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    Clinical Trial Results:
    A Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib Plus Pembrolizumab Versus Pemigatinib Alone Versus Standard of Care as First Line Treatment for Metastatic or Unresectable Urothelial Carcinoma in Cisplatin-Ineligible Participants Whose Tumors Express FGFR3 Mutation or Rearrangement (FIGHT-205)

    Summary
    EudraCT number
    		 2019-000721-50
    Trial protocol
    		 ES   BE   DE   FI   FR   PL   AT   PT   HU   BG   GB   IT  
    Global end of trial date
    18 Apr 2021

    Results information
    Results version number
    		 v2(current)
    This version publication date
    11 Jun 2022
    First version publication date
    02 Apr 2022
    Other versions
    		 v1
    Version creation reason

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    INCB 54828-205
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04003610
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Incyte Corporation
    Sponsor organisation address
    1801 Augustine Cutoff Drive, Wilmington, United States, 19803
    Public contact
    Study Director, Incyte Corporation, 1 18554633463, medinfo@incyte.com
    Scientific contact
    Study Director, Incyte Corporation, 1 18554633463, 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
    18 Apr 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Apr 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The purpose of this study was to evaluate the safety and efficacy of pemigatinib plus pembrolizumab or pemigatinib alone versus the standard of care for participants with metastatic or unresectable urothelial carcinoma who were not eligible to receive cisplatin, were harboring FGFR3 mutation or rearrangement, and who had not received prior treatment.
    Protection of trial subjects
    This study was performed in accordance with ethical principles that have their origin in the Declaration of Helsinki and conducted in adherence to the study Protocol, applicable Good Clinical Practices, and applicable laws and country-specific regulations in which the study was being conducted. An IDMC was formed, but because of slow recruitment and low enrollment (n=7), it was never convened.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    14 May 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Japan: 3
    Worldwide total number of subjects
    7
    EEA total number of subjects
    4
    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)
    1
    From 65 to 84 years
    6
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted (enrolled participants) at 7 sites located in France, Italy, Spain, and Japan.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg
    Arm description
    		 Pemigatinib was self-administered at 13.5 mg QD PO until disease progression during each 21-day treatment cycle. Participants not reaching the target serum phosphate level (> 5.5 mg/dL) during Cycle 1 despite being study drug compliant increased the daily pemigatinib dose to 18 mg starting at Cycle 2 as long as they were not experiencing an ongoing Grade 2 or higher treatment-related AE. Pembrolizumab 200 mg was administered intravenously (IV) on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.
    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
    13.5 mg daily dose; administered once a day every day for each 21-day cycle

    Investigational medicinal product name
    Pembrolizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    200 mg daily; administered on Day 1 of each 21-day cycle for up to 35 cycles

    Arm title
    		 Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Arm description
    		 Participants received either gemcitabine plus carboplatin or pembrolizumab as standard of care. Participants received gemcitabine 1000 mg/meters squared (m^2) IV over 30 minutes on Days 1 and 8 of each 3-week treatment cycle, followed by carboplatin (dosed to target area under the concentration-time curve [AUC] of 5 mg/milliliters [mL]/minute [min] or 4.5 mg/mL/min if required per local guidelines) on Day 1 or Day 2 of each 3-week treatment cycle. Treatment continued for 4 to 6 cycles (or per institutional standards) until disease progression or intolerable toxicity. Pembrolizumab 200 mg was administered IV on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Carboplatin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    AUC 5 mg/mL/min (or AUC 4.5 mg/mL/min if required per local guidelines); administered on Day 1 or Day 2 of each 21-day cycle for up to 6 cycles (minimum of 4 cycles) in absence of progression or per institutional standard

    Investigational medicinal product name
    Gemcitabine
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    1000 mg/m^2 per administration; administered over 30 minutes on Days 1 and 8 of each 3-week cycle for up to 6 cycles (minimum of 4 cycles) in absence of progression or per institutional standard

    Number of subjects in period 1
    		Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Started
    1
    6
    Completed
    0
    0
    Not completed
    1
    6
         Adverse event, serious fatal
    -
    3
         Study Terminated by Sponsor
    -
    3
         Lost to follow-up
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pemigatinib 13.5 mg plus pembrolizumab 200 mg
    Reporting group description
    		 Pemigatinib was self-administered at 13.5 mg QD PO until disease progression during each 21-day treatment cycle. Participants not reaching the target serum phosphate level (> 5.5 mg/dL) during Cycle 1 despite being study drug compliant increased the daily pemigatinib dose to 18 mg starting at Cycle 2 as long as they were not experiencing an ongoing Grade 2 or higher treatment-related AE. Pembrolizumab 200 mg was administered intravenously (IV) on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.

    Reporting group title
    Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Reporting group description
    		 Participants received either gemcitabine plus carboplatin or pembrolizumab as standard of care. Participants received gemcitabine 1000 mg/meters squared (m^2) IV over 30 minutes on Days 1 and 8 of each 3-week treatment cycle, followed by carboplatin (dosed to target area under the concentration-time curve [AUC] of 5 mg/milliliters [mL]/minute [min] or 4.5 mg/mL/min if required per local guidelines) on Day 1 or Day 2 of each 3-week treatment cycle. Treatment continued for 4 to 6 cycles (or per institutional standards) until disease progression or intolerable toxicity. Pembrolizumab 200 mg was administered IV on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.

    Reporting group values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab Total
    Number of subjects
    		 1 6 7
    Age Categorical
    Units: participants
        In Utero
    		 0 0 0
        Pre-term newborn - gestational age < 37 wk
    		 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)
    		 0 1 1
        Elderly (From 65-84 years)
    		 1 5 6
        Elderly 85 years and over
    		 0 0 0
    Sex: Female, Male
    Due to low participant enrollment in the pemigatinib 13.5 mg plus pembrolizumab 200 mg arm, data are not being reported, as doing so may risk participant identification.
    Units: Subjects
        Female
    		 0 4 4
        Male
    		 0 2 2
        Unknown or Not Reported
    		 1 0 1
    Race (NIH/OMB)
    Due to low participant enrollment in the pemigatinib 13.5 mg plus pembrolizumab 200 mg arm, data are not being reported, as doing so may risk participant identification.
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 3 3
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 0 0
        White
    		 0 3 3
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 1 0 1
    Ethnicity (NIH/OMB)
    Due to low participant enrollment in the pemigatinib 13.5 mg plus pembrolizumab 200 mg arm, data are not being reported, as doing so may risk participant identification.
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 0 5 5
        Unknown or Not Reported
    		 1 1 2

    End points

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    End points reporting groups
    Reporting group title
    Pemigatinib 13.5 mg plus pembrolizumab 200 mg
    Reporting group description
    		 Pemigatinib was self-administered at 13.5 mg QD PO until disease progression during each 21-day treatment cycle. Participants not reaching the target serum phosphate level (> 5.5 mg/dL) during Cycle 1 despite being study drug compliant increased the daily pemigatinib dose to 18 mg starting at Cycle 2 as long as they were not experiencing an ongoing Grade 2 or higher treatment-related AE. Pembrolizumab 200 mg was administered intravenously (IV) on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.

    Reporting group title
    Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Reporting group description
    		 Participants received either gemcitabine plus carboplatin or pembrolizumab as standard of care. Participants received gemcitabine 1000 mg/meters squared (m^2) IV over 30 minutes on Days 1 and 8 of each 3-week treatment cycle, followed by carboplatin (dosed to target area under the concentration-time curve [AUC] of 5 mg/milliliters [mL]/minute [min] or 4.5 mg/mL/min if required per local guidelines) on Day 1 or Day 2 of each 3-week treatment cycle. Treatment continued for 4 to 6 cycles (or per institutional standards) until disease progression or intolerable toxicity. Pembrolizumab 200 mg was administered IV on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.

    Primary: Progression-free survival (PFS)

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    End point title
    		 Progression-free survival (PFS) [1]
    End point description
    PFS was defined as the time from the randomization date until the date of disease progression (as measured by a blinded independent central review [BICR] per Response Evaluation Criteria in Solid Tumors version 1.1 [RECIST v1.1]) or death due to any cause, whichever occurred first. -9999/9999=The upper/lower limits of the confidence interval were not estimable because too few participants had disease progression or died.
    End point type
    Primary
    End point timeframe
    up to 130 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: The Sponsor made a decision unrelated to safety to halt study enrollment. Due to early termination of the study with only 7 participants, no analysis of this endpoint was done.
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1
    6
    Units: days
        median (confidence interval 95%)
    1.81 (-9999 to 9999)
    2.12 (1.51 to 9999)
    No statistical analyses for this end point

    Secondary: Overall survival (OS)

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    End point title
    		 Overall survival (OS)
    End point description
    OS was defined as the time from the date of randomization until death due to any cause. -9999/9999=The median and upper/lower limits of the confidence interval were not estimable because too few participants died.
    End point type
    Secondary
    End point timeframe
    up to 225 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1
    6
    Units: days
        median (confidence interval 95%)
    9999 (-9999 to 9999)
    9999 (1.51 to 9999)
    No statistical analyses for this end point

    Secondary: Objective response rate (ORR)

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    End point title
    		 Objective response rate (ORR)
    End point description
    ORR was defined as the proportion of participants with a best overall response of complete response (CR) or partial response (PR) per RECIST v1.1 (as measured by BICR).
    End point type
    Secondary
    End point timeframe
    up to 148 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1 [2]
    6 [3]
    Units: percentage of participants
        number (not applicable)
    0
    0
    Notes
    [2] - Due to low participant enrollment (n=7), efficacy analyses were not conducted.
    [3] - Due to low participant enrollment (n=7), efficacy analyses were not conducted.
    No statistical analyses for this end point

    Secondary: Duration of response (DOR)

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    End point title
    		 Duration of response (DOR)
    End point description
    DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression (per RECIST v1.1) or death, whichever occurred first (as measured by BICR).
    End point type
    Secondary
    End point timeframe
    up to 148 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: days
        median (confidence interval 95%)
    ( to )
    ( to )
    Notes
    [4] - As measured by BICR, there were no responders; thus, DOR was not calculated.
    [5] - As measured by BICR, there were no responders; thus, DOR was not calculated.
    No statistical analyses for this end point

    Secondary: Number of participants with treatment-emergent adverse events

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    End point title
    		 Number of participants with treatment-emergent adverse events
    End point description
    A treatment-emergent adverse event was defined as an adverse event that was either reported for the first time or the worsening of a pre-existing event after the first dose of study drug until 30 days after the last dose of study drug.
    End point type
    Secondary
    End point timeframe
    up to 178 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1
    6
    Units: participants
    1
    6
    No statistical analyses for this end point

    Secondary: EORTC QLQ-C30 score

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    End point title
    		 EORTC QLQ-C30 score
    End point description
    The European Organization for the Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) contains 30 items and measures 5 functional dimensions (i.e., physical, role, emotional, cognitive, and social), 3 symptom items (i.e., fatigue, nausea/vomiting, and pain), 6 single items (i.e., dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, and financial impact), and a global health and quality of life scale. For each scale and single item, a linear transformation was applied to standardize the scores between 0 (worst) and 100 (best) as described in the EORTC QLQ-C30 Scoring Manual. 9999=No participants were analyzed at this time point in this treatment arm. 99999=A standard deviation was not calculated when only 1 participant contributed to the analysis. GHS/QoL=Global Health Status/Quality of Life.
    End point type
    Secondary
    End point timeframe
    up to 160 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1
    5
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Appetite Loss, Baseline (BL), n=1, 5
    33.3 ± 99999
    26.7 ± 36.51
        Appetite Loss, Cycle 3, Day 1, n=0, 2
    9999 ± 9999
    16.7 ± 23.57
        Appetite Loss, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Appetite Loss, Follow Up, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        Cognitive Functioning, Baseline, n=1, 5
    66.7 ± 99999
    66.7 ± 20.41
        Cognitive Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    91.7 ± 11.79
        Cognitive Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        Cognitive Functioning, Follow Up, n=0, 1
    9999 ± 9999
    83.3 ± 99999
        Constipation, Baseline, n=1, 5
    0.0 ± 99999
    40.0 ± 27.89
        Constipation, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    16.7 ± 23.57
        Constipation, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Constipation, Follow Up, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Diarrhea, Baseline, n=1, 5
    0.0 ± 99999
    20.0 ± 44.72
        Diarrhea, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    0.0 ± 0.0
        Diarrhea, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Diarrhea, Follow Up, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Dyspnea, Baseline, n=1, 5
    0.0 ± 99999
    26.7 ± 27.89
        Dyspnea, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    16.7 ± 23.57
        Dyspnea, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Dyspnea, Follow Up, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        Emotional Functioning, Baseline, n=1, 5
    50.0 ± 99999
    73.3 ± 19.00
        Emotional Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    87.5 ± 5.89
        Emotional Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    91.7 ± 99999
        Emotional Functioning, Follow Up, n=0, 1
    9999 ± 9999
    100.0 ± 99999
        Fatigue, Baseline, n=1, 5
    44.4 ± 99999
    46.7 ± 36.35
        Fatigue, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    50.0 ± 39.28
        Fatigue, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Fatigue, Follow Up, n=0, 1
    9999 ± 9999
    55.6 ± 99999
        Financial Difficulties, Baseline, n=1, 5
    33.3 ± 99999
    20.0 ± 18.26
        Financial Difficulties, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    16.7 ± 23.57
        Financial Difficulties, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Financial Difficulties, Follow Up, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        GHS/QoL, Baseline, n=1, 5
    16.7 ± 99999
    51.7 ± 31.95
        GHS/QoL, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    66.7 ± 11.79
        GHS/QoL, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        GHS/QoL, Follow Up, n=0, 1
    9999 ± 9999
    50.0 ± 99999
        Insomnia, Baseline, n=1, 5
    33.3 ± 99999
    46.7 ± 38.01
        Insomnia, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    33.3 ± 47.14
        Insomnia, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Insomnia, Follow Up, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        Nausea and Vomiting, Baseline, n=1, 5
    0.0 ± 99999
    6.7 ± 9.13
        Nausea and Vomiting, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    8.3 ± 11.79
        Nausea and Vomiting, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Nausea and Vomiting, Follow Up, n=0, 1
    9999 ± 9999
    16.7 ± 99999
        Pain, Baseline, n=1, 5
    50.0 ± 99999
    40.0 ± 43.46
        Pain, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    41.7 ± 11.79
        Pain, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Pain, Follow Up, n=0, 1
    9999 ± 9999
    16.7 ± 99999
        Physical Functioning, Baseline, n=1, 5
    73.3 ± 99999
    65.3 ± 26.83
        Physical Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    33.3 ± 47.14
        Physical Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        Physical Functioning, Follow Up, n=0, 1
    9999 ± 9999
    40.0 ± 99999
        Role Functioning, Baseline, n=1, 5
    66.7 ± 99999
    56.7 ± 40.14
        Role Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    33.3 ± 47.14
        Role Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        Role Functioning, Follow Up, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Social Functioning, Baseline, n=1, 5
    66.7 ± 99999
    53.3 ± 36.13
        Social Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    66.7 ± 0.00
        Social Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    66.7 ± 99999
        Social Functioning, Follow Up, n=0, 1
    9999 ± 9999
    83.3 ± 99999
    No statistical analyses for this end point

    Secondary: Change from baseline in the EORTC QLQ-C30 score

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    End point title
    		 Change from baseline in the EORTC QLQ-C30 score
    End point description
    The European Organization for the Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) contains 30 items and measures 5 functional dimensions (i.e., physical, role, emotional, cognitive, and social), 3 symptom items (i.e., fatigue, nausea/vomiting, and pain), 6 single items (i.e., dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, and financial impact), and a global health and quality of life scale. For each scale and single item, a linear transformation was applied to standardize the scores between 0 (worst) and 100 (best) as described in the EORTC QLQ-C30 Scoring Manual. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=No participants were analyzed at this time point in this treatment arm. 99999=A standard deviation was not calculated when only 1 participant contributed to the analysis.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 160 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1
    5
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Appetite Loss, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    -16.7 ± 23.57
        Appetite Loss, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Appetite Loss, Follow Up, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Cognitive Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    33.3 ± 23.57
        Cognitive Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Cognitive Functioning, Follow Up, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Constipation, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    -33.3 ± 0.00
        Constipation, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    -33.3 ± 99999
        Constipation, Follow Up, n=0, 1
    9999 ± 9999
    -33.3 ± 99999
        Diarrhea, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    -50.0 ± 70.71
        Diarrhea, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Diarrhea, Follow Up, n=0, 1
    9999 ± 9999
    -66.7 ± 99999
        Dyspnea, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    0.0 ± 0.00
        Dyspnea, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Dyspnea, Follow Up, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Emotional Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    12.5 ± 29.46
        Emotional Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Emotional Functioning, Follow Up, n=0, 1
    9999 ± 9999
    41.7 ± 99999
        Fatigue, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    -11.1 ± 15.71
        Fatigue, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    11.1 ± 99999
        Fatigue, Follow Up, n=0, 1
    9999 ± 9999
    -44.4 ± 99999
        Financial Difficulties, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    0.0 ± 0.00
        Financial Difficulties, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Financial Difficulties, Follow Up, n=0, 1
    9999 ± 9999
    -33.3 ± 99999
        Global Health Status/QoL, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    33.3 ± 35.36
        Global Health Status/QoL, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    16.7 ± 99999
        Global Health Status/QoL, Follow Up, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Insomnia, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    -16.7 ± 23.57
        Insomnia, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Insomnia, Follow Up, n=0, 1
    9999 ± 9999
    -33.3 ± 99999
        Nausea and Vomiting, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    0.0 ± 0.00
        Nausea and Vomiting, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Nausea and Vomiting, Follow Up, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Pain, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    -8.3 ± 58.93
        Pain, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Pain, Follow Up, n=0, 1
    9999 ± 9999
    -83.3 ± 99999
        Physical Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    -26.7 ± 37.71
        Physical Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Physical Functioning, Follow Up, n=0, 1
    9999 ± 9999
    -13.3 ± 99999
        Role Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    0.0 ± 0.00
        Role Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    0.0 ± 99999
        Role Functioning, Follow Up, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Social Functioning, Cycle 3 Day 1, n=0, 2
    9999 ± 9999
    41.7 ± 11.79
        Social Functioning, Cycle 6 Day 1, n=0, 1
    9999 ± 9999
    33.3 ± 99999
        Social Functioning, Follow Up, n=0, 1
    9999 ± 9999
    66.7 ± 99999
    No statistical analyses for this end point

    Secondary: Number of participants with the indicated EQ-5D-5L dimension scores

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    End point title
    		 Number of participants with the indicated EQ-5D-5L dimension scores
    End point description
    The EQ-5D-5L is a standardized instrument for use as a measure of health outcome. The EQ-5D-5L descriptive system is composed of 5 dimensions (mobility [M], self-care [S-C], usual activities [U A], pain/discomfort [P/D], and anxiety/depression [A/D]). Each dimension has 5 response levels, which are coded by single-digit numbers: 1 = no problems, 2 = slight problems, 3 = moderate problems, 4 = severe problems, 5 = unable to/extreme problems. The EQ-5D-5L also includes a graded (0 [worst overall health] to 100 [best overall health]) vertical visual analog scale that provides a quantitative measure of the participant's perception of their overall health. 9999=No participants were analyzed at this time point in this treatment arm.
    End point type
    Secondary
    End point timeframe
    up to 160 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1
    5
    Units: participants
    number (not applicable)
        A/D, Baseline, Score 1; n=1, 5
    0
    3
        A/D, Baseline, Score 2; n=1, 5
    0
    0
        A/D, Baseline, Score 3; n=1, 5
    1
    1
        A/D, Baseline, Score 4; n=1, 5
    0
    1
        A/D, Baseline, Score 5; n=1, 5
    0
    0
        A/D, Cycle 4 Day 1, Score 1; n=0, 1
    9999
    1
        A/D, Cycle 4 Day 1, Score 2; n=0, 1
    9999
    0
        A/D, Cycle 4 Day 1, Score 3; n=0, 1
    9999
    0
        A/D, Cycle 4 Day 1, Score 4; n=0, 1
    9999
    0
        A/D, Cycle 4 Day 1, Score 5; n=0, 1
    9999
    0
        A/D, Cycle 7 Day 1, Score 1, n=0, 1
    9999
    1
        A/D, Cycle 7 Day 1, Score 2, n=0, 1
    9999
    0
        A/D, Cycle 7 Day 1, Score 3, n=0, 1
    9999
    0
        A/D, Cycle 7 Day 1, Score 4, n=0, 1
    9999
    0
        A/D, Cycle 7 Day 1, Score 5, n=0, 1
    9999
    0
        A/D, Follow Up, Score 1; n=0, 1
    9999
    1
        A/D, Follow Up, Score 2; n=0, 1
    9999
    0
        A/D, Follow Up, Score 3; n=0, 1
    9999
    0
        A/D, Follow Up, Score 4; n=0, 1
    9999
    0
        A/D, Follow Up, Score 5; n=0, 1
    9999
    0
        M, Baseline, Score 1; n=1, 5
    0
    1
        M, Baseline, Score 2; n=1, 5
    0
    2
        M, Baseline, Score 3; n=1, 5
    1
    2
        M, Baseline, Score 4; n=1, 5
    0
    0
        M, Baseline, Score 5; n=1, 5
    0
    0
        M, Cycle 4 Day 1, Score 1; n=0, 1
    9999
    0
        M, Cycle 4 Day 1, Score 2; n=0, 1
    9999
    1
        M, Cycle 4 Day 1, Score 3; n=0, 1
    9999
    0
        M, Cycle 4 Day 1, Score 4; n=0, 1
    9999
    0
        M, Cycle 4 Day 1, Score 5; n=0, 1
    9999
    0
        M, Cycle 7 Day 1, Score 1; n=0, 1
    9999
    0
        M, Cycle 7 Day 1, Score 2; n=0, 1
    9999
    1
        M, Cycle 7 Day 1, Score 3; n=0, 1
    9999
    0
        M, Cycle 7 Day 1, Score 4; n=0, 1
    9999
    0
        M, Cycle 7 Day 1, Score 5; n=0, 1
    9999
    0
        M, Follow Up, Score 1; n=0, 1
    9999
    0
        M, Follow Up, Score 2; n=0, 1
    9999
    0
        M, Follow Up, Score 3; n=0, 1
    9999
    0
        M, Follow Up, Score 4; n=0, 1
    9999
    0
        M, Follow Up, Score 5; n=0, 1
    9999
    1
        P/D, Baseline, Score 1; n=1, 5
    0
    2
        P/D, Baseline, Score 2; n=1, 5
    0
    1
        P/D, Baseline, Score 3; n=1, 5
    1
    0
        P/D, Baseline, Score 4; n=1, 5
    0
    2
        P/D, Baseline, Score 5; n=1, 5
    0
    0
        P/D, Cycle 4 Day 1, Score 1; n=0, 1
    9999
    1
        P/D, Cycle 4 Day 1, Score 2; n=0, 1
    9999
    0
        P/D, Cycle 4 Day 1, Score 3; n=0, 1
    9999
    0
        P/D, Cycle 4 Day 1, Score 4; n=0, 1
    9999
    0
        P/D, Cycle 4 Day 1, Score 5; n=0, 1
    9999
    0
        P/D, Cycle 7 Day 1, Score 1; n=0, 1
    9999
    0
        P/D, Cycle 7 Day 1, Score 2; n=0, 1
    9999
    0
        P/D, Cycle 7 Day 1, Score 3; n=0, 1
    9999
    1
        P/D, Cycle 7 Day 1, Score 4; n=0, 1
    9999
    0
        P/D, Cycle 7 Day 1, Score 5; n=0, 1
    9999
    0
        P/D, Follow Up, Score 1; n=0, 1
    9999
    0
        P/D, Follow Up, Score 2; n=0, 1
    9999
    0
        P/D, Follow Up, Score 3; n=0, 1
    9999
    1
        P/D, Follow Up, Score 4; n=0, 1
    9999
    0
        P/D, Follow Up, Score 5; n=0, 1
    9999
    0
        S-C, Baseline, Score 1; n=1, 5
    0
    3
        S-C, Baseline, Score 2; n=1, 5
    1
    0
        S-C, Baseline, Score 3; n=1, 5
    0
    2
        S-C, Baseline, Score 4; n=1, 5
    0
    0
        S-C, Baseline, Score 5; n=1, 5
    0
    0
        S-C, Cycle 4 Day 1, Score 1; n=0, 1
    9999
    0
        S-C, Cycle 4 Day 1, Score 2; n=0, 1
    9999
    1
        S-C, Cycle 4 Day 1, Score 3; n=0, 1
    9999
    0
        S-C, Cycle 4 Day 1, Score 4; n=0, 1
    9999
    0
        S-C, Cycle 4 Day 1, Score 5; n=0, 1
    9999
    0
        S-C, Cycle 7 Day 1, Score 1; n=0, 1
    9999
    0
        S-C, Cycle 7 Day 1, Score 2; n=0, 1
    9999
    1
        S-C, Cycle 7 Day 1, Score 3; n=0, 1
    9999
    0
        S-C, Cycle 7 Day 1, Score 4; n=0, 1
    9999
    0
        S-C, Cycle 7 Day 1, Score 5; n=0, 1
    9999
    0
        S-C, Follow Up, Score 1; n=0, 1
    9999
    0
        S-C, Follow Up, Score 2; n=0, 1
    9999
    0
        S-C, Follow Up, Score 3; n=0, 1
    9999
    1
        S-C, Follow Up, Score 4; n=0, 1
    9999
    0
        S-C, Follow Up, Score 5; n=0, 1
    9999
    0
        U A, Baseline, Score 1; n=1, 5
    0
    2
        U A, Baseline, Score 2; n=1, 5
    0
    1
        U A, Baseline, Score 3; n=1, 5
    1
    2
        U A, Baseline, Score 4; n=1, 5
    0
    0
        U A, Baseline, Score 5; n=1, 5
    0
    0
        U A, Cycle 4 Day 1, Score 1; n=0, 1
    9999
    0
        U A, Cycle 4 Day 1, Score 2; n=0, 1
    9999
    1
        U A, Cycle 4 Day 1, Score 3; n=0, 1
    9999
    0
        U A, Cycle 4 Day 1, Score 4; n=0, 1
    9999
    0
        U A, Cycle 4 Day 1, Score 5; n=0, 1
    9999
    0
        U A, Cycle 7 Day 1, Score 1; n=0, 1
    9999
    0
        U A, Cycle 7 Day 1, Score 2; n=0, 1
    9999
    1
        U A, Cycle 7 Day 1, Score 3; n=0, 1
    9999
    0
        U A, Cycle 7 Day 1, Score 4; n=0, 1
    9999
    0
        U A, Cycle 7 Day 1, Score 5; n=0, 1
    9999
    0
        U A, Follow Up, Score 1; n=0, 1
    9999
    0
        U A, Follow Up, Score 2; n=0, 1
    9999
    0
        U A, Follow Up, Score 3; n=0, 1
    9999
    0
        U A, Follow Up, Score 4; n=0, 1
    9999
    1
        U A, Follow Up, Score 5; n=0, 1
    9999
    0
    No statistical analyses for this end point

    Secondary: Change from baseline in the EQ-5D-5L EQ Visual Analog Scale score

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    End point title
    		 Change from baseline in the EQ-5D-5L EQ Visual Analog Scale score
    End point description
    The EQ-5D-5L is a standardized instrument for use as a measure of health outcome. The EQ-5D-5L descriptive system is composed of 5 dimensions (mobility, self-case, usual activities, pain/discomfort, and anxiety/depression). Each dimension has 5 response levels, which are coded by single-digit numbers: 1 = no problems, 2 = slight problems, 3 = moderate problems, 4 = severe problems, 5 = unable to/extreme problems. The EQ-5D-5L also includes a graded (0 [worst overall health] to 100 [best overall health]) vertical visual analog scale that provides a quantitative measure of the participant's perception of their overall health. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. 9999=No participants were analyzed at this time point in this treatment arm. 99999=A standard deviation was not calculated when only 1 participant contributed to the analysis.
    End point type
    Secondary
    End point timeframe
    Baseline; up to 160 days
    End point values
    		 Pemigatinib 13.5 mg plus pembrolizumab 200 mg Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Number of subjects analysed
    1
    5
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline, n=1, 5
    40 ± 99999
    52 ± 30.54
        Cycle 4 Day 1, n=0, 1
    9999 ± 9999
    5 ± 99999
        Cycle 7 Day 1, n=0, 1
    9999 ± 9999
    5 ± 99999
        Follow Up, n=0, 1
    9999 ± 9999
    30 ± 99999
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    up to 178 days. Deaths were monitored for up to 225 days. Treatment-emergent adverse events (TEAEs) were monitored for up to 178 days (for up to 30 days after last dose of study drug).
    Adverse event reporting additional description
    Treatment-emergent adverse events, defined as adverse events that were either reported for the first time or the worsening of pre-existing events after the first dose of study drug until 30 days after the last dose of study drug, are reported for the Safety Population (all randomized participants who received at least one dose of study treatment).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab
    Reporting group description
    		 Participants received either gemcitabine plus carboplatin or pembrolizumab as standard of care. Participants received gemcitabine 1000 mg/meters squared (m^2) IV over 30 minutes on Days 1 and 8 of each 3-week treatment cycle, followed by carboplatin (dosed to target area under the concentration-time curve [AUC] of 5 mg/milliliters [mL]/minute [min] or 4.5 mg/mL/min if required per local guidelines) on Day 1 or Day 2 of each 3-week treatment cycle. Treatment continued for 4 to 6 cycles (or per institutional standards) until disease progression or intolerable toxicity. Pembrolizumab 200 mg was administered IV on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.

    Reporting group title
    Pemigatinib 13.5 mg plus pembrolizumab 200 mg
    Reporting group description
    		 Pemigatinib was self-administered at 13.5 milligrams (mg) once a day (QD) orally (PO) until disease progression during each 21-day treatment cycle. Participants not reaching the target serum phosphate level (> 5.5 milligrams per deciliter [mg/dL]) during Cycle 1 despite being study drug compliant increased the daily pemigatinib dose to 18 mg starting at Cycle 2 as long as they were not experiencing an ongoing Grade 2 or higher treatment-related adverse event (AE). Pembrolizumab 200 mg was administered intravenously (IV) on Day 1 of each 21-day treatment cycle for up to 35 cycles or disease progression.

    Serious adverse events
    		 Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab Pemigatinib 13.5 mg plus pembrolizumab 200 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 1 (100.00%)
         number of deaths (all causes)
    3
    0
         number of deaths resulting from adverse events
    0
    0
    Musculoskeletal and connective tissue disorders
    Bone pain
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Gemcitabine 1000 mg/m^2 plus carboplatin or pembrolizumab Pemigatinib 13.5 mg plus pembrolizumab 200 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    1 / 1 (100.00%)
    Vascular disorders
    Vascular pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Malaise
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Oedema peripheral
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Penile pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Delirium
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 1 (100.00%)
         occurrences all number
    1
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Blood alkaline phosphatase increased
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Platelet count decreased
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Neuropathy peripheral
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Hypofibrinogenaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Neutropenia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Stomatitis
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Abdominal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Cheilitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Constipation
         subjects affected / exposed
    3 / 6 (50.00%)
    0 / 1 (0.00%)
         occurrences all number
    3
    0
    Diarrhoea
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Dry mouth
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Nausea
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Vomiting
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Erythema multiforme
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Pruritus
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Dry skin
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Bone pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Escherichia bacteraemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Pyelonephritis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Hyperphosphataemia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Decreased appetite
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Hyperkalaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Hypocalcaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 Apr 2020
    Changes were made to address requests by Health Authorities for certain changes as well as feedback from Scientific Steering Committee to help with enrollment of participants.

    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 Sponsor made a decision unrelated to safety to halt study enrollment. Due to early termination of the study with only 7 participants, no analysis of efficacy endpoints was done.
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