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    Clinical Trial Results:
    An Open-Label, Single-Arm, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Single and Multiple Doses of REGN1500 in Patients with Homozygous Familial Hypercholesterolemia

    Summary
    EudraCT number
    2016-000411-32
    Trial protocol
    NL  
    Global end of trial date
    23 Jul 2018

    Results information
    Results version number
    v1(current)
    This version publication date
    07 Aug 2019
    First version publication date
    07 Aug 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    R1500-CL-1331
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02265952
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Regeneron Pharmaceuticals, Inc.
    Sponsor organisation address
    777 Old Saw Mill River Road, Tarrytown, United States, 10591
    Public contact
    Clinical Trial Information, Regeneron Pharmaceuticals, Inc., clinicaltrials@regeneron.com
    Scientific contact
    Clinical Trial Information, Regeneron Pharmaceuticals, Inc., 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
    09 Aug 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jul 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The purpose of this study was to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by REGN1500 in subjects with Homozygous Familial Hypercholesterolemia (HoFH).
    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 Conference on Harmonisation (ICH) guidelines for Good Clinical Practice (GCP) and applicable regulatory requirements.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    04 Feb 2015
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 4
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    Netherlands: 2
    Worldwide total number of subjects
    9
    EEA total number of subjects
    2
    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)
    9
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study was conducted at five sites in United States, Canada and the Netherlands between 04 Feb 2015 and 23 Jul 2018. A total of 12 subjects were screened in the study. Out of 12 subjects, 9 subjects received the study treatment.

    Pre-assignment
    Screening details
    This study consisted of 2 screening periods: 1st for main study and 2nd for open label extension (OLE) period. Subjects on stable background medical lipid-modifying therapy for at least 4 weeks prior to screening, or who didn't undergo lipid apheresis within 4 weeks prior to screening visit, were directly entered into a 2-week screening period.

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

    Arms
    Arm title
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Arm description
    Subjects received single dose of REGN1500 subcutaneous (SC) injection of 250 milligrams (mg) at Week 0 (Day 1), followed by single dose of REGN1500 intravenous (IV) injection of 15 milligrams per kilogram (mg/kg) at Week 2 (Day 15) and then followed by 4 doses of REGN1500 SC injection of 450 mg once weekly starting from Week 12 (Day 85). Only the first 2 enrolled subjects received 4 weekly REGN1500 450 mg SC doses at weeks 12, 13, 14, and 15 per the protocol. This dose regimen was removed under protocol amendment 4. Subjects were followed for a period of 24 weeks (through Week 26 [Day 183]) after the last dose of study drug in the main study period.
    Arm type
    Experimental

    Investigational medicinal product name
    REGN1500
    Investigational medicinal product code
    REGN1500
    Other name
    Evinacumab
    Pharmaceutical forms
    Concentrate for solution for injection/infusion
    Routes of administration
    Intravenous use, Subcutaneous use
    Dosage and administration details
    Subjects received REGN1500 SC in the abdomen/ IV infusion in the main study period.

    Number of subjects in period 1
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Started
    9
    Completed
    9
    Period 2
    Period 2 title
    Open Label Extension (OLE) Period
    Is this the baseline period?
    No
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Arm title
    REGN1500 300 mg SC/20 mg/kg IV
    Arm description
    Subjects received REGN1500 a SC injection of 300 mg at Week 26 (Day 183), 27(Day 190), 28 (Day 197) and 29 (Day 204) followed by IV injection of 20 mg/kg at Week 38 (Day 267) and every 12 weeks starting at Week 58 (Day 407) through Week 178 (Day 1247) in open-label extension period. Subjects were to be followed for a period of 24 weeks (through Week 214 [Day 1499]) after the last dose of study drug in the OLE treatment period.
    Arm type
    Experimental

    Investigational medicinal product name
    REGN1500
    Investigational medicinal product code
    REGN1500
    Other name
    Evinacumab
    Pharmaceutical forms
    Concentrate for solution for injection/infusion
    Routes of administration
    Intravenous use, Subcutaneous use
    Dosage and administration details
    Subjects received REGN1500 SC in the abdomen/ IV infusion in the open-label treatment period.

    Number of subjects in period 2 [1]
    REGN1500 300 mg SC/20 mg/kg IV
    Started
    8
    Completed
    0
    Not completed
    8
         Physician decision
    7
         Consent withdrawn by subject
    1
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: One subject chose not to enroll in OLE period.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Reporting group description
    Subjects received single dose of REGN1500 subcutaneous (SC) injection of 250 milligrams (mg) at Week 0 (Day 1), followed by single dose of REGN1500 intravenous (IV) injection of 15 milligrams per kilogram (mg/kg) at Week 2 (Day 15) and then followed by 4 doses of REGN1500 SC injection of 450 mg once weekly starting from Week 12 (Day 85). Only the first 2 enrolled subjects received 4 weekly REGN1500 450 mg SC doses at weeks 12, 13, 14, and 15 per the protocol. This dose regimen was removed under protocol amendment 4. Subjects were followed for a period of 24 weeks (through Week 26 [Day 183]) after the last dose of study drug in the main study period.

    Reporting group values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC Total
    Number of subjects
    9 9
    Age categorical
    Units: Subjects
        < 45
    7 7
        ≥ 45 to < 65
    2 2
        ≥ 65 to < 75
    0 0
        < 75
    0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    35.9 ± 9.06 -
    Gender categorical
    Units: Subjects
        Female
    4 4
        Male
    5 5
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    3 3
        Not Hispanic or Latino
    6 6
        Not Reported
    0 0
    Race
    Units: Subjects
        White
    7 7
        Black or African American
    0 0
        Asian
    0 0
        American Indian or Alaska Native
    0 0
        Native Hawaiian or Other Pacific Islander
    0 0
        Other
    0 0
        Not Reported
    2 2
    Baseline Low-Density Lipoprotein Cholesterol (LDL-C)
    Units: Milligram per Deciliter (mg/dL)
        arithmetic mean (standard deviation)
    376.0 ± 240.85 -
    Baseline High-Density Lipoprotein Cholesterol (HDL-C)
    Units: mg/dL
        arithmetic mean (standard deviation)
    38.8 ± 14.24 -
    Baseline Non-High-Density Lipoprotein Cholesterol (Non-HDL-C)
    Units: mg/dL
        arithmetic mean (standard deviation)
    392.1 ± 246.41 -
    Baseline Apolipoprotein B (Apo B)
    Units: mg/dL
        arithmetic mean (standard deviation)
    226.1 ± 131.70 -
    Baseline Apolipoprotein CIII (Apo CIII)
    Units: mg/dL
        arithmetic mean (standard deviation)
    7.976 ± 3.3747 -
    Baseline Triglyceride (TG)
    Units: mg/dL
        arithmetic mean (standard deviation)
    80.0 ± 41.20 -
    Baseline Lipoprotein(a) (Lp[a])
    Units: mg/dL
        arithmetic mean (standard deviation)
    154.9 ± 109.25 -
    Baseline Total Cholesterol (Total -C)
    Units: mg/dL
        arithmetic mean (standard deviation)
    430.9 ± 235.60 -
    Baseline Apolipoprotein A-1 (Apo A-1)
    Units: mg/dL
        arithmetic mean (standard deviation)
    110.4 ± 23.74 -

    End points

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    End points reporting groups
    Reporting group title
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Reporting group description
    Subjects received single dose of REGN1500 subcutaneous (SC) injection of 250 milligrams (mg) at Week 0 (Day 1), followed by single dose of REGN1500 intravenous (IV) injection of 15 milligrams per kilogram (mg/kg) at Week 2 (Day 15) and then followed by 4 doses of REGN1500 SC injection of 450 mg once weekly starting from Week 12 (Day 85). Only the first 2 enrolled subjects received 4 weekly REGN1500 450 mg SC doses at weeks 12, 13, 14, and 15 per the protocol. This dose regimen was removed under protocol amendment 4. Subjects were followed for a period of 24 weeks (through Week 26 [Day 183]) after the last dose of study drug in the main study period.
    Reporting group title
    REGN1500 300 mg SC/20 mg/kg IV
    Reporting group description
    Subjects received REGN1500 a SC injection of 300 mg at Week 26 (Day 183), 27(Day 190), 28 (Day 197) and 29 (Day 204) followed by IV injection of 20 mg/kg at Week 38 (Day 267) and every 12 weeks starting at Week 58 (Day 407) through Week 178 (Day 1247) in open-label extension period. Subjects were to be followed for a period of 24 weeks (through Week 214 [Day 1499]) after the last dose of study drug in the OLE treatment period.

    Primary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) to Week 4 in the Main Study Period

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    End point title
    Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) to Week 4 in the Main Study Period [1]
    End point description
    Percent change was reported for low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 4. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all subjects in the safety analysis set (SAF) who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Primary
    End point timeframe
    Baseline (Week 0) up to Week 4
    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 sensitivity analysis was completed for this study.
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percent Change
        arithmetic mean (standard deviation)
    -49.17 ± 23.136
    No statistical analyses for this end point

    Secondary: Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) to Week 4 in the Main Study Period

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    End point title
    Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) to Week 4 in the Main Study Period
    End point description
    Absolute change in low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 4 was reported. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 4
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: mg/dL
        arithmetic mean (standard deviation)
    -157.34 ± 89.590
    No statistical analyses for this end point

    Secondary: Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Week 2 to Week 4 in the Main Study Period

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    End point title
    Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Week 2 to Week 4 in the Main Study Period
    End point description
    Absolute change in low-density lipoprotein cholesterol (LDL-C) from week 2 to week 4 was reported. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Secondary
    End point timeframe
    Week 2 to Week 4
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Milligram per Deciliter (mg/dL)
        arithmetic mean (standard deviation)
    -63.63 ± 50.970
    No statistical analyses for this end point

    Secondary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Week 2 to Week 4 in the Main Study Period

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    End point title
    Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Week 2 to Week 4 in the Main Study Period
    End point description
    Percent change was reported in low-density lipoprotein cholesterol (LDL-C) from week 2 to week 4. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Secondary
    End point timeframe
    Week 2 to Week 4
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percent Change
        arithmetic mean (standard deviation)
    -30.05 ± 18.503
    No statistical analyses for this end point

    Secondary: Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) Over Time in the Main Study Period

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    End point title
    Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) Over Time in the Main Study Period
    End point description
    Absolute change was reported in low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 26. The efficacy analysis set included all subjects in the safety analysis set (SAF) who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: mg/dL
    arithmetic mean (standard deviation)
        Change at Day 4 (n= 9)
    -39.60 ± 41.677
        Change at Week 1 (n= 9)
    -67.59 ± 46.041
        Change at Week 2 (n= 9)
    -93.71 ± 86.504
        Change at Week 3 (n= 9)
    -129.97 ± 90.533
        Change at Week 4 (n= 9)
    -157.34 ± 89.590
        Change at Week 5 (n= 8)
    -179.18 ± 101.260
        Change at Week 6 (n= 9)
    -183.41 ± 111.313
        Change at Week 8 (n= 9)
    -176.72 ± 118.441
        Change at Week 10 (n= 9)
    -151.19 ± 78.164
        Change at Week 12 (n= 9)
    -109.40 ± 74.108
        Change at Week 13 (n= 2)
    -170.15 ± 105.288
        Change at Week 14 (n= 9)
    -104.16 ± 78.954
        Change at Week 15 (n= 2)
    -186.15 ± 119.147
        Change at Week 16 (n= 9)
    -97.29 ± 179.016
        Change at Week 17 (n= 2)
    -174.20 ± 103.803
        Change at Week 18 (n= 7)
    -62.11 ± 167.004
        Change at Week 20 (n= 2)
    -155.25 ± 79.832
        Change at Week 22 (n= 7)
    -41.37 ± 169.432
        Change at Week 23 (n= 2)
    -155.85 ± 78.984
        Change at Week 25 (n= 2)
    -87.90 ± 23.900
        Change at Week 26 (n= 6)
    -23.98 ± 152.794
    No statistical analyses for this end point

    Secondary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) Over Time in the Main Study Period

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    End point title
    Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) Over Time in the Main Study Period
    End point description
    Percent change was reported in low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 26. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percent Change
    arithmetic mean (standard deviation)
        Percent change at Day 4 (n= 9)
    -12.76 ± 11.086
        Percent change at Week 1 (n= 9)
    -24.00 ± 21.078
        Percent change at Week 2 (n= 9)
    -30.13 ± 24.402
        Percent change at Week 3 (n= 9)
    -41.38 ± 24.986
        Percent change at Week 4 (n= 9)
    -49.17 ± 23.136
        Percent change at Week 5 (n= 8)
    -46.88 ± 14.507
        Percent change at Week 6 (n= 9)
    -52.13 ± 14.768
        Percent change at Week 8 (n= 9)
    -51.59 ± 17.910
        Percent change at Week 10 (n= 9)
    -45.61 ± 13.839
        Percent change at Week 12 (n= 9)
    -36.54 ± 19.281
        Percent change at Week 13 (n= 2)
    -39.84 ± 10.753
        Percent change at Week 14 (n= 9)
    -33.02 ± 18.538
        Percent change at Week 15 (n= 2)
    -43.38 ± 12.814
        Percent change at Week 16 (n= 9)
    -24.98 ± 25.441
        Percent change at Week 17 (n= 2)
    -40.99 ± 9.950
        Percent change at Week 18 (n= 7)
    -13.72 ± 28.424
        Percent change at Week 20 (n= 2)
    -37.17 ± 5.503
        Percent change at Week 22 (n= 7)
    -9.50 ± 38.456
        Percent change at Week 23 (n= 2)
    -37.37 ± 5.217
        Percent change at Week 25 (n= 2)
    -22.12 ± 2.537
        Percent change at Week 26 (n= 6)
    -10.27 ± 25.806
    No statistical analyses for this end point

    Secondary: Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

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    End point title
    Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period
    End point description
    Absolute change in low-density lipoprotein cholesterol (LDL-C) from baseline (week 26) up to week 214 was reported. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for the endpoint at a given time point. (ET= Early Termination; EOS = End of Study)
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) up to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: mg/dL
    arithmetic mean (standard deviation)
        Change at Week 26 (n= 8)
    261.34 ± 178.792
        Change at Week 27 (n= 8)
    -58.73 ± 56.992
        Change at Week 28 (n= 8)
    -88.16 ± 83.196
        Change at Week 29 (n= 8)
    -116.20 ± 102.219
        Change at Week 30 (n= 7)
    -87.80 ± 108.905
        Change at Week 31 (n= 8)
    -144.99 ± 130.764
        Change at Week 32 (n= 8)
    -159.16 ± 128.072
        Change at Week 34 (n= 8)
    -128.38 ± 108.331
        Change at Week 36 (n= 8)
    -105.94 ± 89.990
        Change at Week 38 (n= 8)
    -79.98 ± 97.851
        Change at Week 39 (n= 6)
    -138.28 ± 123.133
        Change at Week 40 (n= 6)
    -127.13 ± 122.808
        Change at Week 41 (n= 6)
    -163.28 ± 137.365
        Change at Week 42 (n= 6)
    -142.65 ± 126.647
        Change at Week 44 (n= 8)
    -140.36 ± 109.275
        Change at Week 46 (n= 8)
    -123.25 ± 112.503
        Change at Week 50 (n= 8)
    -106.61 ± 99.458
        Change at Week 54 (n= 8)
    -13.38 ± 177.649
        Change at Week 58 (n= 6)
    -48.27 ± 93.443
        Change at Week 70 (n= 4)
    -117.93 ± 145.917
        Change at Week 82 (n= 4)
    -63.88 ± 64.738
        Change at Week 94 (n= 1)
    -369.90 ± 99999
        Change at Week 106 (n= 1)
    -290.00 ± 99999
        Change at ET (n= 8)
    -9.78 ± 64.930
        Change at EOS (n= 3)
    95.63 ± 147.100
    No statistical analyses for this end point

    Secondary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

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    End point title
    Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period
    End point description
    Percent change was reported in low-density lipoprotein cholesterol (LDL-C) from baseline (week 26) to week 214 in the OLE period. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had at least 1 post-baseline measure of the lipid panel in the main study period. Here "n" signifies those subjects who were evaluable for this endpoint at a given time point. (ET= Early Termination; EOS = End of Study)
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) up to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: Percent Change
    arithmetic mean (standard deviation)
        Baseline (n= 8)
    261.34 ± 178.792
        Percent change at Week 27 (n= 8)
    -22.49 ± 16.520
        Percent change at Week 28 (n= 8)
    -36.74 ± 20.931
        Percent change at Week 29 (n= 8)
    -44.35 ± 20.817
        Percent change at Week 30 (n= 7)
    -37.37 ± 31.297
        Percent change at Week 31 (n= 8)
    -50.74 ± 20.147
        Percent change at Week 32 (n= 8)
    -60.03 ± 18.502
        Percent change at Week 34 (n= 8)
    -53.03 ± 22.130
        Percent change at Week 36 (n= 8)
    -47.90 ± 21.623
        Percent change at Week 38 (n= 8)
    -37.05 ± 25.341
        Percent change at Week 39 (n= 6)
    -59.83 ± 22.220
        Percent change at Week 40 (n= 6)
    -53.90 ± 20.970
        Percent change at Week 41 (n= 6)
    -66.28 ± 18.555
        Percent change at Week 42 (n= 6)
    -59.59 ± 18.950
        Percent change at Week 44 (n= 8)
    -57.64 ± 18.599
        Percent change at Week 46 (n= 8)
    -48.74 ± 19.717
        Percent change at Week 50 (n= 8)
    -42.31 ± 23.194
        Percent change at Week 54 (n= 8)
    -13.60 ± 44.339
        Percent change at Week 58 (n= 6)
    -12.86 ± 27.645
        Percent change at Week 70 (n= 4)
    -38.61 ± 36.151
        Percent change at Week 82 (n= 4)
    -24.68 ± 30.789
        Percent change at Week 94 (n= 1)
    -81.67 ± 99999
        Percent change at Week 106 (n= 1)
    -64.03 ± 99999
        Percent change at ET (n= 8)
    -17.65 ± 28.813
        Percent change at EOS (n= 3)
    19.59 ± 40.976
    No statistical analyses for this end point

    Secondary: Absolute Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 0) up to Week 26 in the Main Study Period

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    End point title
    Absolute Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 0) up to Week 26 in the Main Study Period
    End point description
    Absolute change was reported for Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline (week 0) up to Week 26. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: mg/dL
    arithmetic mean (standard deviation)
        Apo B: Change at Week 2 (n = 9)
    -53.0 ± 56.29
        Apo B: Change at Week 3 (n = 9)
    -81.0 ± 54.97
        Apo B: Change at Week 4 (n = 9)
    -96.3 ± 56.01
        Apo B: Change at Week 5 (n = 8)
    -104.6 ± 61.43
        Apo B: Change at Week 6 (n = 9)
    -100.7 ± 71.12
        Apo B: Change at Week 8 (n = 9)
    -96.8 ± 67.36
        Apo B: Change at Week 12 (n = 9)
    -60.0 ± 63.61
        Apo B: Change at Week 14 (n = 9)
    -61.7 ± 64.18
        Apo B: Change at Week 16 (n = 9)
    -62.6 ± 98.77
        Apo B: Change at Week 18 (n = 7)
    -28.6 ± 80.58
        Apo B: Change at Week 20 (n = 2)
    -114.0 ± 67.88
        Apo B: Change at Week 22 (n = 7)
    -22.7 ± 77.82
        Apo B: Change at Week 23 (n = 2)
    -119.0 ± 79.20
        Apo B: Change at Week 26 (n = 6)
    -4.7 ± 68.52
        Non-HDL-C: Change at Day 4 (n = 9)
    -44.61 ± 42.322
        Non-HDL-C: Change at Week 1 (n = 9)
    -72.16 ± 46.480
        Non-HDL-C: Change at Week 2 (n = 9)
    -97.12 ± 88.026
        Non-HDL-C: Change at Week 3 (n = 9)
    -138.09 ± 91.574
        Non-HDL-C: Change at Week 4 (n = 9)
    -165.64 ± 93.130
        Non-HDL-C: Change at Week 5 (n = 8)
    -187.46 ± 104.826
        Non-HDL-C: Change at Week 6 (n = 9)
    -191.20 ± 116.337
        Non-HDL-C: Change at Week 8 (n = 9)
    -182.98 ± 121.701
        Non-HDL-C: Change at Week 10 (n = 9)
    -156.98 ± 81.636
        Non-HDL-C: Change at Week 12 (n = 9)
    -116.20 ± 77.582
        Non-HDL-C: Change at Week 13 (n = 2)
    -180.25 ± 116.178
        Non-HDL-C: Change at Week 14 (n = 9)
    -108.58 ± 83.773
        Non-HDL-C: Change at Week 15 (n = 2)
    -196.85 ± 135.270
        Non-HDL-C: Change at Week 16 (n = 9)
    -99.58 ± 186.796
        Non-HDL-C: Change at Week 17 (n = 2)
    -185.40 ± 114.834
        Non-HDL-C: Change at Week 18 (n = 7)
    -60.41 ± 169.805
        Non-HDL-C: Change at Week 20 (n = 2)
    -161.90 ± 85.843
        Non-HDL-C: Change at Week 22 (n = 7)
    -41.16 ± 175.121
        Non-HDL-C: Change at Week 23 (n = 2)
    -163.25 ± 85.065
        Non-HDL-C: Change at Week 25 (n = 2)
    -92.20 ± 29.981
        Non-HDL-C: Change at Week 26 (n = 6)
    -25.47 ± 156.948
        Total-C: Change at Day 4 (n = 9)
    -50.36 ± 41.860
        Total-C: Change at Week 1 (n = 9)
    -81.99 ± 46.513
        Total-C: Change at Week 2 (n = 9)
    -109.03 ± 86.365
        Total-C: Change at Week 3 (n = 9)
    -154.10 ± 86.927
        Total-C: Change at Week 4 (n = 9)
    -180.79 ± 87.304
        Total-C: Change at Week 5 (n = 8)
    -199.11 ± 98.202
        Total-C: Change at Week 6 (n = 9)
    -207.63 ± 107.187
        Total-C: Change at Week 8 (n = 9)
    -195.18 ± 113.993
        Total-C: Change at Week 10 (n = 9)
    -167.87 ± 76.914
        Total-C: Change at Week 12 (n = 9)
    -122.94 ± 76.104
        Total-C: Change at Week 13 (n = 2)
    -188.45 ± 113.349
        Total-C: Change at Week 14 (n = 9)
    -114.68 ± 84.082
        Total-C: Change at Week 15 (n = 2)
    -205.60 ± 136.047
        Total-C: Change at Week 16 (n = 9)
    -105.24 ± 186.103
        Total-C: Change at Week 17 (n = 2)
    -195.00 ± 111.157
        Total-C: Change at Week 18 (n = 7)
    -63.27 ± 166.911
        Total-C: Change at Week 20 (n = 2)
    -170.45 ± 78.135
        Total-C: Change at Week 22 (n = 7)
    -41.30 ± 174.597
        Total-C: Change at Week 23 (n = 2)
    -167.55 ± 80.539
        Total-C: Change at Week 25 (n = 2)
    -90.15 ± 26.234
        Total-C: Change at Week 26 (n = 6)
    -30.77 ± 156.736
        Lp(a): Change at Week 2 (n = 9)
    -22.4 ± 30.18
        Lp(a): Change at Week 3 (n = 9)
    -20.8 ± 30.11
        Lp(a): Change at Week 4 (n = 9)
    -21.1 ± 28.29
        Lp(a): Change at Week 5 (n = 8)
    -30.1 ± 24.91
        Lp(a): Change at Week 6 (n = 9)
    -25.0 ± 31.40
        Lp(a): Change at Week 8 (n = 9)
    -28.9 ± 23.81
        Lp(a): Change at Week 12 (n = 9)
    -20.9 ± 16.30
        Lp(a): Change at Week 14 (n = 9)
    -22.3 ± 21.01
        Lp(a): Change at Week 16 (n = 9)
    -26.9 ± 38.97
        Lp(a): Change at Week 18 (n = 7)
    -29.9 ± 29.27
        Lp(a): Change at Week 20 (n = 2)
    -19.0 ± 46.67
        Lp(a): Change at Week 22 (n = 7)
    -24.3 ± 37.53
        Lp(a): Change at Week 23 (n = 2)
    -12.5 ± 19.09
        Lp(a): Change at Week 26 (n = 6)
    -16.3 ± 37.90
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 0) up to Week 26 in the Main Study Period

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    End point title
    Percent Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 0) up to Week 26 in the Main Study Period
    End point description
    Percent change was reported for Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline (week 0) up to week 26. Apo B, Non-HDL-C, Total-C, and Lp(a) were measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percent Change
    arithmetic mean (standard deviation)
        Apo B: Percent change at Week 2 (n = 9)
    -24.38 ± 20.959
        Apo B: Percent change at Week 3 (n = 9)
    -38.63 ± 22.114
        Apo B: Percent change at Week 4 (n = 9)
    -45.89 ± 18.222
        Apo B: Percent change at Week 5 (n = 8)
    -42.28 ± 13.247
        Apo B: Percent change at Week 6 (n = 9)
    -43.12 ± 14.716
        Apo B: Percent change at Week 8 (n = 9)
    -42.66 ± 14.475
        Apo B: Percent change at Week 12 (n = 9)
    -29.47 ± 21.736
        Apo B: Percent change at Week 14 (n = 9)
    -27.42 ± 18.418
        Apo B: Percent change at Week 16 (n = 9)
    -21.60 ± 24.970
        Apo B: Percent change at Week 18 (n = 7)
    -7.63 ± 24.347
        Apo B: Percent change at Week 20 (n = 2)
    -39.32 ± 6.481
        Apo B: Percent change at Week 22 (n = 7)
    -8.07 ± 30.444
        Apo B: Percent change at Week 23 (n = 2)
    -40.29 ± 10.089
        Apo B: Percent change at Week 26 (n = 6)
    -2.03 ± 25.320
        Non-HDL-C: Percent change at Day 4 (n = 9)
    -13.61 ± 10.721
        Non-HDL-C: Percent change at Week 1 (n = 9)
    -24.12 ± 20.083
        Non-HDL-C: Percent change at Week 2 (n = 9)
    -29.61 ± 23.406
        Non-HDL-C: Percent change at Week 3 (n = 9)
    -41.62 ± 23.969
        Non-HDL-C: Percent change at Week 4 (n = 9)
    -48.91 ± 22.257
        Non-HDL-C: Percent change at Week 5 (n = 8)
    -46.61 ± 14.172
        Non-HDL-C: Percent change at Week 6 (n = 9)
    -51.49 ± 14.344
        Non-HDL-C: Percent change at Week 8 (n = 9)
    -50.65 ± 17.231
        Non-HDL-C: Percent change at Week 10 (n = 9)
    -44.80 ± 13.148
        Non-HDL-C: Percent change at Week 12 (n = 9)
    -36.40 ± 18.711
        Non-HDL-C: Percent change at Week 13 (n = 2)
    -39.84 ± 11.313
        Non-HDL-C: Percent change at Week 14 (n = 9)
    -32.71 ± 18.214
        Non-HDL-C: Percent change at Week 15 (n = 2)
    -43.09 ± 14.482
        Non-HDL-C: Percent change at Week 16 (n = 9)
    -24.22 ± 25.899
        Non-HDL-C: Percent change at Week 17 (n = 2)
    -41.21 ± 10.455
        Non-HDL-C: Percent change at Week 18 (n = 7)
    -12.70 ± 27.798
        Non-HDL-C: Percent change at Week 20 (n = 2)
    -36.71 ± 5.471
        Non-HDL-C: Percent change at Week 22 (n = 7)
    -9.43 ± 37.102
        Non-HDL-C: Percent change at Week 23 (n = 2)
    -37.10 ± 5.138
        Non-HDL-C: Percent change at Week 25 (n = 2)
    -21.86 ± 1.676
        Non-HDL-C: Percent change at Week 26 (n = 6)
    -9.99 ± 25.292
        Total-C: Percent change at Day 4 (n = 9)
    -13.18 ± 8.932
        Total-C: Percent change at Week 1 (n = 9)
    -23.23 ± 16.602
        Total-C: Percent change at Week 2 (n = 9)
    -29.14 ± 20.307
        Total-C: Percent change at Week 3 (n = 9)
    -40.81 ± 21.011
        Total-C: Percent change at Week 4 (n = 9)
    -46.95 ± 19.083
        Total-C: Percent change at Week 5 (n = 8)
    -45.43 ± 12.909
        Total-C: Percent change at Week 6 (n = 9)
    -50.68 ± 13.327
        Total-C: Percent change at Week 8 (n = 9)
    -48.29 ± 15.556
        Total-C: Percent change at Week 10 (n = 9)
    -42.15 ± 10.858
        Total-C: Percent change at Week 12 (n = 9)
    -33.48 ± 16.136
        Total-C: Percent change at Week 13 (n = 2)
    -38.98 ± 10.525
        Total-C: Percent change at Week 14 (n = 9)
    -29.66 ± 15.759
        Total-C: Percent change at Week 15 (n = 2)
    -42.01 ± 14.356
        Total-C: Percent change at Week 16 (n = 9)
    -23.51 ± 25.208
        Total-C: Percent change at Week 17 (n = 2)
    -40.59 ± 9.468
        Total-C: Percent change at Week 18 (n = 7)
    -12.65 ± 26.105
        Total-C: Percent change at Week 20 (n = 2)
    -36.28 ± 3.862
        Total-C: Percent change at Week 22 (n = 7)
    -7.73 ± 33.729
        Total-C: Percent change at Week 23 (n = 2)
    -35.50 ± 4.662
        Total-C: Percent change at Week 25 (n = 2)
    -19.82 ± 1.459
        Total-C: Percent change at Week 26 (n = 6)
    -10.05 ± 22.903
        Lp(a): Percent change at Week 2 (n = 9)
    -11.64 ± 20.323
        Lp(a): Percent change at Week 3 (n = 9)
    -12.25 ± 19.350
        Lp(a): Percent change at Week 4 (n = 9)
    -10.85 ± 24.465
        Lp(a): Percent change at Week 5 (n = 8)
    -23.79 ± 17.908
        Lp(a): Percent change at Week 6 (n = 9)
    -17.12 ± 23.680
        Lp(a): Percent change at Week 8 (n = 9)
    -17.24 ± 17.764
        Lp(a): Percent change at Week 12 (n = 9)
    -13.09 ± 12.614
        Lp(a): Percent change at Week 14 (n = 9)
    -13.54 ± 10.151
        Lp(a): Percent change at Week 16 (n = 9)
    -16.93 ± 24.795
        Lp(a): Percent change at Week 18 (n = 7)
    -19.61 ± 21.686
        Lp(a): Percent change at Week 20 (n = 2)
    -19.72 ± 32.894
        Lp(a): Percent change at Week 22 (n = 7)
    -10.37 ± 29.230
        Lp(a): Percent change at Week 23 (n = 2)
    -10.62 ± 15.373
        Lp(a): Percent change at Week 26 (n = 6)
    3.46 ± 39.400
    No statistical analyses for this end point

    Secondary: Absolute Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

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    End point title
    Absolute Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period
    End point description
    Absolute changein Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline to week 214 in open label extension (OLE) period. The efficacy analysis set included all subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point. (ET = Early Termination; EOS = End of Study)
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) up to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: mg/dL
    arithmetic mean (standard deviation)
        Apo B: Change at Week 30 (n= 7)
    -48.1 ± 61.41
        Apo B: Change at Week 34 (n= 8)
    -65.9 ± 58.96
        Apo B: Change at Week 38 (n= 8)
    -40.9 ± 56.34
        Apo B: Change at Week 42 (n= 6)
    -79.5 ± 71.19
        Apo B: Change at Week 44 (n= 8)
    -78.6 ± 58.93
        Apo B: Change at Week 50 (n= 8)
    -57.8 ± 50.82
        Apo B: Change at Week 70 (n= 4)
    -60.3 ± 78.24
        Apo B: Change at Week 82 (n= 4)
    -38.0 ± 35.66
        Apo B: Change at Week 94 (n= 1)
    -212.0 ± 99999
        Apo B: Change at Week 106 (n= 1)
    -173.0 ± 99999
        Apo B: Change at ET (n= 8)
    -3.5 ± 42.88
        Apo B: Change at EOS (n= 3)
    65.0 ± 100.53
        Non- HDL-C: Change at Week 27 (n= 8)
    -65.06 ± 61.756
        Non- HDL-C: Change at Week 28 (n= 8)
    -95.65 ± 87.499
        Non- HDL-C: Change at Week 29 (n= 8)
    -122.94 ± 106.296
        Non- HDL-C: Change at Week 30 (n= 7)
    -94.33 ± 113.286
        Non- HDL-C: Change at Week 31 (n= 8)
    -152.98 ± 135.977
        Non- HDL-C: Change at Week 32 (n= 8)
    -166.94 ± 133.990
        Non- HDL-C: Change at Week 34 (n= 8)
    -133.69 ± 112.247
        Non- HDL-C: Change at Week 36 (n= 8)
    -111.06 ± 93.635
        Non- HDL-C: Change at Week 38 (n= 8)
    -84.16 ± 101.210
        Non- HDL-C: Change at Week 39 (n= 6)
    -146.35 ± 128.368
        Non- HDL-C: Change at Week 40 (n= 6)
    -134.50 ± 128.198
        Non- HDL-C: Change at Week 41 (n= 6)
    -171.25 ± 143.692
        Non- HDL-C:Change at Week 42 (n= 6)
    -150.28 ± 132.214
        Non- HDL-C: Change at Week 44 (n= 8)
    -149.00 ± 114.152
        Non- HDL-C: Change at Week 46 (n= 8)
    -129.99 ± 116.770
        Non- HDL-C: Change at Week 50 (n= 8)
    -110.83 ± 104.353
        Non- HDL-C: Change at Week 54 (n= 8)
    -14.79 ± 178.797
        Non- HDL-C: Change at Week 58 (n= 6)
    -49.13 ± 97.212
        Non- HDL-C: Change at Week 70 (n= 4)
    -121.83 ± 152.887
        Non- HDL-C: Change at Week 82 (n= 4)
    -69.00 ± 66.715
        Non- HDL-C: Change at Week 94 (n= 1)
    -388.10 ± 99999
        Non- HDL-C: Change at Week 106 (n= 1)
    -303.10 ± 99999
        Non- HDL-C: Change at ET (n= 8)
    -12.45 ± 64.513
        Non- HDL-C: Change at EOS (n= 3)
    102.07 ± 153.301
        Total-C: Change at Week 27 (n= 8)
    -73.80 ± 60.737
        Total-C: Change at Week 28 (n= 8)
    -107.78 ± 83.051
        Total-C: Change at Week 29 (n= 8)
    -134.33 ± 102.667
        Total-C: Change at Week 30 (n= 7)
    -107.61 ± 109.689
        Total-C: Change at Week 31 (n= 8)
    -166.85 ± 130.925
        Total-C: Change at Week 32 (n= 8)
    -181.09 ± 130.048
        Total-C: Change at Week 34 (n= 8)
    -148.90 ± 106.534
        Total-C: Change at Week 36 (n= 8)
    -125.14 ± 89.065
        Total-C: Change at Week 38 (n= 8)
    -93.00 ± 99.558
        Total-C: Change at Week 39 (n= 6)
    -160.38 ± 125.452
        Total-C: Change at Week 40 (n= 6)
    -150.42 ± 124.695
        Total-C: Change at Week 41 (n= 6)
    -188.42 ± 141.249
        Total-C: Change at Week 42 (n= 6)
    -166.80 ± 128.247
        Total-C: Change at Week 44 (n= 8)
    -164.68 ± 110.222
        Total-C: Change at Week 46 (n= 8)
    -144.69 ± 110.682
        Total-C: Change at Week 50 (n= 8)
    -123.04 ± 99.299
        Total-C: Change at Week 54 (n= 8)
    -23.80 ± 180.982
        Total-C: Change at Week 58 (n= 6)
    -54.45 ± 97.160
        Total-C: Change at Week 70 (n= 4)
    -132.55 ± 152.664
        Total-C: Change at Week 82 (n= 4)
    -75.43 ± 67.802
        Total-C: Change at Week 94 (n= 1)
    -392.30 ± 99999
        Total-C: Change at Week 106 (n= 1)
    -303.10 ± 99999
        Total-C: Change at ET (n= 8)
    -20.38 ± 68.809
        Total-C: Change at EOS (n= 3)
    100.40 ± 144.828
        Lp(a): Change at Week 30 (n= 7)
    -9.0 ± 27.59
        Lp(a): Change at Week 34 (n= 8)
    -10.8 ± 24.00
        Lp(a): Change at Week 38 (n= 8)
    -16.0 ± 19.70
        Lp(a): Change at Week 42 (n= 6)
    -0.8 ± 41.36
        Lp(a): Change at Week 44 (n= 8)
    1.5 ± 37.73
        Lp(a): Change at Week 50 (n= 8)
    11.6 ± 48.23
        Lp(a): Change at Week 70 (n= 4)
    36.8 ± 77.92
        Lp(a): Change at Week 82 (n= 4)
    -5.3 ± 27.04
        Lp(a): Change at Week 94 (n= 1)
    -18.0 ± 99999
        Lp(a): Change at Week 106 (n= 1)
    39.0 ± 99999
        Lp(a): Change at Week ET (n= 8)
    11.6 ± 26.72
        Lp(a): Change at EOS (n= 3)
    18.3 ± 18.01
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

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    End point title
    Percent Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period
    End point description
    Percent change was reported in Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline (week 26) up to week 214 in OLE Period. Apo B, Non-HDL-C, Total-C, and Lp(a) were measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here "n" signifies the subjects who were evaluable for this endpoint at a given time point. (ET= Early Termination; EOS = End of Study)
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) up to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: Percent change
    arithmetic mean (standard deviation)
        Apo B: Percent change at Week 30 (n= 7)
    -30.59 ± 25.991
        Apo B: Percent change at Week 34 (n= 8)
    -41.27 ± 19.573
        Apo B: Percent change at Week 38 (n= 8)
    -29.02 ± 25.029
        Apo B: Percent change at Week 42 (n= 6)
    -50.90 ± 17.497
        Apo B: Percent change at Week 44 (n= 8)
    -48.59 ± 16.201
        Apo B: Percent change at Week 50 (n= 8)
    -36.68 ± 19.620
        Apo B: Percent change at Week 70 (n= 4)
    -33.44 ± 26.773
        Apo B: Percent change at Week 82 (n= 4)
    -24.87 ± 17.148
        Apo B: Percent change at Week 94 (n= 1)
    -77.09 ± 99999
        Apo B: Percent change at Week 106 (n= 1)
    -62.91 ± 99999
        Apo B: Percent change at ET (n= 8)
    -11.94 ± 25.708
        Apo B: Percent change at EOS (n= 3)
    22.60 ± 38.988
        Non- HDL-C: Percent change at Week 27 (n= 8)
    -23.62 ± 15.342
        Non- HDL-C: Percent change at Week 28 (n= 8)
    -37.22 ± 19.207
        Non- HDL-C: Percent change at Week 29 (n= 8)
    -44.30 ± 19.766
        Non- HDL-C: Percent change at Week 30 (n= 7)
    -37.90 ± 29.133
        Non- HDL-C: Percent change at Week 31 (n= 8)
    -50.59 ± 19.285
        Non- HDL-C: Percent change at Week 32 (n= 8)
    -59.09 ± 18.200
        Non- HDL-C: Percent change at Week 34 (n= 8)
    -51.55 ± 21.112
        Non- HDL-C: Percent change at Week 36 (n= 8)
    -46.62 ± 20.797
        Non- HDL-C: Percent change at Week 38 (n= 8)
    -36.10 ± 24.473
        Non- HDL-C: Percent change at Week 39 (n= 6)
    -59.25 ± 21.381
        Non- HDL-C: Percent change at Week 40 (n= 6)
    -53.24 ± 20.135
        Non- HDL-C: Percent change at Week 41 (n= 6)
    -65.07 ± 18.375
        Non- HDL-C: Percent change at Week 42 (n= 6)
    -58.92 ± 18.287
        Non- HDL-C: Percent change at Week 44 (n= 8)
    -57.03 ± 17.675
        Non- HDL-C: Percent change at Week 46 (n= 8)
    -48.27 ± 18.674
        Non- HDL-C: Percent change at Week 50 (n= 8)
    -41.16 ± 22.284
        Non- HDL-C: Percent change at Week 54 (n= 8)
    -12.93 ± 42.412
        Non- HDL-C: Percent change at Week 58 (n= 6)
    -12.28 ± 26.060
        Non- HDL-C: Percent change at Week 70 (n= 4)
    -37.68 ± 35.771
        Non- HDL-C: Percent change at Week 82 (n= 4)
    -26.16 ± 27.153
        Non- HDL-C: Percent change at Week 94 (n= 1)
    -81.52 ± 99999
        Non- HDL-C: Percent change at Week 106 (n= 1)
    -63.66 ± 99999
        Non- HDL-C: Percent change at ET (n= 8)
    -17.65 ± 27.339
        Non- HDL-C: Percent change at EOS (n= 3)
    19.98 ± 40.313
        Total-C: Percent change at Week 27 (n= 8)
    -22.24 ± 11.566
        Total-C: Percent change at Week 28 (n= 8)
    -34.78 ± 14.984
        Total-C: Percent change at Week 29 (n= 8)
    -40.57 ± 14.335
        Total-C: Percent change at Week 30 (n= 7)
    -36.37 ± 23.550
        Total-C: Percent change at Week 31 (n= 8)
    -48.83 ± 16.944
        Total-C: Percent change at Week 32 (n= 8)
    -55.05 ± 16.624
        Total-C: Percent change at Week 34 (n= 8)
    -48.67 ± 18.123
        Total-C: Percent change at Week 36 (n= 8)
    -44.16 ± 19.175
        Total-C: Percent change at Week 38 (n= 8)
    -33.29 ± 22.145
        Total-C: Percent change at Week 39 (n= 6)
    -54.40 ± 19.803
        Total-C: Percent change at Week 40 (n= 6)
    -50.13 ± 18.316
        Total-C: Percent change at Week 41 (n= 6)
    -60.87 ± 17.966
        Total-C: Percent change at Week 42 (n= 6)
    -54.88 ± 15.886
        Total-C: Percent change at Week 44 (n= 8)
    -53.41 ± 16.231
        Total-C: Percent change at Week 46 (n= 8)
    -46.06 ± 16.855
        Total-C: Percent change at Week 50 (n= 8)
    -39.04 ± 19.318
        Total-C: Percent change at Week 54 (n= 8)
    -12.82 ± 38.323
        Total-C: Percent change at Week 58 (n= 6)
    -13.78 ± 21.976
        Total-C: Percent change at Week 70 (n= 4)
    -36.63 ± 32.371
        Total-C: Percent change at Week 82 (n= 4)
    -23.09 ± 19.171
        Total-C: Percent change at Week 94 (n= 1)
    -77.50 ± 99999
        Total-C: Percent change at Week 106 (n= 1)
    -59.88 ± 99999
        Total-C: Percent change at ET (n= 8)
    -16.76 ± 25.085
        Total-C: Percent change at EOS (n= 3)
    18.20 ± 33.900
        Lp(a): Percent change at Week 30 (n= 7)
    -3.57 ± 24.610
        Lp(a): Percent change at Week 34 (n= 8)
    -6.43 ± 21.281
        Lp(a): Percent change at Week 38 (n= 8)
    -9.50 ± 14.429
        Lp(a): Percent change at Week 42 (n= 6)
    -4.76 ± 24.227
        Lp(a): Percent change at Week 44 (n= 8)
    -5.13 ± 25.377
        Lp(a): Percent change at Week 50 (n= 8)
    1.02 ± 29.756
        Lp(a): Percent change at Week 70 (n= 4)
    15.80 ± 36.280
        Lp(a): Percent change at Week 82 (n= 4)
    -3.77 ± 16.168
        Lp(a): Percent change at Week 94 (n= 1)
    -11.92 ± 99999
        Lp(a): Percent change at Week 106 (n= 1)
    25.83 ± 99999
        Lp(a): Percent change at ET (n= 8)
    9.96 ± 21.597
        Lp(a): Percent change at EOS (n= 3)
    40.51 ± 39.616
    No statistical analyses for this end point

    Secondary: Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% from Baseline (Week 0) in the Main Study Period

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    End point title
    Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% from Baseline (Week 0) in the Main Study Period
    End point description
    Percentage of subjects who achieved reduction in low-density lipoprotein cholesterol (LDL-C) of greater than or equal to (≥) 25 percent (%) from baseline in the main study period was reported. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percentage of subjects
        number (not applicable)
    100
    No statistical analyses for this end point

    Secondary: Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% from Baseline (Week 26) in the Open Label Extension (OLE) Period

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    End point title
    Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% from Baseline (Week 26) in the Open Label Extension (OLE) Period
    End point description
    Percentage of subjects who achieved a reduction in low-density lipoprotein cholesterol (LDL-C) of ≥ 25% from baseline (week 26) to week 214 was reported. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: Percentage of Subjects
        number (not applicable)
    100
    No statistical analyses for this end point

    Secondary: Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% from Baseline (Week 0) in the Main Study Period

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    End point title
    Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% from Baseline (Week 0) in the Main Study Period
    End point description
    Percentage of subjects who achieved a reduction in low-density lipoprotein cholesterol (LDL-C) of ≥ 50% from baseline (week 0) to week 26 was reported. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percentage of subjects
        number (not applicable)
    77.8
    No statistical analyses for this end point

    Secondary: Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% from Baseline (Week 26) in the Open Label Extension (OLE) Period

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    End point title
    Percentage of Subjects Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% from Baseline (Week 26) in the Open Label Extension (OLE) Period
    End point description
    Percentage of subjects who achieved reduction in low-density lipoprotein cholesterol (LDL-C) of ≥ 50% from baseline (week 26) in the OLE period was reported. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) up to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: Percentage of subjects
        number (not applicable)
    87.5
    No statistical analyses for this end point

    Secondary: Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 0) Over Time in the Main Study Period

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    End point title
    Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 0) Over Time in the Main Study Period
    End point description
    Absolute change was reported in HDL-C, TG, and Apo A-1 from baseline (week 0) up to week 26. The efficacy analysis set included all subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: mg/dL
    arithmetic mean (standard deviation)
        HDL-C: Change at Day 4 (n = 9)
    -5.81 ± 4.789
        HDL-C: Change at Week 1 (n = 9)
    -9.92 ± 6.578
        HDL-C: Change at Week 2 (n = 9)
    -11.99 ± 7.351
        HDL-C: Change at Week 3 (n = 9)
    -16.02 ± 10.737
        HDL-C: Change at Week 4 (n = 9)
    -15.07 ± 10.792
        HDL-C: Change at Week 5 (n = 8)
    -11.66 ± 8.442
        HDL-C: Change at Week 6 (n = 9)
    -16.46 ± 11.868
        HDL-C: Change at Week 8 (n = 9)
    -12.06 ± 11.334
        HDL-C: Change at Week 10 (n = 9)
    -10.94 ± 8.215
        HDL-C: Change at Week 12 (n = 9)
    -6.82 ± 6.811
        HDL-C: Change at Week 13 (n = 2)
    -8.10 ± 2.970
        HDL-C: Change at Week 14 (n = 9)
    -6.18 ± 4.205
        HDL-C: Change at Week 15 (n = 2)
    -8.70 ± 0.566
        HDL-C: Change at Week 16 (n = 9)
    -5.54 ± 5.785
        HDL-C: Change at Week 17 (n = 2)
    -9.40 ± 3.818
        HDL-C: Change at Week 18 (n = 7)
    -2.93 ± 7.435
        HDL-C: Change at Week 20 (n = 2)
    -8.70 ± 8.202
        HDL-C: Change at Week 22 (n = 7)
    -0.30 ± 6.943
        HDL-C: Change at Week 23 (n = 2)
    -4.00 ± 4.384
        HDL-C: Change at Week 25 (n = 2)
    2.50 ± 3.818
        HDL-C: Change at Week 26 (n = 6)
    -5.30 ± 5.266
        TG: Change at Day 4 (n = 9)
    -25.17 ± 18.545
        TG: Change at Week 1 (n = 9)
    -21.41 ± 17.603
        TG: Change at Week 2 (n = 9)
    -15.83 ± 15.908
        TG: Change at Week 3 (n = 9)
    -39.73 ± 31.977
        TG: Change at Week 4 (n = 9)
    -40.81 ± 30.277
        TG: Change at Week 5 (n = 8)
    -42.04 ± 28.887
        TG: Change at Week 6 (n = 9)
    -38.73 ± 35.120
        TG: Change at Week 8 (n = 9)
    -30.37 ± 26.383
        TG: Change at Week 10 (n = 9)
    -28.79 ± 21.095
        TG: Change at Week 12 (n = 9)
    -33.23 ± 32.901
        TG: Change at Week 13 (n = 2)
    -49.15 ± 56.922
        TG: Change at Week 14 (n = 9)
    -22.11 ± 35.348
        TG: Change at Week 15 (n = 2)
    -52.20 ± 82.590
        TG: Change at Week 16 (n = 9)
    -11.20 ± 48.136
        TG: Change at Week 17 (n = 2)
    -55.35 ± 58.195
        TG: Change at Week 18 (n = 7)
    9.77 ± 24.537
        TG: Change at Week 20 (n = 2)
    -33.65 ± 33.729
        TG: Change at Week 22 (n = 7)
    1.90 ± 33.916
        TG: Change at Week 23 (n = 2)
    -36.30 ± 31.254
        TG: Change at Week 25 (n = 2)
    -23.45 ± 34.436
        TG: Change at Week 26 (n = 6)
    -5.87 ± 28.748
        Apo A-1: Change at Week 2 (n = 9)
    -28.6 ± 18.64
        Apo A-1: Change at Week 3 (n = 9)
    -43.3 ± 22.54
        Apo A-1: Change at Week 4 (n = 9)
    -43.4 ± 16.88
        Apo A-1: Change at Week 5 (n = 8)
    -35.8 ± 17.53
        Apo A-1: Change at Week 6 (n = 9)
    -40.7 ± 19.86
        Apo A-1: Change at Week 8 (n = 9)
    -33.6 ± 20.64
        Apo A-1: Change at Week 12 (n = 9)
    -17.2 ± 20.04
        Apo A-1: Change at Week 14 (n = 9)
    -17.7 ± 11.97
        Apo A-1: Change at Week 16 (n = 9)
    -11.3 ± 16.50
        Apo A-1: Change at Week 18 (n = 7)
    -4.9 ± 17.72
        Apo A-1: Change at Week 20 (n = 2)
    -24.0 ± 8.49
        Apo A-1: Change at Week 22 (n = 7)
    1.9 ± 15.42
        Apo A-1: Change at Week 23 (n = 2)
    -23.0 ± 8.49
        Apo A-1: Change at Week 26 (n = 6)
    -10.8 ± 13.83
    No statistical analyses for this end point

    Secondary: Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 0) Over Time in the Main Study Period

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    End point title
    Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 0) Over Time in the Main Study Period
    End point description
    Percent change was reported in HDL-C, TG, and Apo A-1 from baseline (week 0) up to week 26. HDL-C, TG, and Apo A-1 were measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies the subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 26
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percent Change
    arithmetic mean (standard deviation)
        HDL-C: Percent Change at Day 4 (n = 9)
    -14.37 ± 10.830
        HDL-C: Percent Change at Week 1 (n = 9)
    -23.34 ± 10.783
        HDL-C: Percent Change at Week 2 (n = 9)
    -28.75 ± 8.458
        HDL-C: Percent Change at Week 3 (n = 9)
    -36.96 ± 16.495
        HDL-C: Percent Change at Week 4 (n = 9)
    -35.24 ± 16.212
        HDL-C: Percent Change at Week 5 (n = 8)
    -29.74 ± 14.240
        HDL-C: Percent Change at Week 6 (n = 9)
    -37.88 ± 15.864
        HDL-C: Percent Change at Week 8 (n = 9)
    -25.42 ± 23.199
        HDL-C: Percent Change at Week 10 (n = 9)
    -24.57 ± 22.409
        HDL-C: Percent Change at Week 12 (n = 9)
    -13.59 ± 23.189
        HDL-C: Percent Change at Week 13 (n = 2)
    -24.48 ± 6.243
        HDL-C: Percent Change at Week 14 (n = 9)
    -16.81 ± 14.822
        HDL-C: Percent Change at Week 15 (n = 2)
    -26.97 ± 4.895
        HDL-C: Percent Change at Week 16 (n = 9)
    -12.03 ± 16.026
        HDL-C: Percent Change at Week 17 (n = 2)
    -28.34 ± 8.393
        HDL-C: Percent Change at Week 18 (n = 7)
    -1.05 ± 24.171
        HDL-C: Percent Change at Week 20 (n = 2)
    -25.39 ± 22.187
        HDL-C: Percent Change at Week 22 (n = 7)
    3.78 ± 20.466
        HDL-C: Percent Change at Week 23 (n = 2)
    -11.56 ± 12.094
        HDL-C: Percent Change at Week 25 (n = 2)
    8.41 ± 12.698
        HDL-C: Percent Change at Week 26 (n = 6)
    -12.00 ± 11.109
        TG: Percent Change at Day 4 (n = 9)
    -29.61 ± 18.562
        TG: Percent Change at Week 1 (n = 9)
    -23.41 ± 20.150
        TG: Percent Change at Week 2 (n = 9)
    -17.19 ± 18.735
        TG: Percent Change at Week 3 (n = 9)
    -44.38 ± 19.223
        TG: Percent Change at Week 4 (n = 9)
    -46.57 ± 17.014
        TG: Percent Change at Week 5 (n = 8)
    -44.49 ± 16.583
        TG: Percent Change at Week 6 (n = 9)
    -40.03 ± 26.830
        TG: Percent Change at Week 8 (n = 9)
    -30.69 ± 24.800
        TG: Percent Change at Week 10 (n = 9)
    -31.40 ± 16.697
        TG: Percent Change at Week 12 (n = 9)
    -35.38 ± 25.014
        TG: Percent Change at Week 13 (n = 2)
    -35.11 ± 26.733
        TG: Percent Change at Week 14 (n = 9)
    -23.51 ± 28.335
        TG: Percent Change at Week 15 (n = 2)
    -27.77 ± 55.240
        TG: Percent Change at Week 16 (n = 9)
    -6.70 ± 40.326
        TG: Percent Change at Week 17 (n = 2)
    -42.09 ± 22.941
        TG: Percent Change at Week 18 (n = 7)
    23.13 ± 55.349
        TG: Percent Change at Week 20 (n = 2)
    -26.30 ± 11.945
        TG: Percent Change at Week 22 (n = 7)
    8.86 ± 43.819
        TG: Percent Change at Week 23 (n = 2)
    -30.58 ± 6.662
        TG: Percent Change at Week 25 (n = 2)
    -13.62 ± 21.582
        TG: Percent Change at Week 26 (n = 6)
    -1.60 ± 33.318
        Apo A-1: Percent Change at Week 2 (n = 9)
    -24.37 ± 12.794
        Apo A-1: Percent Change at Week 3 (n = 9)
    -36.47 ± 16.930
        Apo A-1: Percent Change at Week 4 (n = 9)
    -38.54 ± 8.906
        Apo A-1: Percent Change at Week 5 (n = 8)
    -31.91 ± 11.491
        Apo A-1: Percent Change at Week 6 (n = 9)
    -34.72 ± 12.342
        Apo A-1: Percent Change at Week 8 (n = 9)
    -29.03 ± 14.765
        Apo A-1: Percent Change at Week 12 (n = 9)
    -11.85 ± 25.176
        Apo A-1: Percent Change at Week 14 (n = 9)
    -17.65 ± 13.824
        Apo A-1: Percent Change at Week 16 (n = 9)
    -9.86 ± 14.372
        Apo A-1: Percent Change at Week 18 (n = 7)
    -1.16 ± 19.598
        Apo A-1: Percent Change at Week 20 (n = 2)
    -22.15 ± 7.965
        Apo A-1: Percent Change at Week 22 (n = 7)
    4.21 ± 17.857
        Apo A-1: Percent Change at Week 23 (n = 2)
    -21.22 ± 7.959
        Apo A-1: Percent Change at Week 26 (n = 6)
    -9.68 ± 11.250
    No statistical analyses for this end point

    Secondary: Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

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    End point title
    Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period
    End point description
    Percent change was reported in HDL-C, TG and Apo A-1 from baseline (week 26) up to week 214. HDL-C, TG and Apo A-1 were measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for the endpoint at a given time point. (ET= Early Termination; EOS = End of Study)
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) up to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: Percent change
    arithmetic mean (standard deviation)
        HDL-C: Percent change at Week 27 (n= 8)
    -21.47 ± 11.815
        HDL-C: Percent change at Week 28 (n= 8)
    -27.60 ± 11.485
        HDL-C: Percent change at Week 29 (n= 8)
    -26.51 ± 16.584
        HDL-C: Percent change at Week 30 (n= 7)
    -27.57 ± 14.566
        HDL-C: Percent change at Week 31 (n= 8)
    -32.81 ± 18.939
        HDL-C: Percent change at Week 32 (n= 8)
    -32.72 ± 14.684
        HDL-C: Percent change at Week 34 (n= 8)
    -34.60 ± 15.971
        HDL-C: Percent change at Week 36 (n= 8)
    -31.14 ± 18.695
        HDL-C: Percent change at Week 38 (n= 8)
    -18.57 ± 15.411
        HDL-C: Percent change at Week 39 (n= 6)
    -32.09 ± 16.062
        HDL-C: Percent change at Week 40 (n= 6)
    -36.25 ± 9.327
        HDL-C: Percent change at Week 41 (n= 6)
    -39.14 ± 13.804
        HDL-C: Percent change at Week 42 (n= 6)
    -38.31 ± 10.670
        HDL-C: Percent change at Week 44 (n= 8)
    -37.01 ± 12.049
        HDL-C: Percent change at Week 46 (n= 8)
    -32.77 ± 13.829
        HDL-C: Percent change at Week 50 (n= 8)
    -25.17 ± 17.957
        HDL-C: Percent change at Week 54 (n= 8)
    -20.16 ± 18.657
        HDL-C: Percent change at Week 58 (n= 6)
    -10.87 ± 14.194
        HDL-C: Percent change at Week 70 (n= 4)
    -24.67 ± 15.473
        HDL-C: Percent change at Week 82 (n= 4)
    -17.56 ± 10.574
        HDL-C: Percent change at Week 94 (n= 1)
    -13.95 ± 99999
        HDL-C: Percent change at Week 106 (n= 1)
    0.00 ± 99999
        HDL-C: Percent change at ET (n= 8)
    -15.08 ± 21.569
        HDL-C: Percent change at EOS (n= 3)
    -0.78 ± 27.385
        Triglycerides: Percent change at Week 27 (n= 8)
    -38.62 ± 26.036
        Triglycerides: Percent change at Week 28 (n= 8)
    -41.76 ± 27.247
        Triglycerides: Percent change at Week 29 (n= 8)
    -41.17 ± 18.721
        Triglycerides: Percent change at Week 30 (n= 7)
    -38.82 ± 23.649
        Triglycerides: Percent change at Week 31 (n= 8)
    -44.86 ± 28.212
        Triglycerides: Percent change at Week 32 (n= 8)
    -45.33 ± 23.671
        Triglycerides: Percent change at Week 34 (n= 8)
    -28.58 ± 23.643
        Triglycerides: Percent change at Week 36 (n= 8)
    -25.89 ± 36.606
        Triglycerides: Percent change at Week 38 (n= 8)
    -24.32 ± 25.386
        Triglycerides: Percent change at Week 39 (n= 6)
    -48.37 ± 26.457
        Triglycerides: Percent change at Week 40 (n= 6)
    -43.36 ± 26.527
        Triglycerides: Percent change at Week 41 (n= 6)
    -46.65 ± 25.386
        Triglycerides: Percent change at Week 42 (n= 6)
    -43.09 ± 29.631
        Triglycerides: Percent change at Week 44 (n= 8)
    -47.98 ± 35.076
        Triglycerides: Percent change at Week 46 (n= 8)
    -37.64 ± 26.234
        Triglycerides: Percent change at Week 50 (n= 8)
    -22.10 ± 27.247
        Triglycerides: Percent change at Week 54 (n= 8)
    -1.08 ± 30.518
        Triglycerides: Percent change at Week 58 (n= 6)
    3.72 ± 25.130
        Triglycerides: Percent change at Week 70 (n= 4)
    -22.28 ± 31.761
        Triglycerides: Percent change at Week 82 (n= 4)
    -36.47 ± 9.998
        Triglycerides: Percent change at Week 94 (n= 1)
    -76.36 ± 99999
        Triglycerides: Percent change at Week 106 (n= 1)
    -57.98 ± 99999
        Triglycerides: Percent change at ET (n= 8)
    -14.52 ± 25.892
        Triglycerides: Percent change at EOS (n= 3)
    30.98 ± 40.511
        Apo A-1: Percent change at Week 30 (n= 7)
    -27.20 ± 12.119
        Apo A-1: Percent change at Week 34 (n= 8)
    -29.05 ± 17.483
        Apo A-1: Percent change at Week 38 (n= 8)
    -11.79 ± 23.939
        Apo A-1: Percent change at Week 42 (n= 6)
    -35.47 ± 9.512
        Apo A-1: Percent change at Week 44 (n= 8)
    -34.28 ± 11.306
        Apo A-1: Percent change at Week 50 (n= 8)
    -21.01 ± 15.471
        Apo A-1: Percent change at Week 70 (n= 4)
    -25.28 ± 11.950
        Apo A-1: Percent change at Week 82 (n= 4)
    -18.98 ± 6.534
        Apo A-1: Percent change at Week 94 (n= 1)
    -22.45 ± 99999
        Apo A-1: Percent change at Week 106 (n= 1)
    3.06 ± 99999
        Apo A-1: Percent change at ET (n= 8)
    -9.63 ± 25.308
        Apo A-1: Percent change at EOS (n= 3)
    11.27 ± 25.093
    No statistical analyses for this end point

    Secondary: Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

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    End point title
    Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) from Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period
    End point description
    Absolute change was reported in HDL-C, TG and Apo A-1 from baseline (week 26) up to week 214. The efficacy analysis set included all subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point. (ET= Early Termination; EOS = End of Study)
    End point type
    Secondary
    End point timeframe
    Baseline (Week 26) up to Week 214
    End point values
    REGN1500 300 mg SC/20 mg/kg IV
    Number of subjects analysed
    8
    Units: mg/dL
    arithmetic mean (standard deviation)
        HDL-C: Change at Week 27 (n= 8)
    -8.78 ± 5.736
        HDL-C: Change at Week 28 (n= 8)
    -12.08 ± 7.244
        HDL-C: Change at Week 29 (n= 8)
    -11.44 ± 7.858
        HDL-C: Change at Week 30 (n= 7)
    -13.29 ± 9.270
        HDL-C: Change at Week 31 (n= 8)
    -13.94 ± 8.655
        HDL-C: Change at Week 32 (n= 8)
    -14.11 ± 8.163
        HDL-C: Change at Week 34 (n= 8)
    -15.10 ± 9.167
        HDL-C: Change at Week 36 (n= 8)
    -14.14 ± 10.426
        HDL-C: Change at Week 38 (n= 8)
    -8.89 ± 7.513
        HDL-C: Change at Week 39 (n= 6)
    -14.02 ± 8.023
        HDL-C: Change at Week 40 (n= 6)
    -15.90 ± 7.171
        HDL-C: Change at Week 41 (n= 6)
    -17.17 ± 8.262
        HDL-C: Change at Week 42 (n= 6)
    -16.65 ± 7.148
        HDL-C: Change at Week 44 (n= 8)
    -15.83 ± 8.167
        HDL-C: Change at Week 46 (n= 8)
    -14.85 ± 10.173
        HDL-C: Change at Week 50 (n= 8)
    -12.25 ± 10.985
        HDL-C: Change at Week 54 (n= 8)
    -9.06 ± 9.107
        HDL-C: Change at Week 58 (n= 6)
    -5.42 ± 6.058
        HDL-C: Change at Week 70 (n= 4)
    -10.83 ± 10.448
        HDL-C: Change at Week 82 (n= 4)
    -6.28 ± 2.655
        HDL-C: Change at Week 94 (n= 1)
    -4.20 ± 99999
        HDL-C: Change at Week 106 (n= 1)
    0.00 ± 99999
        HDL-C: Percent change at ET (n= 8)
    -7.86 ± 8.258
        HDL-C: Change at EOS (n= 3)
    -1.67 ± 10.698
        Triglycerides: Change at Week 27 (n= 8)
    -31.19 ± 28.590
        Triglycerides: Change at Week 28 (n= 8)
    -36.28 ± 30.630
        Triglycerides: Change at Week 29 (n= 8)
    -32.86 ± 25.188
        Triglycerides: Change at Week 30 (n= 7)
    -32.37 ± 30.475
        Triglycerides: Change at Week 31 (n= 8)
    -39.04 ± 30.169
        Triglycerides: Change at Week 32 (n= 8)
    -37.83 ± 30.808
        Triglycerides: Change at Week 34 (n= 8)
    -25.41 ± 26.559
        Triglycerides: Change at Week 36 (n= 8)
    -24.66 ± 28.139
        Triglycerides: Change at Week 38 (n= 8)
    -21.13 ± 25.190
        Triglycerides: Change at Week 39 (n= 6)
    -40.27 ± 32.803
        Triglycerides: Change at Week 40 (n= 6)
    -37.30 ± 33.559
        Triglycerides: Change at Week 41 (n= 6)
    -39.37 ± 33.251
        Triglycerides: Change at Week 42 (n= 6)
    -37.62 ± 33.724
        Triglycerides: Change at Week 44 (n= 8)
    -42.25 ± 32.682
        Triglycerides: Change at Week 46 (n= 8)
    -31.53 ± 28.209
        Triglycerides: Change at Week 50 (n= 8)
    -20.00 ± 27.720
        Triglycerides: Change at Week 54 (n= 8)
    -6.40 ± 26.869
        Triglycerides: Change at Week 58 (n= 6)
    -3.53 ± 21.753
        Triglycerides: Change at Week 70 (n= 4)
    -21.23 ± 35.614
        Triglycerides: Change at Week 82 (n= 4)
    -26.33 ± 21.457
        Triglycerides: Change at Week 94 (n= 1)
    -88.50 ± 99999
        Triglycerides: Change at Week 106 (n= 1)
    -67.20 ± 99999
        Triglycerides: Change at ET (n= 8)
    -11.71 ± 21.343
        Triglycerides: Change at EOS (n= 3)
    30.67 ± 35.390
        Apo A-1: Change at Week 30 (n= 7)
    -34.4 ± 20.18
        Apo A-1: Change at Week 34 (n= 8)
    -35.1 ± 24.70
        Apo A-1: Change at Week 38 (n= 8)
    -18.0 ± 26.54
        Apo A-1: Change at Week 42 (n= 6)
    -43.7 ± 15.71
        Apo A-1: Change at Week 44 (n= 8)
    -39.4 ± 18.12
        Apo A-1: Change at Week 50 (n= 8)
    -26.9 ± 23.56
        Apo A-1: Change at Week 70 (n= 4)
    -30.3 ± 20.12
        Apo A-1: Change at Week 82 (n= 4)
    -21.0 ± 5.83
        Apo A-1: Change at Week 94 (n= 1)
    -22.0 ± 99999
        Apo A-1: Change at Week 106 (n= 1)
    3.0 ± 99999
        Apo A-1: Change at ET (n= 8)
    -15.6 ± 24.34
        Apo A-1: Change at EOS (n= 3)
    7.0 ± 24.98
    No statistical analyses for this end point

    Secondary: Absolute Change in Apolipoprotein CIII (Apo CIII) from Baseline (Week 0) Over Time in the Main Study Period

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    End point title
    Absolute Change in Apolipoprotein CIII (Apo CIII) from Baseline (Week 0) Over Time in the Main Study Period
    End point description
    Absolute change was reported in Apo CIII from baseline (week 0) up to week 16. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies the subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) up to Week 16
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: mg/dL
    arithmetic mean (standard deviation)
        Apo CIII: Change at Day 4 (n = 9)
    -3.729 ± 1.6905
        Apo CIII: Change at Week 1 (n = 9)
    -4.296 ± 1.9829
        Apo CIII: Change at Week 2 (n = 9)
    -4.301 ± 2.2542
        Apo CIII: Change at Week 5 (n = 8)
    -6.000 ± 2.8541
        Apo CIII: Change at Week 6 (n = 1)
    -8.380 ± 99999
        Apo CIII: Change at Week 12 (n = 9)
    -3.772 ± 2.8443
        Apo CIII: Change at Week 14 (n = 9)
    -3.866 ± 3.7327
        Apo CIII: Change at Week 16 (n = 9)
    -2.757 ± 4.4877
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein CIII (Apo CIII) from Baseline (Week 0) Over Time in the Main Study Period

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    End point title
    Percent Change in Apolipoprotein CIII (Apo CIII) from Baseline (Week 0) Over Time in the Main Study Period
    End point description
    Percent change was reported in Apo CIII from baseline (week 0) up to week 16. Apo CIII was measured using conventional units mg/dL. The efficacy analysis set included all subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies the subjects who were evaluable for this endpoint at a given time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 0) to Week 16
    End point values
    REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Number of subjects analysed
    9
    Units: Percent Change
    arithmetic mean (standard deviation)
        Apo CIII: Percent Change at Day 4 (n = 9)
    -46.59 ± 22.129
        Apo CIII: Percent Change at Week 1 (n = 9)
    -55.43 ± 18.541
        Apo CIII: Percent Change at Week 2 (n = 9)
    -56.57 ± 24.815
        Apo CIII: Percent Change at Week 5 (n = 8)
    -75.44 ± 9.667
        Apo CIII: Percent Change at Week 6 (n = 1)
    -91.48 ± 99999
        Apo CIII: Percent Change at Week 12 (n = 9)
    -49.07 ± 25.517
        Apo CIII: Percent Change at Week 14 (n = 9)
    -40.67 ± 44.674
        Apo CIII: Percent Change at Week 16 (n = 9)
    -28.30 ± 54.676
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All adverse events (AEs) were collected from signature of the informed consent form up to the final visit regardless of seriousness or relationship to investigational product.
    Adverse event reporting additional description
    During the main study period, the safety analysis set (SAF) was defined as all enrolled subjects who received at least 1 dose or part of a dose. For OLE, the SAF was defined as all enrolled subjects who received at least 1 dose or part of a dose in the OLE period. The SAF in both study periods was based on the treatment received (as treated).
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    17.0
    Reporting groups
    Reporting group title
    OLE Period: REGN1500 300 mg SC/20 mg/kg IV
    Reporting group description
    Subjects received REGN1500 a SC injection of 300 mg at Week 26 (Day 183), 27(Day 190), 28 (Day 197) and 29 (Day 204) followed by IV injection of 20 mg/kg at Week 38 (Day 267) and every 12 weeks starting at Week 58 (Day 407) through Week 178 (Day 1247) in open-label extension period. Subjects were to be followed for a period of 24 weeks (through Week 214 [Day 1499]) after the last dose of study drug in the OLE treatment period.

    Reporting group title
    Main Study Period: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Reporting group description
    Subjects received single dose of REGN1500 subcutaneous (SC) injection of 250 milligrams (mg) at Week 0 (Day 1), followed by single dose of REGN1500 intravenous (IV) injection of 15 milligrams per kilogram (mg/kg) at Week 2 (Day 15) and then followed by 4 doses of REGN1500 SC injection of 450 mg once weekly starting from Week 12 (Day 85). Only the first 2 enrolled subjects received 4 weekly REGN1500 450 mg SC doses at weeks 12, 13, 14, and 15 per the protocol. This dose regimen was removed under protocol amendment 4. Subjects were followed for a period of 24 weeks (through Week 26 [Day 183]) after the last dose of study drug in the main study period.

    Serious adverse events
    OLE Period: REGN1500 300 mg SC/20 mg/kg IV Main Study Period: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 9 (11.11%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Cardiac disorders
    Coronary artery disease
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Coronary artery stenosis
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Bronchospasm
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    OLE Period: REGN1500 300 mg SC/20 mg/kg IV Main Study Period: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 8 (100.00%)
    9 / 9 (100.00%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Immune system disorders
    Food allergy
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Fatigue
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Chest pain
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Injection site haematoma
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Influenza like illness
         subjects affected / exposed
    2 / 8 (25.00%)
    2 / 9 (22.22%)
         occurrences all number
    2
    3
    Injection site haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Local swelling
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Oedema peripheral
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Crush injury
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Contusion
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Procedural hypotension
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Thermal burn
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    Crystal urine present
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Liver function test abnormal
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Transaminases increased
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Bronchospasm
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Cough
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Epistaxis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Rhinalgia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Upper-Airway cough syndrome
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Headache
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Memory impairment
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Corneal opacity
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Eye inflammation
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Diarrhoea
         subjects affected / exposed
    4 / 8 (50.00%)
    1 / 9 (11.11%)
         occurrences all number
    6
    2
    Faeces discoloured
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Nausea
         subjects affected / exposed
    3 / 8 (37.50%)
    4 / 9 (44.44%)
         occurrences all number
    5
    6
    Vomiting
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Renal and urinary disorders
    Renal failure chronic
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Ecchymosis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Alopecia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Generalised erythema
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Pruritus allergic
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    4 / 8 (50.00%)
    4 / 9 (44.44%)
         occurrences all number
    8
    6
    Muscle spasms
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Musculoskeletal pain
         subjects affected / exposed
    2 / 8 (25.00%)
    2 / 9 (22.22%)
         occurrences all number
    3
    2
    Neck pain
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Myalgia
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Pain in extremity
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    Nasopharyngitis
         subjects affected / exposed
    3 / 8 (37.50%)
    2 / 9 (22.22%)
         occurrences all number
    4
    3
    Influenza
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Pharyngitis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Rhinitis
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Sinusitis
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 9 (11.11%)
         occurrences all number
    2
    2
    Urinary tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 May 2014
    The purpose of this amendment was to include sampling for, and assessment of, low-density lipoprotein receptor (LDLR) function.
    01 Oct 2014
    The purpose of this amendment was to: Allow up to 4 patients who are taking stable, background lomitapide to be enrolled; Allow patients with a known history of HIV to be enrolled. Inclusion of these patients will be dependent on their ability to meet entry criteria for CD4 count, viral load, absence of opportunistic infections or tuberculosis, and stability of their antiretroviral therapy; Require more stringent (double-barrier) contraception; Move the required DNA sample for confirmation of diagnosis of homozygous familial hypercholesterolemia from visit 2 (baseline) to visit 1 (screening); Add review of inclusion/exclusion criteria at visit 2 (baseline); Increase the time after the final date of the clinical study report that sub-study samples may be stored and used for research purposes from up to 10 years to up to 15 years; Remove the 1-day window from visit 2 (baseline); Update exposure data from the ongoing phase 1 study; Clarify the end of treatment and end of study visits for patients who prematurely discontinue from the study; Make minor editorial changes
    24 Feb 2015
    The purpose of this amendment was to: Exclude women of childbearing potential from eligibility in the study, in agreement with the Food and Drug Administration (FDA), based on safety findings from a pilot embryo-fetal toxicity study with REGN1500; Include measurement of follicle-stimulating hormone (FSH) at screening; Remove urine pregnancy testing; Allow for patients to be rescreened on a case-by-case basis; Allow for replacement of patients who discontinued prior to visit 4
    02 Nov 2015
    The purpose of this amendment was to: Remove the 4 weekly 450 mg SC doses; Extend the duration of follow-up/observation period to 24 weeks after the last dose of study drug to ensure patients are monitored for the duration of drug exposure; Allow women of childbearing potential to participate in the study; Update exclusion criteria; Update prohibited and permitted concomitant medications
    14 May 2016
    The purpose of this amendment was to: Add an open-label extension (OLE) period; Update the protocol with new clinical and preclinical data; Revise the PK and ADA variables; Revise the statistical hypothesis and justification of sample size; Increase the number and region of the study sites; Remove the 250 mg/kg subcutaneous (SC) dose from the main period of the study; Include a possible interim analysis; Allow the required DNA sample to be collected at visit 1a or visit 1; Revise the secondary endpoints to reflect the removal of the 4 weekly 450 mg SC doses that was implemented in Amendment 4; Clarify pharmacokinetic (PK) sampling times in the main study period; Clarify anti-drug antibody (ADA) sampling; Update definition of baseline and clarify use of data from unscheduled assessments; Update the safety section of the protocol with the new language in the updated protocol template; State that serum pregnancy testing in the main period of the study will only be performed in women of childbearing potential (WOCBP); Make miscellaneous editorial/formatting revisions

    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.
    At the time patients completed the main study, the open-label extension (OLE) study was not yet open to enrollment. Therefore, there was a lag of between 168 days to 586 days between the end of the main study and the start of the OLE study.
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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