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    Clinical Trial Results:
    A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome

    Summary
    EudraCT number
    		 2011-005535-68
    Trial protocol
    		 DE   NL   GB   ES  
    Global end of trial date
    28 May 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Jul 2016
    First version publication date
    27 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CLCQ908B2302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01514461
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111,
    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
    28 May 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    28 May 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this clinical trial was to demonstrate the superiority of LCQ908 20 mg or LCQ908 40 mg compared to placebo in reducing fasting triglycerides after 12 weeks of treatment in subjects with FCS.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    05 Jul 2012
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 16
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Netherlands: 10
    Country: Number of subjects enrolled
    South Africa: 3
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    United States: 4
    Worldwide total number of subjects
    45
    EEA total number of subjects
    22
    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)
    42
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Among 63 screened patients, 45 (71.4%) patients completed the screening/dietary lead-in phase and got randomized.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet, once daily. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Matching placebo of LCQ908 10 mg, 20 mg and 40 mg

    Arm title
    		 LCQ908 20 mg
    Arm description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 10 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 20 mg tablets, once daily. A low fat diet was followed and recorded in patient diary.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Matching placebo of LCQ908 10 mg, 20 mg and 40 mg

    Investigational medicinal product name
    pradigastat
    Investigational medicinal product code
    LCQ908
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    LCQ908 20 mg taken once daily

    Arm title
    		 LCQ908 40 mg
    Arm description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 40 mg active tablet + one LCQ908 placebo matching to 20mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Matching placebo of LCQ908 10 mg, 20 mg and 40 mg

    Investigational medicinal product name
    pradigastat
    Investigational medicinal product code
    LCQ908
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    LCQ908 20 mg taken once daily

    Number of subjects in period 1
    		Placebo LCQ908 20 mg LCQ908 40 mg
    Started
    15
    15
    15
    Completed
    10
    12
    11
    Not completed
    5
    3
    4
         Adverse event, serious fatal
    1
    -
    -
         Physician decision
    1
    1
    -
         Adverse event, non-fatal
    2
    1
    3
         Patient/guardian decision
    1
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet, once daily. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Reporting group title
    LCQ908 20 mg
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 10 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 20 mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Reporting group title
    LCQ908 40 mg
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 40 mg active tablet + one LCQ908 placebo matching to 20mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Reporting group values
    		 Placebo LCQ908 20 mg LCQ908 40 mg Total
    Number of subjects
    		 15 15 15 45
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 12 15 15 42
        From 65-84 years
    		 3 0 0 3
    Age Continuous |
    Units: Years
        arithmetic mean (standard deviation)
    		 52.9 ( 10.9 ) 42.3 ( 13.61 ) 42.7 ( 10.94 ) -
    Gender, Male/Female
    Units: Participants
        Female
    		 8 7 4 19
        Male
    		 7 8 11 26

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet, once daily. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Reporting group title
    LCQ908 20 mg
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 10 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 20 mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Reporting group title
    LCQ908 40 mg
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 40 mg active tablet + one LCQ908 placebo matching to 20mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Primary: Percent change in fasting triglycerides from baseline to 12 weeks

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    End point title
    		 Percent change in fasting triglycerides from baseline to 12 weeks
    End point description
    Blood samples were collected for a fasting lipid panel, including triglycerides. If the 12-week value was missing, the measurement value at 12 weeks or the last available post-baseline measurement value during the double-blind treatment period was analyzed. Baseline is defined as the average of fasting triglyceride values taken at day -3 and day 1. Adjusted geometric means are calculated by back-transforming the adjusted means from the model and expressing as a percentage change from baseline.
    End point type
    Primary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    14
    14
    12
    Units: percent change
        geometric mean (confidence interval)
    45.6 (6.8 to 98.7)
    3.7 (-24.3 to 42.1)
    -13.9 (-38.9 to 21.3)
    Statistical analysis title
    		 LCQ908 20 mg Vs Placebo
    Comparison groups
    Placebo v LCQ908 20 mg
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 equivalence
    P-value
    		 = 0.0538 [1]
    Method
    		 Mixed models analysis
    Parameter type
    		 % change from reference treatment
    Point estimate
    -28.78
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -55.69
         upper limit
    		 14.46
    Notes
    [1] - Adjusted 1-sided p-value calculated using Dunnett's step down method.
    Statistical analysis title
    		 LCQ908 40mg Vs Placebo
    Comparison groups
    Placebo v LCQ908 40 mg
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 equivalence
    P-value
    		 = 0.0182 [2]
    Method
    		 Mixed models analysis
    Parameter type
    		 % change from reference treatment
    Point estimate
    -40.88
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -63.99
         upper limit
    		 -2.94
    Notes
    [2] - Adjusted 1-sided p-value calculated using Dunnett's step down method.

    Secondary: Percentage of patients responding to investigational treatment by achieving fasting triglycerides (TG) of at least 40% from baseline or final fasting TG < 8.4 mmol/L (750 mg/dL)

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    End point title
    		 Percentage of patients responding to investigational treatment by achieving fasting triglycerides (TG) of at least 40% from baseline or final fasting TG < 8.4 mmol/L (750 mg/dL)
    End point description
    Percentage calculated as (m/n)*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride.
    End point type
    Secondary
    End point timeframe
    Baseline, 12 weeks, 24 weeks, 52 weeks
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    15
    15
    15
    Units: Percentage of participants
    number (not applicable)
        Week 12 (n = 14, 14, 12)
    14.3
    21.4
    50
        Week 24 (n = 13, 14, 12)
    30.8
    35.7
    33.3
        Week 52 (n = 11, 14, 11)
    18.2
    21.4
    27.3
    No statistical analyses for this end point

    Secondary: Percentage of patients responding to investigational treatment by achieving final fasting triglycerides < 8.4 mmol/L (750 mg/dL)

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    End point title
    		 Percentage of patients responding to investigational treatment by achieving final fasting triglycerides < 8.4 mmol/L (750 mg/dL)
    End point description
    Percentage calculated as (m/n)*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride.
    End point type
    Secondary
    End point timeframe
    12 weeks, 24 weeks, 52 weeks
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    15
    15
    15
    Units: Percentage of participants
    number (not applicable)
        Week 12 (n = 14, 14, 12)
    14.3
    14.3
    33.3
        Week 24 (n = 13, 14, 12)
    30.8
    14.3
    16.7
        Week 52 (n = 11, 14, 11)
    18.2
    14.3
    18.2
    No statistical analyses for this end point

    Secondary: Percentage of patients responding to investigational treatment by achieving fasting triglycerides (TG) of at least 40% from baseline

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    End point title
    		 Percentage of patients responding to investigational treatment by achieving fasting triglycerides (TG) of at least 40% from baseline
    End point description
    Percentage calculated as (m/n)*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride.
    End point type
    Secondary
    End point timeframe
    Baseline, 12 weeks, 24 weeks, 52 weeks
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    15
    15
    15
    Units: Percentage of participants
    number (not applicable)
        Week 12 (n = 14, 14, 12)
    0
    14.3
    25
        Week 24 (n = 13, 14, 12)
    15.4
    28.6
    16.7
        Week 52 (n = 11, 14, 11)
    0
    14.3
    27.3
    No statistical analyses for this end point

    Secondary: Percentage of patients achieving fasting triglycerides (TG) target thresholds

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    End point title
    		 Percentage of patients achieving fasting triglycerides (TG) target thresholds
    End point description
    Percentage of patients reaching target values of <1000 mg/dL or target values of < 2000 mg/dL for fasting triglycerides is reported. Pecentage calculated as (m/n)*100; where 'm' The number of patients who reach target values for fasting triglyceride, 'n' the number of patients with non-missing fasting triglyceride.
    End point type
    Secondary
    End point timeframe
    12 weeks, 24 weeks, 52 weeks
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    15
    15
    15
    Units: Percentage of patients
    number (not applicable)
        TG < 1000 mg/dL, week 12 (n=14,14,12)
    14.3
    21.4
    33.3
        TG < 1000 mg/dL, week 24 (n=13,14,12)
    30.8
    21.4
    25
        TG < 1000 mg/dL, week 52 (n=11,14,11)
    27.3
    14.3
    36.4
        TG < 2000 mg/dL, week 12 (n=14,14,12)
    35.7
    50
    83.3
        TG < 2000 mg/dL, week 24 (n=13,14,12)
    38.5
    57.1
    58.3
        TG < 2000 mg/dL, week 52 (n=11,14,11)
    36.4
    50
    63.6
    No statistical analyses for this end point

    Secondary: Percent change from baseline in fasting triglycerides

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    End point title
    		 Percent change from baseline in fasting triglycerides
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline, 24 weeks, 52 weeks
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    15
    15
    15
    Units: Percent change
    geometric mean (confidence interval)
        Week 24 (n=13, 14, 12)
    4.9 (-26.6 to 50.1)
    -15.8 (-40.5 to 19.3)
    5.5 (-28 to 54.5)
        Week 52 (n=11, 14, 11)
    15.2 (-23 to 72.5)
    -6.7 (-36.4 to 36.9)
    4.9 (-31.2 to 60)
    No statistical analyses for this end point

    Secondary: Percent change from baseline for postprandial triglycerides following the standardized meal tolerance test at Week 12

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    End point title
    		 Percent change from baseline for postprandial triglycerides following the standardized meal tolerance test at Week 12
    End point description
    Post prandial peak triglycerides – maximum triglyceride value over 0-24 hours Post prandial triglycerides AUC0-24 – area under the time curve for triglycerides over 0-24 Adjusted geometric means are calculated by back-transforming the adjusted means from the model and expressed as a percentage change from baseline. hours
    End point type
    Secondary
    End point timeframe
    0-24 hours at Baseline, Week 12
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    15
    15
    15
    Units: Percent change
    geometric mean (confidence interval)
        Triglycerides (Peak 0-24h) [n=12, 12, 11)
    56.9 (6.9 to 130.2)
    8.6 (-24.8 to 56.8)
    6.3 (-30.3 to 62.2)
        Triglycerides (AUC 0-24h) [n=12, 12, 11)
    44.5 (-1.3 to 111.5)
    0.8 (-30.1 to 45.1)
    2.8 (-32.4 to 56.3)
    No statistical analyses for this end point

    Secondary: Pharmacokinetics of LCQ908 - Trough Concentration (Cmin) and Observed maximum blood concentration (Cmax)

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    End point title
    		 Pharmacokinetics of LCQ908 - Trough Concentration (Cmin) and Observed maximum blood concentration (Cmax) [3]
    End point description
    Lowest observed blood concentration (Cmin) and observed maximum blood concentration (Cmax) following drug administration derived from non-compartmental analysis using scheduled sampling time for the whole dataset.
    End point type
    Secondary
    End point timeframe
    0, 1, 2, 3, 4, 6, and 24 hours at Week 12
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: PK parameters are only analyzed on study drug (LCQ908), not on placebo.
    End point values
    		 LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    12
    9
    Units: ng/mL
    arithmetic mean (standard deviation)
        Cmin
    312 ( 120 )
    426 ( 224 )
        Cmax
    603 ( 244 )
    745 ( 408 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetics of LCQ908- Area under the plasma concentration time curve AUC (0-24hour)

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    End point title
    		 Pharmacokinetics of LCQ908- Area under the plasma concentration time curve AUC (0-24hour) [4]
    End point description
    The area under the concentration-time curve from time zero to 24 hours after drug administration was calculated by using linear trapezoidal rule.
    End point type
    Secondary
    End point timeframe
    0, 1, 2, 3, 4, 6, and 24 hours at Week 12
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: PK parameters are only analyzed on study drug (LCQ908), not on placebo.
    End point values
    		 LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    12
    9
    Units: ng/mL *hr
        arithmetic mean (standard deviation)
    11000 ( 4100 )
    14300 ( 7390 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetics of LCQ908- Time to reach maximum concentration following drug administration Tmax (hours)

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    End point title
    		 Pharmacokinetics of LCQ908- Time to reach maximum concentration following drug administration Tmax (hours) [5]
    End point description
    End point type
    Secondary
    End point timeframe
    0, 1, 2, 3, 4, 6, and 24 hours at Week 12
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: PK parameters are only analyzed on study drug (LCQ908), not on placebo.
    End point values
    		 LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    12
    9
    Units: hours
        median (full range (min-max))
    6 (0 to 24)
    8 (0 to 24)
    No statistical analyses for this end point

    Secondary: Pharmacokinetics of LCQ908- Average observed blood concentration (Cavg)

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    End point title
    		 Pharmacokinetics of LCQ908- Average observed blood concentration (Cavg) [6]
    End point description
    Average observed blood concentration measured by (AUC0-24)/24.
    End point type
    Secondary
    End point timeframe
    0, 1, 2, 3, 4, 6, and 24 hours at Week 12
    Notes
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: PK parameters are only analyzed on study drug (LCQ908), not on placebo.
    End point values
    		 LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    12
    9
    Units: ng/mL
        arithmetic mean (standard deviation)
    459 ( 171 )
    597 ( 308 )
    No statistical analyses for this end point

    Secondary: Number of patients reported with any adverse event, serious adverse event and death

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    End point title
    		 Number of patients reported with any adverse event, serious adverse event and death
    End point description
    End point type
    Secondary
    End point timeframe
    52 weeks
    End point values
    		 Placebo LCQ908 20 mg LCQ908 40 mg
    Number of subjects analysed
    15
    15
    14
    Units: Participants
        At least one adverse events
    15
    15
    14
        At least one serious adverse event
    6
    6
    3
        Death
    1
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet, once daily. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 placebo matching to 40mg tablet + one LCQ908 placebo matching to 20mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Reporting group title
    LCQ908 40mg
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 40 mg active tablet + one LCQ908 placebo matching to 20mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 10mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Reporting group title
    LCQ908 20mg
    Reporting group description
    		 In period II (0-12 weeks) double-blind treatment: one LCQ908 20 mg active tablet + one LCQ908 placebo matching to 40mg tablet, once daily. No dose titration allowed. In period III (12-52 weeks) double blind treatment: Without down titration, the period II dosing regimen was followed. Following decision to down titrate: one LCQ908 10 mg active tablet + one LCQ908 placebo matching to 40 mg tablet + one LCQ908 placebo matching to 20 mg tablets, once daily. A low fat diet was followed and recorded in patient diary.

    Serious adverse events
    		 Placebo LCQ908 40mg LCQ908 20mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 15 (40.00%)
    3 / 14 (21.43%)
    6 / 15 (40.00%)
         number of deaths (all causes)
    1
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    HEPATIC ENZYME INCREASED
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    MALIGNANT PALATE NEOPLASM
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    INCISIONAL HERNIA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    FEMORAL ARTERY OCCLUSION
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    CARDIAC ARREST
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    CEREBRAL HAEMORRHAGE
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Gastrointestinal disorders
    ABDOMINAL PAIN
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    GASTROINTESTINAL DISORDER
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PANCREATITIS
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    3 / 15 (20.00%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 1
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PANCREATITIS ACUTE
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    HYPERTRANSAMINASAEMIA
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    PULMONARY EMBOLISM
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    PNEUMONIA
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    HYPERGLYCAEMIA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo LCQ908 40mg LCQ908 20mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 15 (100.00%)
    14 / 14 (100.00%)
    15 / 15 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    LIPOMA
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    NEOPLASM SKIN
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    PAPILLOMA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Vascular disorders
    PERIPHERAL ARTERIAL OCCLUSIVE DISEASE
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    HOT FLUSH
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    CYST
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    ASTHENIA
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 14 (14.29%)
    1 / 15 (6.67%)
         occurrences all number
    0
    2
    1
    FATIGUE
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    2
    PYREXIA
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    2 / 15 (13.33%)
         occurrences all number
    1
    1
    2
    MALAISE
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    INFLUENZA LIKE ILLNESS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Immune system disorders
    ALLERGY TO ARTHROPOD STING
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    FOOD ALLERGY
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    ALLERGY TO PLANTS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    DYSPNOEA
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    0
    COUGH
         subjects affected / exposed
    2 / 15 (13.33%)
    4 / 14 (28.57%)
    3 / 15 (20.00%)
         occurrences all number
    2
    4
    3
    SINUS CONGESTION
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    OROPHARYNGEAL PAIN
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    2
    NASAL CONGESTION
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    2
    1
    0
    DYSPNOEA EXERTIONAL
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Psychiatric disorders
    DEPRESSION
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    INSOMNIA
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    STRESS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Investigations
    BLOOD GLUCOSE INCREASED
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    HEPATIC ENZYME INCREASED
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    HAEMOGLOBIN DECREASED
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    CAROTID BRUIT
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    WEIGHT DECREASED
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 14 (14.29%)
    2 / 15 (13.33%)
         occurrences all number
    0
    2
    2
    WEIGHT INCREASED
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    FALL
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    CONTUSION
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    CHEST INJURY
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    LIGAMENT SPRAIN
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    LIMB INJURY
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    MUSCLE RUPTURE
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    SCAR
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    RIB FRACTURE
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    POST PROCEDURAL COMPLICATION
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    SPORTS INJURY
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    TOOTH FRACTURE
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    CARDIAC ARREST
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    PALPITATIONS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    CORONARY ARTERY STENOSIS
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    CARDIAC FAILURE
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    SUPRAVENTRICULAR EXTRASYSTOLES
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Nervous system disorders
    DIZZINESS
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 14 (7.14%)
    3 / 15 (20.00%)
         occurrences all number
    4
    1
    3
    DYSGEUSIA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    LETHARGY
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    HEADACHE
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    4 / 15 (26.67%)
         occurrences all number
    1
    1
    4
    SCIATICA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    2
    SYNCOPE
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Blood and lymphatic system disorders
    LEUKOPENIA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    ANAEMIA
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    1
    THROMBOCYTOPENIA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Ear and labyrinth disorders
    DEAFNESS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Eye disorders
    CONJUNCTIVAL HAEMORRHAGE
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    BLEPHARITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    ABDOMINAL DISCOMFORT
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 14 (14.29%)
    0 / 15 (0.00%)
         occurrences all number
    0
    2
    0
    ABDOMINAL DISTENSION
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    ABDOMINAL RIGIDITY
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    ABDOMINAL PAIN UPPER
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    3
    ABDOMINAL PAIN
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    7 / 15 (46.67%)
         occurrences all number
    1
    2
    14
    BARRETT'S OESOPHAGUS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    CHANGE OF BOWEL HABIT
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    2 / 15 (13.33%)
         occurrences all number
    1
    1
    2
    DENTAL CARIES
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    DEFAECATION URGENCY
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    CONSTIPATION
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    DIARRHOEA
         subjects affected / exposed
    10 / 15 (66.67%)
    10 / 14 (71.43%)
    12 / 15 (80.00%)
         occurrences all number
    13
    34
    56
    DIVERTICULUM
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    ENTERITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    DYSPEPSIA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    DRY MOUTH
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    FAECES DISCOLOURED
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    FREQUENT BOWEL MOVEMENTS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    FLATULENCE
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    1
    FAECES SOFT
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    2
    GASTROOESOPHAGEAL REFLUX DISEASE
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    HYPERCHLORHYDRIA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    PANCREATITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 14 (14.29%)
    3 / 15 (20.00%)
         occurrences all number
    0
    6
    7
    NAUSEA
         subjects affected / exposed
    1 / 15 (6.67%)
    4 / 14 (28.57%)
    5 / 15 (33.33%)
         occurrences all number
    1
    6
    9
    INGUINAL HERNIA
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    RECTAL HAEMORRHAGE
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    3
    TOOTHACHE
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    VOMITING
         subjects affected / exposed
    0 / 15 (0.00%)
    4 / 14 (28.57%)
    6 / 15 (40.00%)
         occurrences all number
    0
    7
    11
    Hepatobiliary disorders
    CHOLELITHIASIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Skin and subcutaneous tissue disorders
    SKIN LESION
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    ROSACEA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    ALOPECIA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    XANTHOMA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    ACNE
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Renal and urinary disorders
    RENAL FAILURE ACUTE
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    DYSURIA
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    ARTHRALGIA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    3 / 15 (20.00%)
         occurrences all number
    0
    1
    3
    BACK PAIN
         subjects affected / exposed
    1 / 15 (6.67%)
    3 / 14 (21.43%)
    1 / 15 (6.67%)
         occurrences all number
    1
    3
    1
    BURSITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    MUSCLE FATIGUE
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    INTERVERTEBRAL DISC PROTRUSION
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    MUSCLE SPASMS
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    MUSCULOSKELETAL PAIN
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    PAIN IN EXTREMITY
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    0
    NECK PAIN
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    MYOPATHY
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    MYALGIA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    TENDONITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    3
    SJOGREN'S SYNDROME
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    BRONCHITIS
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    CONJUNCTIVITIS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    ERYSIPELAS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    EAR INFECTION
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    CYSTITIS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    GASTROENTERITIS
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 14 (7.14%)
    2 / 15 (13.33%)
         occurrences all number
    3
    1
    3
    GASTROENTERITIS VIRAL
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    INFLUENZA
         subjects affected / exposed
    3 / 15 (20.00%)
    4 / 14 (28.57%)
    0 / 15 (0.00%)
         occurrences all number
    3
    4
    0
    HORDEOLUM
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    HAND-FOOT-AND-MOUTH DISEASE
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    JOINT ABSCESS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    LARYNGITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    PHARYNGITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    NASOPHARYNGITIS
         subjects affected / exposed
    3 / 15 (20.00%)
    4 / 14 (28.57%)
    2 / 15 (13.33%)
         occurrences all number
    3
    6
    4
    LOCALISED INFECTION
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    PNEUMONIA
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    SUBCUTANEOUS ABSCESS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    RHINITIS
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    RESPIRATORY TRACT INFECTION
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    3
    TINEA INFECTION
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    TOOTH ABSCESS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    TOOTH INFECTION
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    UPPER RESPIRATORY TRACT INFECTION
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 14 (7.14%)
    1 / 15 (6.67%)
         occurrences all number
    3
    1
    1
    URINARY TRACT INFECTION
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Metabolism and nutrition disorders
    DECREASED APPETITE
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    DIABETES MELLITUS
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    HYPERGLYCAEMIA
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    HYPOCALCAEMIA
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    HYPOGLYCAEMIA
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    HYPOKALAEMIA
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    IRON DEFICIENCY
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    VITAMIN D DEFICIENCY
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Apr 2012
    Amendment 1 Version 1 : This protocol amendment introduced two key secondary objectives to the study protocol: a. To assess the effect of LCQ908 as compared to placebo on fasting triglycerides after 24 and 52 weeks. b. To assess the effect of LCQ908 as compared to placebo on post-prandial triglycerides after 12 weeks.
    20 Jun 2012
    Amendment 1 Version 2 : To incorporate the rational for the amendment 1 version 1 into the body of the protocol, rather than a stand-alone document to meet the requirements of certain Health Authorities.
    23 Jan 2013
    Amendment 2 Version 3: This amendment introduced modifications to three exclusion criteria. Additionally, the reporting of SAEs was updated to reflect the current reporting procedure.
    02 May 2013
    Amendment 3 Version 4: This amendment introduced modifications based on feedback from the FDA regarding previous amendment of the CLCQ908B2302 study protocol in which the exclusion criterion associated with diabetes mellitus (type 1 and 2) was deleted. The modification allowed a more representative patient population to be recruited into the study but has also raised concern at the agency with regard to the potential hypoglycemic events that could be associated with the use of pradigastat in uncontrolled diabetes. Additional information about potential hypoglycemic events was therefore, collected in the case report forms.
    07 Nov 2013
    Amendment 4 Version 5: This amendment incorporated FDA feedback on need to follow the recommendations of the National Research council on the Prevention and Treatment of Missing Data in Clinical Trials. FDA suggested that an alternative method of dealing with missing data in the primary analysis should be defined. Following the changes to the primary analysis required to alter the method of handling missing data, an alternative methodology to the original approach for controlling the family wise type I error rate was also specified in order to reduce the operational burden on statistical programming.

    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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