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    Clinical Trial Results:
    A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia

    Summary
    EudraCT number
    		 2010-023742-79
    Trial protocol
    		 IT  
    Global end of trial date
    01 Dec 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Apr 2018
    First version publication date
    26 Apr 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AEG 733-012
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00943306
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Aegerion Pharmaceuticals, Inc.
    Sponsor organisation address
    One Main Street, Suite 800, Cambridge, United States, 02142
    Public contact
    Agnieszka Jurecka, MD, PhD, Aegerion Pharmaceuticals, +1 857-242-5140, agnieszka.jurecka@aegerion.com
    Scientific contact
    Agnieszka Jurecka, MD, PhD, Aegerion Pharmaceuticals, +1 857-242-5140, agnieszka.jurecka@aegerion.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
    01 Dec 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Dec 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to evaluate the efficacy of lomitapide, as defined by percent change in LDL-C at the maximum tolerated dose at Week 48 compared to baseline, in combination with other lipid lowering therapy in patients with homozygous familial hypercholesterolemia (FH) who completed study UP1002 or 733-005.
    Protection of trial subjects
    Although patients entered this study with a defined personal MTD of lomitapide (based on the MTD established during the first 26 weeks of treatment on Study UP1002/AEGR-733-005), a dose modification schedule was used if the patient required dose reduction in response to an AE or other factor
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    17 Sep 2009
    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
    Italy: 5
    Country: Number of subjects enrolled
    United States: 3
    Country: Number of subjects enrolled
    Canada: 2
    Country: Number of subjects enrolled
    South Africa: 9
    Worldwide total number of subjects
    19
    EEA total number of subjects
    5
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    19
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Patients were eligible for inclusion in the study if they completed Study UP1002/AEGR-733-005 and were willing and able to provide consent and comply with local requirements of the study protocol.

    Pre-assignment
    Screening details
    		 Patients who met any of the stopping rules for study discontinuation at the final Study of UP 1002/AEGR-733-005 were excluded from the study.

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

    Arms
    Arm title
    		 All patients
    Arm description
    		 Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lomitapide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Lomitapide mesylate capsules, 5 mg or 20 mg, were administered orally once per day. During Study UP1002/AEGR-733-005, the dose was initiated at 5 mg/day for 2 weeks and escalated at 4-week intervals (as tolerated) to 60 mg/day. Patients’ specific doses for AEGR-733-012 were carried forward from UP1002/AEGR-733-005, but did not exceed the MTD that patients received during the original study.

    Number of subjects in period 1
    		All patients
    Started
    19
    Completed
    16
    Not completed
    3
         Adverse event, serious fatal
    1
         Physician decision
    1
         Sponsor decision due to non-compliance
    1

    Baseline characteristics

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

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 19 19
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 19 19
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 30.4 ± 11.74 -
    Gender categorical
    Units: Subjects
        Female
    		 9 9
        Male
    		 10 10

    End points

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    End points reporting groups
    Reporting group title
    All patients
    Reporting group description
    		 Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

    Subject analysis set title
    Week 126 Completers Population
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    17 of 19 patients were included in the Week 126 Completers Population. One patient was terminated from treatment prior to Week 126 due to investigator decision. One patient continued treatment beyond Week 126 but did not have the visit conducted as scheduled (the patient temporarily discontinued from treatment due to a planned pregnancy; following delivery of a healthy baby, she returned to the study and completed treatment).

    Subject analysis set title
    Safety Population
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Safety population includes all patients who took at least one dose of study drug.

    Primary: Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 126

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    End point title
    		 Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 126 [1]
    End point description
    Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Primary
    End point timeframe
    Baseline and Week 126
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Descriptive statistics [arithmetic mean, arithmetic standard deviation, median, minimum (min), maximum (max), and number of subjects (N)] will be used to summarize results for the primary efficacy endpoint LDL-C
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -45.5 ± 31.35
    No statistical analyses for this end point

    Secondary: Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 174

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    End point title
    		 Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 174
    End point description
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    -51.0 ± 16.03
    No statistical analyses for this end point

    Secondary: Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 222

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    End point title
    		 Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 222
    End point description
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -58.5 ± 24.25
    No statistical analyses for this end point

    Secondary: Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 246

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    End point title
    		 Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 246
    End point description
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -60.1 ± 18.51
    No statistical analyses for this end point

    Secondary: Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 270

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    End point title
    		 Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 270
    End point description
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -74.0 ± 19.10
    No statistical analyses for this end point

    Secondary: Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 294

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    End point title
    		 Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) - Week 294
    End point description
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -51.1 ± 10.11
    No statistical analyses for this end point

    Secondary: Percent Change in Total Cholesterol - Week 126

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    End point title
    		 Percent Change in Total Cholesterol - Week 126
    End point description
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -43.2 ± 25.35
    No statistical analyses for this end point

    Secondary: Percent Change in Total Cholesterol - Week 174

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    End point title
    		 Percent Change in Total Cholesterol - Week 174
    End point description
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    -46.9 ± 15.21
    No statistical analyses for this end point

    Secondary: Percent Change in Total Cholesterol - Week 222

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    End point title
    		 Percent Change in Total Cholesterol - Week 222
    End point description
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -51.0 ± 21.34
    No statistical analyses for this end point

    Secondary: Percent Change in Total Cholesterol - Week 246

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    End point title
    		 Percent Change in Total Cholesterol - Week 246
    End point description
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -54.1 ± 16.88
    No statistical analyses for this end point

    Secondary: Percent Change in Total Cholesterol - Week 270

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    End point title
    		 Percent Change in Total Cholesterol - Week 270
    End point description
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -65.2 ± 15.97
    No statistical analyses for this end point

    Secondary: Percent Change in Total Cholesterol - Week 294

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    End point title
    		 Percent Change in Total Cholesterol - Week 294
    End point description
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -43.9 ± 5.01
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein B (Apo B) - Week 126

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    End point title
    		 Percent Change in Apolipoprotein B (Apo B) - Week 126
    End point description
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -53.6 ± 23.74
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein B (Apo B) - Week 174

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    End point title
    		 Percent Change in Apolipoprotein B (Apo B) - Week 174
    End point description
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    -59.4 ± 12.60
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein B (Apo B) - Week 222

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    End point title
    		 Percent Change in Apolipoprotein B (Apo B) - Week 222
    End point description
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -65.1 ± 20.72
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein B (Apo B) - Week 246

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    End point title
    		 Percent Change in Apolipoprotein B (Apo B) - Week 246
    End point description
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -65.9 ± 15.76
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein B (Apo B) - Week 270

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    End point title
    		 Percent Change in Apolipoprotein B (Apo B) - Week 270
    End point description
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -76.7 ± 16.09
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein B (Apo B) - Week 294

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    End point title
    		 Percent Change in Apolipoprotein B (Apo B) - Week 294
    End point description
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -60.9 ± 12.17
    No statistical analyses for this end point

    Secondary: Percent Change in Triglycerides - Week 126

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    End point title
    		 Percent Change in Triglycerides - Week 126
    End point description
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -37.5 ± 42.52
    No statistical analyses for this end point

    Secondary: Percent Change in Triglycerides - Week 174

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    End point title
    		 Percent Change in Triglycerides - Week 174
    End point description
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    -31.7 ± 37.09
    No statistical analyses for this end point

    Secondary: Percent Change in Triglycerides - Week 222

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    End point title
    		 Percent Change in Triglycerides - Week 222
    End point description
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -27.6 ± 48.88
    No statistical analyses for this end point

    Secondary: Percent Change in Triglycerides - Week 246

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    End point title
    		 Percent Change in Triglycerides - Week 246
    End point description
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -41.9 ± 33.16
    No statistical analyses for this end point

    Secondary: Percent Change in Triglycerides - Week 270

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    End point title
    		 Percent Change in Triglycerides - Week 270
    End point description
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -48.5 ± 34.24
    No statistical analyses for this end point

    Secondary: Percent Change in Triglycerides - Week 294

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    End point title
    		 Percent Change in Triglycerides - Week 294
    End point description
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    29.2 ± 58.74
    No statistical analyses for this end point

    Secondary: Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 126

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    End point title
    		 Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 126
    End point description
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -47.1 ± 27.83
    No statistical analyses for this end point

    Secondary: Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 174

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    End point title
    		 Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 174
    End point description
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    -53.5 ± 16.42
    No statistical analyses for this end point

    Secondary: Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 222

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    End point title
    		 Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 222
    End point description
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -57.0 ± 24.07
    No statistical analyses for this end point

    Secondary: Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 246

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    End point title
    		 Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 246
    End point description
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -58.8 ± 17.73
    No statistical analyses for this end point

    Secondary: Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 270

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    End point title
    		 Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 270
    End point description
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -71.5 ± 17.65
    No statistical analyses for this end point

    Secondary: Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 294

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    End point title
    		 Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) - Week 294
    End point description
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -46.6 ± 5.19
    No statistical analyses for this end point

    Secondary: Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 126

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    End point title
    		 Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 126
    End point description
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -36.8 ± 43.90
    No statistical analyses for this end point

    Secondary: Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 174

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    End point title
    		 Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 174
    End point description
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    -31.5 ± 36.32
    No statistical analyses for this end point

    Secondary: Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 222

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    End point title
    		 Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 222
    End point description
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -26.3 ± 49.94
    No statistical analyses for this end point

    Secondary: Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 246

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    End point title
    		 Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 246
    End point description
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -41.4 ± 34.20
    No statistical analyses for this end point

    Secondary: Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 270

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    End point title
    		 Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 270
    End point description
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -48.7 ± 33.41
    No statistical analyses for this end point

    Secondary: Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 294

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    End point title
    		 Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) - Week 294
    End point description
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -30.6 ± 59.94
    No statistical analyses for this end point

    Secondary: Percent Change in Lp(a) - Week 126

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    End point title
    		 Percent Change in Lp(a) - Week 126
    End point description
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    5.5 ± 43.62
    No statistical analyses for this end point

    Secondary: Percent Change in Lp(a) - Week 174

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    End point title
    		 Percent Change in Lp(a) - Week 174
    End point description
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    10.2 ± 60.64
    No statistical analyses for this end point

    Secondary: Percent Change in Lp(a) - Week 222

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    End point title
    		 Percent Change in Lp(a) - Week 222
    End point description
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -12.8 ± 49.00
    No statistical analyses for this end point

    Secondary: Percent Change in Lp(a) - Week 246

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    End point title
    		 Percent Change in Lp(a) - Week 246
    End point description
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    13
    Units: percent change
        arithmetic mean (standard deviation)
    3.4 ± 54.22
    No statistical analyses for this end point

    Secondary: Percent Change in Lp(a) - Week 270

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    End point title
    		 Percent Change in Lp(a) - Week 270
    End point description
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -6.6 ± 48.99
    No statistical analyses for this end point

    Secondary: Percent Change in Lp(a) - Week 294

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    End point title
    		 Percent Change in Lp(a) - Week 294
    End point description
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -10.4 ± 35.64
    No statistical analyses for this end point

    Secondary: Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 126

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    End point title
    		 Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 126
    End point description
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -8.3 ± 19.28
    No statistical analyses for this end point

    Secondary: Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 174

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    End point title
    		 Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 174
    End point description
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    3.8 ± 26.51
    No statistical analyses for this end point

    Secondary: Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 222

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    End point title
    		 Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 222
    End point description
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -2.7 ± 21.22
    No statistical analyses for this end point

    Secondary: Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 246

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    End point title
    		 Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 246
    End point description
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    14
    Units: percent change
        arithmetic mean (standard deviation)
    -12.5 ± 19.17
    No statistical analyses for this end point

    Secondary: Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 270

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    End point title
    		 Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 270
    End point description
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -10.3 ± 27.68
    No statistical analyses for this end point

    Secondary: Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 294

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    End point title
    		 Percent Change in High Density Lipoprotein Cholesterol (HDL-C) - Week 294
    End point description
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -23.5 ± 2.27
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein AI (Apo AI) - Week 126

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    End point title
    		 Percent Change in Apolipoprotein AI (Apo AI) - Week 126
    End point description
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) and Week 126 (Week 48 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 126
    End point values
    		 Week 126 Completers Population
    Number of subjects analysed
    17
    Units: percent change
        arithmetic mean (standard deviation)
    -14.0 ± 17.71
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein AI (Apo AI) - Week 174

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    End point title
    		 Percent Change in Apolipoprotein AI (Apo AI) - Week 174
    End point description
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) and Week 174 (Week 96 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 174
    End point values
    		 Safety Population
    Number of subjects analysed
    16
    Units: percent change
        arithmetic mean (standard deviation)
    -8.2 ± 20.12
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein AI (Apo AI) - Week 222

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    End point title
    		 Percent Change in Apolipoprotein AI (Apo AI) - Week 222
    End point description
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) and Week 222 (Week 144 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 222
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -2.7 ± 33.30
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein AI (Apo AI) - Week 246

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    End point title
    		 Percent Change in Apolipoprotein AI (Apo AI) - Week 246
    End point description
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) and Week 246 (Week 168 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 246
    End point values
    		 Safety Population
    Number of subjects analysed
    15
    Units: percent change
        arithmetic mean (standard deviation)
    -16.8 ± 26.62
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein AI (Apo AI) - Week 270

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    End point title
    		 Percent Change in Apolipoprotein AI (Apo AI) - Week 270
    End point description
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) and Week 270 (Week 192 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 270
    End point values
    		 Safety Population
    Number of subjects analysed
    9
    Units: percent change
        arithmetic mean (standard deviation)
    -17.8 ± 20.12
    No statistical analyses for this end point

    Secondary: Percent Change in Apolipoprotein AI (Apo AI) - Week 294

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    End point title
    		 Percent Change in Apolipoprotein AI (Apo AI) - Week 294
    End point description
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) and Week 294 (Week 216 in Study AEGR-733-012).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 294
    End point values
    		 Safety Population
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    -30.5 ± 22.2
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    11.0
    Reporting groups
    Reporting group title
    All patients
    Reporting group description
    		 Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

    Serious adverse events
    		 All patients
    Total subjects affected by serious adverse events
         subjects affected / exposed
    7 / 19 (36.84%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    1
    Investigations
    International normalised ratio increased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Subdural haematoma
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Arteriovenous fistula
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypovolaemic shock
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Aortic valve incompetence
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Coronary artery disease
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Surgical and medical procedures
    Anticoagulant therapy
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Transfusion
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Facial palsy
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Sudden cardiac death
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reflux oesophagitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Hepatotoxicity
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Rhabdomyolysis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 All patients
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    17 / 19 (89.47%)
    Vascular disorders
    Arterial stenosis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Arteriovenous fistula
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Haematoma
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Hypertension
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Hypotension
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Hypovolaemic shock
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Surgical and medical procedures
    Anticoagulant therapy
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Transfusion
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Chest pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Fatigue
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Oedema peripheral
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Sudden cardiac death
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Vestibulitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    3
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Apnoea
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Cough
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Dyspnoea
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Dyspnoea exertional
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Epistaxis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Painful respiration
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Pharyngolaryngeal pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Sinus congestion
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    4
    Depression
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    6
    Stress
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    7
    Aspartate aminotransferase increased
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    4
    Blood potassium increased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Blood pressure increased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Carotid bruit
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    5
    International normalised ratio decreased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    International normalised ratio increased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Liver function test abnormal
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Prothrombin time prolonged
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Transaminases increased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    5
    Vitamin K decreased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Weight decreased
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    White blood cell count decreased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Joint injury
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    3
    Post procedural diarrhoea
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Procedural headache
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Skeletal injury
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Subdural haematoma
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    4 / 19 (21.05%)
         occurrences all number
    12
    Aortic valve incompetence
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Chest pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Coronary artery disease
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Sinus bradycardia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    4
    Facial Palsy
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Headache
         subjects affected / exposed
    6 / 19 (31.58%)
         occurrences all number
    10
    Hypoaesthesia
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Migraine
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Paraesthesia
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    8
    Sensory disturbance
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Syncope
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    3
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Vertigo
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Eye disorders
    Blepharitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    4
    Chapped lips
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Dental caries
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    8 / 19 (42.11%)
         occurrences all number
    25
    Dyspepsia
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Epigastric discomfort
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    4
    Flatulence
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    4
    Gingival bleeding
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Haemorrhoidal haemorrhage
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Hiatus hernia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Intestinal mass
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    3
    Nausea
         subjects affected / exposed
    6 / 19 (31.58%)
         occurrences all number
    17
    Reflux oesophagitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    stomach discomfort
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Toothache
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    5 / 19 (26.32%)
         occurrences all number
    9
    Hepatobiliary disorders
    Hepatotoxicity
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Dry skin
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Hair growth abnormal
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Scar
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    4
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    3
    Back pain
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    3
    Musculoskeletal pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Neck pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Rhabdomyolysis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Tendonitis
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    4
    Gastroenteritis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    4
    Gastrointestinal infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    5 / 19 (26.32%)
         occurrences all number
    8
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    4 / 19 (21.05%)
         occurrences all number
    11
    Sinusitis
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    3
    Tooth abscess
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Urinary tract infection
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Viral infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Carotene decreased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Dehydration
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Iron deficiency
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    4
    Oral intake reduced
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Vitamin E deficiency
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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