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    Clinical Trial Results:
    Obeticholic acid treatment in patients with bile acid diarrhoea: an open-label, pilot study of mechanisms, safety and symptom response.

    Summary
    EudraCT number
    		 2011-003777-28
    Trial protocol
    		 GB  
    Global end of trial date
    28 Feb 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Feb 2020
    First version publication date
    06 Feb 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    OBADIAH1
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01585025
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Imperial College London
    Sponsor organisation address
    South Kensington Campus, London, United Kingdom, SW7 2AZ
    Public contact
    Julian RF Walters, Imperial College London, julian.walters@imperial.ac.uk
    Scientific contact
    Julian RF Walters, Imperial College London, julian.walters@imperial.ac.uk
    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
    02 Feb 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 Jan 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    28 Feb 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective is to define the change over 2 weeks in serum fibroblast growth factor (FGF19) in 3 groups of patients: primary bile acid diarrhoea, secondary bile acid diarrhoea, and a control population of patients with chronic diarrhoea but with normal bile acid retention.
    Protection of trial subjects
    None
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Apr 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
    United Kingdom: 35
    Worldwide total number of subjects
    35
    EEA total number of subjects
    35
    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)
    25
    From 65 to 84 years
    10
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were recruited between 2012 and 2014 at Gastrointestinal Outpatient Clinics at Hammersmith and Charing Cross Hospitals

    Pre-assignment
    Screening details
    		 35 eligible participants enrolled in the study

    Period 1
    Period 1 title
    Overall (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Primary Bile acid diarrhoea
    Arm description
    		 Participants with SeHCAT <10% without other causes such as Crohn's disease and/or ileal resection
    Arm type
    		 Experimental

    Investigational medicinal product name
    Obeticholic acid
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Day -14 to Day 0 subjects stopped their usual diarrhoeal medication. Day 1 to Day 15 Obeticholic acid 25mg tablet administered to subjects once daily in the morning. Day 16 to day 28 normal diarrhoeal medication may be re-commenced.

    Arm title
    		 Secondary Bile acid diarrhoea
    Arm description
    		 Participants with Crohn's disease or ileal resection
    Arm type
    		 Experimental

    Investigational medicinal product name
    Obeticholic acid
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Day -14 to Day 0 subjects stopped their usual diarrhoeal medication. Day 1 to Day 15 Obeticholic acid 25mg tablet administered to subjects once daily in the morning. Day 16 to day 28 normal diarrhoeal medication may be re-commenced.

    Arm title
    		 Idiopathic Diarrhoea Controls
    Arm description
    		 Participants with Chronic diarrhoea with SeHCAT >15% and no Crohn's or ileal resection
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Obeticholic acid
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Day -14 to Day 0 subjects stopped their usual diarrhoeal medication. Day 1 to Day 15 Obeticholic acid 25mg tablet administered to subjects once daily in the morning. Day 16 to day 28 normal diarrhoeal medication may be re-commenced.

    Number of subjects in period 1
    		Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls
    Started
    10
    13
    12
    Completed
    10
    10
    8
    Not completed
    0
    3
    4
         Consent withdrawn by subject
    -
    3
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Primary Bile acid diarrhoea
    Reporting group description
    		 Participants with SeHCAT <10% without other causes such as Crohn's disease and/or ileal resection

    Reporting group title
    Secondary Bile acid diarrhoea
    Reporting group description
    		 Participants with Crohn's disease or ileal resection

    Reporting group title
    Idiopathic Diarrhoea Controls
    Reporting group description
    		 Participants with Chronic diarrhoea with SeHCAT >15% and no Crohn's or ileal resection

    Reporting group values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Total
    Number of subjects
    		 10 13 12 35
    Age categorical
    Units: Subjects
        Age 18-74
    		 10 10 8 28
        Not recorded
    		 0 3 4 7
    Age continuous
    Units: years
        median (full range (min-max))
    		 47 (24 to 74) 45 (27 to 75) 39 (25 to 68) -
    Gender categorical
    Units: Subjects
        Female
    		 7 7 3 17
        Male
    		 3 3 5 11
        Not recorded
    		 0 3 4 7
    Subject analysis sets

    Subject analysis set title
    Primary Bile acid diarrhoea baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Day 0, baseline analyses

    Subject analysis set title
    Secondary Bile acid diarrhoea baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Day 0, baseline analyses

    Subject analysis set title
    Idiopathic Diarrhoea Controls baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Day 0, baseline analyses

    Subject analysis sets values
    		 Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects
    10
    10
    8
    Age categorical
    Units: Subjects
        Age 18-74
    10
    10
    8
        Not recorded
    0
    3
    4
    Age continuous
    Units: years
        median (full range (min-max))
    47 (24 to 74)
    45 (27 to 75)
    39 (25 to 68)
    Gender categorical
    Units: Subjects
        Female
    7
    7
    3
        Male
    3
    3
    5
        Not recorded
    0
    3
    4

    End points

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    End points reporting groups
    Reporting group title
    Primary Bile acid diarrhoea
    Reporting group description
    		 Participants with SeHCAT <10% without other causes such as Crohn's disease and/or ileal resection

    Reporting group title
    Secondary Bile acid diarrhoea
    Reporting group description
    		 Participants with Crohn's disease or ileal resection

    Reporting group title
    Idiopathic Diarrhoea Controls
    Reporting group description
    		 Participants with Chronic diarrhoea with SeHCAT >15% and no Crohn's or ileal resection

    Subject analysis set title
    Primary Bile acid diarrhoea baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Day 0, baseline analyses

    Subject analysis set title
    Secondary Bile acid diarrhoea baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Day 0, baseline analyses

    Subject analysis set title
    Idiopathic Diarrhoea Controls baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Day 0, baseline analyses

    Primary: Changes in Fasting FGF19

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    End point title
    		 Changes in Fasting FGF19
    End point description
    End point type
    Primary
    End point timeframe
    Day 0, day 15
    End point values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects analysed
    10
    10
    8
    10
    10
    8
    Units: pg/ml
        median (inter-quartile range (Q1-Q3))
    237 (116 to 302)
    46 (24 to 72)
    194 (126 to 344)
    133 (102 to 168)
    32 (24 to 42)
    116 (57 to 186)
    Statistical analysis title
    		 FGF19 Primary Bile acid
    Statistical analysis description
    Compared the baseline to treatment
    Comparison groups
    Primary Bile acid diarrhoea v Primary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.007
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 FGF19 Secondary Bile acid
    Statistical analysis description
    Compared the baseline to treatment
    Comparison groups
    Secondary Bile acid diarrhoea v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.11
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 FGF19 Idiopathic Diarrhoea Control
    Statistical analysis description
    Compared the baseline to treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Idiopathic Diarrhoea Controls baseline
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 FGF19 overall
    Statistical analysis description
    Comparison between the groups
    Comparison groups
    Idiopathic Diarrhoea Controls v Primary Bile acid diarrhoea v Secondary Bile acid diarrhoea v Idiopathic Diarrhoea Controls baseline v Primary Bile acid diarrhoea baseline v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    56
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 = 0.0004
    Method
    		 Kruskal-wallis
    Confidence interval

    Secondary: Changes in 6h Response (AUC) of FGF19 to OCA

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    End point title
    		 Changes in 6h Response (AUC) of FGF19 to OCA
    End point description
    Change in dynamic response of FGF19 in 6 hours following OCA administration; at start and end of 15 day OCA test period.
    End point type
    Secondary
    End point timeframe
    Day 0, day 15
    End point values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects analysed
    10
    10
    8
    10
    10
    8
    Units: pg/ml
        median (inter-quartile range (Q1-Q3))
    3825 (2515 to 6129)
    457 (316 to 1016)
    2099 (1972 to 2341)
    3945 (2609 to 4834)
    401 (267 to 1086)
    1644 (1247 to 2776)
    Statistical analysis title
    		 FGF19 6h response Primary Bile acid
    Statistical analysis description
    Comparison baseline to treatment
    Comparison groups
    Primary Bile acid diarrhoea v Primary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.72
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 FGF19 6h response Secondary Bile acid
    Statistical analysis description
    Comparison baseline to treatment
    Comparison groups
    Secondary Bile acid diarrhoea v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.51
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 FGF19 6h response Idiopathic Control
    Statistical analysis description
    Comparison baseline to treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Idiopathic Diarrhoea Controls baseline
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.13
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Overall FGF19 6h response
    Statistical analysis description
    Comparison between all treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Primary Bile acid diarrhoea v Secondary Bile acid diarrhoea v Idiopathic Diarrhoea Controls baseline v Primary Bile acid diarrhoea baseline v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    56
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 = 0.13
    Method
    		 Kruskal-wallis
    Confidence interval

    Secondary: Changes in Fasting 7α-hydroxy-4-cholesten-3-one (C4)

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    End point title
    		 Changes in Fasting 7α-hydroxy-4-cholesten-3-one (C4)
    End point description
    Change in fasting 7α-hydroxy-4-cholesten-3-one before and after 15 day administration of OCA.
    End point type
    Secondary
    End point timeframe
    Day 0, day 15
    End point values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects analysed
    10
    10
    8
    10
    10
    8
    Units: microgram/L
        median (inter-quartile range (Q1-Q3))
    3 (1 to 17)
    56 (14 to 122)
    1 (1 to 3)
    16 (11 to 37)
    104 (48 to 134)
    9 (3 to 14)
    Statistical analysis title
    		 C4 Primary Bile Acid
    Statistical analysis description
    Comparison baseline to treatment
    Comparison groups
    Primary Bile acid diarrhoea v Primary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.03
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 C4 Secondary Bile Acid
    Statistical analysis description
    Comparison baseline to treatment
    Comparison groups
    Secondary Bile acid diarrhoea v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.11
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 C4 Idiopathic control
    Statistical analysis description
    Comparison baseline to treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Idiopathic Diarrhoea Controls baseline
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.02
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 C4 Overall
    Statistical analysis description
    Comparison between treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Primary Bile acid diarrhoea v Secondary Bile acid diarrhoea v Idiopathic Diarrhoea Controls baseline v Primary Bile acid diarrhoea baseline v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    56
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 = 0.001
    Method
    		 Kruskal-wallis
    Confidence interval

    Secondary: Changes in Serum Total Bile Acids

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    End point title
    		 Changes in Serum Total Bile Acids
    End point description
    Dynamic changes of total bile acids over 6 hour period following OCA administration before and after 15 day OCA period.
    End point type
    Secondary
    End point timeframe
    Day 0, day 15
    End point values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects analysed
    10
    10
    8
    10
    10
    8
    Units: micromol/L
        median (inter-quartile range (Q1-Q3))
    0.9 (0.9 to 3)
    2.5 (1.0 to 4.0)
    1.0 (0.9 to 1.8)
    1.5 (1.0 to 4.0)
    2.5 (1.0 to 6.0)
    1.5 (1.0 to 2.8)
    Statistical analysis title
    		 Bile acid Primary Bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Primary Bile acid diarrhoea v Primary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.13
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Bile acid Secondary Bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Secondary Bile acid diarrhoea v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Bile acid Idiopathic control
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Idiopathic Diarrhoea Controls baseline
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Bile acid Overall
    Statistical analysis description
    Comparison between treatments
    Comparison groups
    Idiopathic Diarrhoea Controls v Primary Bile acid diarrhoea v Secondary Bile acid diarrhoea v Idiopathic Diarrhoea Controls baseline v Primary Bile acid diarrhoea baseline v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    56
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 = 0.04
    Method
    		 Kruskal-wallis
    Confidence interval

    Secondary: Changes in Stool Frequency (weekly number of stools)

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    End point title
    		 Changes in Stool Frequency (weekly number of stools)
    End point description
    End point type
    Secondary
    End point timeframe
    Week 2, week 4
    End point values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects analysed
    10
    10
    7
    10
    10
    7
    Units: stools per week
        median (inter-quartile range (Q1-Q3))
    14 (9 to 27)
    17 (17 to 42)
    18 (13 to 19)
    23 (11 to 27)
    23 (14 to 44)
    15 (11 to 17)
    Statistical analysis title
    		 Weekly stool primary bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Primary Bile acid diarrhoea v Primary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.03
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Weekly stool secondary bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Secondary Bile acid diarrhoea v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.17
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Weekly stool idiopathic control
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Idiopathic Diarrhoea Controls baseline
    Number of subjects included in analysis
    14
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.31
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Weekly stool overall
    Statistical analysis description
    Comparison between treatments
    Comparison groups
    Idiopathic Diarrhoea Controls v Secondary Bile acid diarrhoea v Primary Bile acid diarrhoea v Idiopathic Diarrhoea Controls baseline v Primary Bile acid diarrhoea baseline v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    54
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Changes in Mean Stool Form

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    End point title
    		 Changes in Mean Stool Form
    End point description
    Change in mean stool form reported per week between week 2 (baseline) and week 4 (week 2 of treatment) using the Bristol Stool Form Scale (range of scores 1 to 7). High scores are a worse outcome (7=liquid stools).
    End point type
    Secondary
    End point timeframe
    week 2, week 4
    End point values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects analysed
    10
    10
    7
    10
    10
    7
    Units: Score on Bristol scale
        median (inter-quartile range (Q1-Q3))
    4.3 (4.0 to 4.8)
    5.6 (4.6 to 6.7)
    4.9 (4.1 to 5.0)
    5.2 (4.5 to 5.6)
    6.0 (5.3 to 6.9)
    4.9 (4.3 to 5.9)
    Statistical analysis title
    		 Mean stool form Primary Bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Primary Bile acid diarrhoea v Primary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.05
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Mean stool form Secondary Bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Secondary Bile acid diarrhoea v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.04
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Mean stool form Idiopathic control
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Idiopathic Diarrhoea Controls baseline
    Number of subjects included in analysis
    14
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.74
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Mean stool form overall
    Statistical analysis description
    Comparison between treatments
    Comparison groups
    Idiopathic Diarrhoea Controls v Primary Bile acid diarrhoea v Secondary Bile acid diarrhoea v Idiopathic Diarrhoea Controls baseline v Primary Bile acid diarrhoea baseline v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    54
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.01
    Method
    		 Kruskal-wallis
    Confidence interval

    Secondary: Change in Stool Index

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    End point title
    		 Change in Stool Index
    End point description
    Change in index calculated on a weekly basis, between week 2 (baseline) and week 4 (week 2 of treatment). The index calculated as ([weekly stool frequency x mean Bristol Stool Form Scale score] = Loperamide use [weekly mg x 3]). Individual scores ranged from 25 to 1095, with higher scores being worse.
    End point type
    Secondary
    End point timeframe
    week 2, week 4
    End point values
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls Primary Bile acid diarrhoea baseline Secondary Bile acid diarrhoea baseline Idiopathic Diarrhoea Controls baseline
    Number of subjects analysed
    10
    10
    7
    10
    10
    7
    Units: index score
        median (inter-quartile range (Q1-Q3))
    76 (44 to 104)
    127 (47 to 321)
    83 (65 to 101)
    113 (81 to 144)
    132 (72 to 473)
    96 (69 to 100)
    Statistical analysis title
    		 Stool index primary bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Primary Bile acid diarrhoea v Primary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.005
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Stool index secondary bile acid
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Secondary Bile acid diarrhoea v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.03
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Stool index idiopathic control
    Statistical analysis description
    Comparison between baseline to treatment
    Comparison groups
    Idiopathic Diarrhoea Controls v Idiopathic Diarrhoea Controls baseline
    Number of subjects included in analysis
    14
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.61
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Stool index overall
    Statistical analysis description
    Comparison between treatments
    Comparison groups
    Idiopathic Diarrhoea Controls v Primary Bile acid diarrhoea v Secondary Bile acid diarrhoea v Idiopathic Diarrhoea Controls baseline v Primary Bile acid diarrhoea baseline v Secondary Bile acid diarrhoea baseline
    Number of subjects included in analysis
    54
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0007
    Method
    		 Kruskal-wallis
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    6 weeks
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    10
    Reporting groups
    Reporting group title
    Primary Bile acid diarrhoea
    Reporting group description
    		 Participants with SeHCAT <10% without other causes such as Crohn's disease and/or ileal resection

    Reporting group title
    Secondary Bile acid diarrhoea
    Reporting group description
    		 Participants with Crohn's disease or ileal resection

    Reporting group title
    Idiopathic Diarrhoea Controls
    Reporting group description
    		 Participants with Chronic diarrhoea with SeHCAT >15% and no Crohn's or ileal resection

    Serious adverse events
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Primary Bile acid diarrhoea Secondary Bile acid diarrhoea Idiopathic Diarrhoea Controls
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 10 (0.00%)
    3 / 10 (30.00%)
    1 / 8 (12.50%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 10 (20.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    2
    1
    Gastrointestinal disorders
    Constipation/abdominal pain
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (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? 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.
    Recruitment in the idiopathic chronic diarrhoea control group did not reach the prespecified number of 10 due to dropouts. Of the 8 subjects recruited, one failed to return diaries that could be analyzed.
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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