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    Clinical Trial Results:
    A Phase 2, Randomized, Double-Blind, Placebo Controlled Study Evaluating the Safety, Tolerability, and Efficacy of GS-9674 in Subjects with Primary Biliary Cholangitis Without Cirrhosis

    Summary
    EudraCT number
    		 2016-002443-42
    Trial protocol
    		 GB   AT  
    Global end of trial date
    04 Sep 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    19 Sep 2020
    First version publication date
    19 Sep 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GS-US-427-4024
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02943447
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Gilead Sciences
    Sponsor organisation address
    333 Lakeside Drive, Foster City, CA, United States, 94404
    Public contact
    Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com
    Scientific contact
    Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    04 Sep 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Sep 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Sep 2019
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to evaluate the safety and tolerability of cilofexor in adults with primary biliary cholangitis (PBC).
    Protection of trial subjects
    The protocol and consent/assent forms were submitted by each investigator to a duly constituted Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for review and approval before study initiation. All revisions to the consent/assent forms (if applicable) after initial IEC/IRB approval were submitted by the investigator to the IEC/IRB for review and approval before implementation in accordance with regulatory requirements. This study was conducted in accordance with recognized international scientific and ethical standards, including but not limited to the International Conference on Harmonization guideline for Good Clinical Practice (ICH GCP) and the original principles embodied in the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Dec 2016
    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 States: 35
    Country: Number of subjects enrolled
    Canada: 21
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Austria: 7
    Worldwide total number of subjects
    71
    EEA total number of subjects
    15
    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)
    63
    From 65 to 84 years
    8
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were enrolled at study sites in United States, Canada, United Kingdom, and Austria. The first participant was screened on 01 December 2016. The last study visit occurred on 4 September 2019.

    Pre-assignment
    Screening details
    		 130 participants were screened.

    Period 1
    Period 1 title
    Blinded Study Phase
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Blinded Study Phase: Cilofexor 100 mg
    Arm description
    		 Cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Cilofexor
    Investigational medicinal product code
    Other name
    GS-9674
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    100 mg tablet administered orally once daily, with food

    Investigational medicinal product name
    Placebo to match cilofexor 30 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Tablet(s) administered orally once daily, with food

    Arm title
    		 Blinded Study Phase: Cilofexor 30 mg
    Arm description
    		 Cilofexor 30 mg tablet + placebo to match cilofexor 100 mg tablet orally once daily for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Cilofexor
    Investigational medicinal product code
    Other name
    GS-9674
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    30 mg tablet administered orally once daily, with food

    Investigational medicinal product name
    Placebo to match cilofexor 100 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Tablet(s) administered orally once daily, with food

    Arm title
    		 Blinded Study Phase: Placebo
    Arm description
    		 Placebo to match cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo to match cilofexor 100 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Tablet(s) administered orally once daily, with food

    Investigational medicinal product name
    Placebo to match cilofexor 30 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Tablet(s) administered orally once daily, with food

    Number of subjects in period 1
    		Blinded Study Phase: Cilofexor 100 mg Blinded Study Phase: Cilofexor 30 mg Blinded Study Phase: Placebo
    Started
    28
    30
    13
    Completed
    23
    28
    12
    Not completed
    5
    2
    1
         Adverse event
    3
    -
    -
         Investigator`s discretion
    1
    -
    -
         Withdrew consent
    1
    2
    1
    Period 2
    Period 2 title
    Open-Label Extension Phase
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 OLE Phase: From Cilofexor 100 mg
    Arm description
    		 Following Blinded Study Phase, participants in the Cilofexor 100 mg group, willing to enter Open-Label Extension (OLE) Phase received open-label cilofexor 100 mg tablet orally once daily for 97.4 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Cilofexor 100 mg
    Investigational medicinal product code
    Other name
    GS-9674
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Tablet(s) administered orally once daily, with food

    Arm title
    		 OLE Phase: From Cilofexor 30 mg
    Arm description
    		 Following Blinded Study Phase, participants in the Cilofexor 30 mg group, willing to enter OLE Phase received open-label cilofexor 100 mg tablet orally once daily for 96.1 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Cilofexor 100 mg
    Investigational medicinal product code
    Other name
    GS-9674
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Tablet(s) administered orally once daily, with food

    Arm title
    		 OLE Phase: From Placebo
    Arm description
    		 Following Blinded Study Phase, participants in the Placebo group, willing to enter OLE Phase received open-label cilofexor 100 mg tablet orally once daily for 97.3 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Cilofexor 100 mg
    Investigational medicinal product code
    Other name
    GS-9674
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Tablet(s) administered orally once daily, with food

    Number of subjects in period 2
    		OLE Phase: From Cilofexor 100 mg OLE Phase: From Cilofexor 30 mg OLE Phase: From Placebo
    Started
    23
    28
    12
    Completed
    5
    3
    2
    Not completed
    18
    25
    10
         Study terminated by sponsor
    10
    12
    6
         Adverse event
    4
    7
    3
         Withdrew consent
    1
    1
    1
         Lost to follow-up
    -
    1
    -
         Lack of efficacy
    3
    4
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Blinded Study Phase: Cilofexor 100 mg
    Reporting group description
    		 Cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.

    Reporting group title
    Blinded Study Phase: Cilofexor 30 mg
    Reporting group description
    		 Cilofexor 30 mg tablet + placebo to match cilofexor 100 mg tablet orally once daily for 12 weeks.

    Reporting group title
    Blinded Study Phase: Placebo
    Reporting group description
    		 Placebo to match cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.

    Reporting group values
    		 Blinded Study Phase: Cilofexor 100 mg Blinded Study Phase: Cilofexor 30 mg Blinded Study Phase: Placebo Total
    Number of subjects
    		 28 30 13 71
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 54 ( 9.8 ) 57 ( 6.3 ) 58 ( 5.9 ) -
    Gender categorical
    Units: Subjects
        Female
    		 28 26 12 66
        Male
    		 0 4 1 5
    Race
    Not Permitted = local regulators did not allow collection of race information.
    Units: Subjects
        Asian
    		 2 0 0 2
        Black or African American
    		 0 1 0 1
        White
    		 26 27 13 66
        Not Permitted
    		 0 1 0 1
        Other
    		 0 1 0 1
    Ethnicity
    Not Permitted = local regulators did not allow collection of ethnicity information.
    Units: Subjects
        Hispanic or Latino
    		 1 1 2 4
        Not Hispanic or Latino
    		 26 28 11 65
        Not Permitted
    		 1 1 0 2

    End points

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    End points reporting groups
    Reporting group title
    Blinded Study Phase: Cilofexor 100 mg
    Reporting group description
    		 Cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.

    Reporting group title
    Blinded Study Phase: Cilofexor 30 mg
    Reporting group description
    		 Cilofexor 30 mg tablet + placebo to match cilofexor 100 mg tablet orally once daily for 12 weeks.

    Reporting group title
    Blinded Study Phase: Placebo
    Reporting group description
    		 Placebo to match cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.
    Reporting group title
    OLE Phase: From Cilofexor 100 mg
    Reporting group description
    		 Following Blinded Study Phase, participants in the Cilofexor 100 mg group, willing to enter Open-Label Extension (OLE) Phase received open-label cilofexor 100 mg tablet orally once daily for 97.4 weeks.

    Reporting group title
    OLE Phase: From Cilofexor 30 mg
    Reporting group description
    		 Following Blinded Study Phase, participants in the Cilofexor 30 mg group, willing to enter OLE Phase received open-label cilofexor 100 mg tablet orally once daily for 96.1 weeks.

    Reporting group title
    OLE Phase: From Placebo
    Reporting group description
    		 Following Blinded Study Phase, participants in the Placebo group, willing to enter OLE Phase received open-label cilofexor 100 mg tablet orally once daily for 97.3 weeks.

    Primary: Percentage of Participants Experiencing Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) in the Blinded Study Phase

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    End point title
    		 Percentage of Participants Experiencing Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) in the Blinded Study Phase [1]
    End point description
    The Safety Analysis Set included all participants who took at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    First dose date up to Week 12 + 30 days
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical comparison was planned or performed.
    End point values
    		 Blinded Study Phase: Cilofexor 100 mg Blinded Study Phase: Cilofexor 30 mg Blinded Study Phase: Placebo
    Number of subjects analysed
    28
    30
    13
    Units: percentage of participants
    number (not applicable)
        TEAEs
    89.3
    76.7
    84.6
        TESAEs
    0.0
    3.3
    0.0
    No statistical analyses for this end point

    Primary: Percentage of Participants Experiencing TEAEs and TESAEs in the Open-Label Extension (OLE) Phase

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    End point title
    		 Percentage of Participants Experiencing TEAEs and TESAEs in the Open-Label Extension (OLE) Phase [2]
    End point description
    The OLE Analysis Set included all participants who took at least 1 dose of study drug in the OLE Phase.
    End point type
    Primary
    End point timeframe
    First dose date in the OLE phase up to last dose date (Maximum: 97.4 weeks) + 30 days
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical comparison was planned or performed.
    End point values
    		 OLE Phase: From Cilofexor 100 mg OLE Phase: From Cilofexor 30 mg OLE Phase: From Placebo
    Number of subjects analysed
    23
    28
    12
    Units: percentage of participants
    number (not applicable)
        TEAEs
    95.7
    89.3
    100.0
        TESAEs
    4.3
    0.0
    0.0
    No statistical analyses for this end point

    Primary: Percentage of Participants Who Experienced Graded Laboratory Abnormalities in the Blinded Study Phase

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    End point title
    		 Percentage of Participants Who Experienced Graded Laboratory Abnormalities in the Blinded Study Phase [3]
    End point description
    Treatment-emergent laboratory abnormalities were defined as values that increase at least one toxicity grade from baseline. The most severe graded abnormality from all tests was counted for each participant. Participants in the Safety Analysis Set were analyzed.
    End point type
    Primary
    End point timeframe
    First dose date up to Week 12 + 30 days
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical comparison was planned or performed.
    End point values
    		 Blinded Study Phase: Cilofexor 100 mg Blinded Study Phase: Cilofexor 30 mg Blinded Study Phase: Placebo
    Number of subjects analysed
    28
    30
    13
    Units: percentage of participants
    number (not applicable)
        Any Graded Laboratory Abnormality
    85.7
    86.7
    92.3
        Grade 4 or above Laboratory Abnormalities
    0.0
    3.3
    0.0
    No statistical analyses for this end point

    Primary: Percentage of Participants Who Experienced Graded Laboratory Abnormalities in the OLE Phase

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    End point title
    		 Percentage of Participants Who Experienced Graded Laboratory Abnormalities in the OLE Phase [4]
    End point description
    Treatment-emergent laboratory abnormalities were defined as values that increase at least one toxicity grade from baseline. The most severe graded abnormality from all tests was counted for each participant. Participants in the OLE Analysis Set were analyzed.
    End point type
    Primary
    End point timeframe
    First dose date in the OLE phase up to last dose date (Maximum: 97.4 weeks) + 30 days
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical comparison was planned or performed.
    End point values
    		 OLE Phase: From Cilofexor 100 mg OLE Phase: From Cilofexor 30 mg OLE Phase: From Placebo
    Number of subjects analysed
    23
    28
    12
    Units: percentage of participants
    number (not applicable)
        Any Graded Laboratory Abnormality
    91.3
    96.4
    100.0
        Grade 4 or above Laboratory Abnormalities
    0.0
    0.0
    0.0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Blinded Study Phase: First dose date up to Week 12 + 30 days; Open-Label Extension (OLE) Phase: First dose date in the (OLE) Phase up to last dose date (Maximum: 97.4 weeks) + 30 days
    Adverse event reporting additional description
    The Safety Analysis Set for Blinded Study phase included all participants who took at least 1 dose of study drug in Blinded Study phase and the OLE Analysis Set included all participants who took at least 1 dose of study drug in OLE phase.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Blinded Study Phase: Cilofexor 100 mg
    Reporting group description
    		 Blinded Study Phase: Cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.

    Reporting group title
    Blinded Study Phase: Cilofexor 30 mg
    Reporting group description
    		 Blinded Study Phase: Cilofexor 30 mg tablet + placebo to match cilofexor 100 mg tablet orally once daily for 12 weeks.

    Reporting group title
    Blinded Study Phase: Placebo
    Reporting group description
    		 Blinded Study Phase: Placebo to match cilofexor 100 mg tablet + placebo to match cilofexor 30 mg tablet orally once daily for 12 weeks.

    Reporting group title
    OLE Phase: From Cilofexor 100 mg
    Reporting group description
    		 OLE Phase: Following Blinded Study Phase, participants in the Cilofexor 100 mg group, willing to enter OLE Phase received open-label cilofexor 100 mg tablet orally once daily for 97.4 weeks.

    Reporting group title
    OLE Phase: From Cilofexor 30 mg
    Reporting group description
    		 OLE Phase: Following Blinded Study Phase, participants in the Cilofexor 30 mg group, willing to enter OLE Phase received open-label cilofexor 100 mg tablet orally once daily for 96.1 weeks.

    Reporting group title
    OLE Phase: From Placebo
    Reporting group description
    		 OLE Phase: Following Blinded Study Phase, participants in the Placebo group, willing to enter OLE Phase received open-label cilofexor 100 mg tablet orally once daily for 97.3 weeks.

    Serious adverse events
    		 Blinded Study Phase: Cilofexor 100 mg Blinded Study Phase: Cilofexor 30 mg Blinded Study Phase: Placebo OLE Phase: From Cilofexor 100 mg OLE Phase: From Cilofexor 30 mg OLE Phase: From Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Invasive ductal breast carcinoma
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Blinded Study Phase: Cilofexor 100 mg Blinded Study Phase: Cilofexor 30 mg Blinded Study Phase: Placebo OLE Phase: From Cilofexor 100 mg OLE Phase: From Cilofexor 30 mg OLE Phase: From Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    21 / 28 (75.00%)
    18 / 30 (60.00%)
    11 / 13 (84.62%)
    20 / 23 (86.96%)
    25 / 28 (89.29%)
    12 / 12 (100.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    2
    0
    1
    Flushing
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    5 / 28 (17.86%)
    2 / 30 (6.67%)
    3 / 13 (23.08%)
    3 / 23 (13.04%)
    4 / 28 (14.29%)
    2 / 12 (16.67%)
         occurrences all number
    5
    2
    3
    3
    4
    2
    Pyrexia
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    1 / 28 (3.57%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    1
    0
    1
    0
    Influenza like illness
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Peripheral swelling
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    2 / 28 (7.14%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    Cyst
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Suprapubic pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    2 / 28 (7.14%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    2
    3
    0
    Rhinorrhoea
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    2
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    1 / 23 (4.35%)
    2 / 28 (7.14%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    1
    2
    0
    Personality change
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Investigations
    Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    1 / 28 (3.57%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    1
    Blood cholesterol increased
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Joint injury
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Limb injury
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Procedural anxiety
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 28 (14.29%)
    4 / 30 (13.33%)
    4 / 13 (30.77%)
    2 / 23 (8.70%)
    1 / 28 (3.57%)
    0 / 12 (0.00%)
         occurrences all number
    5
    4
    4
    2
    1
    0
    Somnolence
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    1
    0
    0
    0
    Tension headache
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    0
    0
    2
    Hyperaesthesia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 28 (0.00%)
    2 / 30 (6.67%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    3
    0
    2
    0
    0
    Eye disorders
    Dry eye
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    2 / 28 (7.14%)
    1 / 12 (8.33%)
         occurrences all number
    2
    0
    0
    0
    2
    1
    Eye pruritus
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    4 / 28 (14.29%)
    4 / 30 (13.33%)
    1 / 13 (7.69%)
    1 / 23 (4.35%)
    6 / 28 (21.43%)
    4 / 12 (33.33%)
         occurrences all number
    5
    4
    1
    1
    7
    4
    Nausea
         subjects affected / exposed
    3 / 28 (10.71%)
    2 / 30 (6.67%)
    3 / 13 (23.08%)
    3 / 23 (13.04%)
    2 / 28 (7.14%)
    2 / 12 (16.67%)
         occurrences all number
    3
    2
    3
    3
    2
    2
    Abdominal pain
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 30 (6.67%)
    1 / 13 (7.69%)
    1 / 23 (4.35%)
    3 / 28 (10.71%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    1
    1
    4
    1
    Constipation
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 30 (3.33%)
    2 / 13 (15.38%)
    2 / 23 (8.70%)
    0 / 28 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    3
    1
    2
    2
    0
    2
    Vomiting
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    1 / 28 (3.57%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    0
    1
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    0
    1
    0
    2
    Dry mouth
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    2 / 28 (7.14%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    1
    2
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    0 / 28 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    1
    0
    0
    1
    0
    2
    Abdominal distension
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    2 / 28 (7.14%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    3
    0
    Dyspepsia
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    0
    0
    0
    0
    Abdominal discomfort
         subjects affected / exposed
    0 / 28 (0.00%)
    2 / 30 (6.67%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Barrett`s oesophagus
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Gingival bleeding
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    13 / 28 (46.43%)
    6 / 30 (20.00%)
    3 / 13 (23.08%)
    4 / 23 (17.39%)
    11 / 28 (39.29%)
    6 / 12 (50.00%)
         occurrences all number
    14
    6
    3
    5
    18
    7
    Pruritus generalised
         subjects affected / exposed
    4 / 28 (14.29%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    1 / 28 (3.57%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    0
    1
    1
    0
    Rash
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    2 / 28 (7.14%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    2
    2
    0
    Rash maculo-papular
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    0
    1
    0
    1
    Rash papular
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    3 / 23 (13.04%)
    2 / 28 (7.14%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    3
    2
    1
    Pain in extremity
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    3 / 28 (10.71%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    2
    3
    1
    Arthralgia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    3 / 28 (10.71%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    0
    1
    3
    2
    Back pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    3 / 23 (13.04%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Bone pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Musculoskeletal pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Neck pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    1 / 28 (3.57%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    1
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    4 / 28 (14.29%)
    5 / 30 (16.67%)
    1 / 13 (7.69%)
    3 / 23 (13.04%)
    4 / 28 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    5
    6
    1
    4
    6
    1
    Urinary tract infection
         subjects affected / exposed
    2 / 28 (7.14%)
    2 / 30 (6.67%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    2 / 28 (7.14%)
    2 / 12 (16.67%)
         occurrences all number
    2
    2
    0
    2
    2
    2
    Influenza
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    0 / 28 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    2
    0
    0
    1
    0
    2
    Sinusitis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    1 / 28 (3.57%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    1
    1
    1
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 30 (3.33%)
    0 / 13 (0.00%)
    1 / 23 (4.35%)
    1 / 28 (3.57%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    1
    1
    1
    Bronchitis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    2 / 23 (8.70%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    2
    0
    1
    Abscess neck
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Carbuncle
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    1 / 13 (7.69%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Conjunctivitis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Infected skin ulcer
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Oral candidiasis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Skin infection
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 30 (0.00%)
    0 / 13 (0.00%)
    0 / 23 (0.00%)
    0 / 28 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    2

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    30 Sep 2016
    • Duration of stable ursodeoxycholic acid (UDCA) use was increased from 6 months to 12 months prior to screening to ensure stability of UDCA-induced alkaline phosphatase (ALP) reductions. • Clarification was added that adults with nonalcoholic steatohepatitis should be excluded. • Additional visits were added for safety monitoring during the OLE phase. • New safety and pharmacokinetic (PK) data from nonclinical studies were added. • The risk/benefit assessment was updated with data from a phase 1 study (GS-US-402-1851) reinforcing the positive benefit/risk ratio. • Magnetic resonance elastography was added as an exploratory measure of liver stiffness. • Inclusion criteria for platelet count, albumin, and creatinine clearance (CLcr) were revised to further ensure adults with advanced liver disease would not be enrolled. • Changes to restrictions on concomitant medications were made based on additional drug-drug interaction study data. • Guidelines for drug-induced liver injury (DILI) monitoring and study drug stopping rules were clarified, and creatine phosphokinase (CPK) testing was added. • Preclinical embryofetal toxicity data were added. Requirements for pregnancy testing and contraceptive use were updated.
    21 Dec 2016
    • Final toxicology data were added to support dosing of subjects beyond 12 weeks. • Inclusion criteria were revised to remove CLcr, since serum creatinine was a more appropriate criterion to use in this population. • Concomitant medication guidelines were clarified regarding timing of use of bile acid sequestrants. • Requirements for cirrhosis assessment at screening were clarified. • Frequency of pregnancy testing during the OLE phase was increased.
    09 Feb 2017
    • Elevated ALP as a criterion for close observation for drug-induced liver injury (DILI) was removed, since elevated ALP is one of the characteristics of PBC. Criteria for close observation were clarified.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    04 Sep 2019
    The study was terminated because of the availability of alternate therapies for primary biliary cholangitis (PBC).
    -

    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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    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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