    47
    Notes
    [10] - Safety Population: all enrolled participants who received at least 1 dose of study drug
    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 considered drug related, that occurs after a participant provides informed consent. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 Grades 1 through 4. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: moderate; minimal, local, or noninvasive intervention indicated; limiting 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 intervention indicated.
    End point type
    Secondary
    End point timeframe
    up to 2543 days
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    47 [11]
    Units: participants
    39
    Notes
    [11] - Safety Population
    No statistical analyses for this end point

    Other pre-specified: Percentage of participants who achieved CR as determined by central review committee (CRC) assessment

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    End point title
    		 Percentage of participants who achieved CR as determined by central review committee (CRC) assessment
    End point description
    In addition, responses were assessed by CRC based on Myeloid/Lymphoid Neoplasm International Working Group (MLN IWG) response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions. Confidence intervals were calculated based on the exact method for binomial distribution.
    End point type
    Other pre-specified
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [12]
    Units: percentage of participants
        number (confidence interval 95%)
    68.9 (53.35 to 81.83)
    Notes
    [12] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Other pre-specified: Percentage of participants who achieved a best overall response of CR or PR as determined by CRC assessment

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    End point title
    		 Percentage of participants who achieved a best overall response of CR or PR as determined by CRC assessment
    End point description
    In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions. Confidence intervals were calculated based on the exact method for binomial distribution.
    End point type
    Other pre-specified
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [13]
    Units: percentage of participants
        number (confidence interval 95%)
    75.6 (60.46 to 87.12)
    Notes
    [13] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Other pre-specified: Percentage of participants who achieved a CCyR as assessed by CRC assessment

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    End point title
    		 Percentage of participants who achieved a CCyR as assessed by CRC assessment
    End point description
    In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions. Confidence intervals were calculated based on the exact method for binomial distribution.
    End point type
    Other pre-specified
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [14]
    Units: percentage of participants
        number (confidence interval 95%)
    73.3 (58.06 to 85.40)
    Notes
    [14] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Other pre-specified: Percentage of participants who achieved a PCyR as assessed by CRC assessment

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    End point title
    		 Percentage of participants who achieved a PCyR as assessed by CRC assessment
    End point description
    In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions. Confidence intervals were calculated based on the exact method for binomial distribution.
    End point type
    Other pre-specified
    End point timeframe
    up to 2513 days (120 21-day treatment cycles)
    End point values
    		 Pemigatinib 13.5 mg
    Number of subjects analysed
    45 [15]
    Units: percentage of participants
        number (confidence interval 95%)
    15.6 (6.49 to 29.46)
    Notes
    [15] - Full Efficacy-Evaluable Population
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    up to 2730 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 study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    Pemigatinib 13.5 mg
    Reporting group description
    		 Pemigatinib 13.5 milligrams (mg) was self-administered once daily (QD) as oral tablets on an intermittent dosing (ID) schedule or continuous dosing (CD) schedule. Participants started pemigatinib 13.5 mg on the ID schedule (i.e., 2 weeks on/1 week off) as per the initial Protocol and on the CD schedule in 21-day cycles following a Protocol amendment.

    Serious adverse events
    		 Pemigatinib 13.5 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    27 / 47 (57.45%)
         number of deaths (all causes)
    16
         number of deaths resulting from adverse events
    6
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Malignant neoplasm progression
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Vascular disorders
    Aortic stenosis
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General physical health deterioration
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pain
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Multiple organ dysfunction syndrome
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Pyrexia
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Oedema genital
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Confusional state
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Hip fracture
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Humerus fracture
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Femur fracture
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Fall
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Intentional overdose
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Subdural haematoma
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin abrasion
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    2 / 47 (4.26%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Atrial flutter
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Atrioventricular block complete
         subjects affected / exposed
    2 / 47 (4.26%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Heart failure with preserved ejection fraction
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    2 / 47 (4.26%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 1
    Nervous system disorders
    Syncope
         subjects affected / exposed
    2 / 47 (4.26%)
         occurrences causally related to treatment / all
    1 / 3
         deaths causally related to treatment / all
    0 / 0
    Seizure
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Upper gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypertransaminasaemia
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences causally related to treatment / all
    1 / 4
         deaths causally related to treatment / all
    0 / 1
    Bladder tamponade
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bladder obstruction
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nephrolithiasis
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Postrenal failure
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Urinary retention
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Spinal pain
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Spinal flattening
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Erysipelas
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Endocarditis
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Corneal abscess
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia pseudomonal
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia aspiration
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 47 (4.26%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Infection
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Urinary tract infection
         subjects affected / exposed
    2 / 47 (4.26%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Pyelonephritis
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Stenotrophomonas infection
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pseudomonas infection
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Calciphylaxis
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypercalcaemia
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Hyperphosphataemia
         subjects affected / exposed
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Pemigatinib 13.5 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    46 / 47 (97.87%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    8
    Hypotension
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    6
    General disorders and administration site conditions
    Chills
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    5
    Asthenia
         subjects affected / exposed
    10 / 47 (21.28%)
         occurrences all number
    12
    Fatigue
         subjects affected / exposed
    11 / 47 (23.40%)
         occurrences all number
    15
    Oedema peripheral
         subjects affected / exposed
    11 / 47 (23.40%)
         occurrences all number
    15
    Non-cardiac chest pain
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Pyrexia
         subjects affected / exposed
    10 / 47 (21.28%)
         occurrences all number
    19
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    9 / 47 (19.15%)
         occurrences all number
    11
    Dyspnoea
         subjects affected / exposed
    10 / 47 (21.28%)
         occurrences all number
    15
    Epistaxis
         subjects affected / exposed
    16 / 47 (34.04%)
         occurrences all number
    23
    Oropharyngeal pain
         subjects affected / exposed
    9 / 47 (19.15%)
         occurrences all number
    12
    Nasal congestion
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    7
    Upper-airway cough syndrome
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    7
    Confusional state
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Depression
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Insomnia
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    8
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    11
    Blood creatinine increased
         subjects affected / exposed
    8 / 47 (17.02%)
         occurrences all number
    11
    Blood alkaline phosphatase increased
         subjects affected / exposed
    11 / 47 (23.40%)
         occurrences all number
    14
    Aspartate aminotransferase increased
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    10
    Weight decreased
         subjects affected / exposed
    8 / 47 (17.02%)
         occurrences all number
    9
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    8
    Fall
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    10
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Nervous system disorders
    Ageusia
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    7
    Dizziness
         subjects affected / exposed
    9 / 47 (19.15%)
         occurrences all number
    16
    Dysgeusia
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    7
    Headache
         subjects affected / exposed
    12 / 47 (25.53%)
         occurrences all number
    15
    Hypogeusia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Paraesthesia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Neuropathy peripheral
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Memory impairment
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    6
    Tremor
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    16 / 47 (34.04%)
         occurrences all number
    27
    Leukopenia
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    6
    Neutropenia
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    9
    Thrombocytopenia
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    8
    Eye disorders
    Conjunctival hyperaemia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Corneal erosion
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Detachment of retinal pigment epithelium
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Dry eye
         subjects affected / exposed
    20 / 47 (42.55%)
         occurrences all number
    22
    Blepharitis
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    4
    Cataract
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    5
    Entropion
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Epiretinal membrane
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    4
    Eye discharge
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Eye pain
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Keratitis
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    6
    Lacrimation increased
         subjects affected / exposed
    8 / 47 (17.02%)
         occurrences all number
    9
    Photophobia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Macular oedema
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    5
    Punctate keratitis
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Visual acuity reduced
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Vision blurred
         subjects affected / exposed
    10 / 47 (21.28%)
         occurrences all number
    10
    Trichiasis
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    8
    Subretinal fluid
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    6
    Aphthous ulcer
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Abdominal pain
         subjects affected / exposed
    16 / 47 (34.04%)
         occurrences all number
    25
    Abdominal pain upper
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    9
    Dyspepsia
         subjects affected / exposed
    10 / 47 (21.28%)
         occurrences all number
    11
    Constipation
         subjects affected / exposed
    20 / 47 (42.55%)
         occurrences all number
    29
    Diarrhoea
         subjects affected / exposed
    29 / 47 (61.70%)
         occurrences all number
    51
    Dry mouth
         subjects affected / exposed
    19 / 47 (40.43%)
         occurrences all number
    20
    Dysphagia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Gastrooesophageal reflux disease
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    5
    Haemorrhoids
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    5
    Oral pain
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    4
    Nausea
         subjects affected / exposed
    17 / 47 (36.17%)
         occurrences all number
    19
    Mouth ulceration
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    6
    Vomiting
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    19
    Stomatitis
         subjects affected / exposed
    21 / 47 (44.68%)
         occurrences all number
    51
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    13
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    26 / 47 (55.32%)
         occurrences all number
    29
    Dermatitis
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Dry skin
         subjects affected / exposed
    12 / 47 (25.53%)
         occurrences all number
    13
    Erythema
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    7
    Hyperhidrosis
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    6
    Hyperkeratosis
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    4
    Ingrowing nail
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    5
    Palmar-plantar erythrodysaesthesia syndrome
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    16
    Onychomadesis
         subjects affected / exposed
    8 / 47 (17.02%)
         occurrences all number
    8
    Onycholysis
         subjects affected / exposed
    8 / 47 (17.02%)
         occurrences all number
    9
    Onychoclasis
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    4
    Onychalgia
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    6
    Nail bed tenderness
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    4
    Nail discolouration
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    4
    Nail disorder
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    5
    Nail dystrophy
         subjects affected / exposed
    8 / 47 (17.02%)
         occurrences all number
    8
    Skin exfoliation
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    5
    Rash
         subjects affected / exposed
    12 / 47 (25.53%)
         occurrences all number
    19
    Pruritus
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    9
    Skin ulcer
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    9
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    5
    Dysuria
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    5
    Pollakiuria
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    14
    Arthralgia
         subjects affected / exposed
    10 / 47 (21.28%)
         occurrences all number
    20
    Flank pain
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Pain in extremity
         subjects affected / exposed
    13 / 47 (27.66%)
         occurrences all number
    16
    Neck pain
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    6
    Muscle spasms
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    7
    Muscular weakness
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    4
    Musculoskeletal pain
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    5
    Myalgia
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    9
    Infections and infestations
    Conjunctivitis
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    7
    Cystitis
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    12
    Candida infection
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    COVID-19
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Bronchitis
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Erysipelas
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    5
    Fungal infection
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Pharyngitis
         subjects affected / exposed
    6 / 47 (12.77%)
         occurrences all number
    6
    Paronychia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Oral herpes
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Oral candidiasis
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Nasopharyngitis
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Pneumonia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    4
    Urinary tract infection
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    11
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Sinusitis
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    7
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    16 / 47 (34.04%)
         occurrences all number
    19
    Dehydration
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Hypercalcaemia
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    8
    Hyperglycaemia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Hyperphosphataemia
         subjects affected / exposed
    35 / 47 (74.47%)
         occurrences all number
    59
    Hyperuricaemia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    3
    Hypoalbuminaemia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    6
    Hypocalcaemia
         subjects affected / exposed
    4 / 47 (8.51%)
         occurrences all number
    5
    Hypokalaemia
         subjects affected / exposed
    7 / 47 (14.89%)
         occurrences all number
    14
    Hypomagnesaemia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    5
    Hyponatraemia
         subjects affected / exposed
    3 / 47 (6.38%)
         occurrences all number
    8
    Hypophosphataemia
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    7
    Vitamin D deficiency
         subjects affected / exposed
    5 / 47 (10.64%)
         occurrences all number
    6

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Aug 2016
    The primary purpose of this Protocol Amendment was to provide additional clinical data from the ongoing INCB 53828-101 study, refine the inclusion criteria to better define the population, and to amend the pharmacokinetic (PK) and electrocardiogram (ECG) sampling timepoint. requirements
    12 Dec 2016
    The primary purpose of this amendment was to update language based on Regulatory Agencies' comments. Updates included but were not limited to clarification of inclusion and exclusion criteria and guidance for dose reductions.
    17 May 2018
    The purpose of this amendment was to add language to allow for continuous administration of INCB054828. Updated clinical data were added to support continuous administration. Other modifications were made based on new preclinical and/or clinical data.
    22 May 2019
    The main purpose of this amendment was to modify the primary and secondary study efficacy endpoints and to revise the proposed response criteria. Other modifications were made to include treatment-naive participants and to update the Protocol with program-level standard language.
    02 Jul 2020
    The main purpose of this amendment was to include updated language for comprehensive eye examination, per Regulatory Agency feedback. Other modifications were made to include a long-term treatment visit schedule option for participants with stable response, to update the Protocol with program-level standard language and post-transplant follow-up, and to provide additional language clarifications.
    13 Jul 2023
    The main purpose of this amendment was to modify the long-term treatment visit schedule to start at Cycle 18 and to update the assessments in the post-transplant follow-up period. This amendment also incorporated changes from country-specific adaptations.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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