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    Clinical Trial Results:
    A Randomized Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH)

    Summary
    EudraCT number
    		 2014-000972-24
    Trial protocol
    		 SE   NL   BE   DK   DE  
    Global end of trial date
    02 Jul 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Apr 2016
    First version publication date
    07 Apr 2016
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    D3561C00004
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Astra Zeneca R&D
    Sponsor organisation address
    Pepparedsleden 1, Mölndal, Sweden, 43183
    Public contact
    Stefan C. Carlsson, Astra Zeneca R&D, Stefan.Carlsson@astrazeneca.com
    Scientific contact
    Stefan C. Carlsson, Astra Zeneca R&D Mölndal R&D, Stefan.Carlsson@astrazeneca.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    17 Jul 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    02 Jul 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Jul 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to assess the efficacy of rosuvastatin 20 mg on LDL-C, compared to placebo, after 6 weeks of treatment in pediatric HoFH patients
    Protection of trial subjects
    None
    Background therapy
    		 Ezetimibe and apheresis treatments were allowed during the study if initiated before study enrolment
    Evidence for comparator
    		 -
    Actual start date of recruitment
    03 Nov 2014
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 12 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Sweden: 1
    Country: Number of subjects enrolled
    Israel: 3
    Country: Number of subjects enrolled
    Malaysia: 3
    Country: Number of subjects enrolled
    Netherlands: 5
    Country: Number of subjects enrolled
    Taiwan: 4
    Country: Number of subjects enrolled
    Denmark: 1
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Canada: 2
    Worldwide total number of subjects
    20
    EEA total number of subjects
    8
    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)
    7
    Adolescents (12-17 years)
    13
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 20 patients recruited at centers within 8 countries (Belgium, Canada, Denmark, Israel, Malaysia, Sweden, Taiwan, and The Netherlands) that participated in the study. FSI date: 03-Nov-2014.

    Pre-assignment
    Screening details
    		 20 enrolled, 3 withdrew consent, 3 did not fulfil eligibility criteria. Among the 14 left, 10 went through 4 week lead-in phase and 4 did not.

    Period 1
    Period 1 title
    Screening
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Screening
    Arm description
    		 -
    Arm type
    		 Screening

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Screening
    Started
    20
    Completed
    14
    Not completed
    6
         3 consent 3 criteria
    6
    Period 2
    Period 2 title
    Lead-in
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Lead-in
    Arm description
    		 -
    Arm type
    		 Lead-in

    Investigational medicinal product name
    Rosuvastatin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet, Tablet
    Routes of administration
    Oral use, Oral use
    Dosage and administration details
    10mg QED

    Number of subjects in period 2
    		Lead-in
    Started
    14
    Completed
    14
    Period 3
    Period 3 title
    Cross-over
    Is this the baseline period?
    		 Yes [1]
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Arm title
    		 Cross-over
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Rosuvastatin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    20mg QED

    Notes
    [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period.
    Justification: Period 1 is not the baseline period. Rather baseline is defined as the last measurement before the first dose of randomized study drug in the cross-over phase.
    Number of subjects in period 3 [2]
    		Cross-over
    Started
    14
    Completed
    13
    Not completed
    1
         Consent withdrawn by subject
    1
    Notes
    [2] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: The number of subjects reported in the basline period is not the same as the worldwide number enrolled in the trial as 6 of the 20 subjects did not have baseline measurements.
    Period 4
    Period 4 title
    Maintenance
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Maintenance
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Rosuvastatin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    20mg QED

    Number of subjects in period 4
    		Maintenance
    Started
    13
    Completed
    13

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cross-over
    Reporting group description
    		 Safety Analysis Set

    Reporting group values
    		 Cross-over Total
    Number of subjects
    		 14 14
    Age Categorical
    Age Groups are Children (6-9 years) and Adolescents (10-17 years)
    Units: Subjects
        Children (6-9 years)
    		 4 4
        Adolescents (10-17 years)
    		 10 10
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 10.9 ± 2.7 -
    Gender Categorical
    Units: Subjects
        Female
    		 7 7
        Male
    		 7 7
    Subject analysis sets

    Subject analysis set title
    C-FAS PLACEBO
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Cross-over Full Analysis Set, 6 weeks of Placebo

    Subject analysis set title
    C-FAS ROSUVASTATIN
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Cross-over Full Analysis Set, 6 weeks of rosuvastatin

    Subject analysis sets values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects
    13
    13
    Age Categorical
    Age Groups are Children (6-9 years) and Adolescents (10-17 years)
    Units: Subjects
        Children (6-9 years)
    4
    4
        Adolescents (10-17 years)
    9
    9
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    10.9 ± 2.7
    10.9 ± 2.7
    Gender Categorical
    Units: Subjects
        Female
    6
    6
        Male
    7
    7

    End points

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    End points reporting groups
    Reporting group title
    Screening
    Reporting group description
    		 -
    Reporting group title
    Lead-in
    Reporting group description
    		 -
    Reporting group title
    Cross-over
    Reporting group description
    		 -
    Reporting group title
    Maintenance
    Reporting group description
    		 -

    Subject analysis set title
    C-FAS PLACEBO
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Cross-over Full Analysis Set, 6 weeks of Placebo

    Subject analysis set title
    C-FAS ROSUVASTATIN
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Cross-over Full Analysis Set, 6 weeks of rosuvastatin

    Primary: LDL-C during Cross-over phase

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    End point title
    		 LDL-C during Cross-over phase
    End point description
    7 patients had 6 weeks of rosuvastatin followed by 6 weeks of placebo and 6 patients had 6 weeks of placebo followed by 6 weeks of rosuvastatin
    End point type
    Primary
    End point timeframe
    LDL-C
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: mg/dL
    number (standard deviation)
        mg/dL
    481.4
    396
        mmol/L
    12.47
    10.26
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log LDL-C at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.005
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -22.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -33.5
         upper limit
    		 -9.1

    Secondary: LDL-C during Cross-over phase, non-apheresis

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    End point title
    		 LDL-C during Cross-over phase, non-apheresis
    End point description
    Efficacy in terms of low density lipoprotein cholesterol (LDL C) following 6 weeks rosuvastatin 20 mg or placebo treatment in patients not treated with Apheresis
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    6
    6
    Units: mg/dL
    arithmetic mean (standard deviation)
        mg/dL
    594.7 ± 203.6
    479.8 ± 239.07
        mmol/L
    15.4 ± 5.274
    12.43 ± 6.191
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log LDL-C at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.08
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -26.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -48.7
         upper limit
    		 6

    Secondary: HDL-C

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    End point title
    		 HDL-C
    End point description
    Efficacy in terms of high density lipoprotein cholesterol (HDL C)
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: mg/dL
    arithmetic mean (standard deviation)
        mg/dL
    33.7 ± 8.47
    35.5 ± 7.29
        mmol/L
    0.87 ± 0.218
    0.92 ± 0.189
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log HDL-C at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.314
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    7.4
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.4
         upper limit
    		 24.5

    Secondary: Triglycerides during Cross-over phase

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    End point title
    		 Triglycerides during Cross-over phase
    End point description
    Efficacy in terms of triglycerides (TG)
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: mg/dL
    arithmetic mean (standard deviation)
        mg/dL
    119.5 ± 52.67
    79.8 ± 24.48
        mmol/L
    1.35 ± 0.595
    0.9 ± 0.277
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log triglycerides at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.004
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -30.4
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -44.2
         upper limit
    		 -13.3

    Secondary: LDL-C/HDL-C during Cross-over phase

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    End point title
    		 LDL-C/HDL-C during Cross-over phase
    End point description
    Efficacy in terms of low density lipoprotein cholesterol (LDL C) / high density lipoprotein cholesterol (HDL C)
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: N/A
        arithmetic mean (standard deviation)
    15.6 ± 9.1317
    12.208 ± 7.8638
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log LDL-C/HDL-C at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.006
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -27.6
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -41.2
         upper limit
    		 -11

    Secondary: TC/HDL-C during Cross-over phase

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    End point title
    		 TC/HDL-C during Cross-over phase
    End point description
    Efficacy in terms of total cholesterol (TC) / high density lipoprotein cholesterol (HDL C)
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: N/A
        arithmetic mean (standard deviation)
    17.416 ± 9.5454
    13.704 ± 8.0414
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log TC/HDL-C at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.005
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -25.6
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -38.1
         upper limit
    		 -10.5

    Secondary: Non-HDL-C/HDL-C during Cross-over phase

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    End point title
    		 Non-HDL-C/HDL-C during Cross-over phase
    End point description
    Efficacy in terms of non-high density lipoprotein cholesterol (non-HDL C) / HDL C
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: N/A
        arithmetic mean (standard deviation)
    16.416 ± 9.5454
    12.704 ± 8.0414
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log Non-HDL-C/HDL-C at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.005
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -28.2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -41.7
         upper limit
    		 -11.4

    Secondary: Apo A-1 during Cross-over phase

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    End point title
    		 Apo A-1 during Cross-over phase
    End point description
    Efficacy in terms of apolipoprotein A-1 (Apo A-1)
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: mg/dL
    arithmetic mean (standard deviation)
        mg/dL
    100 ± 17.65
    103.5 ± 17.28
        g/L
    1 ± 0.177
    1.03 ± 0.173
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log Apo A-1 at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.383
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    4.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -5.5
         upper limit
    		 14.6

    Secondary: Apo B/Apo A-1 during Cross-over phase

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    End point title
    		 Apo B/Apo A-1 during Cross-over phase
    End point description
    Efficacy in terms of apolipoprotein B (Apo B) / apolipoprotein A-1 (Apo A-1)
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: N/A
        arithmetic mean (standard deviation)
    2.873 ± 1.4669
    2.408 ± 1.3259
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log Apo B/Apo A-1 at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.013
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -20.4
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -32.8
         upper limit
    		 -5.6

    Secondary: LDL-C from End of Placebo Period to End of Study

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    End point title
    		 LDL-C from End of Placebo Period to End of Study
    End point description
    Change in low density lipoprotein cholesterol (LDL C) from end of placebo period to 6, 12, and 18 weeks of therapy with rosuvastatin 20 mg
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6), Day 84 (week 12), Day 126 (week 18) and Day 168 (week 24)
    End point values
    		 Cross-over
    Number of subjects analysed
    13
    Units: mg/dL
    arithmetic mean (standard deviation)
        LDL-C eop (n=13) mg/dL
    481.4 ± 184.9
        LDL-C 6 wks after eop (n=13) mg/dL
    409.7 ± 209.97
        LDL-C 12 wks after eop (n=11) mg/dL
    372.5 ± 187.11
        LDL-C 18 wks after eop (n=5) mg/dL
    441.8 ± 260.31
        LDL-C eop (n=13) mmol/L
    12.47 ± 4.79
        LDL-C 6 wks after eop (n=13) mmol/L
    10.61 ± 5.438
        LDL-C 12 wks after eop (n=11) mmol/L
    9.65 ± 4.845
        LDL-C 18 wks after eop (n=5) mmol/L
    11.44 ± 6.74
    No statistical analyses for this end point

    Secondary: Rosuvastatin Trough Concentrations

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    End point title
    		 Rosuvastatin Trough Concentrations
    End point description
    Pharmacokinetic profile in terms of trough concentrations
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6), Day 84 (week 12), Day 126 (week 18)
    End point values
    		 Cross-over Maintenance
    Number of subjects analysed
    11 [1]
    13 [2]
    Units: ng/mL
    arithmetic mean (standard deviation)
        ng/mL
    7.387 ± 8.11
    4.482 ± 3.5
    Notes
    [1] - Measurement taken after 6 weeks active treatment (rosuvastatin) in cross-over phase
    [2] - Measurement taken after 6 weeks active treatment (rosuvastatin) in maintenance phase
    No statistical analyses for this end point

    Secondary: Total Cholesterol during Cross-over phase

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    End point title
    		 Total Cholesterol during Cross-over phase
    End point description
    Efficacy in terms of total cholesterol (TC)
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: mg/dL
    arithmetic mean (standard deviation)
        mg/dL
    539 ± 184.91
    447.6 ± 195.46
        mmol/L
    13.96 ± 4.79
    11.59 ± 5.063
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log TC at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.003
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -20.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -29.7
         upper limit
    		 -9.1

    Secondary: Non-HDL-C during Cross-over phase

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    End point title
    		 Non-HDL-C during Cross-over phase
    End point description
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: mg/dL
    arithmetic mean (standard deviation)
        mg/dL
    505.3 ± 186.39
    412.1 ± 198.62
        mmol/L
    13.09 ± 4.826
    10.67 ± 5.144
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log Non-HDL-C at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.003
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative Diff in geometric LS means
    Point estimate
    -22.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -33.7
         upper limit
    		 -10.3

    Secondary: Apo B during Cross-over phase

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    End point title
    		 Apo B during Cross-over phase
    End point description
    End point type
    Secondary
    End point timeframe
    Samples taken at Day 42 (week 6) and Day 84 (week 12)
    End point values
    		 C-FAS PLACEBO C-FAS ROSUVASTATIN
    Number of subjects analysed
    13
    13
    Units: mg/dL
    arithmetic mean (standard deviation)
        mg/dL
    267.9 ± 86.33
    234.9 ± 107.02
        g/L
    2.68 ± 0.863
    2.35 ± 1.07
    Statistical analysis title
    		 Mixed model analysis
    Statistical analysis description
    The repsonse was log Apo B at 6 weeks and 12 weeks of the Cross-over phase and the fixed factors where treatment and period. Subject/patient was a random factor
    Comparison groups
    C-FAS PLACEBO v C-FAS ROSUVASTATIN
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.024
    Method
    		 Mixed models analysis
    Parameter type
    		 Relative diff in geometric LS means
    Point estimate
    -17.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -29.2
         upper limit
    		 -2.9

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs will be collected from the time of signature of informed consent throughout the efficacy maintenance phase and including the follow-up of unresolved adverse events; SAEs will be recorded up to 30 days after the last dose of study drug
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Cross-over
    Reporting group description
    		 -

    Reporting group title
    Lead-in
    Reporting group description
    		 Optional Lead-in phase

    Reporting group title
    Maintenance
    Reporting group description
    		 Maintenance phase

    Reporting group title
    Screening
    Reporting group description
    		 -

    Serious adverse events
    		 Cross-over Lead-in Maintenance Screening
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    1 / 20 (5.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Nervous system disorders
    presyncope
    Additional description: Several presyncope episodes
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Cross-over Lead-in Maintenance Screening
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 14 (28.57%)
    3 / 11 (27.27%)
    1 / 13 (7.69%)
    0 / 20 (0.00%)
    Investigations
    Blood bicarbonate decreased
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 11 (0.00%)
    1 / 13 (7.69%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    1
    0
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    2 / 14 (14.29%)
    2 / 11 (18.18%)
    0 / 13 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    2
    0
    0
    Peripheral swelling
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Chest pain
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 11 (9.09%)
    0 / 13 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Infections and infestations
    Influenza
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 11 (0.00%)
    0 / 13 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 Jul 2014
    1) Patients entering the study on 20 mg or more of rosuvastatin are permitted to receive rosuvastatin 20 mg during the lead-in phase of the study. 2) Patients entering the study on 10 mg or more of rosuvastatin who do not require washout of any dyslipidemia medication, are permitted to forego the lead-in phase and proceed directly to the cross-over phase of the study. 3) PK samples will be drawn at Visits 4, 5, and 6, instead of Visits 6 and 7. 4) Temperature is not required to be taken orally – i.e., it will be permitted to be taken by any route (oral, ear, axillary, rectal).
    08 Dec 2014
    1) Statistical updates were made regarding the primary and secondary analyses and the addition of subgroup analyses. 2) “or currently lactating” was added to Exclusion Criterion #21. 3) An assessment of urinary albumin/creatinine ratio was added to the procedures identified in the study plan (Table 1) and to Table 2 for the urinalysis laboratory variables. Additionally, the estimated glomerular filtration rate by Schwartz formula was added to the clinical chemistry assessments listed in Table 2. 4) Text was added regarding the management of repeat/unscheduled laboratories. 5) Text regarding laboratory values outside the reference limits was modified. 6) Appendix E was updated to reflect lab management changes; additional details were added to provide guidance on the actions that should occur if creatine kinase (CK) was found to be >10xULN, and for CK 5-10xULN.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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