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    Clinical Trial Results:
    A Phase 2 Study of REGN2810, a Fully Human Monoclonal Antibody to Programmed Death-1, in Patients With Advanced Basal Cell Carcinoma Who Experienced Progression of Disease on Hedgehog Pathway Inhibitor Therapy, or Were Intolerant of Prior Hedgehog Pathway Inhibitor Therapy

    Summary
    EudraCT number
    		 2016-003122-16
    Trial protocol
    		 BE   DE   AT   GR   ES   FR   IT  
    Global end of trial date
    27 Apr 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 May 2024
    First version publication date
    09 May 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    R2810-ONC-1620
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03132636
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Regeneron Pharmaceuticals, Inc.
    Sponsor organisation address
    777 Old Saw Mill River Road, Tarrytown, NY, United States, 10591
    Public contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.com
    Scientific contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.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
    27 Apr 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Apr 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the trial was to estimate the objective response rate (ORR) for metastatic Basal Cell Carcinoma (BCC) (group 1) and for unresectable locally advanced BCC (group 2) when treated with cemiplimab as a monotherapy
    Protection of trial subjects
    It is the responsibility of both the sponsor and the investigator(s) to ensure that this clinical study is conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki, and that are consistent with the ICH guidelines for GCP and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    29 Jun 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Austria: 1
    Country: Number of subjects enrolled
    Belgium: 10
    Country: Number of subjects enrolled
    Canada: 4
    Country: Number of subjects enrolled
    France: 23
    Country: Number of subjects enrolled
    Germany: 25
    Country: Number of subjects enrolled
    Greece: 7
    Country: Number of subjects enrolled
    Italy: 14
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    Switzerland: 3
    Country: Number of subjects enrolled
    United States: 42
    Worldwide total number of subjects
    138
    EEA total number of subjects
    89
    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)
    58
    From 65 to 84 years
    68
    85 years and over
    12

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 138 participants were enrolled into 1 of 2 groups: (1) participants with metastatic Basal Cell Carcinoma (mBCC) or (2) with unresectable locally advanced BCC (laBCC) who experienced progression of disease on Hedgehog inhibitor (HHI) therapy, or response no better than stable disease for at least 9 mos. or were intolerant of prior HHI therapy.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Group 1: Metastatic BCC (mBCC)
    Arm description
    		 Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.
    Arm type
    		 Experimental

    Investigational medicinal product name
    cemiplimab
    Investigational medicinal product code
    REGN2810
    Other name
    Libtayo
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received open-label cemiplimab as an IV infusion over approximately 30 minutes

    Arm title
    		 Group 2: Unresectable Locally Advanced BCC (laBCC)
    Arm description
    		 Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
    Arm type
    		 Experimental

    Investigational medicinal product name
    cemiplimab
    Investigational medicinal product code
    REGN2810
    Other name
    Libtayo
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received open-label cemiplimab as an IV infusion over approximately 30 minutes

    Number of subjects in period 1
    		Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Started
    54
    84
    Completed Treatment
    11
    28
    Completed
    6
    19
    Not completed
    48
    65
         Adverse event, serious fatal
    4
    7
         Physician decision
    1
    -
         Subject decision
    1
    9
         Withdrawal of consent
    2
    5
         Sponsor decision
    -
    1
         Non compliance with protocol by participant
    1
    1
         Lost insurance coverage
    1
    -
         Adverse event, non-fatal
    1
    2
         Did not re-consent
    1
    -
         Lost to follow-up
    3
    2
         Progressive disease
    33
    36
         Related to radiological outcomes
    -
    1
         Unable to come to site for continued visits
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Group 1: Metastatic BCC (mBCC)
    Reporting group description
    		 Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.

    Reporting group title
    Group 2: Unresectable Locally Advanced BCC (laBCC)
    Reporting group description
    		 Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.

    Reporting group values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC) Total
    Number of subjects
    		 54 84 138
    Age categorical
    Units: Participants
        Adults (18-64 years)
    		 27 31 58
        From 65-74 years
    		 18 19 37
        75 years and over
    		 9 34 43
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 63.8 ( 11.09 ) 69.1 ( 12.84 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 16 28 44
        Male
    		 38 56 94
    Race/Ethnicity, Customized
    Units: Subjects
        Race : White
    		 47 57 104
        Race : Not Reported
    		 1 0 1
        Race : Missing
    		 6 27 33
    Race/Ethnicity, Customized
    Units: Subjects
        Ethnicity : Not Hispanic or Latino
    		 46 56 102
        Ethnicity : Hispanic or Latino
    		 2 1 3
        Ethnicity : Missing
    		 6 27 33

    End points

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    End points reporting groups
    Reporting group title
    Group 1: Metastatic BCC (mBCC)
    Reporting group description
    		 Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.

    Reporting group title
    Group 2: Unresectable Locally Advanced BCC (laBCC)
    Reporting group description
    		 Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.

    Primary: Objective Response Rate (ORR) as assessed by Independent Central Review (ICR)

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    End point title
    		 Objective Response Rate (ORR) as assessed by Independent Central Review (ICR) [1]
    End point description
    ORR was defined as percentage of participants with best overall response of complete response (CR) or partial response (PR) according to RECIST v1.1 assessed as per ICR assessment. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<) 10 millimeter (mm) (< 1 centimeter [cm]). PR: At least a 30 percent (%) decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. ORR was determined by Clopper-Pearson method. Full analysis set (FAS) (included all enrolled participants for each group who passed screening and were deemed to be eligible for this study)
    End point type
    Primary
    End point timeframe
    Up to 1422 days (approximately 46 months)
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: ORR was determined by Clopper-Pearson method.
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Percentage of Participants
        number (confidence interval 95%)
    22.2 (12.0 to 35.6)
    32.1 (22.4 to 43.2)
    No statistical analyses for this end point

    Secondary: Objective Response Rate (ORR) per Investigator assessment

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    End point title
    		 Objective Response Rate (ORR) per Investigator assessment
    End point description
    ORR was defined as percentage of participants with best overall response of complete response (CR) or partial response (PR) according to RECIST v1.1 per Investigator assessment. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<) 10 millimeter (mm) (< 1 centimeter [cm]). PR: At least a 30 percent (%) decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. ORR was determined by Clopper-Pearson method. The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.
    End point type
    Secondary
    End point timeframe
    Up to 1422 days (approximately 46 months)
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Percentage of Participants
        number (confidence interval 95%)
    25.9 (15.0 to 39.7)
    36.9 (26.6 to 48.1)
    No statistical analyses for this end point

    Secondary: Duration of Response (DOR) as assessed by ICR

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    End point title
    		 Duration of Response (DOR) as assessed by ICR
    End point description
    DOR per ICR was determined for participants with best overall response of CR or PR. DOR measured from the time measurement criteria are first met for CR/PR (whichever was first recorded) until first date of recurrent or progressive disease (PD) (photographic or radiographic), or death due to any cause. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm (<1 cm). PR: At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (includes baseline sum if that is the smallest on study). DOR determined by Kaplan-Meier estimate. FAS included all enrolled participants for each group who passed screening and were deemed eligible; "Number analyzed" signifies those participants with confirmed CR or PR.
    End point type
    Secondary
    End point timeframe
    Up to 48 months
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    12
    27
    Units: Months
        median (confidence interval 95%)
    99999 (9.8 to 99999)
    99999 (15.5 to 99999)
    No statistical analyses for this end point

    Secondary: Duration of Response (DOR) per Investigator assessment

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    End point title
    		 Duration of Response (DOR) per Investigator assessment
    End point description
    DOR per investigator assessment was determined for participants with best overall response of CR or PR. DOR was measured from the time measurement criteria are first met for CR/PR (whichever was first recorded) until the first date of recurrent or progressive disease (PD) (photographic or radiographic), or death due to any cause. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm (<1 cm). PR: At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). DOR was determined by Kaplan-Meier estimate. "Number analyzed" signifies those participants with confirmed CR or PR.
    End point type
    Secondary
    End point timeframe
    Up to 48 months
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    14
    31
    Units: Months
        median (confidence interval 95%)
    99999 (9.8 to 99999)
    19.6 (16.7 to 99999)
    No statistical analyses for this end point

    Secondary: Complete Response (CR) Rate as assessed by ICR

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    End point title
    		 Complete Response (CR) Rate as assessed by ICR
    End point description
    CR rate was determined by the percentage of participants with best overall response of CR. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm (<1 cm). CR rate 95% confidence interval determined by Clopper-Pearson exact confidence interval. The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.
    End point type
    Secondary
    End point timeframe
    Up to 48 months
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Percentage of Participants
        number (confidence interval 95%)
    3.7 (0.5 to 12.7)
    8.3 (3.4 to 16.4)
    No statistical analyses for this end point

    Secondary: Complete Response (CR) Rate per Investigator assessment

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    End point title
    		 Complete Response (CR) Rate per Investigator assessment
    End point description
    CR rate was determined by the percentage of participants with best overall response of CR. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm (<1 cm). CR rate 95% confidence interval determined by Clopper-Pearson exact confidence interval. The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.
    End point type
    Secondary
    End point timeframe
    From date of treatment until best objective response of CR, up to 60 months (approximately 260 weeks)
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Percentage of Participants
        number (confidence interval 95%)
    3.7 (0.5 to 12.7)
    8.3 (3.4 to 16.4)
    No statistical analyses for this end point

    Secondary: Progression Free Survival (PFS) as assessed by ICR

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    End point title
    		 Progression Free Survival (PFS) as assessed by ICR
    End point description
    PFS was defined as the time from start of treatment until the first date of recurrent or PD (photographic or radiographic), or death due to any cause, whichever occurred first, was determined by IRC. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). PFS was determined by Kaplan-Meier estimate. FAS (all enrolled participants for each group who passed screening and were deemed to be eligible for this study)
    End point type
    Secondary
    End point timeframe
    Up to 60 months
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Months
        median (confidence interval 95%)
    10.1 (4.2 to 15.9)
    16.5 (8.6 to 21.8)
    No statistical analyses for this end point

    Secondary: Progression Free Survival (PFS) per Investigator assessment

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    End point title
    		 Progression Free Survival (PFS) per Investigator assessment
    End point description
    PFS was defined as the time from start of treatment until the first date of recurrent or PD (photographic or radiographic), or death due to any cause, whichever occurred first, was determined by IRC. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). PFS was determined by Kaplan-Meier estimate. The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.
    End point type
    Secondary
    End point timeframe
    Up to 60 months
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Months
        median (confidence interval 95%)
    6.6 (4.2 to 8.3)
    18.1 (10.4 to 21.3)
    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 measured as time from the start of treatment until death due to any cause. Participants who did not die were censored at the last date that participant was documented to be alive. OS was calculated based on Kaplan-Meier estimate. The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.
    End point type
    Secondary
    End point timeframe
    Up to 60 months
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Months
        median (confidence interval 95%)
    49.9 (28.4 to 99999)
    99999 (99999 to 99999)
    No statistical analyses for this end point

    Secondary: Change from Baseline of Patient-reported Outcomes in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)

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    End point title
    		 Change from Baseline of Patient-reported Outcomes in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)
    End point description
    EORTC QLQ-C30: a 30-item questionnaire used to assess overall QoL in cancer patients & consists of 15 domains: 1 Global Health Status (GHS)/QoL scale, 5 functional scales (Physical, role, cognitive, emotional, social), 9 symptom scales/items (Fatigue, nausea & vomiting, pain, dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, financial impact). Most items scored 1 ("not at all") - 4 ("very much") except items contributing to GHS/QoL (scored 1 ("very poor") - 7 ("excellent"). Linear transformation applied to raw scores (all transformed scores lie between 0-100). For GHS/QoL & 5 functional scales: higher score indicates “better” quality of life/functioning & a positive change from baseline indicates improvement. Symptom scales/items: higher score indicates a “worse” level of symptoms/problems, negative change from baseline indicates improvement. Number ("n") signifies those participants who were evaluable at specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1 of Cycle 1); Day 1 of Cycles 2 to 9 (C2D1, C3D1, C4D1, C5D1, C6D1, C7D1, C8D1, C9D1) (Cycles 1-5 [Each cycle of 9 weeks], Cycles 6 to 9 [Each cycle of 12 weeks])
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Physical Functioning: Change at C2D1 (n=43; n=75)
    -4.88 ( 14.274 )
    -1.55 ( 12.101 )
        Physical Functioning: Change at C3D1 (n=29; n=65)
    -1.78 ( 13.481 )
    -0.56 ( 15.947 )
        Physical Functioning: Change at C4D1 (n=26; n=55)
    -6.60 ( 12.828 )
    -3.79 ( 17.814 )
        Physical Functioning: Change at C5D1 (n=20; n=50)
    1.08 ( 10.033 )
    -2.86 ( 17.063 )
        Physical Functioning: Change at C6D1 (n=19; n=40)
    -1.67 ( 9.734 )
    0.33 ( 12.073 )
        Physical Functioning: Change at C7D1 (n=13; n=33)
    -0.38 ( 11.142 )
    -0.20 ( 15.410 )
        Physical Functioning: Change at C8D1 (n=10; n=33)
    -1.33 ( 12.090 )
    -3.03 ( 13.549 )
        Physical Functioning: Change at C9D1 (n=11; n=29)
    -2.27 ( 8.765 )
    -5.06 ( 20.444 )
        Role Functioning: Change at C2D1 (n=54; n=84)
    -2.71 ( 27.442 )
    -3.11 ( 20.264 )
        Role Functioning: Change at C3D1 (n=29; n=65)
    5.17 ( 27.854 )
    -4.87 ( 25.297 )
        Role Functioning: Change at C4D1 (n=26; n=55)
    -3.21 ( 25.394 )
    -6.06 ( 26.326 )
        Role Functioning: Change at C5D1 (n=20; n=50)
    5.00 ( 22.361 )
    -5.33 ( 26.393 )
        Role Functioning: Change at C6D1 (n=19; n=40)
    6.14 ( 21.667 )
    -3.33 ( 16.963 )
        Role Functioning: Change at C7D1 (n=13; n=33)
    12.82 ( 24.677 )
    -7.07 ( 16.682 )
        Role Functioning: Change at C8D1 (n=10; n=33)
    15.00 ( 25.398 )
    -4.04 ( 19.557 )
        Role Functioning: Change at C9D1 (n=11; n=29)
    9.09 ( 22.808 )
    -9.77 ( 30.052 )
        Emotional Functioning: Change at C2D1 (n=54; n=74)
    3.17 ( 19.638 )
    1.95 ( 20.483 )
        Emotional Functioning: Change at C3D1 (n=29; n=64)
    1.15 ( 18.730 )
    1.56 ( 18.538 )
        Emotional Functioning: Change at C4D1 (n=26; n=54)
    3.53 ( 22.007 )
    1.39 ( 21.277 )
        Emotional Functioning: Change at C5D1 (n=20; n=49)
    5.83 ( 24.046 )
    -4.59 ( 19.622 )
        Emotional Functioning: Change at C6D1 (n=19; n=40)
    7.02 ( 20.650 )
    1.04 ( 19.809 )
        Emotional Functioning: Change at C7D1 (n=13; n=33)
    3.85 ( 18.199 )
    -4.63 ( 19.576 )
        Emotional Functioning: Change at C8D1 (n=11; n=33)
    16.67 ( 25.000 )
    3.11 ( 19.810 )
        Emotional Functioning: Change at C9D1 (n=11; n=29)
    5.30 ( 17.189 )
    2.11 ( 17.246 )
        Cognitive Functioning: Change at C2D1 (n=54; n=74)
    0.78 ( 13.586 )
    -2.48 ( 26.985 )
        Cognitive Functioning: Change at C3D1 (n=29; n=64)
    -1.72 ( 16.871 )
    -4.17 ( 23.382 )
        Cognitive Functioning: Change at C4D1 (n=26; n=54)
    -0.64 ( 15.261 )
    -4.94 ( 21.385 )
        Cognitive Functioning: Change at C5D1 (n=20; n=49)
    -4.17 ( 14.178 )
    -6.46 ( 25.188 )
        Cognitive Functioning: Change at C6D1 (n=19; n=40)
    -0.88 ( 12.998 )
    -1.25 ( 19.017 )
        Cognitive Functioning: Change at C7D1 (n=13; n=33)
    -2.56 ( 11.479 )
    -5.56 ( 18.942 )
        Cognitive Functioning: Change at C8D1 (n=11; n=33)
    -6.06 ( 11.237 )
    1.52 ( 17.362 )
        Cognitive Functioning: Change at C9D1 (n=11; n=29)
    -10.61 ( 25.025 )
    -2.30 ( 23.873 )
        Social Functioning: Change at C2D1 (n=54; n=74)
    0.39 ( 19.412 )
    4.50 ( 21.422 )
        Social Functioning: Change at C3D1 (n=29; n=64)
    3.45 ( 16.891 )
    0.78 ( 21.088 )
        Social Functioning: Change at C4D1 (n=26; n=54)
    3.85 ( 24.179 )
    1.85 ( 23.272 )
        Social Functioning: Change at C5D1 (n=20; n=49)
    3.33 ( 22.685 )
    0.34 ( 23.445 )
        Social Functioning: Change at C6D1 (n=19; n=40)
    10.53 ( 16.860 )
    0.00 ( 24.749 )
        Social Functioning: Change at C7D1 (n=13; n=33)
    12.82 ( 22.724 )
    0.00 ( 18.162 )
        Social Functioning: Change at C8D1 (n=11; n=33)
    10.61 ( 15.407 )
    2.02 ( 23.848 )
        Social Functioning: Change at C9D1 (n=11; n=29)
    9.09 ( 13.670 )
    -2.30 ( 27.359 )
        Fatigue: Change at C2D1 (n=54; n=84)
    5.17 ( 22.919 )
    6.44 ( 24.542 )
        Fatigue: Change at C3D1 (n=29; n=65)
    1.92 ( 18.322 )
    6.58 ( 24.901 )
        Fatigue: Change at C4D1 (n=26; n=55)
    -2.56 ( 21.154 )
    7.58 ( 25.912 )
        Fatigue: Change at C5D1 (n=20; n=50)
    -5.56 ( 24.048 )
    9.67 ( 23.404 )
        Fatigue: Change at C6D1 (n=19; n=40)
    -5.85 ( 19.376 )
    7.78 ( 17.649 )
        Fatigue: Change at C7D1 (n=13; n=33)
    -1.71 ( 19.692 )
    14.14 ( 20.838 )
        Fatigue: Change at C8D1 (n=10; n=33)
    -7.78 ( 24.595 )
    9.09 ( 19.534 )
        Fatigue: Change at C9D1 (n=11; n=29)
    -1.01 ( 16.607 )
    12.64 ( 20.080 )
        Nausea/Vomiting: Change at C2D1 (n=54, n=84)
    -0.78 ( 8.095 )
    0.22 ( 12.703 )
        Nausea/Vomiting: Change at C3D1 (n=29, n=65)
    0.00 ( 13.363 )
    -1.79 ( 12.884 )
        Nausea/Vomiting: Change at C4D1 (n=26, n=55)
    0.00 ( 16.330 )
    0.30 ( 13.414 )
        Nausea/Vomiting: Change at C5D1 (n=20, n=50)
    0.83 ( 10.080 )
    1.00 ( 13.640 )
        Nausea/Vomiting: Change at C6D1 (n=19, n=40)
    -0.88 ( 3.824 )
    1.67 ( 13.503 )
        Nausea/Vomiting: Change at C7D1 (n=13, n=33)
    -1.28 ( 14.372 )
    3.03 ( 13.472 )
        Nausea/Vomiting: Change at C8D1 (n=10, n=33)
    0.00 ( 0.000 )
    0.51 ( 12.832 )
        Nausea/Vomiting: Change at C9D1 (n=11, n=29)
    0.00 ( 0.000 )
    -2.30 ( 13.890 )
        Pain: Change at C2D1 (n=54, n=84)
    -2.33 ( 30.338 )
    -0.22 ( 26.351 )
        Pain: Change at C3D1 (n=29, n=65)
    -9.77 ( 22.056 )
    -1.54 ( 30.151 )
        Pain: Change at C4D1 (n=26, n=55)
    0.00 ( 29.439 )
    -4.85 ( 25.795 )
        Pain: Change at C5D1 (n=20, n=50)
    -4.17 ( 25.291 )
    -4.00 ( 25.098 )
        Pain: Change at C6D1 (n=19, n=40)
    -14.04 ( 29.535 )
    -2.92 ( 24.427 )
        Pain: Change at C7D1 (n=13, n=33)
    -21.79 ( 29.174 )
    -4.04 ( 24.661 )
        Pain: Change at C8D1 (n=11, n=33)
    -13.64 ( 25.624 )
    -7.58 ( 25.716 )
        Pain: Change at C9D1 (n=11, n=29)
    -19.70 ( 27.707 )
    -5.17 ( 24.030 )
        Dyspnoea: Change at C2D1 (n=42, n=84)
    2.38 ( 17.097 )
    1.33 ( 24.162 )
        Dyspnoea: Change at C3D1 (n=28, n=65)
    3.57 ( 16.578 )
    -0.51 ( 23.930 )
        Dyspnoea: Change at C4D1 (n=25, n=55)
    0.00 ( 16.667 )
    2.42 ( 24.724 )
        Dyspnoea: Change at C5D1 (n=19, n=49)
    3.51 ( 21.928 )
    0.00 ( 28.054 )
        Dyspnoea: Change at C6D1 (n=18, n=40)
    1.85 ( 13.873 )
    -1.67 ( 19.900 )
        Dyspnoea: Change at C7D1 (n=12, n=33)
    2.78 ( 30.011 )
    2.02 ( 21.952 )
        Dyspnoea: Change at C8D1 (n=10, n=33)
    16.67 ( 36.004 )
    2.02 ( 21.952 )
        Dyspnoea: Change at C9D1 (n=10, n=29)
    6.67 ( 26.294 )
    2.30 ( 17.663 )
        Insomnia: Change at C2D1 (n=54, n=84)
    -2.33 ( 26.622 )
    0.44 ( 24.195 )
        Insomnia: Change at C3D1 (n=29, n=65)
    -6.90 ( 24.200 )
    -0.51 ( 26.016 )
        Insomnia: Change at C4D1 (n=26, n=55)
    -10.26 ( 29.468 )
    -1.82 ( 19.687 )
        Insomnia: Change at C5D1 (n=20, n=50)
    -15.00 ( 31.484 )
    1.33 ( 30.087 )
        Insomnia: Change at C6D1 (n=19, n=40)
    -17.54 ( 32.142 )
    -4.17 ( 24.093 )
        Insomnia: Change at C7D1 (n=13, n=33)
    -7.69 ( 41.172 )
    1.01 ( 28.241 )
        Insomnia: Change at C8D1 (n=10, n=33)
    -13.33 ( 54.885 )
    3.03 ( 25.500 )
        Insomnia: Change at C9D1 (n=11, n=29)
    -6.06 ( 38.925 )
    5.75 ( 25.306 )
        Appetite loss: Change at C2D1 (n=54, n=74)
    4.65 ( 26.807 )
    -2.25 ( 27.215 )
        Appetite loss: Change at C3D1 (n=29, n=62)
    -2.30 ( 15.252 )
    -4.30 ( 22.163 )
        Appetite loss: Change at C4D1 (n=26, n=54)
    -1.28 ( 30.523 )
    -3.09 ( 26.117 )
        Appetite loss: Change at C5D1 (n=20, n=49)
    3.33 ( 21.357 )
    -1.36 ( 30.398 )
        Appetite loss: Change at C6D1 (n=19, n=39)
    -1.75 ( 17.476 )
    -2.56 ( 29.004 )
        Appetite loss: Change at C7D1 (n=13, n=33)
    -5.13 ( 12.518 )
    -1.01 ( 30.601 )
        Appetite loss: Change at C8D1 (n=10, n=32)
    -3.33 ( 10.541 )
    -2.08 ( 31.609 )
        Appetite loss: Change at C9D1 (n=11, n=28)
    -3.03 ( 17.979 )
    1.19 ( 30.741 )
        Constipation: Change at C2D1 (n=54, n=74)
    1.55 ( 19.181 )
    2.25 ( 21.603 )
        Constipation: Change at C3D1 (n=29, n=63)
    0.00 ( 21.822 )
    1.06 ( 20.712 )
        Constipation: Change at C4D1 (n=26, n=54)
    -2.56 ( 18.674 )
    1.23 ( 21.440 )
        Constipation: Change at C5D1 (n=20, n=49)
    5.00 ( 16.312 )
    -0.68 ( 22.037 )
        Constipation: Change at C6D1 (n=19, n=40)
    0.00 ( 15.713 )
    -1.67 ( 18.413 )
        Constipation: Change at C7D1 (n=13, n=33)
    5.13 ( 18.490 )
    4.04 ( 13.838 )
        Constipation: Change at C8D1 (n=10, n=33)
    13.33 ( 23.307 )
    -2.02 ( 16.540 )
        Constipation: Change at C9D1 (n=11, n=29)
    0.00 ( 14.907 )
    1.15 ( 22.683 )
        Diarhoea: Change at C2D1 (n=54, n=74)
    3.88 ( 14.924 )
    0.45 ( 24.360 )
        Diarhoea: Change at C3D1 (n=29, n=64)
    1.15 ( 14.037 )
    -1.04 ( 20.547 )
        Diarhoea: Change at C4D1 (n=26, n=54)
    3.85 ( 23.715 )
    -1.85 ( 19.870 )
        Diarhoea: Change at C5D1 (n=20, n=49)
    6.67 ( 20.520 )
    -0.68 ( 23.064 )
        Diarhoea: Change at C6D1 (n=19, n=40)
    3.51 ( 18.904 )
    -1.67 ( 18.413 )
        Diarhoea: Change at C7D1 (n=13, n=33)
    0.00 ( 13.608 )
    1.01 ( 19.516 )
        Diarhoea: Change at C8D1 (n=11, n=33)
    0.00 ( 14.907 )
    -1.01 ( 19.516 )
        Diarhoea: Change at C9D1 (n=11, n=29)
    0.00 ( 14.907 )
    -2.30 ( 21.696 )
        Financial Problems: Change at C2D1 (n=54, n=74)
    -3.10 ( 17.539 )
    -3.15 ( 25.385 )
        Financial Problems: Change at C3D1 (n=28, n=63)
    0.00 ( 18.144 )
    -4.23 ( 24.313 )
        Financial Problems: Change at C4D1 (n=26, n=54)
    -5.13 ( 22.494 )
    -4.94 ( 23.710 )
        Financial Problems: Change at C5D1 (n=20, n=49)
    0.00 ( 18.732 )
    -6.80 ( 22.546 )
        Financial Problems: Change at C6D1 (n=19, n=40)
    -1.75 ( 23.501 )
    -5.83 ( 27.099 )
        Financial Problems: Change at C7D1 (n=13, n=33)
    -2.56 ( 21.350 )
    1.01 ( 19.516 )
        Financial Problems: Change at C8D1 (n=11, n=33)
    -3.03 ( 17.979 )
    1.01 ( 19.516 )
        Financial Problems: Change at C9D1 (n=11, n=29)
    -6.06 ( 20.101 )
    -2.30 ( 23.454 )
        Global health status: Change at C2D1 (n=54, n=72)
    -3.68 ( 21.845 )
    2.55 ( 15.298 )
        Global health status: Change at C3D1 (n=29, n=62)
    3.74 ( 14.362 )
    -2.55 ( 19.823 )
        Global health status: Change at C4D1 (n=26, n=51)
    -2.24 ( 14.636 )
    -0.49 ( 18.136 )
        Global health status: Change at C5D1 (n=20, n=48)
    7.50 ( 14.023 )
    -1.91 ( 21.210 )
        Global health status: Change at C6D1 (n=19, n=38)
    10.96 ( 11.802 )
    4.17 ( 19.447 )
        Global health status: Change at C7D1 (n=13, n=32)
    16.03 ( 22.428 )
    -3.13 ( 19.715 )
        Global health status: Change at C8D1 (n=11, n=31)
    9.09 ( 13.670 )
    2.15 ( 21.727 )
        Global health status: Change at C9D1 (n=11, n=28)
    8.33 ( 20.412 )
    -7.14 ( 28.211 )
    No statistical analyses for this end point

    Secondary: Change from Baseline of Patient-reported Outcomes in Skindex-16 Questionnaire

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    End point title
    		 Change from Baseline of Patient-reported Outcomes in Skindex-16 Questionnaire
    End point description
    Skindex-16 questionnaire: 16 questions related to quality of life in cancer patients. Each item is rated on a 7-point Likert scale (0=never bothered - 6=always bothered). Each raw score is multiplied by 16.667 to transform all responses to a linear scale from 0 (no effect) to 100 (effect experienced all the time). Responses are categorized into 3 subscales: symptom, emotional & functional; respective scores are expressed in a linear scale from 0 to 100. Symptoms scale score is an average of items 1 - 4 expressed in a linear scale from 0 - 100, Emotions scale score is an average of items 5 - 11 expressed in a linear scale from 0 - 100 & Functioning scale score is an average of items 12 - 16 expressed in a linear scale from 0 - 100. A negative change from baseline indicates an improvement compared to baseline. FAS (all enrolled participants); Number ("n") signifies those participants who were evaluable at specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1 of Cycle 1); Day 1 of Cycles 2 to 9 (Cycles 1-5 [Each cycle of 9 weeks], Cycles 6 to 9 [Each cycle of 12 weeks])
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Emotions: Change at C2D1 (n=42, n=69)
    -6.90 ( 24.422 )
    -8.93 ( 27.767 )
        Emotions: Change at C3D1 (n=26, n=63)
    -7.92 ( 18.033 )
    -8.60 ( 25.641 )
        Emotions: Change at C4D1 (n=22, n=51)
    -3.97 ( 17.175 )
    -11.45 ( 24.215 )
        Emotions: Change at C5D1 (n=17, n=46)
    -0.14 ( 12.835 )
    -10.25 ( 24.646 )
        Emotions: Change at C6D1 (n=16, n=35)
    -9.08 ( 22.824 )
    -19.73 ( 27.304 )
        Emotions: Change at C7D1 (n=12, n=30)
    6.55 ( 17.053 )
    -13.65 ( 27.132 )
        Emotions: Change at C8D1 (n=10, n=30)
    -0.71 ( 8.478 )
    -13.97 ( 25.001 )
        Emotions: Change at C9D1 (n=11, n=28)
    5.70 ( 14.711 )
    -15.08 ( 31.843 )
        Symptoms: Change at C2D1 (n=54, n=84)
    0.99 ( 18.788 )
    -1.31 ( 21.607 )
        Symptoms: Change at C3D1 (n=26, n=64)
    3.85 ( 16.580 )
    -0.26 ( 24.158 )
        Symptoms: Change at C4D1 (n=22, n=52)
    5.30 ( 18.464 )
    -6.62 ( 23.917 )
        Symptoms: Change at C5D1 (n=17, n=47)
    -0.98 ( 15.205 )
    -4.11 ( 18.062 )
        Symptoms: Change at C6D1 (n=16, n=36)
    -1.56 ( 16.307 )
    -1.85 ( 21.419 )
        Symptoms: Change at C7D1 (n=12, n=30)
    4.17 ( 23.233 )
    0.69 ( 24.518 )
        Symptoms: Change at C8D1 (n=10, n=31)
    5.00 ( 17.213 )
    -2.96 ( 24.395 )
        Symptoms: Change at C9D1 (n=11, n=28)
    8.33 ( 21.246 )
    -3.42 ( 24.455 )
        Functioning: Change at C2D1 (n=54, n=84)
    -3.49 ( 19.183 )
    -4.98 ( 23.650 )
        Functioning: Change at C3D1 (n=26, n=63)
    -5.00 ( 18.166 )
    -4.76 ( 20.203 )
        Functioning: Change at C4D1 (n=22, n=51)
    -8.18 ( 24.119 )
    -5.82 ( 23.275 )
        Functioning: Change at C5D1 (n=17, n=47)
    -7.06 ( 20.746 )
    -3.76 ( 16.369 )
        Functioning: Change at C6D1 (n=16, n=35)
    -12.08 ( 27.022 )
    -11.14 ( 18.113 )
        Functioning: Change at C7D1 (n=12, n=30)
    -3.89 ( 19.583 )
    -6.00 ( 15.767 )
        Functioning: Change at C8D1 (n=10, n=30)
    -7.33 ( 23.402 )
    -7.00 ( 17.926 )
        Functioning: Change at C9D1 (n=11, n=28)
    -9.39 ( 21.072 )
    -5.60 ( 20.965 )
    No statistical analyses for this end point

    Secondary: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs

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    End point title
    		 Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs are defined as AEs that developed or worsened during the on-treatment period and treatment-related AEs that occur during post-treatment period. A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. Any TEAE included participants with both serious and non-serious TEAEs. The safety analysis set (SAF) included all enrolled participants who received any study drug for each group.
    End point type
    Secondary
    End point timeframe
    Up to 1422 days (approximately 46 months)
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Participants
        Participants with any TEAEs
    51
    83
        Participants with any Serious TEAEs
    16
    31
    No statistical analyses for this end point

    Secondary: Serum Concentration at Pre-infusion (Ctrough)

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    End point title
    		 Serum Concentration at Pre-infusion (Ctrough)
    End point description
    Ctrough of cemiplimab was reported; Pharmacokinetic (PK) analysis set (included all participants who received any cemiplimab and had at least one non-missing post-baseline measurement of cemiplimab concentration in serum); Number ("n") signifies those participants who were evaluable at specific time points.
    End point type
    Secondary
    End point timeframe
    At pre-infusion on Cycle 1 Day 22 and Cycle 3 Day 1 (Each cycle of 9 weeks)
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: Milligram per Liter (mg/L)
    arithmetic mean (standard deviation)
        Cycle 1 Day 22 (n=50, n=78)
    28.3 ( 18.4 )
    29.8 ( 12.0 )
        Cycle 3 Day 1 (n=32, n=66)
    60.6 ( 28.2 )
    68.6 ( 32.8 )
    No statistical analyses for this end point

    Secondary: Serum Concentration at End of Infusion (Cmax)

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    End point title
    		 Serum Concentration at End of Infusion (Cmax)
    End point description
    Cmax of cemiplimab was reported; PK analysis set (included all participants who received any cemiplimab and had at least one non-missing post-baseline measurement of cemiplimab concentration in serum); Number ("n") signifies those participants who were evaluable at specific time points
    End point type
    Secondary
    End point timeframe
    At end-of-infusion (within 10 minutes after the end of infusion) on Cycle 1 Day 1 and Cycle 3 Day 1 (Each cycle of 9 weeks)
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    54
    84
    Units: mg/L
    arithmetic mean (standard deviation)
        Cycle 1 Day 1 (n=47, n=81)
    103 ( 25.2 )
    104 ( 45.5 )
        Cycle 3 Day 1 (n=54, n=84)
    160 ( 52.7 )
    192 ( 91.6 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Anti-Drug Antibody (ADA) Status

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    End point title
    		 Number of Participants With Anti-Drug Antibody (ADA) Status
    End point description
    Immunogenicity was characterized by ADA responses & titers. Responses categories: Negative - ADA negative response at all time points, regardless of missing samples; Pre-existing immunoreactivity - ADA positive response at baseline with all post first dose negative results or positive response at baseline with all post first dose ADA responses < 9-fold over baseline titer levels; Treatment-boosted response - positive response in the assay post first dose, ≥ 9-fold over baseline titer levels, when baseline results are positive; Treatment-emergent response - ADA positive response in the cemiplimab ADA assay post first dose when baseline results = negative or missing. The anti-drug antibody set included all participants who received cemiplimab and who had at least 1 non-missing result in the ADA assay after the first dose of the study drug.
    End point type
    Secondary
    End point timeframe
    Cycle 1: Days 1 and 43; Cycles 3 and 5: Day 1 (Each cycle of 9 weeks)
    End point values
    		 Group 1: Metastatic BCC (mBCC) Group 2: Unresectable Locally Advanced BCC (laBCC)
    Number of subjects analysed
    52
    81
    Units: Participants
        Negative ADA
    50
    74
        Pre-Existing ADA
    2
    2
        Treatment-emergent ADA
    0
    5
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All Adverse events (AEs) were collected from the time of informed consent signature until 105 days after the last dose of study drug (up to 2129 days)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Group 2: Unresectable Locally Advanced BCC (laBCC)
    Reporting group description
    		 Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.

    Reporting group title
    Group 1: Metastatic BCC (mBCC)
    Reporting group description
    		 Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.

    Serious adverse events
    		 Group 2: Unresectable Locally Advanced BCC (laBCC) Group 1: Metastatic BCC (mBCC)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    33 / 84 (39.29%)
    18 / 54 (33.33%)
         number of deaths (all causes)
    21
    22
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Brain neoplasm malignant
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Infected neoplasm
         subjects affected / exposed
    2 / 84 (2.38%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Basal cell carcinoma
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lymphoproliferative disorder
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Meningioma
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Phlebitis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Peripheral ischaemia
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypotension
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypertensive crisis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General physical health deterioration
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Sarcoidosis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary oedema
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Haemoptysis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Pleural effusion
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonitis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory failure
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Delirium
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Investigations
    Weight decreased
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Tibia fracture
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Procedural pain
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Multiple fractures
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infusion related reaction
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Fall
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Upper limb fracture
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Radial head dislocation
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Wound haemorrhage
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac failure
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Supraventricular tachycardia
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Acute coronary syndrome
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Atrial fibrillation
         subjects affected / exposed
    0 / 84 (0.00%)
    2 / 54 (3.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Autoimmune myocarditis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Autoimmune pericarditis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune-mediated myocarditis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    2 / 84 (2.38%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Cerebrovascular accident
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cerebrospinal fluid leakage
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Brain oedema
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Headache
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Facial paralysis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Somnolence
         subjects affected / exposed
    1 / 84 (1.19%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Haemorrhage intracranial
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Dizziness
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Pancytopenia
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lymphadenopathy mediastinal
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Anaemia
         subjects affected / exposed
    2 / 84 (2.38%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ear and labyrinth disorders
    Ear disorder
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Colitis
         subjects affected / exposed
    2 / 84 (2.38%)
    2 / 54 (3.70%)
         occurrences causally related to treatment / all
    2 / 2
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Erosive oesophagitis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune-mediated enterocolitis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Constipation
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Immune-mediated hepatitis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Autoimmune hepatitis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Dermal cyst
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    2 / 84 (2.38%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Urinary retention
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Endocrine disorders
    Adrenal insufficiency
         subjects affected / exposed
    2 / 84 (2.38%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypophysitis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Dupuytren's contracture
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Back pain
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Oral candidiasis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatitis C
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    4 / 84 (4.76%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Soft tissue infection
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Clostridium difficile colitis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Atypical pneumonia
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Arthritis bacterial
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Wound infection staphylococcal
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Subcutaneous abscess
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Clostridium difficile infection
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infection
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Erysipelas
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia staphylococcal
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Skin infection
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Type 2 diabetes mellitus
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cachexia
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Group 2: Unresectable Locally Advanced BCC (laBCC) Group 1: Metastatic BCC (mBCC)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    76 / 84 (90.48%)
    50 / 54 (92.59%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Seborrhoeic keratosis
         subjects affected / exposed
    5 / 84 (5.95%)
    1 / 54 (1.85%)
         occurrences all number
    5
    1
    Basal cell carcinoma
         subjects affected / exposed
    8 / 84 (9.52%)
    3 / 54 (5.56%)
         occurrences all number
    9
    5
    Tumour haemorrhage
         subjects affected / exposed
    9 / 84 (10.71%)
    1 / 54 (1.85%)
         occurrences all number
    12
    1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    9 / 84 (10.71%)
    12 / 54 (22.22%)
         occurrences all number
    15
    33
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    5 / 84 (5.95%)
    2 / 54 (3.70%)
         occurrences all number
    7
    2
    Fatigue
         subjects affected / exposed
    25 / 84 (29.76%)
    24 / 54 (44.44%)
         occurrences all number
    35
    32
    Pain
         subjects affected / exposed
    2 / 84 (2.38%)
    3 / 54 (5.56%)
         occurrences all number
    2
    4
    Pyrexia
         subjects affected / exposed
    5 / 84 (5.95%)
    7 / 54 (12.96%)
         occurrences all number
    7
    9
    Oedema peripheral
         subjects affected / exposed
    5 / 84 (5.95%)
    7 / 54 (12.96%)
         occurrences all number
    5
    9
    Asthenia
         subjects affected / exposed
    17 / 84 (20.24%)
    5 / 54 (9.26%)
         occurrences all number
    27
    6
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    10 / 84 (11.90%)
    4 / 54 (7.41%)
         occurrences all number
    14
    5
    Dyspnoea
         subjects affected / exposed
    11 / 84 (13.10%)
    4 / 54 (7.41%)
         occurrences all number
    18
    5
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    3 / 84 (3.57%)
    3 / 54 (5.56%)
         occurrences all number
    3
    3
    Investigations
    Weight increased
         subjects affected / exposed
    2 / 84 (2.38%)
    8 / 54 (14.81%)
         occurrences all number
    2
    12
    Alanine aminotransferase increased
         subjects affected / exposed
    4 / 84 (4.76%)
    3 / 54 (5.56%)
         occurrences all number
    6
    3
    Blood creatinine increased
         subjects affected / exposed
    8 / 84 (9.52%)
    4 / 54 (7.41%)
         occurrences all number
    9
    4
    Weight decreased
         subjects affected / exposed
    7 / 84 (8.33%)
    5 / 54 (9.26%)
         occurrences all number
    7
    6
    Blood creatine phosphokinase increased
         subjects affected / exposed
    6 / 84 (7.14%)
    4 / 54 (7.41%)
         occurrences all number
    8
    5
    Injury, poisoning and procedural complications
    Infusion related reaction
         subjects affected / exposed
    0 / 84 (0.00%)
    3 / 54 (5.56%)
         occurrences all number
    0
    3
    Fall
         subjects affected / exposed
    5 / 84 (5.95%)
    4 / 54 (7.41%)
         occurrences all number
    5
    7
    Nervous system disorders
    Headache
         subjects affected / exposed
    12 / 84 (14.29%)
    7 / 54 (12.96%)
         occurrences all number
    14
    8
    Paraesthesia
         subjects affected / exposed
    4 / 84 (4.76%)
    3 / 54 (5.56%)
         occurrences all number
    4
    3
    Dizziness
         subjects affected / exposed
    7 / 84 (8.33%)
    5 / 54 (9.26%)
         occurrences all number
    8
    5
    Blood and lymphatic system disorders
    Leukocytosis
         subjects affected / exposed
    6 / 84 (7.14%)
    1 / 54 (1.85%)
         occurrences all number
    6
    1
    Anaemia
         subjects affected / exposed
    13 / 84 (15.48%)
    6 / 54 (11.11%)
         occurrences all number
    19
    7
    Eye disorders
    Cataract
         subjects affected / exposed
    5 / 84 (5.95%)
    0 / 54 (0.00%)
         occurrences all number
    6
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    20 / 84 (23.81%)
    20 / 54 (37.04%)
         occurrences all number
    33
    26
    Abdominal pain
         subjects affected / exposed
    6 / 84 (7.14%)
    4 / 54 (7.41%)
         occurrences all number
    7
    4
    Dry mouth
         subjects affected / exposed
    3 / 84 (3.57%)
    4 / 54 (7.41%)
         occurrences all number
    3
    4
    Constipation
         subjects affected / exposed
    5 / 84 (5.95%)
    12 / 54 (22.22%)
         occurrences all number
    6
    14
    Vomiting
         subjects affected / exposed
    6 / 84 (7.14%)
    7 / 54 (12.96%)
         occurrences all number
    10
    9
    Nausea
         subjects affected / exposed
    13 / 84 (15.48%)
    6 / 54 (11.11%)
         occurrences all number
    19
    9
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    6 / 84 (7.14%)
    4 / 54 (7.41%)
         occurrences all number
    6
    4
    Rash maculo-papular
         subjects affected / exposed
    6 / 84 (7.14%)
    5 / 54 (9.26%)
         occurrences all number
    7
    6
    Dermatitis
         subjects affected / exposed
    5 / 84 (5.95%)
    0 / 54 (0.00%)
         occurrences all number
    6
    0
    Dry skin
         subjects affected / exposed
    7 / 84 (8.33%)
    5 / 54 (9.26%)
         occurrences all number
    7
    6
    Actinic keratosis
         subjects affected / exposed
    7 / 84 (8.33%)
    3 / 54 (5.56%)
         occurrences all number
    10
    3
    Pruritus
         subjects affected / exposed
    20 / 84 (23.81%)
    8 / 54 (14.81%)
         occurrences all number
    24
    13
    Eczema
         subjects affected / exposed
    5 / 84 (5.95%)
    5 / 54 (9.26%)
         occurrences all number
    5
    7
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    3 / 84 (3.57%)
    4 / 54 (7.41%)
         occurrences all number
    3
    5
    Endocrine disorders
    Hyperthyroidism
         subjects affected / exposed
    2 / 84 (2.38%)
    5 / 54 (9.26%)
         occurrences all number
    2
    7
    Hypothyroidism
         subjects affected / exposed
    8 / 84 (9.52%)
    4 / 54 (7.41%)
         occurrences all number
    9
    4
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    16 / 84 (19.05%)
    9 / 54 (16.67%)
         occurrences all number
    24
    10
    Neck pain
         subjects affected / exposed
    2 / 84 (2.38%)
    4 / 54 (7.41%)
         occurrences all number
    4
    4
    Back pain
         subjects affected / exposed
    5 / 84 (5.95%)
    5 / 54 (9.26%)
         occurrences all number
    6
    5
    Myalgia
         subjects affected / exposed
    3 / 84 (3.57%)
    5 / 54 (9.26%)
         occurrences all number
    3
    6
    Pain in extremity
         subjects affected / exposed
    4 / 84 (4.76%)
    5 / 54 (9.26%)
         occurrences all number
    5
    9
    Muscle spasms
         subjects affected / exposed
    5 / 84 (5.95%)
    3 / 54 (5.56%)
         occurrences all number
    5
    3
    Infections and infestations
    Conjunctivitis
         subjects affected / exposed
    5 / 84 (5.95%)
    0 / 54 (0.00%)
         occurrences all number
    5
    0
    Urinary tract infection
         subjects affected / exposed
    10 / 84 (11.90%)
    4 / 54 (7.41%)
         occurrences all number
    11
    5
    Bronchitis
         subjects affected / exposed
    6 / 84 (7.14%)
    0 / 54 (0.00%)
         occurrences all number
    7
    0
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 84 (7.14%)
    3 / 54 (5.56%)
         occurrences all number
    6
    3
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    2 / 84 (2.38%)
    6 / 54 (11.11%)
         occurrences all number
    2
    8
    Hypokalaemia
         subjects affected / exposed
    4 / 84 (4.76%)
    4 / 54 (7.41%)
         occurrences all number
    6
    4
    Hypoalbuminaemia
         subjects affected / exposed
    6 / 84 (7.14%)
    1 / 54 (1.85%)
         occurrences all number
    12
    2
    Decreased appetite
         subjects affected / exposed
    14 / 84 (16.67%)
    6 / 54 (11.11%)
         occurrences all number
    18
    6

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Nov 2016
    The purposes of this amendment were to: Note regional laboratory testing for bicarbonate, Add a window for the duration of the REGN2810 infusion, Update the contraception language in the exclusion criteria
    23 Mar 2017
    The purpose of the amendment was to revise the protocol based on regulatory agency advice and the internal program review by the sponsor.
    03 Jul 2017
    Modified exclusion criteria; Additional safety guidance language added; Added an adverse event of special interest (AESI) to the list of AESIs
    29 Jul 2019
    Clarified the details of data cut for the primary analysis for Group 2; Added an interim analysis for Group 1; Clarified eligibility for retreatment; Posttreatment follow-up was extended; Neutralizing antibody analysis was included; Revised exclusion criteria; Modifications for consistency and clarity, and administrative updates

    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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