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    Clinical Trial Results:
    Efficacy and Safety of Evinacumab in Adult Patients with Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

    Summary
    EudraCT number
    		 2021-000437-13
    Trial protocol
    		 AT   NL   DE  
    Global end of trial date
    15 Feb 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Mar 2024
    First version publication date
    06 Mar 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    R1500-HTG-20118
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Regeneron
    Sponsor organisation address
    777 Old Saw Mill River Road, Tarrytown, United States, 10591
    Public contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 8447346643, clinicaltrials@regeneron.com
    Scientific contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc, 9144093597 8447346643, donell.carey@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
    15 Feb 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Feb 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to determine the proportion of patients with elevated TGs, without FCS due to LoF mutations in LPL, and a history of HTG-associated AP* who experience a recurrent episode of AP after treatment with evinacumab versus placebo.
    Protection of trial subjects
    This study was 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 Good Clinical Practice (GCP) and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 Jul 2021
    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: 2
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    United States: 18
    Worldwide total number of subjects
    21
    EEA total number of subjects
    1
    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)
    21
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 40 participants were screened, of which 21 participants met the eligibility criteria and were randomized in 1:1 to receive either evinacumab or matched placebo. The sponsor terminated the study early due to enrollment issues.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Data analyst, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Randomized 1:1
    Arm type
    		 Placebo

    Investigational medicinal product name
    Matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    First dose on day 1, with subsequent doses administered approximately every 4 weeks (Q4W)

    Arm title
    		 Evinacumab
    Arm description
    		 Participants received evinacumab 20 milligrams per kilogram (mg/kg) IV infusion Q4W starting from Day 1 up to 52 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Evinacumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    First dose on day 1, with subsequent doses administered approximately every 4 weeks (Q4W)

    Number of subjects in period 1
    		Placebo Evinacumab
    Started
    10
    11
    Completed
    7
    5
    Not completed
    3
    6
         Subject Decision
    -
    4
         Lost to follow-up
    3
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Randomized 1:1

    Reporting group title
    Evinacumab
    Reporting group description
    		 Participants received evinacumab 20 milligrams per kilogram (mg/kg) IV infusion Q4W starting from Day 1 up to 52 weeks.

    Reporting group values
    		 Placebo Evinacumab Total
    Number of subjects
    		 10 11 21
    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)
    		 10 11 21
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 46.0 ± 15.22 45.3 ± 9.46 -
    Sex: Female, Male
    Units: participants
        Female
    		 3 2 5
        Male
    		 7 9 16
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 2 3 5
        Not Hispanic or Latino
    		 8 8 16
        Unknown or Not Reported
    		 0 0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 1 1 2
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 1 1
        White
    		 9 9 18
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Randomized 1:1

    Reporting group title
    Evinacumab
    Reporting group description
    		 Participants received evinacumab 20 milligrams per kilogram (mg/kg) IV infusion Q4W starting from Day 1 up to 52 weeks.

    Primary: Percentage of Participants with at least One Positively Adjudicated Acute Pancreatitis (AP) Episode

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    End point title
    		 Percentage of Participants with at least One Positively Adjudicated Acute Pancreatitis (AP) Episode [1]
    End point description
    Adjudicated AP episode was determined by an independent acute pancreatitis adjudication committee (APAC). Suspected AP episodes was reviewed by 2 independent physicians. Percentage of participants with at least 1 positively adjudicated AP episode during the 52 weeks was reported.
    End point type
    Primary
    End point timeframe
    Baseline to 52 weeks
    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: No statistical analyses for this end point
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    10
    11
    Units: Percentage
        number (not applicable)
    10.0
    27.3
    No statistical analyses for this end point

    Secondary: Percent change in fasting triglycerides (TGs) - (from baseline to week 52)

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    End point title
    		 Percent change in fasting triglycerides (TGs) - (from baseline to week 52)
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [2]
    1
    Units: Percent
        number (not applicable)
    -92.88
    Notes
    [2] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein C3 (ApoC3) from Baseline to Week 52

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    End point title
    		 Percent Change in Apolipoprotein C3 (ApoC3) from Baseline to Week 52
    End point description
    Apolipoproteins transport lipids through the body by binding with fat and cholesterol to form lipoproteins. ApoC3 was a component of very-low-density lipoproteins (VLDL), high-density lipoprotein (HDL), and triglyceride-rich chylomicrons and regulates lipid metabolism. Percent change in ApoC3 from Baseline to Week 52 was reported.
    End point type
    Secondary
    End point timeframe
    Baseline to week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [3]
    1
    Units: Percent
        number (not applicable)
    -73.78
    Notes
    [3] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Percent change in non-high-density lipoprotein cholesterol (non-HDL-C) - (from baseline to week 52)

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    End point title
    		 Percent change in non-high-density lipoprotein cholesterol (non-HDL-C) - (from baseline to week 52)
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [4]
    1
    Units: Percent
        number (not applicable)
    -60.12
    Notes
    [4] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Percent change in Apolipoprotein B48 (ApoB48) from baseline to week 52

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    End point title
    		 Percent change in Apolipoprotein B48 (ApoB48) from baseline to week 52
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [5]
    1
    Units: Percent
        number (not applicable)
    -92.09
    Notes
    [5] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Percent change in total cholesterol (TC) - (baseline to week 52)

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    End point title
    		 Percent change in total cholesterol (TC) - (baseline to week 52)
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [6]
    1
    Units: Percent
        number (not applicable)
    -57.62
    Notes
    [6] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Percent change in Apolipoprotein B100 (ApoB100) levels from baseline to week 52

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    End point title
    		 Percent change in Apolipoprotein B100 (ApoB100) levels from baseline to week 52
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [7]
    1
    Units: Percent
        number (not applicable)
    225.14
    Notes
    [7] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Percent Change in Nuclear Magnetic Resonance (NMR)-Determined Particle Size and Number From Baseline to Week 52

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    End point title
    		 Percent Change in Nuclear Magnetic Resonance (NMR)-Determined Particle Size and Number From Baseline to Week 52
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [8]
    0 [9]
    Units: Percent
        arithmetic mean (standard deviation)
    ±
    ±
    Notes
    [8] - Early termination of study due to low feasibility
    [9] - Early termination of study due to low feasibility
    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
    End point type
    Secondary
    End point timeframe
    From start of study drug administration up to off drug follow-up (up to Week 72)
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    10
    11
    Units: Participants
        Participants with TEAEs
    10
    7
        Participants with Serious TEAEs
    4
    5
    No statistical analyses for this end point

    Secondary: Number of Independently Adjudicated Positive Episodes of Acute Pancreatitis (AP) Per Participant

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    End point title
    		 Number of Independently Adjudicated Positive Episodes of Acute Pancreatitis (AP) Per Participant
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 52 weeks
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    10
    11
    Units: Number of Episodes
        arithmetic mean (standard deviation)
    0.1 ± 0.32
    0.4 ± 0.67
    No statistical analyses for this end point

    Secondary: Number of Participants With TEAEs Based on Severity

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    End point title
    		 Number of Participants With TEAEs Based on Severity
    End point description
    AE was defined any untoward medical occurrence in a participant administered with a study drug, which does not necessarily had a causal relationship with this treatment. TEAEs are defined as AEs that developed or worsened during the treatment period. Severity of TEAEs was graded according to the following scale: Mild: Does not interfere in a significant manner with the participants normal functioning level, Moderate: Produces some impairment of functioning but is not hazardous to health and Severe: Produces significant impairment of functioning or incapacitation and is a definite hazard to the participants health.
    End point type
    Secondary
    End point timeframe
    From start of study drug administration up to off drug follow-up (up to Week 72)
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    10
    7
    Units: Participants
    number (not applicable)
        Participants with Mild TEAEs
    3
    3
        Participants with Moderate TEAEs
    3
    1
        Participants with Severe TEAEs
    4
    3
    No statistical analyses for this end point

    Secondary: Number of Participants With Clinically Significant Changes from Baseline in Laboratory Parameters

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    End point title
    		 Number of Participants With Clinically Significant Changes from Baseline in Laboratory Parameters
    End point description
    Clinical laboratory parameters included biochemistry, hematology and urinalysis. The number of participants with clinically significant changes from baseline in laboratory parameters were reported. Clinical significance was determined by the investigator.
    End point type
    Secondary
    End point timeframe
    From start of study drug administration up to off drug follow-up (up to Week 72)
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    10
    11
    Units: Participants
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA)

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    End point title
    		 Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA)
    End point description
    Treatment-Emergent ADA was defined as any positive post baseline assay response when baseline results were negative or missing. Treatment-Emergent ADA responses were further classified as: Persistent (a positive result in the ADA assay detected in at least 2 consecutive post baseline samples separated by at least a 16-week post baseline period [based on] nominal sampling time], with no ADA-negative results in-between, regardless of any missing samples); Indeterminate (a positive result in the ADA assay at the last collection time point only, regardless of any missing samples); Transient (not persistent/indeterminate, regardless of any missing samples). Number of participants with positive treatment-emergent ADA response during Week 52 were reported.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    8
    7
    Units: Participants
    number (not applicable)
        Persistent: Treatment-Emergent Response
    0
    0
        Transient: Treatment-Emergent Response
    0
    0
        Indeterminate: Treatment-Emergent Response
    0
    1
    No statistical analyses for this end point

    Secondary: Number of Participants With Positive Neutralizing Antibodies (NAb)

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    End point title
    		 Number of Participants With Positive Neutralizing Antibodies (NAb)
    End point description
    NAb positive was defined as presence of at least one positive nAb sample.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [10]
    0 [11]
    Units: Participants
    Notes
    [10] - Early termination of study due to low feasibility
    [11] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Percent Change in Fasting High-density Lipoprotein Cholesterol (HDL-C) From Baseline to Week 52

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    End point title
    		 Percent Change in Fasting High-density Lipoprotein Cholesterol (HDL-C) From Baseline to Week 52
    End point description
    Percent Change in fasting HDL-C from Baseline to Week 52 was reported.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [12]
    1
    Units: Percent change
        number (not applicable)
    52.94
    Notes
    [12] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Concentration of Total Evinacumab in Serum

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    End point title
    		 Concentration of Total Evinacumab in Serum [13]
    End point description
    Concentration of total evinacumab in serum by time at Pre-dose and End of Infusion were analyzed and reported.
    End point type
    Secondary
    End point timeframe
    Pre-dose and End of Infusion (EOI) at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 36, 40, 44, and 48; Pre-dose at Weeks 28 and 52
    Notes
    [13] - 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: No statistical analyses for this end point
    End point values
    		 Evinacumab
    Number of subjects analysed
    10
    Units: milligrams per liter (mg/L)
    arithmetic mean (standard deviation)
        Week 0: Pre-dose
    1.39 ± 4.40
        Week 0: EOI (End of Infusion)
    573 ± 95.7
        Week 4: Pre-dose
    97.6 ± 37.4
        Week 4: EOI (End of Infusion)
    638 ± 286
        Week 8: Pre-dose
    133 ± 88.6
        Week 8: EOI (End of Infusion)
    724 ± 123
        Week 12: Pre-dose
    158 ± 81.1
        Week 12: EOI (End of Infusion)
    749 ± 215
        Week 16: Pre-dose
    201 ± 68.7
        Week 16: EOI (End of Infusion)
    999.99 ± 999.99
        Week 20: Pre-dose
    999.99 ± 999.99
        Week 20: EOI (End of Infusion)
    999.99 ± 999.99
        Week 24: Pre-dose
    999.99 ± 999.99
        Week 24: EOI (End of Infusion)
    999.99 ± 999.99
        Week 28: Pre-dose
    999.99 ± 999.99
        Week 32: Pre-dose
    999.99 ± 999.99
        Week 32: EOI (End of Infusion)
    999.99 ± 999.99
        Week 36: Pre-dose
    99.1 ± 171
        Week 36: EOI (End of Infusion)
    999.99 ± 999.99
        Week 40: Pre-dose
    999.99 ± 999.99
        Week 40: EOI (End of Infusion)
    999.99 ± 999.99
        Week 44: Pre-dose
    999.99 ± 999.99
        Week 44: EOI (End of Infusion)
    999.99 ± 999.99
        Week 48: Pre-dose
    999.99 ± 999.99
        Week 48: EOI (End of Infusion)
    999.99 ± 999.99
        Week 52: Pre-dose
    999.99 ± 999.99
    No statistical analyses for this end point

    Secondary: Percent Change in Fasting Low Density Lipoprotein (LDL-C) From Baseline to Week 52

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    End point title
    		 Percent Change in Fasting Low Density Lipoprotein (LDL-C) From Baseline to Week 52
    End point description
    LDL-C levels were determined in beta-quantification with ultracentrifugation method. Percent change in fasting LDL-C from Baseline to Week 52 was reported.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 52
    End point values
    		 Placebo Evinacumab
    Number of subjects analysed
    0 [14]
    1
    Units: Percent
        number (not applicable)
    695.00
    Notes
    [14] - Early termination of study due to low feasibility
    No statistical analyses for this end point

    Secondary: Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum

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    End point title
    		 Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum [15]
    End point description
    Concentration of total ANGPTL3 in serum by time were analyzed and reported.
    End point type
    Secondary
    End point timeframe
    Pre-dose and End of Infusion (EOI) at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 36, 40, 44, and 48; Pre-dose at Weeks 28 and 52
    Notes
    [15] - 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: No statistical analyses for this end point
    End point values
    		 Evinacumab
    Number of subjects analysed
    10
    Units: mg/L
    arithmetic mean (standard deviation)
        Week 0: Pre-Dose
    0.0907 ± 0.0272
        Week 0: EOI (End of Infusion)
    0.211 ± 0.0362
        Week 4: Pre-Dose
    0.248 ± 0.105
        Week 4: EOI (End of Infusion)
    0.306 ± 0.0992
        Week 8: Pre-Dose
    0.243 ± 0.0811
        Week 8: EOI (End of Infusion)
    0.298 ± 0.0831
        Week 12: Pre-Dose
    0.281 ± 0.0851
        Week 12: EOI (End of Infusion)
    0.322 ± 0.0672
        Week 16: Pre-Dose
    0.230 ± 0.0560
        Week 16: EOI (End of Infusion)
    999.99 ± 999.99
        Week 20: Pre-Dose
    999.99 ± 999.99
        Week 20: EOI (End of Infusion)
    999.99 ± 999.99
        Week 24: Pre-Dose
    999.99 ± 999.99
        Week 24: EOI (End of Infusion)
    999.99 ± 999.99
        Week 28: Pre-Dose
    999.99 ± 999.99
        Week 32: Pre-Dose
    999.99 ± 999.99
        Week 32: EOI (End of Infusion)
    999.99 ± 999.99
        Week 36: Pre-Dose
    0.162 ± 0.107
        Week 36: EOI (End of Infusion)
    999.99 ± 999.99
        Week 40: Pre-Dose
    999.99 ± 999.99
        Week 40: EOI (End of Infusion)
    999.99 ± 999.99
        Week 44: Pre-Dose
    999.99 ± 999.99
        Week 44: EOI (End of Infusion)
    999.99 ± 999.99
        Week 48: Pre-Dose
    999.99 ± 999.99
        Week 48: EOI (End of Infusion)
    999.99 ± 999.99
        Week 52: Pre-Dose
    999.99 ± 999.99
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From start of study drug administration up to off drug follow-up (72 weeks)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    Evinacumab 20 mg
    Reporting group description
    		 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Randomized 1:1

    Serious adverse events
    		 Evinacumab 20 mg Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 11 (45.45%)
    4 / 10 (40.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Acute myocardial infarction
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Anaphylactic reaction
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Pancreatitis acute
         subjects affected / exposed
    2 / 11 (18.18%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    3 / 3
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 10 (20.00%)
         occurrences causally related to treatment / all
    1 / 1
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Abdominal pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Respiratory syncytial virus infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Evinacumab 20 mg Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 11 (72.73%)
    8 / 10 (80.00%)
    Vascular disorders
    Hot flush
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Fatigue
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Pedal pulse decreased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Epicondylitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Limb injury
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Dizziness
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Somnolence
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Abdominal pain upper
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 10 (20.00%)
         occurrences all number
    0
    2
    Vomiting
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Pancreatic pseudocyst
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Nausea
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 10 (20.00%)
         occurrences all number
    1
    2
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Duodenitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Abdominal pain
         subjects affected / exposed
    4 / 11 (36.36%)
    3 / 10 (30.00%)
         occurrences all number
    5
    6
    Dyspepsia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    3
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Hand-foot-and-mouth disease
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Wound infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Cellulitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    Bronchitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences all number
    1
    0
    COVID-19
         subjects affected / exposed
    3 / 11 (27.27%)
    1 / 10 (10.00%)
         occurrences all number
    3
    1
    Metabolism and nutrition disorders
    Food intolerance
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1
    Hypoglycaemia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    16 Dec 2021
    Protocol Amendment 2

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    15 Feb 2023
    Study Termination
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    The sponsor terminated the study early due to enrollment issues.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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