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    Clinical Trial Results:
    An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

    Summary
    EudraCT number
    		 2017-003170-13
    Trial protocol
    		 NL   NO   CZ   FR   GR   GB   AT   IT  
    Global end of trial date
    13 Apr 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Oct 2023
    First version publication date
    27 Oct 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    R1500-CL-1719
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03409744
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Regeneron Pharmaceuticals, Inc.
    Sponsor organisation address
    777 Old Saw Mill River Road, Tarrytown, NY, United States, 10591
    Public contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.com
    Scientific contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002298-PIP01-17
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Apr 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Apr 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    An open-label study of long-term treatment with evinacumab (15 mg/kg IV every 4 weeks), in participants with homozygous familial hypercholesterolemia.
    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 ICH guidelines for GCP and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    13 Mar 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
    Austria: 5
    Country: Number of subjects enrolled
    Canada: 11
    Country: Number of subjects enrolled
    Czechia: 3
    Country: Number of subjects enrolled
    Australia: 4
    Country: Number of subjects enrolled
    France: 13
    Country: Number of subjects enrolled
    Greece: 6
    Country: Number of subjects enrolled
    Italy: 14
    Country: Number of subjects enrolled
    Japan: 11
    Country: Number of subjects enrolled
    Netherlands: 9
    Country: Number of subjects enrolled
    South Africa: 14
    Country: Number of subjects enrolled
    Ukraine: 7
    Country: Number of subjects enrolled
    United States: 19
    Worldwide total number of subjects
    116
    EEA total number of subjects
    50
    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)
    14
    Adults (18-64 years)
    93
    From 65 to 84 years
    9
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 118 participants were planned to be enrolled. 116 participants were enrolled & treated. Reasons for screen fail were: 1 participant was unwilling to use protocol defined contraception, and 1 participant had a Low-density lipoprotein cholesterol (LDL-C) level less than the lower limit required for inclusion.

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

    Arms
    Arm title
    		 Total evinacumab
    Arm description
    		 Includes all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    Arm type
    		 Experimental

    Investigational medicinal product name
    evinacumab
    Investigational medicinal product code
    REGN1500
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    15 milligrams per kilogram (mg/kg) administered intravenously (IV) every 4 weeks (Q4W)

    Number of subjects in period 1
    		Total evinacumab
    Started
    116
    Completed
    106
    Not completed
    10
         Adverse event, serious fatal
    2
         Physician decision
    5
         Pregnancy
    1
         Lost to follow-up
    1
         Protocol deviation
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 -

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 116 116
    Age Categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 38.8 ( 15.92 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 57 57
        Male
    		 59 59
    Race (NIH/OMB)
    Units: Subjects
        White
    		 80 80
        Black or African American
    		 4 4
        Asian
    		 12 12
        American Indian or Alaska Native
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Not Reported
    		 11 11
        Other
    		 9 9
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 6 6
        Not Hispanic or Latino
    		 100 100
        Not Reported
    		 10 10

    End points

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    End points reporting groups
    Reporting group title
    Total evinacumab
    Reporting group description
    		 Includes all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.

    Subject analysis set title
    Total evinacumab
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Includes all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.

    Primary: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Up to Week 216

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    End point title
    		 Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Up to Week 216 [1]
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Primary
    End point timeframe
    Up to 216 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: Descriptive statistics were used for this endpoint.
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: Number of participants
        Participants with any TEAE
    93
        Participants with at least one serious TEAE
    27
    No statistical analyses for this end point

    Secondary: Percent change in Low-Density Lipoprotein Cholesterol (LDL-C) over time

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    End point title
    		 Percent change in Low-Density Lipoprotein Cholesterol (LDL-C) over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: milligrams per deciliter (mg/dL)
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -46.45 ( 35.504 )
        Week 24 (n=86)
    -43.64 ( 37.606 )
        Week 48 (n=95)
    -43.88 ( 36.037 )
        Week 72 (n=92)
    -45.17 ( 31.571 )
        Week 96 (n=66)
    -38.00 ( 52.859 )
        Week 120 (n=39)
    -33.10 ( 65.888 )
    No statistical analyses for this end point

    Secondary: Absolute change in LDL-C over time

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    End point title
    		 Absolute change in LDL-C over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -142.1 ( 114.61 )
        Week 24 (n=86)
    -132.0 ( 124.37 )
        Week 48 (n=95)
    -132.8 ( 133.29 )
        Week 72 (n=92)
    -135.1 ( 121.91 )
        Week 96 (n=66)
    -131.4 ( 129.29 )
        Week 120 (n=39)
    -132.5 ( 132.03 )
    No statistical analyses for this end point

    Secondary: Percent change in Apolipoprotein B (Apo B) over time

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    End point title
    		 Percent change in Apolipoprotein B (Apo B) over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -39.74 ( 25.338 )
        Week 24 (n=86)
    -36.98 ( 27.574 )
        Week 48 (n=96)
    -35.85 ( 29.998 )
        Week 72 (n=93)
    -37.60 ( 27.292 )
        Week 96 (n=66)
    -32.54 ( 39.969 )
        Week 120 (n=39)
    -27.73 ( 51.722 )
    No statistical analyses for this end point

    Secondary: Absolute change in Apo B over time

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    End point title
    		 Absolute change in Apo B over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -78.0 ( 59.78 )
        Week 24 (n=86)
    -70.5 ( 61.96 )
        Week 48 (n=96)
    -69.4 ( 69.95 )
        Week 72 (n=93)
    -73.9 ( 61.51 )
        Week 96 (n=66)
    -71.5 ( 66.66 )
        Week 120 (n=39)
    -70.7 ( 76.05 )
    No statistical analyses for this end point

    Secondary: Percent change in non-High-Density Lipoprotein Cholesterol (HDL-C) over time

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    End point title
    		 Percent change in non-High-Density Lipoprotein Cholesterol (HDL-C) over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -48.47 ( 28.369 )
        Week 24 (n=86)
    -46.14 ( 29.117 )
        Week 48 (n=95)
    -45.15 ( 33.566 )
        Week 72 (n=92)
    -46.69 ( 28.534 )
        Week 96 (n=66)
    -40.88 ( 46.588 )
        Week 120 (n=39)
    -35.85 ( 58.374 )
    No statistical analyses for this end point

    Secondary: Absolute change in non-HDL-C over time

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    End point title
    		 Absolute change in non-HDL-C over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -153.7 ( 115.66 )
        Week 24 (n=86)
    -143.9 ( 125.03 )
        Week 48 (n=95)
    -144.1 ( 136.95 )
        Week 72 (n=92)
    -147.3 ( 122.76 )
        Week 96 (n=66)
    -144.5 ( 131.15 )
        Week 120 (n=39)
    -142.9 ( 136.65 )
    No statistical analyses for this end point

    Secondary: Percent change in Total Cholesterol (TC) over time

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    End point title
    		 Percent change in Total Cholesterol (TC) over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -47.04 ( 20.722 )
        Week 24 (n=86)
    -44.17 ( 24.224 )
        Week 48 (n=95)
    -43.78 ( 27.838 )
        Week 72 (n=93)
    -44.58 ( 25.700 )
        Week 96 (n=66)
    -40.33 ( 38.163 )
        Week 120 (n=39)
    -36.92 ( 47.469 )
    No statistical analyses for this end point

    Secondary: Absolute change in TC over time

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    End point title
    		 Absolute change in TC over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=109)
    -167.3 ( 115.76 )
        Week 24 (n=86)
    -157.4 ( 125.93 )
        Week 48 (n=95)
    -158.2 ( 136.51 )
        Week 72 (n=93)
    -160.7 ( 123.09 )
        Week 96 (n=66)
    -157.2 ( 132.40 )
        Week 120 (n=39)
    -153.8 ( 138.26 )
    No statistical analyses for this end point

    Secondary: Percent change in Triglycerides (TGs) over time

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    End point title
    		 Percent change in Triglycerides (TGs) over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=107)
    -45.97 ( 26.024 )
        Week 24 (n=84)
    -46.93 ( 24.582 )
        Week 48 (n=94)
    -43.25 ( 37.443 )
        Week 72 (n=92)
    -46.50 ( 25.826 )
        Week 96 (n=66)
    -48.28 ( 24.136 )
        Week 120 (n=36)
    -42.06 ( 36.945 )
    No statistical analyses for this end point

    Secondary: Absolute change in TGs over time

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    End point title
    		 Absolute change in TGs over time
    End point description
    The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
    End point type
    Secondary
    End point timeframe
    Up to 120 weeks
    End point values
    		 Total evinacumab
    Number of subjects analysed
    116
    Units: mg/dL
    arithmetic mean (standard deviation)
        Week 8 (n=107)
    -64.4 ( 89.77 )
        Week 24 (n=84)
    -68.1 ( 97.29 )
        Week 48 (n=94)
    -66.7 ( 97.02 )
        Week 72 (n=92)
    -68.8 ( 91.52 )
        Week 96 (n=66)
    -75.2 ( 107.49 )
        Week 120 (n=36)
    -66.7 ( 110.95 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From signing of Informed Consent to week 216
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Overall
    Reporting group description
    		 Includes all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.

    Serious adverse events
    		 Overall
    Total subjects affected by serious adverse events
         subjects affected / exposed
    27 / 116 (23.28%)
         number of deaths (all causes)
    2
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Prostate cancer
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Peripheral artery stenosis
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Arteriosclerosis
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Aortic stenosis
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    2 / 116 (1.72%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Food allergy
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Ovarian cyst ruptured
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Mental status changes
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Rib fracture
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Scapula fracture
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular pseudoaneurysm
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cervical vertebral fracture
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Arteriovenous fistula site complication
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Coronary artery occlusion
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Cardiac valve disease
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac failure congestive
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac failure chronic
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac failure acute
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Cardiac arrest
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Atrial fibrillation
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Acute myocardial infarction
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Coronary artery disease
         subjects affected / exposed
    2 / 116 (1.72%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Aortic valve disease
         subjects affected / exposed
    2 / 116 (1.72%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Angina unstable
         subjects affected / exposed
    2 / 116 (1.72%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Angina pectoris
         subjects affected / exposed
    2 / 116 (1.72%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Supravalvular aortic stenosis
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Coronary artery stenosis
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Ischaemic stroke
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Spinal epidural haematoma
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Glaucoma
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cataract
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Intestinal ischaemia
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Hepatitis acute
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Renal infarct
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Oesophageal candidiasis
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 116 (0.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Overall
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    79 / 116 (68.10%)
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    6 / 116 (5.17%)
         occurrences all number
    6
    Nervous system disorders
    Headache
         subjects affected / exposed
    19 / 116 (16.38%)
         occurrences all number
    29
    Dizziness
         subjects affected / exposed
    7 / 116 (6.03%)
         occurrences all number
    9
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    10 / 116 (8.62%)
         occurrences all number
    16
    Influenza like illness
         subjects affected / exposed
    17 / 116 (14.66%)
         occurrences all number
    23
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    10 / 116 (8.62%)
         occurrences all number
    21
    Abdominal pain
         subjects affected / exposed
    7 / 116 (6.03%)
         occurrences all number
    10
    Toothache
         subjects affected / exposed
    7 / 116 (6.03%)
         occurrences all number
    10
    Vomiting
         subjects affected / exposed
    7 / 116 (6.03%)
         occurrences all number
    11
    Nausea
         subjects affected / exposed
    14 / 116 (12.07%)
         occurrences all number
    17
    Gastrooesophageal reflux disease
         subjects affected / exposed
    6 / 116 (5.17%)
         occurrences all number
    6
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    6 / 116 (5.17%)
         occurrences all number
    7
    Cough
         subjects affected / exposed
    12 / 116 (10.34%)
         occurrences all number
    16
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    15 / 116 (12.93%)
         occurrences all number
    22
    Pain in extremity
         subjects affected / exposed
    8 / 116 (6.90%)
         occurrences all number
    10
    Back pain
         subjects affected / exposed
    14 / 116 (12.07%)
         occurrences all number
    20
    Myalgia
         subjects affected / exposed
    7 / 116 (6.03%)
         occurrences all number
    10
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    10 / 116 (8.62%)
         occurrences all number
    16
    Upper respiratory tract infection
         subjects affected / exposed
    9 / 116 (7.76%)
         occurrences all number
    14
    Nasopharyngitis
         subjects affected / exposed
    23 / 116 (19.83%)
         occurrences all number
    42
    COVID-19
         subjects affected / exposed
    19 / 116 (16.38%)
         occurrences all number
    20
    Gastroenteritis
         subjects affected / exposed
    10 / 116 (8.62%)
         occurrences all number
    11

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    20 Jul 2018
    The primary purpose of this amendment was to revise the participant population to allow participants with HoFH who had not participated in a previous evinacumab study to enter this study; minor editorial revisions; clarification of wording; added assessments to schedule of events; clarification of inclusion/exclusion criteria and study population
    11 Oct 2019
    After completion of the 2A and 2B versions of the protocol, a country-specific version was required, which became R1500-CL-1719 2B FR. Subsequently, the R1500-CL-1719 2B FR was amended to become R1500-CL-1719 3B FR. To avoid confusion and maintain the sequence, the numbering of present amendment became R1500-CL-1719 4B.
    01 Nov 2019
    The primary purpose for this amendment was to increase the number of participants from 100 to 120 and to add objectives to assess efficacy and safety in adolescent participants with HoFH. This also resulted in changes to the statistical analyses.
    29 May 2020
    Editorial revisions; clarified exclusion criteria; updated schedule of events and study design

    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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    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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