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    Clinical Trial Results:
    A Phase 2, Randomized, Placebo-Controlled Study of Safety and Efficacy, Following Repeat-Dose Administration of Evinacumab (anti-ANGPTL3) in Patients with Severe Hypertriglyceridemia (sHTG) at Risk for Acute Pancreatitis

    Summary
    EudraCT number
    2016-003307-62
    Trial protocol
    GB   IT  
    Global end of trial date
    23 Jul 2020

    Results information
    Results version number
    v1(current)
    This version publication date
    06 Aug 2021
    First version publication date
    06 Aug 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    R1500-HTG-1522
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03452228
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Regeneron Pharmaceuticals, Inc.
    Sponsor organisation address
    777 Old Saw Mill River Rd., 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
    23 Jul 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jul 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Primary objectives: Determine change in triglyceride (TG) levels following 12 wks of repeated intravenous (IV) doses of evinacumab in subset of subjects with a documented history of sHTG (TG ≥1000 milligrams per decilter (mg/dL), a TG level of at least 500 mg/dL at screening, a history of acute pancreatitis & w/out loss-of-function (LOF) mutations in genes in lipoprotein lipase (LPL) pathway; Assess whether reduction in TG of at least 40% from baseline placebo period was achieved. Secondary objectives: Determine percent change from baseline in TG levels following evinacumab overall & in subgroups; Assess changes in reported abdominal/gastrointestinal (GI) symptoms, dietary habits, & symptom/dietary impact measures; Assess degree of pancreatic injury/inflammation at baseline & following 12 & 24 wks of treatment; Evaluate total evinacumab, total ANGPTL3, & anti-drug antibody (ADA) during evinacumab treatment & follow-up periods; Evaluate safety & tolerability of evinacumab
    Protection of trial subjects
    It is the responsibility of both the sponsor and the investigator(s) to ensure that this clinical study will be conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki, and that are consistent with the International Council for Harmonisation (ICH) guidelines for GCP and applicable regulatory requirements.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    14 Aug 2018
    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
    Canada: 11
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    United Kingdom: 6
    Country: Number of subjects enrolled
    United States: 30
    Worldwide total number of subjects
    52
    EEA total number of subjects
    5
    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)
    50
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    A total of 17 centers enrolled subjects in Italy, Canada, United Kingdom of Great Britain and Northern Ireland, and United States.

    Pre-assignment
    Screening details
    The study consisted of a screening period of up to 37 days.

    Period 1
    Period 1 title
    12-week double-blind
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Data analyst, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    DB Placebo IV Q4W/SB Evinacumab
    Arm description
    Subjects received placebo matching evinacumab intravenously (IV) every 4 weeks (Q4W) on days 1, 29, and 57 during the 12-week double-blind treatment period (DBTP). During the 12-week single-blind treatment period (SBTP), subjects received evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Intravenous (IV) placebo

    Arm title
    DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Arm description
    Subjects received evinacumab 15 mg/kg IV Q4W on days 1, 29, and 57 during the 12-week DBTP. During the 12-week SBTP, subjects continued to receive evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.
    Arm type
    Experimental

    Investigational medicinal product name
    evinacumab
    Investigational medicinal product code
    REGN1500
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Intravenous (IV) evinacumab

    Number of subjects in period 1
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Started
    17
    35
    Randomized and Treated
    16
    35
    Completed
    15
    32
    Not completed
    2
    3
         Physician decision
    1
    -
         Adverse event, non-fatal
    -
    2
         Lost to follow-up
    1
    1
    Period 2
    Period 2 title
    12-week single-blind
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Single blind
    Roles blinded
    Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    DB Placebo/SB Evinacumab IV 15mg/kg Q4W
    Arm description
    Subjects received placebo matching evinacumab intravenously (IV) every 4 weeks (Q4W) on days 1, 29, and 57 during the 12-week double-blind treatment period (DBTP). During the 12-week single-blind treatment period (SBTP), subjects received evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.
    Arm type
    Experimental

    Investigational medicinal product name
    evinacumab
    Investigational medicinal product code
    REGN1500
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Intravenous (IV) evinacumab

    Arm title
    DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Arm description
    Subjects received evinacumab 15 mg/kg IV Q4W on days 1, 29, and 57 during the 12-week DBTP. During the 12-week SBTP, subjects continued to receive evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.
    Arm type
    Experimental

    Investigational medicinal product name
    evinacumab
    Investigational medicinal product code
    REGN1500
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Intravenous (IV) evinacumab

    Number of subjects in period 2
    DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Started
    15
    32
    Completed SBTP
    15
    32
    Completed off-treatment follow-up
    14
    29
    Completed
    14
    29
    Not completed
    1
    3
         Consent withdrawn by subject
    1
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    DB Placebo IV Q4W/SB Evinacumab
    Reporting group description
    Subjects received placebo matching evinacumab intravenously (IV) every 4 weeks (Q4W) on days 1, 29, and 57 during the 12-week double-blind treatment period (DBTP). During the 12-week single-blind treatment period (SBTP), subjects received evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Reporting group title
    DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Reporting group description
    Subjects received evinacumab 15 mg/kg IV Q4W on days 1, 29, and 57 during the 12-week DBTP. During the 12-week SBTP, subjects continued to receive evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Reporting group values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab Total
    Number of subjects
    17 35 52
    Age categorical
    Units: Subjects
        In utero
    0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0
        Newborns (0-27 days)
    0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0
        Children (2-11 years)
    0 0 0
        Adolescents (12-17 years)
    0 0 0
        Adults (18-64 years)
    15 35 50
        From 65-84 years
    2 0 2
        85 years and over
    0 0 0
    Sex: Female, Male
    Units: Subjects
        Female
    7 17 24
        Male
    10 18 28
    Race
    Units: Subjects
        White
    13 29 42
        Black or African American
    0 1 1
        Asian
    1 5 6
        American Indian or Alaska Native
    0 0 0
        Native Hawaiian or other Pacific Islander
    0 0 0
        Not Reported
    0 0 0
        Other
    3 0 3

    End points

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    End points reporting groups
    Reporting group title
    DB Placebo IV Q4W/SB Evinacumab
    Reporting group description
    Subjects received placebo matching evinacumab intravenously (IV) every 4 weeks (Q4W) on days 1, 29, and 57 during the 12-week double-blind treatment period (DBTP). During the 12-week single-blind treatment period (SBTP), subjects received evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Reporting group title
    DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Reporting group description
    Subjects received evinacumab 15 mg/kg IV Q4W on days 1, 29, and 57 during the 12-week DBTP. During the 12-week SBTP, subjects continued to receive evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.
    Reporting group title
    DB Placebo/SB Evinacumab IV 15mg/kg Q4W
    Reporting group description
    Subjects received placebo matching evinacumab intravenously (IV) every 4 weeks (Q4W) on days 1, 29, and 57 during the 12-week double-blind treatment period (DBTP). During the 12-week single-blind treatment period (SBTP), subjects received evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Reporting group title
    DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Reporting group description
    Subjects received evinacumab 15 mg/kg IV Q4W on days 1, 29, and 57 during the 12-week DBTP. During the 12-week SBTP, subjects continued to receive evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Subject analysis set title
    Actual Cohort 1 DB Placebo IV Q4W/SB evinacumab
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Actual Cohort 1 (DBTP 12 weeks placebo; SBTP 12 weeks evinacumab) includes subjects with homozygous or compound heterozygous loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data.

    Subject analysis set title
    Actual Cohort 1 DB/SB Evinacumab IV 15mg/kg Q4W
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Actual Cohort 1 (DBTP/SBTP evinacumab) includes subjects with homozygous or compound heterozygous loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data.

    Subject analysis set title
    Actual Cohort 2 DB Placebo IV Q4W/SB evinacumab
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Actual Cohort 2 (DBTP 12 weeks placebo; SBTP 12 weeks evinacumab) includes subjects with heterozygous loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data.

    Subject analysis set title
    Actual Cohort 2 DB/SB Evinacumab IV 15mg/kg Q4W
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Actual Cohort 2 (DBTP/SBTP evinacumab) includes subjects with heterozygous loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data.

    Subject analysis set title
    Actual Cohort 3 DB Placebo IV Q4W/SB evinacumab
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Actual Cohort 3 (DBTP 12 weeks placebo; SBTP 12 weeks evinacumab) includes subjects without loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data.

    Subject analysis set title
    Actual Cohort 3 DB/SB Evinacumab IV 15mg/kg Q4W
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Actual Cohort 3 (DBTP/SBTP evinacumab) includes subjects without loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data.

    Primary: Percent lowering of triglyceride (TG) levels from baseline following 12 weeks of repeated IV doses of evinacumab (Actual Cohort 3)

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    End point title
    Percent lowering of triglyceride (TG) levels from baseline following 12 weeks of repeated IV doses of evinacumab (Actual Cohort 3)
    End point description
    Actual cohort 3 includes subjects without loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data. All subjects included received 12 weeks of evinacumab treatment, regardless of DBTP treatment assignment. For subjects randomized to evinacumab treatment group, baseline (Week 0) was defined as the geometric mean of all available TG results at day -28, day -14 and week 0; for subjects randomized to placebo treatment group, baseline (Week 12) was defined as the geometric mean of all available TG results at weeks 6, 8, and 12. Full analysis set (FAS) includes all randomized subjects who received any double-blind/single-blind study drug; Mixed-effect repeated measures model (MMRM) analysis
    End point type
    Primary
    End point timeframe
    12 weeks
    End point values
    Actual Cohort 3 DB Placebo IV Q4W/SB evinacumab Actual Cohort 3 DB/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    5
    14
    Units: Percent Change
        least squares mean (standard error)
    19.2 ± 99.1
    -37.2 ± 42.9
    Statistical analysis title
    SB Evinacumab, DB Evinacumab
    Statistical analysis description
    Mixed-effect repeated measures model (MMRM) analyzed subject's natural log transformation TG ratio (each evinacumab treatment visit/baseline) with the fixed categorical effects of evinacumab treatment visit (i.e. exposed to evinacumab visits only), actual cohort, and evinacumab treatment visit by actual cohort interaction, as well as the continuous covariates of the log transformation of baseline TG and the log transformation of baseline TG by evinacumab treatment visit interaction.
    Comparison groups
    Actual Cohort 3 DB/SB Evinacumab IV 15mg/kg Q4W v Actual Cohort 3 DB Placebo IV Q4W/SB evinacumab
    Number of subjects included in analysis
    19
    Analysis specification
    Pre-specified
    Analysis type
    other
    Method
    Parameter type
    Least Squares (LS) Mean
    Point estimate
    -27.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -71.2
         upper limit
    84.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    37.4

    Secondary: Percent TG lowering from baseline following 12 weeks of repeated IV doses of evinacumab (Actual Cohort 2)

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    End point title
    Percent TG lowering from baseline following 12 weeks of repeated IV doses of evinacumab (Actual Cohort 2)
    End point description
    Actual cohort 2 includes subjects with heterozygous loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data. All subjects included received 12 weeks of evinacumab treatment, regardless of DBTP treatment assignment. For subjects randomized to evinacumab treatment group, baseline (Week 0) was defined as the geometric mean of all available TG results at day -28, day -14 and week 0; for subjects randomized to placebo treatment group, baseline (Week 12) was defined as the geometric mean of all available TG results at weeks 6, 8, and 12. Full analysis set (FAS) includes all randomized subjects who received any double-blind/single-blind study drug; Mixed-effect repeated measures model (MMRM) analysis
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Actual Cohort 2 DB Placebo IV Q4W/SB evinacumab Actual Cohort 2 DB/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    6
    9
    Units: Percent Change
        least squares mean (standard error)
    -45.1 ± 41.6
    -16.3 ± 66.2
    Statistical analysis title
    SB evinacumab, DB evinacumab
    Statistical analysis description
    Mixed-effect repeated measures model (MMRM) analyzed subject's natural log transformation TG ratio (each evinacumab treatment visit/baseline) with the fixed categorical effects of evinacumab treatment visit (i.e. exposed to evinacumab visits only), actual cohort, and evinacumab treatment visit by actual cohort interaction, as well as the continuous covariates of the log transformation of baseline TG and the log transformation of baseline TG by evinacumab treatment visit interaction.
    Comparison groups
    Actual Cohort 2 DB Placebo IV Q4W/SB evinacumab v Actual Cohort 2 DB/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects included in analysis
    15
    Analysis specification
    Pre-specified
    Analysis type
    other
    Method
    Parameter type
    Least Squares (LS) Mean
    Point estimate
    -30.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -74.4
         upper limit
    90.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    38.1

    Secondary: Percent TG lowering from baseline following 12 weeks of repeated IV doses of evinacumab (Actual Cohort 1)

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    End point title
    Percent TG lowering from baseline following 12 weeks of repeated IV doses of evinacumab (Actual Cohort 1)
    End point description
    Actual cohort 1 includes subjects with homozygous or compound heterozygous loss-of-function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway as was determined based on genotype data. All subjects included received 12 weeks of evinacumab treatment, regardless of DBTP treatment assignment. For subjects randomized to evinacumab treatment group, baseline (Week 0) was defined as the geometric mean of all available TG results at day -28, day -14 and week 0; for subjects randomized to placebo treatment group, baseline (Week 12) was defined as the geometric mean of all available TG results at weeks 6, 8, and 12. Full analysis set (FAS) includes all randomized subjects who received any double-blind/single-blind study drug; Mixed-effect repeated measures model (MMRM) analysis; 99999=not evaluable
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Actual Cohort 1 DB Placebo IV Q4W/SB evinacumab Actual Cohort 1 DB/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    4
    12
    Units: Percent Change
        least squares mean (standard error)
    99999 ± 99999
    -25.7 ± 26.6
    Statistical analysis title
    SB evinacumab, DB evinacumab
    Statistical analysis description
    Mixed-effect repeated measures model (MMRM) analyzes patient’s natural log transformation TG ratio (each evinacumab treatment visit/baseline) with the fixed categorical effects of evinacumab treatment visit (i.e. exposed to evinacumab visits only), as well as the continuous covariates of the log transformation of baseline TG and the log transformation of baseline TG by evinacumab treatment visit interaction.
    Comparison groups
    Actual Cohort 1 DB Placebo IV Q4W/SB evinacumab v Actual Cohort 1 DB/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    other
    Method
    Parameter type
    Least Squares (LS) Mean
    Point estimate
    -31.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -65.2
         upper limit
    34
    Variability estimate
    Standard error of the mean
    Dispersion value
    22.4

    Secondary: Percent TG lowering from baseline following 2 to 24 weeks of repeated IV doses of evinacumab (Actual Cohorts 1,2, and 3)

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    End point title
    Percent TG lowering from baseline following 2 to 24 weeks of repeated IV doses of evinacumab (Actual Cohorts 1,2, and 3)
    End point description
    Includes subjects initially randomized to evinacumab per cohort during the DBTP (FAS DBTP and SBTP); Actual cohort was determined based on genotype data. Baseline fasting TG is defined as the mean of the last 2 measurements during the placebo run-in (day -28, day -14) and week 0; n=number of subjects evaluable at that time point.
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    Actual Cohort 1 DB/SB Evinacumab IV 15mg/kg Q4W Actual Cohort 2 DB/SB Evinacumab IV 15mg/kg Q4W Actual Cohort 3 DB/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    11
    9
    12
    Units: Percent Change
    median (inter-quartile range (Q1-Q3))
        Week 2 (n=11, 9, 11)
    -28.3 (-40.3 to -6.5)
    -77.5 (-81.2 to -58.9)
    -74.4 (-84.2 to -37.1)
        Week 4 (n=11, 9, 12)
    -19.0 (-69.6 to -2.7)
    -48.0 (-76.2 to -29.2)
    -70.3 (-83.4 to -50.2)
        Week 6 (n=11, 9, 11)
    -47.1 (-59.1 to -5.6)
    -84.6 (-85.7 to -61.3)
    -71.3 (-87.0 to -39.5)
        Week 8 (n=11, 9, 12)
    -29.5 (-72.0 to 4.8)
    -69.2 (-72.2 to -58.4)
    -62.5 (-84.8 to 48.0)
        Week 12 (n=11, 9, 12)
    -27.7 (-68.5 to 2.2)
    -64.8 (-84.5 to -41.8)
    -81.7 (-90.5 to -21.7)
        Week 16 (n=11, 9, 12)
    -16.2 (-56.7 to 29.3)
    -71.3 (-80.8 to -57.1)
    -80.4 (-87.2 to -37.3)
        Week 20 (n=11, 9, 12)
    -37.2 (-49.9 to -8.8)
    -65.5 (-70.2 to -45.8)
    -75.7 (-88.1 to -6.6)
        Week 24 (n=11, 9, 12)
    -7.7 (-31.8 to 9.1)
    -75.7 (-82.3 to -57.1)
    -71.4 (-86.5 to -54.0)
    No statistical analyses for this end point

    Secondary: Percent TG lowering from baseline following 2 to 24 weeks of repeated IV doses of evinacumab overall

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    End point title
    Percent TG lowering from baseline following 2 to 24 weeks of repeated IV doses of evinacumab overall [1]
    End point description
    Percent TG lowering from baseline following 2 to 24 weeks of repeated IV doses of evinacumab in subjects randomized to evinacumab during the DBTP. Baseline fasting TG is defined as the mean of the last 2 measurements during the placebo run-in (day -28, day -14) and week 0; n=number of subjects evaluable at that time point; 99999=not applicable to treatment period; Double-blind safety analysis set: Randomized population who received at least 1 dose or part of a dose of double-blind study drug; Single-blind safety analysis set: Randomized population who received at least 1 dose or part of a dose of single-blind study drug
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    Notes
    [1] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Endpoint includes only subjects randomized to evinacumab treatment group.
    End point values
    DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    35 [2]
    32 [3]
    Units: Percent Change
    median (inter-quartile range (Q1-Q3))
        Week 2 (n=31)
    -58.86 (-78.62 to -27.62)
    99999 (99999 to 99999)
        Week 4 (n=32)
    -48.60 (-77.35 to -14.40)
    99999 (99999 to 99999)
        Week 6 (n=31)
    -61.25 (-85.38 to -39.51)
    99999 (99999 to 99999)
        Week 8 (n=32)
    -60.08 (-80.96 to -3.14)
    99999 (99999 to 99999)
        Week 12 (n=32)
    -56.59 (-87.09 to -12.34)
    99999 (99999 to 99999)
        Week 16 (n=32)
    99999 (99999 to 99999)
    -63.10 (-82.83 to -15.47)
        Week 20 (n=32)
    99999 (99999 to 99999)
    -47.86 (-82.53 to -21.52)
        Week 24 (n=32)
    99999 (99999 to 99999)
    -57.90 (-83.85 to -6.24)
    Notes
    [2] - Double-Blind Safety Analysis Set - DBTP
    [3] - Single-Blind Safety Analysis Set - SBTP
    No statistical analyses for this end point

    Secondary: Change from baseline in subject reported abdominal and gastrointestinal symptoms scale

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    End point title
    Change from baseline in subject reported abdominal and gastrointestinal symptoms scale
    End point description
    Abdominal and gastrointestinal (GI) daily symptoms were assessed using the Hypertriglyceridemia and Acute Pancreatitis Symptom Scale (HAP-SS), a 19-item measure of symptoms that has a 24-hour recall period and consists of a total score and four domain scores: pain; abdominal symptoms; physical symptoms; other symptoms, each scored 0 (no symptoms) to 100 (severe symptoms). Patient-reported outcomes (PRO) analysis set: all randomized subjects who received any double-blind study treatment with a baseline and at least 1 non-missing post-baseline PRO evaluation; n=number of subjects evaluable at that time point; 99999=not applicable to treatment period
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    12
    9
    23
    19
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Week 12 (DBTP) n=12; n=23
    -0.69 ± 6.930
    99999 ± 99999
    -0.74 ± 5.102
    99999 ± 99999
        Week 24 (SBTP) n=9; n=19
    99999 ± 99999
    -2.47 ± 5.860
    99999 ± 99999
    1.00 ± 4.319
    No statistical analyses for this end point

    Secondary: Change from baseline in patient reported daily dietary habits and impact questionnaire

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    End point title
    Change from baseline in patient reported daily dietary habits and impact questionnaire
    End point description
    Dietary habits and impact were measured by the Hypertriglyceridemia and Acute Pancreatitis Dietary Behavior (HAP-DB), a 6-item measure of dietary behavior that has a 24-hour recall period using a 5-point frequency Likert scale (1=None of the time to 5=All of the time) and a dietary impact total score, ranging from 6 (no impact) to 30 (severe impact). Patient-reported outcomes (PRO) analysis set: all randomized subjects who received any double-blind study treatment with a baseline and at least 1 non-missing post-baseline PRO evaluation. n=number of subjects evaluable at that time point; 99999=not applicable to treatment period
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    12
    12
    29
    30
    Units: Scores on a Scale
    arithmetic mean (standard deviation)
        Week 12 (DBTP) n=12; n=23
    -2.267 ± 5.5897
    99999 ± 99999
    2.021 ± 15.2152
    99999 ± 99999
        Week 24 (SBTP) n=9; n=19
    99999 ± 99999
    -2.519 ± 6.4489
    99999 ± 99999
    -0.656 ± 8.4428
    No statistical analyses for this end point

    Secondary: Degree of pancreatic injury/inflammation through 18^F-2-Fluoro-2-Deoxy-D Glucose positron emission tomography (18^F-FDG PET): maximum Standardized Uptake Value (SUVmax) at baseline (DBTP only)

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    End point title
    Degree of pancreatic injury/inflammation through 18^F-2-Fluoro-2-Deoxy-D Glucose positron emission tomography (18^F-FDG PET): maximum Standardized Uptake Value (SUVmax) at baseline (DBTP only)
    End point description
    PET analysis set: All randomized subjects who received any double-blind study treatment with a baseline and a post-baseline PET evaluation; PET imaging performed during the DBTP only (per protocol).
    End point type
    Secondary
    End point timeframe
    Baseline
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Number of subjects analysed
    12
    25
    Units: gram/milliliter (g/ml)
        arithmetic mean (standard deviation)
    2.318 ± 0.6168
    2.738 ± 0.6027
    No statistical analyses for this end point

    Secondary: Change from baseline in degree of pancreatic injury/inflammation through 18^F-FDG-PET: SUVmax following 12 weeks of treatment with evinacumab (DBTP only)

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    End point title
    Change from baseline in degree of pancreatic injury/inflammation through 18^F-FDG-PET: SUVmax following 12 weeks of treatment with evinacumab (DBTP only)
    End point description
    PET analysis set: All randomized subjects who received any double-blind study treatment with a baseline and a post-baseline PET evaluation; PET imaging performed during the DBTP only (per protocol).
    End point type
    Secondary
    End point timeframe
    Week 12
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Number of subjects analysed
    12
    25
    Units: g/ml
        arithmetic mean (standard deviation)
    0.576 ± 1.0628
    0.185 ± 0.5830
    No statistical analyses for this end point

    Secondary: Degree of pancreatic injury/inflammation through 18^F-FDG PET: SUVmean at baseline (DBTP only)

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    End point title
    Degree of pancreatic injury/inflammation through 18^F-FDG PET: SUVmean at baseline (DBTP only)
    End point description
    PET analysis set: All randomized subjects who received any double-blind study treatment with a baseline and a post-baseline PET evaluation; PET imaging performed during the DBTP only (per protocol).
    End point type
    Secondary
    End point timeframe
    Baseline
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Number of subjects analysed
    12
    25
    Units: g/ml
        arithmetic mean (standard deviation)
    1.212 ± 0.2513
    1.478 ± 0.2000
    No statistical analyses for this end point

    Secondary: Change from baseline in degree of pancreatic injury/inflammation through 18^F-FDG PET: SUVmean following 12 weeks of treatment with evinacumab (DBTP only)

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    End point title
    Change from baseline in degree of pancreatic injury/inflammation through 18^F-FDG PET: SUVmean following 12 weeks of treatment with evinacumab (DBTP only)
    End point description
    PET analysis set: All randomized subjects who received any double-blind study treatment with a baseline and a post-baseline PET evaluation; PET imaging performed during the DBTP only (per protocol).
    End point type
    Secondary
    End point timeframe
    Week 12
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Number of subjects analysed
    12
    25
    Units: g/ml
        arithmetic mean (standard deviation)
    0.175 ± 0.2117
    -0.007 ± 0.2312
    No statistical analyses for this end point

    Secondary: Degree of pancreatic injury/inflammation through Diffusion Weighted-Magnetic Resonance Imaging (DW-MRI) at baseline as assessed by apparent diffusion coefficient (ADC)

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    End point title
    Degree of pancreatic injury/inflammation through Diffusion Weighted-Magnetic Resonance Imaging (DW-MRI) at baseline as assessed by apparent diffusion coefficient (ADC)
    End point description
    MRI analysis set: All randomized subjects who received any double-blind study treatment with a baseline and at least 1 post-baseline MRI evaluation.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0)
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Number of subjects analysed
    14
    28
    Units: Square-millimeters per second (mm2/sec)
        arithmetic mean (standard deviation)
    0.00144 ± 0.0003033
    0.00154 ± 0.000257
    No statistical analyses for this end point

    Secondary: Change from baseline to degree of pancreatic injury/inflammation through DW-MRI following 12 weeks of treatment with evinacumab as assessed by ADC (DBTP)

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    End point title
    Change from baseline to degree of pancreatic injury/inflammation through DW-MRI following 12 weeks of treatment with evinacumab as assessed by ADC (DBTP)
    End point description
    MRI analysis set: All randomized subjects who received any double-blind study treatment with a baseline and at least 1 post-baseline MRI evaluation.
    End point type
    Secondary
    End point timeframe
    Week 12
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Number of subjects analysed
    13
    26
    Units: mm2/sec
        arithmetic mean (standard deviation)
    -0.00007 ± 0.000107
    0.00001 ± 0.00133
    No statistical analyses for this end point

    Secondary: Change from baseline to degree of pancreatic injury/inflammation through DW-MRI following 24 weeks of treatment with evinacumab as assessed by ADC (SBTP)

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    End point title
    Change from baseline to degree of pancreatic injury/inflammation through DW-MRI following 24 weeks of treatment with evinacumab as assessed by ADC (SBTP)
    End point description
    MRI analysis set: All randomized subjects who received any double-blind study treatment with a baseline and at least 1 post-baseline MRI evaluation.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    13
    24
    Units: mm2/sec
        arithmetic mean (standard deviation)
    -0.00002 ± 0.000125
    0.00000 ± 0.000186
    No statistical analyses for this end point

    Secondary: Total evinacumab concentration in serum

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    End point title
    Total evinacumab concentration in serum
    End point description
    Pharmacokinetic (PK) analysis set: All randomized subjects who received any study drug and have at least 1 non-missing measurement of evinacumab concentration following the first dose of the study drug. Double-blind treatment period (DBTP) is until week 12 Pre-Dose (PD); single-blind treatment period (SBTP) is from week 12 End of Infusion (EOI) until Week 24; n= number of subjects at each time point for each treatment group; 99999=not applicable to treatment period
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    16
    15
    35
    32
    Units: mg/L
    arithmetic mean (standard deviation)
        Week 4 PD (DBTP) n=14;n=31
    0 ± 0
    99999 ± 99999
    73.6 ± 38.3
    99999 ± 99999
        Week 4 EOI (DBTP) n=14;n=30
    0.334 ± 1.25
    99999 ± 99999
    590 ± 153
    99999 ± 99999
        Week 8 PD (DBTP) n=13;n=31
    0 ± 0
    99999 ± 99999
    109 ± 57.2
    99999 ± 99999
        Week 8 EOI (DBTP) n=13;n=32
    28.8 ± 104
    99999 ± 99999
    586 ± 119
    99999 ± 99999
        Week 12 PD (DBTP) n=14;n=31
    0.00639 ± 0.0239
    99999 ± 99999
    124 ± 81.4
    99999 ± 99999
        Week 12 EOI (SBTP) n=13;n=32
    99999 ± 99999
    544 ± 159
    99999 ± 99999
    662 ± 146
        Week 20 PD (SBTP) n=14;n=32
    99999 ± 99999
    113 ± 51.9
    99999 ± 99999
    160 ± 119
        Week 20 EOI (SBTP) n=14;n=32
    99999 ± 99999
    675 ± 146
    99999 ± 99999
    680 ± 199
        Week 24 (Post last dose 4 weeks) n=15;n=32
    99999 ± 99999
    121 ± 76.7
    99999 ± 99999
    134 ± 77.2
        Week 28 (Post last dose 8 weeks) n=12;n=28
    99999 ± 99999
    43.1 ± 47.8
    99999 ± 99999
    40.2 ± 44.6
        Week 36 (Post last dose 16 weeks) n=10;n=23
    99999 ± 99999
    0.239 ± 0.250
    99999 ± 99999
    1.98 ± 7.41
        Week 44 (Post last dose 24 weeks) n=12;n=27
    99999 ± 99999
    0.0190 ± 0.0464
    99999 ± 99999
    0.0680 ± 0.102
    No statistical analyses for this end point

    Secondary: Total Angiopoietin-Like 3 (ANGPTL3) concentration in serum

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    End point title
    Total Angiopoietin-Like 3 (ANGPTL3) concentration in serum
    End point description
    Total target analysis set: All randomized subjects who received any study drug and have at least 1 non-missing measurement of total ANGPTL3 concentration following the first dose of study drug. Treatment assignments for the DBTP are based on the treatment received (placebo or evinacumab). Double-blind treatment period (DBTP) is until week 12 Pre-Dose (PD); single-blind treatment period (SBTP) is from week 12 End of Infusion (EOI) until Week 24; n= number of subjects at each time point for each treatment group; 99999=not applicable to treatment period
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    16
    15
    35
    32
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 4 PD (DBTP) n=14;n=31
    0.111 ± 0.0272
    99999 ± 99999
    0.265 ± 0.0932
    99999 ± 99999
        Week 4 EOI (DBTP) n=14;n=30
    0.106 ± 0.0205
    99999 ± 99999
    0.404 ± 0.132
    99999 ± 99999
        Week 8 PD (DBTP) n=13;n=31
    0.114 ± 0.0333
    99999 ± 99999
    0.307 ± 0.0758
    99999 ± 99999
        Week 8 EOI (DBTP) n=13;n=32
    0.137 ± 0.0977
    99999 ± 99999
    0.415 ± 0.0875
    99999 ± 99999
        Week 12 PD (DBTP) n=14;n=31
    0.107 ± 0.0273
    99999 ± 99999
    0.310 ± 0.0911
    99999 ± 99999
        Week 12 EOI (SBTP) n=13;n=32
    99999 ± 99999
    0.250 ± 0.0940
    99999 ± 99999
    0.394 ± 0.102
        Week 20 PD (SBTP) n=14;n=32
    99999 ± 99999
    0.302 ± 0.0973
    99999 ± 99999
    0.346 ± 0.101
        Week 20 EOI (SBTP) n=14;n=32
    99999 ± 999999
    0.407 ± 0.144
    99999 ± 99999
    0.422 ± 0.118
        Week 24 Post Last Dose 4 Weeks n=15;n=32
    99999 ± 99999
    0.294 ± 0.0954
    99999 ± 99999
    0.331 ± 0.113
        Week 28 Post Last Dose 8 Weeks n=12;n=28
    99999 ± 99999
    0.311 ± 0.0701
    99999 ± 99999
    0.265 ± 0.0984
        Week 36 Post Last Dose 16 Weeks n=10;n=23
    99999 ± 99999
    0.134 ± 0.0487
    99999 ± 99999
    0.193 ± 0.0969
        Week 44 Post Last Dose 24 Weeks n=12;n=27
    99999 ± 99999
    0.107 ± 0.0322
    99999 ± 99999
    0.131 ± 0.0489
    No statistical analyses for this end point

    Secondary: Number of subjects with Anti-Drug (evinacumab) Antibody (ADA)

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    End point title
    Number of subjects with Anti-Drug (evinacumab) Antibody (ADA)
    End point description
    ADA analysis set: All treated subjects who received any amount of study drug (active or placebo [safety analysis set]) and had at least 1 non-missing anti-evinacumab antibody result following the first dose of study drug or placebo. The ADA analysis set is based on the actual treatment received (as treated) rather than as randomized; Summary of ADA status ADA category by DBTP treatment group for Overall Study.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Number of subjects analysed
    15
    34
    Units: Subjects
    number (not applicable)
        Negative any Time (Overall Study)
    14
    29
        Pre-existing Immunoreactivity (Overall Study)
    1
    4
        Treatment-Boosted Response (Overall Study)
    0
    0
        Treatment-Emergent Response (Overall Study)
    0
    1
    No statistical analyses for this end point

    Secondary: Number of subjects with at least one Treatment-Emergent Adverse Event (TEAE)

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    End point title
    Number of subjects with at least one Treatment-Emergent Adverse Event (TEAE)
    End point description
    Double-blind Safety Analysis Set: Randomized population who received at least 1 dose or part of a dose of double-blind study drug; Single-blind Safety Analysis Set (SBTP): Randomized population who received at least 1 dose or part of a dose of single-blind study drug.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    16
    15
    35
    32
    Units: Subjects
        number (not applicable)
    11
    13
    25
    25
    No statistical analyses for this end point

    Secondary: Number of subjects with at least one Serious Adverse Event (SAE)

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    End point title
    Number of subjects with at least one Serious Adverse Event (SAE)
    End point description
    Double-blind Safety Analysis Set: Randomized population who received at least 1 dose or part of a dose of double-blind study drug; Single-blind Safety Analysis Set: Randomized population who received at least 1 dose or part of a dose of single-blind study drug.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    16
    15
    35
    32
    Units: Subjects
        number (not applicable)
    3
    4
    4
    11
    No statistical analyses for this end point

    Secondary: Number of subjects with liver function laboratory abnormalities

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    End point title
    Number of subjects with liver function laboratory abnormalities
    End point description
    Number of subjects with treatment-emergent potentially clinically significant variables (PCSV) during the treatment-emergent adverse event (TEAE) period reported; Double-blind safety analysis set (SAF): Randomized population who received at least 1 dose or part of a dose of double-blind study drug. Subjects analyzed according to treatment received (placebo or evinacumab); Single-Blind SAF: Randomized population who received at least 1 dose or part of a dose of single-blind study drug (SBTP); [Alanine aminotransferase (ALT); Aspartate aminotransferase (AST); Baseline (BL); Upper limit of normal (ULN)]
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    DB Placebo IV Q4W/SB Evinacumab DB Placebo/SB Evinacumab IV 15mg/kg Q4W DB Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Evinacumab/SB Evinacumab IV 15mg/kg Q4W
    Number of subjects analysed
    16
    15
    35
    32
    Units: Subjects
    number (not applicable)
        ALT >2 ULN and <=3 ULN and <=2 ULN at baseline
    0
    0
    1
    0
        ALT >3 ULN and <=5 ULN and <=3 ULN at BL
    0
    0
    2
    0
        ALT >5 ULN and <=10 ULN and <=5 ULN at BL
    0
    1
    0
    1
        ALT 10 ULN and <=20 ULN and <=10 ULN at BL
    0
    0
    0
    0
        ALT >20 ULN and <=20 ULN at BL
    0
    0
    0
    0
        AST >2 ULN and <=3 ULN and <=2 ULN at BL
    0
    0
    2
    0
        AST >3 ULN and <=5 ULN and <=3 ULN at BL
    0
    1
    1
    0
        AST >5 ULN and <=10 ULN and <=5 ULN at BL
    0
    0
    0
    1
        AST >10 ULN and <=20 ULN and <=10 ULN at BL
    0
    0
    0
    0
        AST >20 ULN and <=20 ULN at BL
    0
    0
    0
    0
        Bilirubin >1.5 ULN - <=2 ULN and =<1.5 ULN at BL
    0
    0
    0
    0
        Bilirubin >2 ULN and =< 2.0 ULN at BL
    0
    0
    0
    0
        Alkaline Phosphatase >1.5 ULN and =< 1.5 ULN at BL
    0
    0
    0
    2
        (ALT>3ULN&TB>2ULN)&(ALT<=3ULN or TB<=2ULN) at BL
    0
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Day 1 up to Last Single-Blind (SB) dose + 168 days (24 weeks)
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    DBTP Placebo IV Q4W/SB Evinacumab
    Reporting group description
    Subjects received placebo matching evinacumab intravenously (IV) every 4 weeks (Q4W) on days 1, 29, and 57 during the 12-week double-blind treatment period (DBTP). During the 12-week single-blind treatment period (SBTP), subjects received evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Reporting group title
    DBTP Evinacumab IV 15mg/kg Q4W/SB Evinacumab
    Reporting group description
    Subjects received evinacumab 15 mg/kg IV Q4W on days 1, 29, and 57 during the 12-week DBTP. During the 12-week SBTP, subjects continued to receive evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Reporting group title
    DB Placebo/SBTP Evinacumab IV 15mg/kg Q4W
    Reporting group description
    Subjects received placebo matching evinacumab intravenously (IV) every 4 weeks (Q4W) on days 1, 29, and 57 during the 12-week double-blind treatment period (DBTP). During the 12-week single-blind treatment period (SBTP), subjects received evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Reporting group title
    DB Evinacumab/SBTP Evinacumab IV 15mg/kg Q4W
    Reporting group description
    Subjects received evinacumab 15 mg/kg IV Q4W on days 1, 29, and 57 during the 12-week DBTP. During the 12-week SBTP, subjects continued to receive evinacumab 15 mg/kg IV Q4W on days 85, 113, and 141.

    Serious adverse events
    DBTP Placebo IV Q4W/SB Evinacumab DBTP Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Placebo/SBTP Evinacumab IV 15mg/kg Q4W DB Evinacumab/SBTP Evinacumab IV 15mg/kg Q4W
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 16 (18.75%)
    4 / 35 (11.43%)
    4 / 15 (26.67%)
    11 / 32 (34.38%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Prostate cancer
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Ischaemic stroke
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Pancreatitis acute
         subjects affected / exposed
    2 / 16 (12.50%)
    3 / 35 (8.57%)
    4 / 15 (26.67%)
    8 / 32 (25.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 3
    0 / 4
    0 / 14
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Abdominal pain
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Ileus
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Abdominal pain upper
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Liver injury
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypertriglyceridaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolic acidosis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Clostridium difficile infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Viral infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    DBTP Placebo IV Q4W/SB Evinacumab DBTP Evinacumab IV 15mg/kg Q4W/SB Evinacumab DB Placebo/SBTP Evinacumab IV 15mg/kg Q4W DB Evinacumab/SBTP Evinacumab IV 15mg/kg Q4W
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    11 / 16 (68.75%)
    20 / 35 (57.14%)
    13 / 15 (86.67%)
    22 / 32 (68.75%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    0
    0
    0
    2
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Melanocytic naevus
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    1 / 15 (6.67%)
    1 / 32 (3.13%)
         occurrences all number
    1
    1
    1
    1
    Fatigue
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    1 / 15 (6.67%)
    2 / 32 (6.25%)
         occurrences all number
    1
    1
    1
    2
    Vessel puncture site pain
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Psychiatric disorders
    Delirium
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Insomnia
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences all number
    1
    1
    0
    1
    Injury, poisoning and procedural complications
    Infusion related reaction
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    2
    0
    3
    0
    Contusion
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    2 / 15 (13.33%)
    0 / 32 (0.00%)
         occurrences all number
    0
    2
    3
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    2 / 15 (13.33%)
    0 / 32 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Blood magnesium decreased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    0
    0
    0
    2
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Atelectasis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hiccups
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nasal congestion
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    1 / 15 (6.67%)
    1 / 32 (3.13%)
         occurrences all number
    0
    1
    2
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 16 (12.50%)
    4 / 35 (11.43%)
    2 / 15 (13.33%)
    2 / 32 (6.25%)
         occurrences all number
    2
    6
    3
    2
    Migraine
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sciatica
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Dizziness
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Eye disorders
    Vision blurred
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 16 (6.25%)
    4 / 35 (11.43%)
    2 / 15 (13.33%)
    4 / 32 (12.50%)
         occurrences all number
    1
    4
    2
    6
    Diarrhoea
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    2 / 15 (13.33%)
    0 / 32 (0.00%)
         occurrences all number
    1
    1
    4
    0
    Nausea
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    2 / 15 (13.33%)
    1 / 32 (3.13%)
         occurrences all number
    1
    1
    2
    1
    Abdominal distension
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    1 / 15 (6.67%)
    3 / 32 (9.38%)
         occurrences all number
    0
    1
    1
    4
    Diverticulum
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Dyspepsia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Enteritis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Eructation
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Flatulence
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Food poisoning
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    1 / 32 (3.13%)
         occurrences all number
    0
    0
    1
    1
    Gastritis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences all number
    1
    1
    0
    1
    Oesophagitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pancreatic failure
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    2 / 32 (6.25%)
         occurrences all number
    0
    0
    2
    2
    Abdominal discomfort
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    0 / 15 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    0
    2
    0
    2
    Constipation
         subjects affected / exposed
    0 / 16 (0.00%)
    3 / 35 (8.57%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences all number
    0
    4
    0
    1
    Pancreatitis acute
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    0 / 15 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    0
    1
    0
    4
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    1 / 32 (3.13%)
         occurrences all number
    0
    0
    1
    1
    Azotaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Renal cyst
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Renal impairment
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences all number
    1
    0
    0
    1
    Pruritus
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Bursitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Musculoskeletal pain
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nodal osteoarthritis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Back pain
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    0 / 15 (0.00%)
    1 / 32 (3.13%)
         occurrences all number
    0
    4
    0
    1
    Muscle spasms
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    2 / 32 (6.25%)
         occurrences all number
    0
    0
    0
    2
    Metabolism and nutrition disorders
    Diabetes mellitus
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    2 / 15 (13.33%)
    1 / 32 (3.13%)
         occurrences all number
    1
    1
    2
    1
    Type 2 diabetes mellitus
         subjects affected / exposed
    1 / 16 (6.25%)
    2 / 35 (5.71%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    1
    2
    1
    0
    Hypocalcaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Metabolic acidosis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 16 (6.25%)
    2 / 35 (5.71%)
    1 / 15 (6.67%)
    3 / 32 (9.38%)
         occurrences all number
    1
    2
    1
    3
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    1 / 32 (3.13%)
         occurrences all number
    1
    0
    1
    1
    Urinary tract infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    4 / 32 (12.50%)
         occurrences all number
    1
    0
    1
    5
    Bronchitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    2 / 32 (6.25%)
         occurrences all number
    0
    0
    1
    2
    Clostridium difficile infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Gastroenteritis
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Gastroenteritis viral
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 15 (0.00%)
    0 / 32 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Herpes zoster
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Influenza
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    1 / 15 (6.67%)
    1 / 32 (3.13%)
         occurrences all number
    0
    1
    1
    1
    Laryngitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oral candidiasis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sinusitis
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    1 / 15 (6.67%)
    0 / 32 (0.00%)
         occurrences all number
    0
    2
    1
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Jun 2017
    Increased sample size and number of sites; Changed classification of study from phase 1b to phase 2; Added to rationale; Modified inclusion/exclusion criteria; Clarified description of cohorts; Updated enrollment procedure; Updated language regarding study drug concentration and anti-drug antibody concentration; Modified Schedule of Events, Prohibited Medications, Permitted Medications and Procedures; Clarified primary efficacy analysis; Updated contraception language; Added study drug discontinuation and early termination details; Added sites will provide urine pregnancy tests to female patients of childbearing potential to be used at home; Added pregnancy reporting for female partners of male patients; Added an Independent Data Monitoring Committee
    31 Jul 2017
    Clarified the age range of the patient population; Modified Schedule of Events; Clarified blinding of lipid measurements and analysis sets
    20 Sep 2017
    Updated ADA variables; Modified a secondary objective/endpoint; Allowed more flexibility for a planned interim analysis; Allowed other select individuals at the sponsor to have access to unblinded data for safety or other data review; Modified Schedule of Events; Expanded list of Adverse Events of Special Interest (AESIs) for evinacumab
    18 Jan 2018
    Updated descriptions of the cohorts; Updated text regarding blinding for drug product and placebo
    15 Oct 2019
    Amended to require consistent use of a condom for all sexually active males in response to recent nonclinical findings in the rabbit.

    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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