Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43853   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Phase 4, Double Blind, Randomized, Placebo Controlled, Multicenter Study Evaluating the Effect of Obeticholic Acid on Clinical Outcomes in Subjects with Primary Biliary Cholangitis. The COBALT Study Clinical Outcomes with Obeticholic Acid in Liver Treatment (COBALT)

    Summary
    EudraCT number
    2014-005012-42
    Trial protocol
    HU   LT   AT   BE   DK   FI   GB   EE   ES   NL   FR   DE   BG   PT  
    Global end of trial date
    23 Dec 2021

    Results information
    Results version number
    v1(current)
    This version publication date
    05 Jan 2023
    First version publication date
    05 Jan 2023
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    747-302
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02308111
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Intercept Pharmaceuticals, Inc.
    Sponsor organisation address
    305 Madison Avenue, Morristown, New Jersey, United States, 07960
    Public contact
    Steven Lauder, Intercept Pharmaceuticals, Inc., steven.lauder@interceptpharma.com
    Scientific contact
    Medical Information, Intercept Pharmaceuticals, Inc., medinfo@interceptpharma.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
    28 Sep 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Dec 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Dec 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The objective of this study is to assess the long-term efficacy of obeticholic acid (OCA) compared to placebo, in conjunction with the established local standard of care, on clinical outcomes in participants with primary biliary cholangitis (PBC) as measured by time to the first occurrence of any of the following adjudicated events, derived as composite event endpoints of death (all-cause), liver transplant, model of end-stage liver disease (MELD) ≥15, uncontrolled ascites, or hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy, or spontaneous bacterial peritonitis; and as expanded composite event endpoints (including events mentioned above with additional events of MELD-Na ≥15, portal hypertension syndromes, progression to decompensated liver disease, and Progression to clinical evidence of portal hypertension without decompensation).
    Protection of trial subjects
    This study was conducted in accordance with the International Council on Harmonisation (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the countries in which the study was conducted.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    26 Dec 2014
    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: 61
    Country: Number of subjects enrolled
    Argentina: 38
    Country: Number of subjects enrolled
    Canada: 31
    Country: Number of subjects enrolled
    Australia: 16
    Country: Number of subjects enrolled
    Hong Kong: 9
    Country: Number of subjects enrolled
    Israel: 12
    Country: Number of subjects enrolled
    Korea, Republic of: 6
    Country: Number of subjects enrolled
    Switzerland: 7
    Country: Number of subjects enrolled
    Chile: 3
    Country: Number of subjects enrolled
    Brazil: 3
    Country: Number of subjects enrolled
    Mexico: 1
    Country: Number of subjects enrolled
    Turkey: 2
    Country: Number of subjects enrolled
    Netherlands: 11
    Country: Number of subjects enrolled
    Poland: 19
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    Sweden: 4
    Country: Number of subjects enrolled
    United Kingdom: 26
    Country: Number of subjects enrolled
    Austria: 1
    Country: Number of subjects enrolled
    Belgium: 3
    Country: Number of subjects enrolled
    Denmark: 9
    Country: Number of subjects enrolled
    Estonia: 4
    Country: Number of subjects enrolled
    Finland: 5
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Germany: 9
    Country: Number of subjects enrolled
    Hungary: 10
    Country: Number of subjects enrolled
    Italy: 24
    Country: Number of subjects enrolled
    Lithuania: 9
    Worldwide total number of subjects
    334
    EEA total number of subjects
    119
    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)
    280
    From 65 to 84 years
    54
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    A total of 334 participant were randomised into the study. The study was terminated early as the Data Monitoring Committee made the recommendation to not pursue further enrollment given the lack of feasibility for this post-marketing study as designed. At termination, the study randomised <80% of planned enrollment.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Obeticholic Acid
    Arm description
    Participants received obeticholic acid (OCA) 5 mg for a minimum of 3 months and titrating up to 10 mg for the remainder of the study (based on tolerability and Child-Pugh [CP] Score). Non-cirrhotic and classified as CP Class A: 5 mg tablet of OCA once daily, titrating up to a maximum of 10 mg OCA once daily based on tolerability at 3 months for the duration of the study (majority of participants). Cirrhotic and classified as CP Class B and C: 5 mg tablet of OCA once weekly for at least 3 months, titrating up to a maximum dose and frequency of 10 mg twice weekly based on tolerability and biochemical response for the duration of the study.
    Arm type
    Experimental

    Investigational medicinal product name
    Obeticholic Acid
    Investigational medicinal product code
    747-302
    Other name
    6alpha-ethylchenodeoxycholic acid (6-ECDCA), INT-747
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Non-cirrhotic and classified as CP Class A: 5 mg tablet of OCA once daily, titrating up to a maximum of 10 mg OCA once daily based on tolerability at 3 months for the duration of the study (majority of participants). Cirrhotic and classified as CP Class B and C: 5 mg tablet of OCA once weekly for at least 3 months, subsequently titrating up to a maximum dose and frequency of 10 mg OCA twice weekly based on tolerability and biochemical response for the duration of the study.

    Arm title
    Placebo
    Arm description
    Participants received one tablet daily (or a lower frequency depending on CP score) for the remainder of the study.
    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
    Participant received one tablet of placebo daily (or a lower frequency depending on CP score) for the remainder of the study.

    Number of subjects in period 1
    Obeticholic Acid Placebo
    Started
    168
    166
    Completed
    0
    0
    Not completed
    168
    166
         Adverse event, serious fatal
    9
    6
         Physician decision
    6
    17
         Liver transplant
    3
    3
         Study Terminated by Sponsor
    72
    61
         Site closure
    3
    3
         Consent withdrawn by subject
    28
    38
         Initiated Commercial OCALIVA
    6
    8
         Adverse event, non-fatal
    31
    19
         Liver transplant waitlist
    2
    2
         Non-compliance with study drug
    3
    1
         Lost to follow-up
    2
    7
         COVID-19 pandemic limitation
    2
    -
         Early termination as subject not met criteria
    1
    -
         Protocol deviation
    -
    1

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Obeticholic Acid
    Reporting group description
    Participants received obeticholic acid (OCA) 5 mg for a minimum of 3 months and titrating up to 10 mg for the remainder of the study (based on tolerability and Child-Pugh [CP] Score). Non-cirrhotic and classified as CP Class A: 5 mg tablet of OCA once daily, titrating up to a maximum of 10 mg OCA once daily based on tolerability at 3 months for the duration of the study (majority of participants). Cirrhotic and classified as CP Class B and C: 5 mg tablet of OCA once weekly for at least 3 months, titrating up to a maximum dose and frequency of 10 mg twice weekly based on tolerability and biochemical response for the duration of the study.

    Reporting group title
    Placebo
    Reporting group description
    Participants received one tablet daily (or a lower frequency depending on CP score) for the remainder of the study.

    Reporting group values
    Obeticholic Acid Placebo Total
    Number of subjects
    168 166 334
    Age categorical
    Units: Subjects
        Between 18 and 65 years
    140 140 280
        >=65 years
    28 26 54
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    53.4 ± 10.28 53.9 ± 10.41 -
    Gender categorical
    Units: Subjects
        Female
    151 149 300
        Male
    17 17 34
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    24 18 42
        Not Hispanic or Latino
    138 139 277
        Unknown or Not Reported
    6 9 15
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    1 2 3
        Asian
    11 9 20
        Native Hawaiian or Other Pacific Islander
    0 0 0
        Black or African American
    2 2 4
        White
    146 143 289
        More than one race
    4 1 5
        Unknown or Not Reported
    4 9 13

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Obeticholic Acid
    Reporting group description
    Participants received obeticholic acid (OCA) 5 mg for a minimum of 3 months and titrating up to 10 mg for the remainder of the study (based on tolerability and Child-Pugh [CP] Score). Non-cirrhotic and classified as CP Class A: 5 mg tablet of OCA once daily, titrating up to a maximum of 10 mg OCA once daily based on tolerability at 3 months for the duration of the study (majority of participants). Cirrhotic and classified as CP Class B and C: 5 mg tablet of OCA once weekly for at least 3 months, titrating up to a maximum dose and frequency of 10 mg twice weekly based on tolerability and biochemical response for the duration of the study.

    Reporting group title
    Placebo
    Reporting group description
    Participants received one tablet daily (or a lower frequency depending on CP score) for the remainder of the study.

    Subject analysis set title
    Obeticholic Acid (ITT)
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    Intent-to-Treat (ITT) population consisted of all randomized participants who received at least 1 dose of OCA.

    Subject analysis set title
    Placebo (ITT)
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    ITT population consisted of all randomized participants who received at least 1 dose of placebo.

    Subject analysis set title
    Obeticholic Acid (Safety)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The safety population consisted of all participants who received any amount of OCA. Treatment assignment based on the treatment received before any initiation of commercial OCA.

    Subject analysis set title
    Placebo (Safety)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The safety population consisted of all participants who received any amount of placebo.

    Subject analysis set title
    Obeticholic Acid (PK)
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    The pharmacokinetic (PK) population consisted of all OCA participants who had at least 1 confirmed fasted analyzable sample. Participants fasted for approximately 8 hours prior to the visit and had no major protocol deviations that potentially affected exposure levels.

    Primary: Time to the First Occurrence of Composite Endpoint

    Close Top of page
    End point title
    Time to the First Occurrence of Composite Endpoint
    End point description
    To assess the effect of OCA, compared to placebo in conjunction with the established local standard of care, on clinical outcomes in participants with PBC as measured by time to the first occurrence of any of the following adjudicated events, derived as a composite event endpoint of death, liver transplant, model of end-stage liver disease (MELD) ≥15, uncontrolled ascites, or hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy (as defined by a West Haven score of >=2), or spontaneous bacterial peritonitis. The clinical events distribution was estimated using the Kaplan-Meier methodology. Point estimates and 95% confidence intervals (CIs) for the clinical events distribution percentiles (25th and 50th) are provided. Here 9999 represents the data that was not calculable due to insufficient clinical events. The 95% CI limits were not estimable due to an insufficient number of participants with clinical events, as indicated by 9999.
    End point type
    Primary
    End point timeframe
    Time to accrue approximately 127 primary endpoint events, up to End of Study (EOS)
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: days
    number (confidence interval 95%)
        25th Percentile
    1092 (670 to 1464)
    970 (688 to 1342)
        50th Percentile
    9999 (1910 to 9999)
    9999 (9999 to 9999)
    Statistical analysis title
    Time to the First Occurrence of Composite Endpoint
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.954
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.68
         upper limit
    1.51

    Primary: Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint)

    Close Top of page
    End point title
    Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint)
    End point description
    Primary clinical outcome event is the first occurrence of the following events: death, liver transplant, MELD score >=15 (MELD-Na score >=12 baseline), MELD-Na score >=15 (MELD-Na score <12 baseline), hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy, spontaneous bacterial peritonitis (confirmed by diagnostic paracentesis), or bacterial empyema, uncontrolled or refractory ascites (requiring large volume paracentesis), portal hypertension syndromes, progression to decompensated liver disease, and progression to clinical evidence of portal hypertension without decompensation (for participants without decompensation or clinical evidence of portal hypertension at baseline). 71 endpoint events were observed in the OCA arm, and 80 were observed in the Placebo arm. The clinical events distribution was estimated using the Kaplan-Meier methodology. Point estimates and 95% CIs for the clinical events distribution percentiles (25th and 50th) are provided.
    End point type
    Primary
    End point timeframe
    Time to accrue approximately 127 primary endpoint events, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168 [1]
    166 [2]
    Units: days
    number (confidence interval 95%)
        25th Percentile
    370 (261 to 638)
    450 (358 to 569)
        50th Percentile
    1827 (1198 to 9999)
    1102 (841 to 9999)
    Notes
    [1] - 9999 = Upper 95% CI not estimated due to an insufficient number of participants with clinical events
    [2] - 9999 = Upper 95% CI not estimated due to an insufficient number of participants with clinical events
    Statistical analysis title
    Primary Clinical Event (Expanded Endpoint)
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.304
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.84
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.61
         upper limit
    1.16

    Secondary: Time to First Occurrence of Fatal Event (All-Cause)

    Close Top of page
    End point title
    Time to First Occurrence of Fatal Event (All-Cause)
    End point description
    The results represent the ratio of OCA to placebo. The fatal events distribution was estimated using the Kaplan-Meier methodology. Point estimates and 95% CIs for the fatal events distribution percentiles (25th and 50th) are provided. Here 9999 represents the data that was not calculable due to insufficient clinical events. The 95% CI limits were not estimable due to an insufficient number of participants with clinical events, as indicated by 9999.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomisation until the date of death from any cause, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: days
    number (confidence interval 95%)
        25th Percentile
    9999 (9999 to 9999)
    9999 (9999 to 9999)
        50th Percentile
    9999 (9999 to 9999)
    9999 (9999 to 9999)
    Statistical analysis title
    Time to First Occurrence Fatal Event (All-Cause)
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.568
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.26
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.57
         upper limit
    2.78

    Secondary: Time to First Occurrence of Liver Transplant

    Close Top of page
    End point title
    Time to First Occurrence of Liver Transplant
    End point description
    The effect of OCA compared to placebo on time to occurrence of a liver transplant was assessed. The results represented the ratio of OCA to placebo. A hazard ratio <1 indicated an advantage for OCA. The event of interest was summarized through Cumulative Incidence Function (CIF) estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomisation until the date of first documented liver transplant or date of death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.18 (0.11 to 0.26)
    0.16 (0.09 to 0.23)
    Statistical analysis title
    Time to First Occurrence of Liver Transplant
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.769
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.59
         upper limit
    2.07

    Secondary: Time to First Occurrence of Hospitalization Due to Hepatic Events

    Close Top of page
    End point title
    Time to First Occurrence of Hospitalization Due to Hepatic Events
    End point description
    Hospitalization events include new onset or recurrent variceal bleed, hepatic encephalopathy (as defined by a West Haven score of >=2), spontaneous bacterial peritonitis (confirmed by diagnostic paracentesis OR presence of >250/mm^3 polymorph leucocytes [PMNs] in the ascitic fluid), bacterial empyema is confirmed by diagnostic thoracentesis OR presence of >250/mm^3 PMNs in the pleural fluid. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomisation until the date of hospitalization, liver transplant or death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.11 (0.06 to 0.17)
    0.18 (0.10 to 0.26)
    Statistical analysis title
    Occurrence of Hospitalization Due to Hepatic Event
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.599
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.84
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.42
         upper limit
    1.67

    Secondary: Time to First Occurrence of Uncontrolled or Refractory Ascites

    Close Top of page
    End point title
    Time to First Occurrence of Uncontrolled or Refractory Ascites
    End point description
    Uncontrolled or refractory ascites are defined as diuretic-resistant ascites requiring large-volume paracentesis. The effect of OCA compared to placebo on time to the first occurrence of uncontrolled or refractory ascites was assessed. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of first documented uncontrolled or refractory ascites, liver transplant, or date of death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.02 (0.01 to 0.05)
    0.04 (0.01 to 0.08)
    Statistical analysis title
    Occurrence of Uncontrolled or Refractory Ascites
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.745
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.78
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.18
         upper limit
    3.43

    Secondary: Time to First Occurrence of MELD Score ≥15

    Close Top of page
    End point title
    Time to First Occurrence of MELD Score ≥15
    End point description
    The MELD score is useful in assessing participants with significant decompensation, and the MELD score is now used by the United Network for Organ Sharing in the United States and Eurotransplants to manage organ allocation for liver transplantation. The MELD score is derived from the participant’s serum total bilirubin, serum creatinine, and International Normalized Ratio (INR), as appropriate, to predict survival. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of first documented MELD Score ≥15, liver transplant or date of death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.13 (0.07 to 0.19)
    0.18 (0.11 to 0.25)
    Statistical analysis title
    Time to First Occurrence of MELD Score ≥15
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.437
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.78
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.43
         upper limit
    1.44

    Secondary: Time To First Occurrence Of Severe Decompensating Events of Expanded Composite Endpoint

    Close Top of page
    End point title
    Time To First Occurrence Of Severe Decompensating Events of Expanded Composite Endpoint
    End point description
    The first occurrence of the key secondary clinical event refers to the first occurrence of the following events: death, liver transplant, MELD-Na score >=15 if MELD-Na< 12 at baseline, MELD score >=15 if MELD-Na >=12 at baseline, uncontrolled or refractory ascites, portal hypertension syndromes (hepatorenal syndrome, portopulmonary syndrome, hepatopulmonary syndrome) or hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy, spontaneous bacterial peritonitis, or bacterial empyema. The clinical events distribution was estimated using the Kaplan-Meier methodology. Point estimates and 95% CIs for the clinical events distribution percentiles (25th and 50th) are provided. Here 9999 represents the data that was not calculable due to insufficient clinical events. The 95% CI limits were not estimable due to an insufficient number of participants with clinical events, as indicated by 9999.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: days
    number (confidence interval 95%)
        25th Percentile
    1092 (670 to 1408)
    929 (679 to 1342)
        50th Percentile
    9999 (1910 to 9999)
    9999 (9999 to 9999)
    Statistical analysis title
    Severe Decompensating Events Expanded Composite
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.898
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.69
         upper limit
    1.52

    Secondary: Time To Liver Transplant Or Death (All-Cause)

    Close Top of page
    End point title
    Time To Liver Transplant Or Death (All-Cause)
    End point description
    The effect of OCA compared to placebo on time to liver transplant or death (all-cause) was assessed. The events distribution was estimated using the Kaplan-Meier methodology. Point estimates and 95% CIs for the clinical events distribution percentiles (25th and 50th) are provided. Here 9999 represents the data that was not calculable due to insufficient clinical events. The 95% CI limits were not estimable due to an insufficient number of participants with clinical events, as indicated by 9999
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomisation until the date of first documented liver transplant or date of death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: days
    number (confidence interval 95%)
        25th Percentile
    1580 (1275 to 9999)
    1803 (1206 to 9999)
        50th Percentile
    9999 (9999 to 9999)
    9999 (9999 to 9999)
    Statistical analysis title
    Time To Liver Transplant Or Death (All-cause)
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.594
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.15
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.69
         upper limit
    1.91

    Secondary: Time To Development Of Varix/Varices

    Close Top of page
    End point title
    Time To Development Of Varix/Varices
    End point description
    The effect of OCA compared to placebo on time to development of varix/varices was assessed. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of first documented development of varix/varices, liver transplant or death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.07 (0.03 to 0.12)
    0.09 (0.04 to 0.17)
    Statistical analysis title
    Time To Development Of Varix/Varices
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.838
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.91
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.37
         upper limit
    2.24

    Secondary: Time To Liver-Related Death

    Close Top of page
    End point title
    Time To Liver-Related Death
    End point description
    The effect of OCA compared to placebo on time to liver-related death was assessed. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of first liver-related or non-liver-related death, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.03 (0.01 to 0.07)
    0.04 (0.01 to 0.09)
    Statistical analysis title
    Time To Liver-Related Death
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.98
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.26
         upper limit
    4.04

    Secondary: Time To Liver-Related Death Or Liver Transplant

    Close Top of page
    End point title
    Time To Liver-Related Death Or Liver Transplant
    End point description
    The effect of OCA compared to placebo on time to liver-related death or liver transplant was assessed. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of liver transplant, liver-related death or non-liver-related death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.20 (0.13 to 0.29)
    0.19 (0.12 to 0.27)
    Statistical analysis title
    Time To Liver-Related Death Or Liver Transplant
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.933
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.58
         upper limit
    1.83

    Secondary: Time To Liver-Related Death, Liver Transplant, Or MELD Score ≥15

    Close Top of page
    End point title
    Time To Liver-Related Death, Liver Transplant, Or MELD Score ≥15
    End point description
    The effect of OCA compared to placebo on time to liver-related death, liver transplant, or MELD Score ≥15 was assessed. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of liver transplant, liver-related death, non-liver-related death from any cause or MELD Score ≥15, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
        number (confidence interval 95%)
    0.25 (0.17 to 0.33)
    0.29 (0.21 to 0.37)
    Statistical analysis title
    Liver-related death, transplant, MELD Score ≥15
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.468
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.84
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.53
         upper limit
    1.34

    Secondary: Progression To Cirrhosis (for Noncirrhotic Subjects at Baseline)

    Close Top of page
    End point title
    Progression To Cirrhosis (for Noncirrhotic Subjects at Baseline)
    End point description
    When a participant is identified as noncirrhotic at the Baseline and exhibited any signs or symptoms associated with progression to cirrhosis, the participant was assessed by Fibroscan® TE where available. The event of interest was summarized through CIF estimate at 5 years.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomization until the date of cirrhosis, liver transplant or death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    78
    62
    Units: score on a scale
        number (confidence interval 95%)
    0.07 (0.02 to 0.16)
    0.15 (0.06 to 0.27)
    Statistical analysis title
    Progression To Cirrhosis (Noncirrhotic Subject)
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    140
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.19
    Method
    Cochran-Mantel-Haenszel
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.43
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.13
         upper limit
    1.41
    Statistical analysis title
    Progression To Cirrhosis (Noncirrhotic Subject)
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    140
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.178
    Method
    Gray’s Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.43
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.13
         upper limit
    1.41

    Secondary: Time To Occurrence Of Hepatocellular Carcinoma (HCC)

    Close Top of page
    End point title
    Time To Occurrence Of Hepatocellular Carcinoma (HCC)
    End point description
    The effect of OCA compared to placebo on time to occurrence of HCC was assessed. The event of interest was summarized through CIF estimate at 5 years. Here 9999 represents the data that was not calculable due to insufficient clinical events. The 95% CI limits were not estimable due to an insufficient number of participants with clinical events, as indicated by 9999.
    End point type
    Secondary
    End point timeframe
    Time to first occurrence from date of randomisation until the date of HCC diagnosis, liver transplant or death from any cause, whichever came first, up to EOS
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168 [3]
    166
    Units: score on a scale
        number (confidence interval 95%)
    9999 (9999 to 9999)
    0.01 (0 to 0.05)
    Notes
    [3] - 9999 = 95% CI limits not estimated due to an insufficient number of participants with clinical event
    Statistical analysis title
    Time To Occurrence Of Hepatocellular Carcinoma
    Comparison groups
    Obeticholic Acid (ITT) v Placebo (ITT)
    Number of subjects included in analysis
    334
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.443
    Method
    Gray's Test
    Parameter type
    Hazard ratio (HR)
    Point estimate
    0.43
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.05
         upper limit
    3.54

    Secondary: Change From Baseline To Month 24 Of Total Bilirubin

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of Total Bilirubin
    End point description
    Liver biochemistry, which includes total bilirubin, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using mixed model repeated measures (MMRM), including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the interactive web review board (IWRS) as fixed effects and baseline values as a covariate. Month 6: OCA (n=149); Placebo (n=153) Month 12: OCA (n=126); Placebo (n=138) Month 24: OCA (n=97); Placebo (n=101)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: mg/dL
    least squares mean (standard error)
        Month 6
    0.10 ± 0.069
    0.17 ± 0.069
        Month 12
    0.26 ± 0.113
    0.29 ± 0.111
        Month 24
    0.30 ± 0.141
    0.63 ± 0.138
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 24 Of Direct Bilirubin

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of Direct Bilirubin
    End point description
    Liver biochemistry, which includes direct bilirubin, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using MMRM, including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the IWRS as fixed effects and baseline values as a covariate. Month 6: OCA (n=147); Placebo (n=147) Month 12: OCA (n=121); Placebo (n=138) Month 24: OCA (n=96); Placebo (n=97)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: mg/dL
    least squares mean (standard error)
        Month 6
    0.11 ± 0.054
    0.14 ± 0.054
        Month 12
    0.13 ± 0.073
    0.22 ± 0.071
        Month 24
    0.19 ± 0.107
    0.48 ± 0.106
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 24 Of Aspartate Aminotransferase (AST)

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of Aspartate Aminotransferase (AST)
    End point description
    Liver biochemistry, which includes AST, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using MMRM, including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the IWRS as fixed effects and baseline values as a covariate. Month 6: OCA (n=147); Placebo (n=150) Month 12: OCA (n=124); Placebo (n=138) Month 24: OCA (n=97); Placebo (n=102)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: U/L
    least squares mean (standard error)
        Month 6
    -14.5 ± 2.21
    -0.6 ± 2.19
        Month 12
    -11.8 ± 2.36
    -5.4 ± 2.27
        Month 24
    -14.8 ± 2.78
    -6.0 ± 2.72
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 24 Of Alanine Aminotransferase (ALT)

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of Alanine Aminotransferase (ALT)
    End point description
    Liver biochemistry, which includes ALT, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using MMRM, including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the IWRS as fixed effects and baseline values as a covariate. Month 6: OCA (n=149); Placebo (n=153) Month 12: OCA (n=126); Placebo (n=138) Month 24: OCA (n=97); Placebo (n=100)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: U/L
    least squares mean (standard error)
        Month 6
    -24.3 ± 2.62
    -7.4 ± 2.59
        Month 12
    -20.5 ± 3.49
    -12.8 ± 3.37
        Month 24
    -28.5 ± 2.92
    -19.4 ± 2.87
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 24 Of Alkaline Phosphatase (ALP)

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of Alkaline Phosphatase (ALP)
    End point description
    Liver biochemistry, which includes ALP, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using MMRM, including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the IWRS as fixed effects and baseline values as a covariate. Month 6: OCA (n=150); Placebo (n=152) Month 12: OCA (n=126); Placebo (n=138) Month 24: OCA (n=98); Placebo (n=102)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: U/L
    least squares mean (standard error)
        Month 6
    -134.3 ± 10.86
    -37.4 ± 10.85
        Month 12
    -144.8 ± 12.05
    -68.8 ± 11.72
        Month 24
    -156.4 ± 14.93
    -113.1 ± 14.60
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 24 Of Gamma-glutamyl Transferase (GGT)

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of Gamma-glutamyl Transferase (GGT)
    End point description
    Liver biochemistry, which includes GGT, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using MMRM, including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the IWRS as fixed effects and baseline values as a covariate. Month 6: OCA (n=150); Placebo (n=152) Month 12: OCA (n=126); Placebo (n=138) Month 24: OCA (n=98); Placebo (n=102)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: U/L
    least squares mean (standard error)
        Month 6
    -155.5 ± 15.04
    -47.0 ± 14.99
        Month 12
    -152.2 ± 18.24
    -63.4 ± 17.71
        Month 24
    -175.6 ± 22.49
    -127.7 ± 22.04
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 24 Of Albumin

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of Albumin
    End point description
    Liver biochemistry, which includes albumin, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using MMRM, including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the IWRS as fixed effects and baseline values as a covariate. Month 6: OCA (n=150); Placebo (n=153) Month 12: OCA (n=126); Placebo (n=138) Month 24: OCA (n=98); Placebo (n=102)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: g/L
    least squares mean (standard error)
        Month 6
    -0.4 ± 0.19
    -0.1 ± 0.19
        Month 12
    -0.3 ± 0.23
    -0.3 ± 0.22
        Month 24
    -0.1 ± 0.29
    -1.1 ± 0.28
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 24 Of INR

    Close Top of page
    End point title
    Change From Baseline To Month 24 Of INR
    End point description
    The coagulation test, which includes INR, was assessed to evaluate biochemical triggers that would prompt an immediate reevaluation of participants for potential hepatic injury or hepatic decompensation. Analysis was performed using MMRM, including treatment group, time, treatment group by time interaction, and randomization stratification factors as entered in the IWRS as fixed effects and baseline values as a covariate. Month 6: OCA (n=148); Placebo (n=146) Month 12: OCA (n=124); Placebo (n=136) Month 24: OCA (n=93); Placebo (n=98)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 24
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: units on a scale
    least squares mean (standard error)
        Month 6
    0.02 ± 0.014
    0.02 ± 0.014
        Month 12
    0.01 ± 0.009
    0.02 ± 0.008
        Month 24
    0.03 ± 0.014
    0.06 ± 0.014
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of MELD Score

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of MELD Score
    End point description
    The MELD score is useful in assessing participants with significant decompensation, and the MELD score is now used by the United Network for Organ Sharing in the United States and Eurotransplants to manage organ allocation for liver transplantation. The MELD score is derived from the participant’s serum total bilirubin, serum creatinine, and INR, as appropriate, to predict survival. Month 12: OCA (n=121); Placebo (n=134) Month 24: OCA (n=91); Placebo (n=97) Month 36: OCA (n=71); Placebo (n=59) Month 48: OCA (n=48); Placebo (n=34) Month 60: OCA (n=31); Placebo (n=20) Month 72: OCA (n=7); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
    median (inter-quartile range (Q1-Q3))
        Month 12
    0 (-0.60 to 0.60)
    0 (-0.50 to 1.30)
        Month 24
    0 (-1.00 to 0.70)
    0.20 (-0.50 to 1.70)
        Month 36
    0 (-1.00 to 0.50)
    0.60 (-0.50 to 2.90)
        Month 48
    0 (-0.95 to 1.45)
    0.40 (-0.80 to 1.60)
        Month 60
    0 (-1.30 to 0.40)
    0 (-1.20 to 1.85)
        Month 72
    0 (-0.80 to 5.20)
    1.95 (-1.40 to 3.10)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of MELD-Na Score

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of MELD-Na Score
    End point description
    The MELD score is useful in assessing participants with significant decompensation, and the MELD score is now used by the United Network for Organ Sharing in the United States and Eurotransplants to manage organ allocation for liver transplantation. The MELD score is derived from the participant’s serum total bilirubin, serum creatinine, and INR, as appropriate, to predict survival. Month 12: OCA (n=121); Placebo (n=132) Month 24: OCA (n=91); Placebo (n=97) Month 36: OCA (n=71); Placebo (n=59) Month 48: OCA (n=48); Placebo (n=34) Month 60: OCA (n=31); Placebo (n=20) Month 72: OCA (n=6); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
    median (inter-quartile range (Q1-Q3))
        Month 12
    0 (-1.0 to 0.0)
    0 (-1.0 to 1.0)
        Month 24
    0 (-2.0 to 0)
    0 (-1.0 to 1.0)
        Month 36
    0 (-2.0 to 0)
    0 (-1.0 to 2.0)
        Month 48
    0 (-2.0 to 2.0)
    0 (-2.0 to 2.0)
        Month 60
    -1.0 (-3.0 to 0)
    0 (-2.5 to 1.5)
        Month 72
    0 (-2.0 to 0)
    0.5 (-3.5 to 1.5)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of CP Score

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of CP Score
    End point description
    Child-Pugh Score (Pugh 1973, Lucey 1997) was calculated and reported within the electronic data capture (EDC) system based on data entered into the CRF by adding the scores from the 5 factors and could have ranged from 5 to15. A total score of 5 to 6 was considered Grade A (mild, well-compensated disease); 7 to 9 was Grade B (moderate, significant functional compromise); and 10 and above was Grade C (severe, decompensated disease). Month 12: OCA (n=126); Placebo (n=135) Month 24: OCA (n=93); Placebo (n=97) Month 36: OCA (n=71); Placebo (n=57) Month 48: OCA (n=47); Placebo (n=35) Month 60: OCA (n=31); Placebo (n=20) Month 72: OCA (n=7); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
    median (inter-quartile range (Q1-Q3))
        Month 12
    0 (0 to 0)
    0 (0 to 1.0)
        Month 24
    0 (0 to 0)
    0 (0 to 1.0)
        Month 36
    0 (0 to 0)
    0 (0 to 1.0)
        Month 48
    0 (0 to 0)
    0 (0 to 1.0)
        Month 60
    0 (0 to 0)
    0 (0 to 1.0)
        Month 72
    0 (0 to 2.0)
    1.0 (0 to 1.0)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of Mayo Risk Score (MRS)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of Mayo Risk Score (MRS)
    End point description
    Mayo Risk Score (MRS) was calculated and reported within the EDC system. Calculation of MRS included Investigator assessment of peripheral edema and the use of diuretic therapy, which was assessed during the adverse event and concomitant medicine review at the scheduled visits and entered into the CRF, as well as total bilirubin, albumin, and prothrombin time results obtained from the central laboratory data. Month 12: OCA (n=117); Placebo (n=128) Month 24: OCA (n=80); Placebo (n=91) Month 36: OCA (n=65); Placebo (n=53) Month 48: OCA (n=43); Placebo (n=29) Month 60: OCA (n=28); Placebo (n=18) Month 72: OCA (n=6); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
    median (inter-quartile range (Q1-Q3))
        Month 12
    0 (-0.380 to 0.320)
    0.080 (-0.205 to 0.535)
        Month 24
    -0.080 (-0.270 to 0.390)
    0.140 (-0.170 to 0.660)
        Month 36
    -0.040 (-0.410 to 0.280)
    0.050 (-0.240 to 0.650)
        Month 48
    0.030 (-0.430 to 0.620)
    0.210 (-0.240 to 0.810)
        Month 60
    -0.145 (-0.575 to 0.160)
    -0.060 (-0.490 to 0.790)
        Month 72
    -0.130 (-0.460 to 0.320)
    0.990 (0.240 to 1.165)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of Immunoglobulin-M (IgM)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of Immunoglobulin-M (IgM)
    End point description
    Markers of inflammation, which include IgM, were assessed. Month 12: OCA (n=124); Placebo (n=135) Month 24: OCA (n=97); Placebo (n=101) Month 36: OCA (n=72); Placebo (n=59) Month 48: OCA (n=48); Placebo (n=35) Month 60: OCA (n=32); Placebo (n=21) Month 72: OCA (n=7); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: g/L
    median (inter-quartile range (Q1-Q3))
        Month 12
    -0.305 (-0.955 to 0.025)
    -0.160 (-0.720 to 0.200)
        Month 24
    -0.470 (-1.250 to -0.090)
    -0.230 (-0.630 to 0.170)
        Month 36
    -0.700 (-1.440 to -0.025)
    -0.330 (-1.270 to 0)
        Month 48
    -0.525 (-1.615 to 0.005)
    -0.690 (-1.540 to 0.090)
        Month 60
    -0.745 (-1.545 to -0.075)
    -0.350 (-1.050 to -0.150)
        Month 72
    -1.590 (-4.100 to 0.050)
    -0.640 (-1.060 to 0.645)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of C-reactive Protein (CRP)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of C-reactive Protein (CRP)
    End point description
    Markers of inflammation, which include CRP, were assessed. Month 12: OCA (n=127); Placebo (n=137) Month 24: OCA (n=98); Placebo (n=101) Month 36: OCA (n=74); Placebo (n=59) Month 48: OCA (n=51); Placebo (n=36) Month 60: OCA (n=33); Placebo (n=22) Month 72: OCA (n=7); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: mg/L
    median (inter-quartile range (Q1-Q3))
        Month 12
    -0.140 (-1.320 to 1.360)
    0.400 (-0.760 to 2.720)
        Month 24
    -0.280 (-1.210 to 1.110)
    0.290 (-1.450 to 2.950)
        Month 36
    -0.525 (-2.160 to 0.630)
    0.340 (-1.460 to 3.830)
        Month 48
    -0.260 (-1.700 to 2.180)
    -0.180 (-1.200 to 2.025)
        Month 60
    -0.720 (-3.020 to 0.740)
    -0.730 (-2.700 to 1.650)
        Month 72
    -0.100 (-2.740 to 2.570)
    0.765 (-2.630 to 4.715)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of Tumor Necrosis Factor-α (TNF-α)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of Tumor Necrosis Factor-α (TNF-α)
    End point description
    Markers of inflammation, which include TNF-α, were assessed. Month 12: OCA (n=117); Placebo (n=123) Month 24: OCA (n=91); Placebo (n=95) Month 36: OCA (n=66); Placebo (n=56) Month 48: OCA (n=46); Placebo (n=33) Month 60: OCA (n=29); Placebo (n=19) Month 72: OCA (n=6); Placebo (n=3)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: pg/mL
    median (inter-quartile range (Q1-Q3))
        Month 12
    0 (-0.362 to 0.580)
    0.058 (-0.236 to 0.791)
        Month 24
    0.203 (-0.213 to 0.758)
    0.323 (-0.039 to 0.654)
        Month 36
    0.055 (-0.304 to 0.627)
    0.296 (-0.078 to 0.750)
        Month 48
    0.165 (-0.195 to 0.814)
    0.539 (0.113 to 0.918)
        Month 60
    -0.009 (-0.765 to 0.596)
    0.419 (-0.153 to 0.753)
        Month 72
    0.046 (-0.390 to 0.175)
    0.616 (-1.318 to 0.788)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of Fibroblast Growth Factor-19 (FGF-19)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of Fibroblast Growth Factor-19 (FGF-19)
    End point description
    Markers of hepatic fibrosis, which include FGF-19, were assessed. Month 12: OCA (n=124); Placebo (n=132) Month 24: OCA (n=95); Placebo (n=97) Month 36: OCA (n=69); Placebo (n=59) Month 48: OCA (n=47); Placebo (n=34) Month 60: OCA (n=31); Placebo (n=22) Month 72: OCA (n=5); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: pg/mL
    median (inter-quartile range (Q1-Q3))
        Month 12
    73.50 (2.85 to 208.00)
    -2.80 (-57.65 to 36.50)
        Month 24
    72.00 (1.00 to 211.00)
    -0.40 (-63.00 to 61.40)
        Month 36
    39.90 (-17.70 to 132.00)
    -2.00 (-51.00 to 50.80)
        Month 48
    56.00 (-24.00 to 219.00)
    -9.45 (-59.90 to 39.30)
        Month 60
    14.40 (-30.00 to 183.00)
    -1.45 (-83.40 to 156.00)
        Month 72
    -106.90 (-172.30 to -1.00)
    -62.00 (-239.00 to -26.50)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of Cytokeratin-18 (CK-18)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of Cytokeratin-18 (CK-18)
    End point description
    Markers of inflammation, which include CK-18, were assessed. Month 12: OCA (n=126); Placebo (n=133) Month 24: OCA (n=97); Placebo (n=100) Month 36: OCA (n=72); Placebo (n=59) Month 48: OCA (n=50); Placebo (n=35) Month 60: OCA (n=33); Placebo (n=21) Month 72: OCA (n=7); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: U/L
    median (inter-quartile range (Q1-Q3))
        Month 12
    -34.450 (-138.010 to 45.800)
    9.100 (-78.540 to 123.770)
        Month 24
    -57.660 (-158.960 to 8.470)
    17.545 (-54.630 to 165.835)
        Month 36
    -45.935 (-171.225 to 18.715)
    6.680 (-112.010 to 76.110)
        Month 48
    -81.035 (-158.900 to 33.550)
    0.290 (-130.710 to 79.410)
        Month 60
    -117.660 (-232.770 to 0)
    -56.390 (-159.780 to 77.020)
        Month 72
    -182.590 (-207.020 to 17.060)
    -32.745 (-199.555 to 78.070)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of 7α-hydroxy-4-cholesten-3-one (C4)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of 7α-hydroxy-4-cholesten-3-one (C4)
    End point description
    Markers of hepatic fibrosis, which include C4, were assessed. Month 12: OCA (n=116); Placebo (n=132) Month 24: OCA (n=96); Placebo (n=98) Month 36: OCA (n=71); Placebo (n=60) Month 48: OCA (n=49); Placebo (n=34) Month 60: OCA (n=33); Placebo (n=22) Month 72: OCA (n=7); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: ng/mL
    median (inter-quartile range (Q1-Q3))
        Month 12
    -2.222 (-8.110 to 0.002)
    -0.250 (-2.691 to 1.523)
        Month 24
    -3.700 (-10.119 to -0.885)
    -0.861 (-5.500 to 0.900)
        Month 36
    -3.826 (-9.380 to 0.182)
    -1.225 (-5.620 to 1.161)
        Month 48
    -4.220 (-9.870 to 0)
    -1.168 (-12.240 to 1.691)
        Month 60
    -3.250 (-10.334 to -0.110)
    -1.161 (-9.591 to 0.660)
        Month 72
    -7.500 (-10.914 to -0.690)
    -0.947 (-15.281 to -0.277)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of Enhanced Liver Fibrosis (ELF)

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of Enhanced Liver Fibrosis (ELF)
    End point description
    Liver fibrosis was assessed using ELF test. The ELF test assessed: hyaluronic acid, procollagen3 N-terminal peptide, and a tissue inhibitor of metalloproteinase 1. Month 12: OCA (n=126); Placebo (n=127) Month 24: OCA (n=95); Placebo (n=95) Month 36: OCA (n=71); Placebo (n=59) Month 48: OCA (n=48); Placebo (n=34) Month 60: OCA (n=33); Placebo (n=21) Month 72: OCA (n=7); Placebo (n=4)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: score on a scale
    median (inter-quartile range (Q1-Q3))
        Month 12
    0.10 (-0.50 to 0.60)
    0.10 (-0.20 to 0.70)
        Month 24
    0 (-0.70 to 0.60)
    0.20 (-0.40 to 1.00)
        Month 36
    -0.20 (-0.90 to 0.70)
    0 (-0.60 to 0.70)
        Month 48
    0.25 (-0.45 to 0.80)
    0.10 (-0.70 to 1.10)
        Month 60
    -0.20 (-1.20 to 0.60)
    0.10 (-1.00 to 1.00)
        Month 72
    0 (-0.80 to 2.80)
    -0.20 (-1.10 to 0.45)
    No statistical analyses for this end point

    Secondary: Change From Baseline To Month 72 Of Liver Stiffness - Transient Elastography

    Close Top of page
    End point title
    Change From Baseline To Month 72 Of Liver Stiffness - Transient Elastography
    End point description
    Hepatic stiffness was measured using non-invasive transient Elastography with a Fibroscan® TE device. Month 12: OCA (n=88); Placebo (n=90) Month 24: OCA (n=67); Placebo (n=67) Month 36: OCA (n=49); Placebo (n=41) Month 48: OCA (n=31); Placebo (n=19) Month 60: OCA (n=19); Placebo (n=14) Month 72: OCA (n=5); Placebo (n=3)
    End point type
    Secondary
    End point timeframe
    Baseline up to Month 72
    End point values
    Obeticholic Acid (ITT) Placebo (ITT)
    Number of subjects analysed
    168
    166
    Units: kPa
    median (inter-quartile range (Q1-Q3))
        Month 12
    0.25 (-2.40 to 4.05)
    0.90 (-1.80 to 7.30)
        Month 24
    -0.60 (-2.60 to 3.60)
    1.50 (-2.20 to 15.60)
        Month 36
    0.30 (-3.40 to 5.50)
    2.00 (-1.60 to 9.10)
        Month 48
    0.70 (-2.20 to 4.00)
    1.00 (-2.80 to 11.90)
        Month 60
    0.20 (-2.50 to 6.00)
    -1.35 (-4.70 to 1.90)
        Month 72
    -2.40 (-2.50 to 2.70)
    3.20 (-2.20 to 8.70)
    No statistical analyses for this end point

    Secondary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Close Top of page
    End point title
    Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
    End point description
    An adverse event (AE) was defined as any unfavorable and unintended sign (for example, including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product or procedure, whether or not considered related to the medicinal product or procedure, which occurred during the course of the clinical study. TEAEs were defined as AEs that occurred on or after the date and time of study drug administration, or those that first occurred before dosing but worsened in frequency or severity after study drug administration. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the ‘Reported Adverse Events’ Section.
    End point type
    Secondary
    End point timeframe
    Baseline up to the last IP dose plus 30 days and prior to commercial OCA initiation date
    End point values
    Obeticholic Acid (Safety) Placebo (Safety)
    Number of subjects analysed
    168
    166
    Units: participants
        TEAEs
    162
    158
        SAE
    53
    53
    No statistical analyses for this end point

    Secondary: Pharmacokinetic (PK) Population: Fasting Trough Concentrations Of OCA By Dose Regimen

    Close Top of page
    End point title
    Pharmacokinetic (PK) Population: Fasting Trough Concentrations Of OCA By Dose Regimen
    End point description
    The fasting trough PK concentrations of OCA of different dose regimens taken throughout the study are reported. Here 9999 represents data that were not available.
    End point type
    Secondary
    End point timeframe
    Months 3, 6, 9, 12, 24, 36, 48, and 60
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        5 mg QD (Month 3)
    119 ± 185
        5 mg QD (Month 6)
    102 ± 186
        5 mg QD (Month 9)
    175 ± 33.0
        5 mg QD (Month 12)
    117 ± 153
        5 mg QD (Month 24)
    113 ± 121
        5 mg QD (Month 36)
    84.5 ± 169
        5 mg QD (Month 48)
    203 ± 181
        5 mg QD (Month 60)
    7.96 ± 173
        5 mg QOD (Month 3)
    123 ± 191
        5 mg QOD (Month 6)
    103 ± 232
        5 mg QOD (Month 9)
    396 ± 9999
        5 mg QOD (Month 12)
    103 ± 97.2
        5 mg QOD (Month 24)
    134 ± 17.1
        5 mg QOD (Month 48)
    307 ± 9999
        5 mg QW (Month 3)
    57.3 ± 199
        5 mg QW (Month 6)
    31.0 ± 125
        5 mg QW (Month 12)
    73.5 ± 302
        5 mg QW (Month 24)
    171 ± 109
        5 mg QW (Month 36)
    245 ± 34.8
        5 mg QW (Month 48)
    141 ± 216
        5 mg Q2W (Month 3)
    61.9 ± 54.7
        5 mg Q2W (Month 6)
    108 ± 57.1
        5 mg Q2W (Month 12)
    187 ± 30.8
        5 mg Q2W (Month 24)
    208 ± 61.0
        5 mg Q2W (Month 36)
    225 ± 159
        5 mg Q2W (Month 48)
    502 ± 59.6
        5 mg Q2W (Month 60)
    7.32 ± 9999
        5 mg other regimens (Month 3)
    50.3 ± 1730
        5 mg other regimens (Month 6)
    79.6 ± 68.2
        5 mg other regimens (Month 12)
    20.6 ± 500
        5 mg other regimens (Month 24)
    91.1 ± 236
        10 mg QD (Month 6)
    126 ± 161
        10 mg QD (Month 9)
    51.1 ± 167
        10 mg QD (Month 12)
    86.9 ± 188
        10 mg QD (Month 24)
    85.6 ± 152
        10 mg QD (Month 36)
    79.9 ± 143
        10 mg QD (Month 48)
    81.2 ± 161
        10 mg QD (Month 60)
    48.6 ± 323
        10 mg QOD (Month 12)
    102 ± 576
        10 mg QOD (Month 24)
    25.5 ± 9999
        10 mg QOD (Month 48)
    9.08 ± 9999
        10 mg Q2W (Month 6)
    538 ± 9999
        10 mg Q2W (Month 9)
    566 ± 9999
        10 mg Q2W (Month 12)
    41.7 ± 45.6
        10 mg Q2W (Month 24)
    62.5 ± 35.9
        10 mg Q2W (Month 36)
    96.3 ± 132
        10 mg Q2W (Month 48)
    70.9 ± 218
        10 mg Q2W (Month 60)
    83.8 ± 9999
        10 mg Q3D (Month 12)
    0.861 ± 9999
    No statistical analyses for this end point

    Secondary: PK Population: Serial Concentration of OCA By Dose Regimen

    Close Top of page
    End point title
    PK Population: Serial Concentration of OCA By Dose Regimen
    End point description
    On Month 9, blood samples were collected at predose, 0.5 h, 0.75 h, 1 h, 1.5 h, 2 h, 2.5 h, 3 h, 4h, 5 h, and 6 h and PK serial concentrations at different dose regimen are reported. Here 9999 represents data that were not available.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    13
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        5 mg QD (Predose)
    61.2 ± 315
        5 mg QD (0.5 h)
    109 ± 66.1
        5 mg QD (0.75 h)
    158 ± 35.6
        5 mg QD (1 h)
    173 ± 56.4
        5 mg QD (1.5 h)
    137 ± 53.2
        5 mg QD (2 h)
    100 ± 100
        5 mg QD (2.5 h)
    90.1 ± 93.0
        5 mg QD (3 h)
    64.7 ± 47.1
        5 mg QD (4 h)
    71.6 ± 179
        5 mg QD (5 h)
    193 ± 150
        5 mg QD (6 h)
    199 ± 203
        5 mg QOD (Predose)
    396 ± 9999
        5 mg QOD (0.5 h)
    236 ± 9999
        5 mg QOD (0.75 h)
    211 ± 9999
        5 mg QOD (1 h)
    217 ± 9999
        5 mg QOD (1.5 h)
    232 ± 9999
        5 mg QOD (2 h)
    243 ± 9999
        5 mg QOD (2.5 h)
    240 ± 9999
        5 mg QOD (3 h)
    171 ± 9999
        5 mg QOD (4 h)
    117 ± 9999
        5 mg QOD (5 h)
    282 ± 9999
        5 mg QOD (6 h)
    544 ± 9999
        10 mg QD (Predose)
    64.2 ± 139
        10 mg QD (0.5 h)
    142 ± 55.8
        10 mg QD (0.75 h)
    176 ± 55.5
        10 mg QD (1 h)
    196 ± 65.5
        10 mg QD (1.5 h)
    254 ± 64.0
        10 mg QD (2 h)
    233 ± 54.0
        10 mg QD (2.5 h)
    206 ± 80.2
        10 mg QD (3 h)
    217 ± 90.7
        10 mg QD (4 h)
    151 ± 74.4
        10 mg QD (5 h)
    180 ± 80.0
        10 mg QD (6 h)
    214 ± 77.9
        10 mg Q2W (Predose)
    370 ± 65.8
        10 mg Q2W (0.5 h)
    490 ± 52.6
        10 mg Q2W (P0.75 h)
    593 ± 68.2
        10 mg Q2W (1 h)
    633 ± 69.5
        10 mg Q2W (1.5 h)
    425 ± 9999
        10 mg Q2W (2 h)
    769 ± 64.5
        10 mg Q2W (2.5 h)
    716 ± 75.1
        10 mg Q2W (3 h)
    602 ± 77.2
        10 mg Q2W (4 h)
    422 ± 116
        10 mg Q2W (5 h)
    758 ± 84.4
        10 mg Q2W (6 h)
    728 ± 169
    No statistical analyses for this end point

    Secondary: PK Population: Area Under The Concentration-Time Curve (AUC) From 0 to 6 Hours Post-dose (AUC0-6h) Of Participants Who Received 5 mg QD OCA and With CP Score=Non-Cirrhotic (NC)

    Close Top of page
    End point title
    PK Population: Area Under The Concentration-Time Curve (AUC) From 0 to 6 Hours Post-dose (AUC0-6h) Of Participants Who Received 5 mg QD OCA and With CP Score=Non-Cirrhotic (NC)
    End point description
    On Month 9, blood samples were collected at predose, 0.5h, 0.75 h, 1 h, 1.5 h, 2 h, 2.5 h, 3 h, 4h, 5 h, and 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [4]
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    758 ± 9999
    Notes
    [4] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: AUC From 0 to 24 Hours Post-dose (AUC0-24h) Of Participants Who Received 5 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: AUC From 0 to 24 Hours Post-dose (AUC0-24h) Of Participants Who Received 5 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose, 0.5h, 0.75 h, 1 h, 1.5 h, 2 h, 2.5 h, 3 h, 4h, 5 h, and 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [5]
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    3710 ± 9999
    Notes
    [5] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Maximum Observed Concentration (Cmax) Of Participants Who Received 5mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Maximum Observed Concentration (Cmax) Of Participants Who Received 5mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    317 ± 9999
    No statistical analyses for this end point

    Secondary: PK Population: Time to Cmax (Tmax) Of Participants Who Received 5mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Time to Cmax (Tmax) Of Participants Who Received 5mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: hours
        median (full range (min-max))
    6.0 (6.0 to 6.0)
    No statistical analyses for this end point

    Secondary: PK Population: Metabolite to Parent Ratio of AUC0-6h (MRAUC) Of Participants Who Received 5mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Metabolite to Parent Ratio of AUC0-6h (MRAUC) Of Participants Who Received 5mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    15.9 ± 9999
    No statistical analyses for this end point

    Secondary: PK Population: Metabolite to Parent Ration of Cmax (MRCmax) Of Participants Who Received 5mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Metabolite to Parent Ration of Cmax (MRCmax) Of Participants Who Received 5mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    8.93 ± 9999
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 5 mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 5 mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    889 ± 133
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 5 mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 5 mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    4040 ± 129
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 5mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 5mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    275 ± 117
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 5mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 5mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: hours
        median (full range (min-max))
    3.38 (0.750 to 6.0)
    No statistical analyses for this end point

    Secondary: PK Population: Concentration at 24 Hours Post-dose (Ctrough) Of Participants Who Received 5mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: Concentration at 24 Hours Post-dose (Ctrough) Of Participants Who Received 5mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    149 ± 22.2
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 5mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 5mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    8.41 ± 50.8
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 5mg QD OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 5mg QD OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with CP Score=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    242 ± 9999
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 5 mg QOD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 5 mg QOD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [6]
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    1480 ± 9999
    Notes
    [6] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 5 mg QOD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 5 mg QOD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with CP Score=NC
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    9940 ± 9999
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 5mg QOD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 5mg QOD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [7]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    544 ± 9999
    Notes
    [7] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 5mg QOD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 5mg QOD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: hours
        median (full range (min-max))
    6.0 (6.0 to 6.0)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 5mg QOD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 5mg QOD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [8]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    396 ± 9999
    Notes
    [8] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 5mg QOD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 5mg QOD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [9]
    Units: unit on a scale
        geometric mean (geometric coefficient of variation)
    26.6 ± 9999
    Notes
    [9] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 5mg QOD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 5mg QOD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [10]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    15.8 ± 9999
    Notes
    [10] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    1090 ± 98.4
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    3820 ± 91.7
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    289 ± 74.5
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: hours
        median (full range (min-max))
    1.50 (1.50 to 3.00)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    4
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    50.6 ± 148
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    13.8 ± 151
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With CP Score=NC

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With CP Score=NC
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QOD OCA with CP Score=NC.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    4.02 ± 107
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    1310 ± 11.9
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With CPS Score=A

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With CPS Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    4280 ± 26.1
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    334 ± 6.71
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With CPS Score=A On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A. Month 9

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With CPS Score=A On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A. Month 9
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: hours
        median (full range (min-max))
    1.50 (0.750 to 2.50)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    55.2 ± 414
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With CP Score=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with CP Score=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    2.94 ± 15.5
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With CP Score=A

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With CP Score=A
    End point description
    PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With CP Score=A
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    1.48 ± 21.8
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 10 mg Q2W OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 10 mg Q2W OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with CP Score=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    3770 ± 84.9
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 10 mg Q2W OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 10 mg Q2W OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with CP Score=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    13900 ± 113
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 10 mg Q2W OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 10 mg Q2W OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with CP Score=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    916 ± 101
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 10 mg Q2W OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 10 mg Q2W OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with CP Score=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: hour
        median (full range (min-max))
    4.0 (2.0 to 6.0)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 10 mg Q2W OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 10 mg Q2W OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with CP Score=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [11]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    566 ± 9999
    Notes
    [11] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 10 mg Q2W OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 10 mg Q2W OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with CP Score=B
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    12.6 ± 450
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 10 mg Q2W OCA and With CP Score=B

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 10 mg Q2W OCA and With CP Score=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with CP Score=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    7.13 ± 315
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 5 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 5 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    1170 ± 68.0
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 5 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 5 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    5490 ± 60.2
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 5 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 5 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    410 ± 37.4
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 5 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 5 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: hour
        median (full range (min-max))
    6.0 (6.0 to 6.0)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 5 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 5 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [12]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    174 ± 9999
    Notes
    [12] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 5 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 5 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    13.7 ± 21.1
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 5 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 5 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    7.15 ± 32.3
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 5 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 5 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [13]
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    436 ± 9999
    Notes
    [13] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 5 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 5 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [14]
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    2000 ± 9999
    Notes
    [14] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 5 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 5 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [15]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    143 ± 9999
    Notes
    [15] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 5 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 5 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: hour
        median (full range (min-max))
    0.750 (0.750 to 0.750)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 5 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 5 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    176 ± 47.9
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 5 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 5 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [16]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    5.99 ± 9999
    Notes
    [16] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 5 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 5 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [17]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    2.48 ± 9999
    Notes
    [17] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 5 mg QOD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 5 mg QOD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [18]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    1480 ± 9999
    Notes
    [18] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 5 mg QOD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 5 mg QOD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [19]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    9940 ± 9999
    Notes
    [19] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 5 mg QOD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 5 mg QOD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [20]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    544 ± 9999
    Notes
    [20] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 5 mg QOD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 5 mg QOD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1
    Units: hour
        median (full range (min-max))
    6.0 (6.0 to 6.0)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 5 mg QOD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 5 mg QOD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [21]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    396 ± 9999
    Notes
    [21] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 5 mg QOD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 5 mg QOD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [22]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    26.6 ± 9999
    Notes
    [22] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 5 mg QOD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 5 mg QOD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 5 mg QOD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [23]
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    15.8 ± 9999
    Notes
    [23] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point description
    PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    886 ± 55.6
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    2860 ± 35.5
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=A
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    240 ± 35.1
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: hour
        median (full range (min-max))
    1.50 (1.50 to 3.00)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    4
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    25.8 ± 188
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    6.04 ± 162
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With MELD Category=A

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With MELD Category=A
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=A.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    1.92 ± 50.6
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 10 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    1610 ± 44.6
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 10 mg QD OCA and With MELD Category=B
    End point description
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    5700 ± 48.7
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 10 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    403 ± 39.4
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 10 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: hours
        median (full range (min-max))
    1.50 (0.750 to 2.50)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 10 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    136 ± 14.9
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 10 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    6.73 ± 212
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 10 mg QD OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg QD OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    3
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    3.11 ± 147
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-6h Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: AUC0-6h Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    3770 ± 84.9
    No statistical analyses for this end point

    Secondary: PK Population: AUC0-24h Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: AUC0-24h Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: h*ng/mL
        geometric mean (geometric coefficient of variation)
    13900 ± 113
    No statistical analyses for this end point

    Secondary: PK Population: Cmax Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Cmax Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    916 ± 101
    No statistical analyses for this end point

    Secondary: PK Population: Tmax Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Tmax Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: hour
        median (full range (min-max))
    4.0 (2.0 to 6.0)
    No statistical analyses for this end point

    Secondary: PK Population: Ctrough Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: Ctrough Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [24]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    566 ± 9999
    Notes
    [24] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: MRAUC Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: MRAUC Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    12.6 ± 450
    No statistical analyses for this end point

    Secondary: PK Population: MRCmax Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B

    Close Top of page
    End point title
    PK Population: MRCmax Of Participants Who Received 10 mg Q2W OCA and With MELD Category=B
    End point description
    On Month 9, blood samples were collected at predose to 6 h for PK analysis in participants who received 10 mg Q2W OCA with MELD Category=B.
    End point type
    Secondary
    End point timeframe
    Month 9
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    2
    Units: units on a scale
        geometric mean (geometric coefficient of variation)
    7.13 ± 315
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg QD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg QD OCA
    End point description
    The trough concentration of OCA in the cirrhotic participants who received 5 mg QD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 3, 6, 12, 24, and 48
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [25]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 3
    295 ± 9999
        Month 6
    291 ± 166
        Month 12
    227 ± 127
        Month 24
    127 ± 9999
        Month 48
    849 ± 9999
    Notes
    [25] - No variation was calculated for Months 3, 24, and 48.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg QD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg QD OCA
    End point description
    The trough concentration of OCA in the non-cirrhotic participants who received 5 mg QD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 3, 6, 9, 12, and 24
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [26]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 3
    77.4 ± 157
        Month 6
    55.9 ± 85.1
        Month 9
    127 ± 9999
        Month 12
    96.0 ± 94.7
        Month 24
    45.4 ± 693
    Notes
    [26] - No variation was calculated on Month 9.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg QOD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg QOD OCA
    End point description
    The trough concentration of OCA in the cirrhotic participants who received 5 mg QOD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 6, 12, and 24
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [27]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 6
    1050 ± 9999
        Month 12
    48.0 ± 9999
        Month 24
    134 ± 17.1
    Notes
    [27] - No variation was calculated on Months 6 and 12.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg QOD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg QOD OCA
    End point description
    The trough concentration of OCA in the non-cirrhotic participants who received 5 mg QOD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 3, 6, and 12
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [28]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 3
    97.2 ± 53.9
        Month 6
    85.4 ± 9999
        Month 12
    200 ± 184
    Notes
    [28] - No variation was calculated on Month 6.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg QW OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg QW OCA
    End point description
    The trough concentration of OCA in the cirrhotic participants who received 5 mg QW OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 6 and 12
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [29]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 6
    56.7 ± 9999
        Month 12
    116 ± 395
    Notes
    [29] - No variation was calculated on Month 6.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg QW OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg QW OCA
    End point description
    At month 12, the trough concentration of OCA in the non-cirrhotic participants who received 5 QW QOD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Month 12
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [30]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    179 ± 9999
    Notes
    [30] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg Q2W OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 5 mg Q2W OCA
    End point description
    At Month 6, the trough concentration of OCA in the cirrhotic participants who received 5 mg Q2W OCA was reported.
    End point type
    Secondary
    End point timeframe
    Month 6
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [31]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    131 ± 9999
    Notes
    [31] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg Q2W OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 5 mg Q2W OCA
    End point description
    The trough concentration of OCA in the non-cirrhotic participants who received 5 Q2W QOD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 3, 6, 12, 24, 36, and 48
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [32]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 3
    86.0 ± 9999
        Month 6
    122 ± 9999
        Month 12
    250 ± 9999
        Month 24
    497 ± 9999
        Month 36
    117 ± 9999
        Month 48
    271 ± 9999
    Notes
    [32] - No variation was calculated at all timepoints.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 10 mg QD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 10 mg QD OCA
    End point description
    The trough concentration of OCA in the cirrhotic participants who received 10 mg QD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 6, 9, 12, 24, 36, and 60
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [33]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 6
    142 ± 242
        Month 9
    7.74 ± 9999
        Month 12
    133 ± 489
        Month 24
    396 ± 61.7
        Month 36
    346 ± 9999
        Month 60
    290 ± 9999
    Notes
    [33] - No variation was calculated on Months 9, 36, and 60.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 10 mg QD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 10 mg QD OCA
    End point description
    The trough concentration of OCA in the non-cirrhotic participants who received 10 QD QOD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 6, 9, 12, 24, and 36
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Month 6
    127 ± 601
        Month 9
    74.5 ± 10.6
        Month 12
    73.3 ± 102
        Month 24
    100 ± 216
        Month 36
    98.5 ± 1730
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 10 mg QOD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 10 mg QOD OCA
    End point description
    The trough concentration of OCA in the Cirrhotic participants who received 10 mg QOD OCA was reported.
    End point type
    Secondary
    End point timeframe
    Months 3, 6, 9, 12, 24, 36, 48, and 60
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    149 [34]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    0 ± 0
    Notes
    [34] - No participants was tested for this outcome measure.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 10 mg QOD OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 10 mg QOD OCA
    End point description
    At Month 12, the trough concentration of OCA in the non-cirrhotic participants who received 10 mg OCA QOD was reported.
    End point type
    Secondary
    End point timeframe
    Month 12
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [35]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    26.9 ± 9999
    Notes
    [35] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 10 mg Q2W OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Cirrhotic Participants Who Received 10 mg Q2W OCA
    End point description
    At Month 6, the trough concentration of OCA in the cirrhotic participants who received 10 mg Q2W OCA was reported.
    End point type
    Secondary
    End point timeframe
    Month 6
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [36]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    538 ± 9999
    Notes
    [36] - No variation was calculated.
    No statistical analyses for this end point

    Secondary: PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 10 mg Q2W OCA

    Close Top of page
    End point title
    PK Population: Trough Concentrations Of Non-Cirrhotic Participants Who Received 10 mg Q2W OCA
    End point description
    At Month 6, the trough concentration of OCA in the non-cirrhotic participants who received 10 mg Q2W OCA was reported.
    End point type
    Secondary
    End point timeframe
    Month 6
    End point values
    Obeticholic Acid (PK)
    Number of subjects analysed
    1 [37]