| Primary | Double-blind Phase: Percent Change From Baseline in Serum Alkaline Phosphatase (ALP) | Blood samples were collected for the analysis of serum ALP. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Percent change from Baseline was calculated as Post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | Modified Intent-to-Treat (mITT) Population (N=161) comprised of all randomized participants who received at least 1 dose of investigational product and had at least 1 post-Baseline ALP evaluation taken ≤7 days after their last dose of investigational product. | Posted | | Mean | Standard Deviation | Percent change | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). | | OG002 | OCA 50 mg | Participants were randomized to receive OCA 50 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). | | OG003 | Placebo | Participants were randomized to receive placebo orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
| | Units | Counts |
|---|
| Participants | - OG00038
- OG00147
- OG00239
- OG003
|
| | Title | Denominators | Categories |
|---|
| | | Title | Measurements |
|---|
| - OG000-23.7± 17.8
- OG001-24.7± 17.9
- OG002-21.0± 27.6
- OG003
|
|
| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
|---|
| All tests: Descriptive methods were used with nominal p values provided and 95% confidence intervals. | Wilcoxon (Mann-Whitney) | Hierarchical model applied and pairwise comparisons of the 10mg treatment group versus the placebo group, then 25 mg and 50 mg were performed. | <0.0001 | Initial Phase 2 study: no a priori estimate of treatment effect available. Effect size estimated. | | | | | | | | | | | | | Other | | |
|
| Secondary | Double-blind Phase: Absolute Values for Serum ALP, Aspartate Aminotransferase (AST), Alanine Transaminase (ALT) and Gamma-glutamyl Transferase (GGT) Levels | Blood samples were collected for the analysis of serum chemistry parameters including ALP, AST, ALT and GGT. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | Intent-to-Treat (ITT) Population (N=165) comprises all randomized participants who received at least 1 dose of investigational product. Only those participants with data available at specified timepoints has been presented. | Posted | | Mean | Standard Deviation | Units per liter | | Baseline and at Days 15, 29, 57 and 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Percent Change From Baseline in Serum ALP, AST, ALT and GGT Levels | Blood samples were collected for the analysis of serum chemistry parameters including ALP, AST, ALT and GGT. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Percent change from Baseline was calculated as Post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=164). | Posted | | Mean | Standard Deviation | Percent change | | Baseline and at Days 15, 29, 57 and 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | LTSE Phase: Mean Percent Change From Baseline in Serum ALP, AST, ALT and GGT Levels | Blood samples were collected for the analysis of serum chemistry parameters including ALP, AST, ALT and GGT. Baseline was defined as the last observed value before the first dose of investigational product (Day 85). Percent change from Baseline was calculated as Post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | Safety Population (N=78) comprised of all randomized participants who received at least 1 dose of investigational product. Only those participants with data available at specified timepoints has been presented (n=69). | Posted | | Mean | Standard Deviation | Percent change | | Baseline and at 3, 6, 9 and 12 Months | | | | ID | Title | Description |
|---|
| OG000 | LTSE OCA Total | A total of 78 participants from the double-blind phase entered the open-label LTSE phase of the study. Seven participants entered the LTSE within 2 weeks of completing the double-blind phase. Participants initially started dosing at less than or equal to (<=) 10 mg daily OCA and doses up to 50 mg daily were evaluated. Entry of 71 participants from double-blind phase to LTSE phase was delayed up to 10 months due to administrative reasons. All participants received open-label OCA during the LTSE phase of the study |
| |
| Secondary | Double-blind Phase: Absolute Values for Albumin Levels | Blood samples were collected for the analysis of serum chemistry parameter: Albumin. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented. | Posted | | Mean | Standard Deviation | Grams per liter | | Baseline and at Days 15, 29, 57 and 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Percent Change From Baseline in Albumin Levels | Blood samples were collected for the analysis of serum chemistry parameter: Albumin. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Percent change from Baseline was calculated as Post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented. | Posted | | Mean | Standard Deviation | Percent change | | Baseline and at Days 15, 29, 57 and 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Absolute Values for Conjugated (Direct) Bilirubin | Blood samples were collected for the analysis of serum chemistry parameter: Conjugated (Direct) bilirubin. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented. | Posted | | Mean | Standard Deviation | Micromoles per liter | | Baseline and at Days 15, 29, 57 and 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Percent Change From Baseline in Conjugated (Direct) Bilirubin | Blood samples were collected for the analysis of serum chemistry parameter: Direct bilirubin. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Percent change from Baseline was calculated as Post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=164). | Posted | | Mean | Standard Deviation | Percent change | | Baseline and at Days 15, 29, 57 and 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | LTSE Phase: Mean Percent Change From Baseline in Total and Conjugated (Direct) Bilirubin | Blood samples were collected for the analysis of serum chemistry parameters Total and direct bilirubin. Baseline was defined as the last observed value before the first dose of investigational product (Day 85). Percent change from Baseline was calculated as Post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | Safety Population (N=78). Only those participants with data available at specified timepoints has been presented (n=69) | Posted | | Mean | Standard Deviation | Percent change | | Baseline and at 3, 6, 9 and 12 Months | | | | ID | Title | Description |
|---|
| OG000 | LTSE OCA Total | A total of 78 participants from the double-blind phase entered the open-label LTSE phase of the study. Seven participants entered the LTSE within 2 weeks of completing the double-blind phase. Participants initially started dosing at less than or equal to (<=) 10 mg daily OCA and doses up to 50 mg daily were evaluated. Entry of 71 participants from double-blind phase to LTSE phase was delayed up to 10 months due to administrative reasons. All participants received open-label OCA during the LTSE phase of the study |
| |
| Secondary | Double-blind Phase: Change From Baseline in Enhanced Liver Fibrosis (ELF) Score | Enhanced Liver Fibrosis (ELF) combines measurements of tissue inhibitor of metalloprotein-ases-1 (TIMP-1), amino-terminal pro-peptide of type III procollagen (PIIINP), and hyaluronic acid (HA). The ELF score is calculated as: 2.278 + 0.851 ln (HA) + 0.751 ln (PIIINP) + 0.394 ln (TIMP-1). An ELF score of less than 7.7 indicates no to mild fibrosis; ≥ 7.7 - < 9.8: Moderate fibrosis; ≥ 9.8 - < 11.3: Severe fibrosis; ≥ 11.3: Cirrhosis. A negative change from Baseline indicates decreased fibrosis. Higher the ELF is associated with higher fibrosis stages and greater the risk of progression. A minimum and maximum value for the scale range does not exist for this assessment. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=146). | Posted | | Mean | Standard Deviation | Scores on a scale | | Baseline and up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). |
|
| Secondary | Double-blind Phase: Change From Baseline in HA, P3NP, and TIMP-1 Levels | Blood samples were collected for the analysis of extracellular matrix markers, including TIMP-1, PIIINP, and HA. The ELF score has been reported to show good correlations with fibrosis stages in chronic liver disease, with higher ELF scores associated with higher fibrosis stages. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=146). | Posted | | Mean | Standard Deviation | Nanograms per milliliter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Absolute Values for Biomarkers of Hepatic Inflammation: C-reactive Protein | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including C-reactive protein. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n-144). | Posted | | Mean | Standard Deviation | Milligrams per milliliter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Change From Baseline Values for Biomarkers of Hepatic Inflammation: C-reactive Protein | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including C-reactive protein. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=114). | Posted | | Mean | Standard Deviation | Milligrams per milliliter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Absolute Values for Biomarkers of Hepatic Inflammation: Non-essential Fatty Acid (NEFA) | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including NEFA. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=143). | Posted | | Mean | Standard Deviation | Millimoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Change From Baseline Values for Biomarkers of Hepatic Inflammation: NEFA | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including NEFA. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=112). | Posted | | Mean | Standard Deviation | Millimoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Absolute Values for Biomarkers of Hepatic Inflammation: Tumor Necrosis Factor Alpha (TNF-alpha) | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including TNF-alpha. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=144). | Posted | | Mean | Standard Deviation | Nanograms per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Change From Baseline Values for Biomarkers of Hepatic Inflammation: TNF-alpha | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including TNF-alpha. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=119). | Posted | | Mean | Standard Deviation | Nanograms per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Absolute Values for Biomarkers of Hepatic Inflammation: TNF-beta and Tumor Growth Factor Beta (TGF-beta) | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including TNF-beta and TGF-beta. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N-165). Only those participants with data available at specified timepoints has been presented (142). | Posted | | Mean | Standard Deviation | Picomoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Change From Baseline Values for Biomarkers of Hepatic Inflammation: TNF-beta and TGF-beta | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including TNF-beta and TGF-beta. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=120). | Posted | | Mean | Standard Deviation | Picomoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Absolute Values for Biomarkers of Hepatic Inflammation: Bile Acids and Glutathion | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including Bile acids and glutathion. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=144). | Posted | | Mean | Standard Deviation | Micromoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
|
| Secondary | Double-blind Phase: Change From Baseline Values for Biomarkers of Hepatic Inflammation: Bile Acids and Glutathion | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including Bile acids and glutathion. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=119). | Posted | | Mean | Standard Deviation | Micromoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
|---|
| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Absolute Values for Biomarkers of Hepatic Inflammation: Immunoglobulin M (IgM) | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including IgM. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified timepoints has been presented (n=144). | Posted | | Mean | Standard Deviation | Grams per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Change From Baseline Values for Biomarkers of Hepatic Inflammation: IgM | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including IgM. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=119). | Posted | | Mean | Standard Deviation | Grams per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Absolute Values for Biomarkers of Hepatic Inflammation: Osteopontin | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including Osteopontin. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (165). Only those participants with data available at specified timepoints has been presented (n=144). | Posted | | Mean | Standard Deviation | Micrograms per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Change From Baseline Values for Biomarkers of Hepatic Inflammation: Osteopontin | Blood samples were collected for the analysis of biomarkers of hepatic inflammation including Osteopontin. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (165). Only those participants with data available at specified time points has been presented (n=119). | Posted | | Mean | Standard Deviation | Micrograms per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Absolute Values for Total Endogenous Bile Acids | Serum samples were collected for the analysis total endogenous bile acids. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=143). | Posted | | Mean | Standard Deviation | Micromoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Change From Baseline in Total Endogenous Bile Acids | Serum samples were collected for the analysis of total endogenous bile acids. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline is defined as post-dose visit value minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=121). | Posted | | Mean | Standard Deviation | Micromoles per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Absolute Values for Fibroblast Growth Factor 19 (FGF19) | FGF-19 is a protein secreted by the gastro-intestine under farnesoid X receptor (FXR) control. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=143). | Posted | | Mean | Standard Deviation | Nanograms per liter | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Percent Change From Baseline Values for FGF19 | FGF-19 is a protein secreted by the gastro-intestine under FXR control. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Percent change from Baseline was calculated as Post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=126). | Posted | | Mean | Standard Deviation | Percent change | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Change From Baseline to Day 85 in Quality of Life as Determined by Short Form-36 (SF-36) Scale | The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the physical component summary (PCS) score of the SF-36. Items 5-8 primarily contribute to the mental component summary (MCS) score of the SF-36. These eight dimensions can be summarized numerically into two scores: PCS and MCS; with score range from 0=worst to 100=best, with higher scores indicating better health. Increases from baseline indicate improvement. Baseline was defined as Day 0. Change from Baseline was defined as value of post Baseline minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=153). | Posted | | Mean | Standard Deviation | Scores on a scale | | Baseline and Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). |
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| Secondary | LTSE Phase: Absolute Values of Quality of Life as Determined by SF-36 Scale | The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the physical component summary (PCS) score of the SF-36. Items 5-8 primarily contribute to the mental component summary (MCS) score of the SF-36. These eight dimensions can be summarized numerically into two scores: PCS and MCS; with score range from 0=worst to 100=best, with higher scores indicating better health. Increases from baseline indicate improvement. Baseline was defined as Day 85 | | Posted | | Mean | Standard Deviation | Scores on a scale | | Baseline and at 3, 6, 9 and 12 Months | | | | ID | Title | Description |
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| OG000 | LTSE OCA Total | A total of 78 participants from the double-blind phase entered the open-label LTSE phase of the study. Seven participants entered the LTSE within 2 weeks of completing the double-blind phase. Participants initially started dosing at less than or equal to (<=) 10 mg daily OCA and doses up to 50 mg daily were evaluated. Entry of 71 participants from double-blind phase to LTSE phase was delayed up to 10 months due to administrative reasons. All participants received open-label OCA during the LTSE phase of the study |
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| Secondary | Double-blind Phase: Change From Baseline in Quality of Life (QoL) as Determined by Primary Biliary Cirrhosis 40 (PBC-40) Scale | PBC-40 is a disease-specific quality of life questionnaire,which consists of 5 Domains(score ranges):general symptoms (score range 7-35), itch (3-15), fatigue (11-55), cognitive function (6-30) and emotional (1-15); higher scores indicated worse outcomes.The 40 questions from the PBC-40 questionnaire were scored from 1 (lowest impact of PBC on QoL) to 5 (representing highest impact); Higher scores indicate worse quality of life. Outcomes of 'never', 'not at all' or 'strongly agree' are scored with 1, 'always', 'very much' or 'strongly disagree' with 5, with following exceptions: For questions 34-39 outcomes were scored in reverse,i.e., 'strongly agree' with 5, and 'strongly disagree' with 1. If less than 50% of the questions per domain were not answered, missing values were imputed by mean of the available question scores for that domain.If for any item multiple answers are given, most severe was used. Baseline = Day 0. Change from Baseline=post Baseline minus Baseline value. | Safety Population. Only those participants with data available at specified time points has been presented (n=163). | Posted | | Mean | Standard Deviation | Scores on a scale | | Baseline and at Days 29, 57 and 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). |
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| Secondary | LTSE Phase: Change From Baseline in Quality of Life as Determined by PBC-40 Scale | PBC-40 is a disease-specific quality of life questionnaire, which consists of 5 Domains(score ranges):general symptoms (score range 7-35), itch (3-15), fatigue (11-55), cognitive function (6-30) and emotional (1-15); higher scores indicated worse outcomes.The 40 questions from the PBC-40 questionnaire were scored from 1 (lowest impact of PBC on QoL) to 5 (representing highest impact); Higher scores indicate worse quality of life. Outcomes of 'never', 'not at all' or 'strongly agree' are scored with 1, 'always', 'very much' or 'strongly disagree' with 5, with following exceptions: For questions 34-39 outcomes were scored in reverse,i.e., 'strongly agree' with 5, and 'strongly disagree' with 1. If less than 50% of the questions per domain were not answered, missing values were imputed by mean of the available question scores for that domain.If for any item multiple answers are given, most severe was used. Baseline = Day 85. Change from Baseline=post Baseline minus Baseline value. | Safety Population (N=78). Only those participants with data available at specified time points has been presented (n=66). | Posted | | Mean | Standard Deviation | Scores on a scale | | Baseline and at 6 and 12 months | | | | ID | Title | Description |
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| OG000 | LTSE OCA Total | A total of 78 participants from the double-blind phase entered the open-label LTSE phase of the study. Seven participants entered the LTSE within 2 weeks of completing the double-blind phase. Participants initially started dosing at less than or equal to (<=) 10 mg daily OCA and doses up to 50 mg daily were evaluated. Entry of 71 participants from double-blind phase to LTSE phase was delayed up to 10 months due to administrative reasons. All participants received open-label OCA during the LTSE phase of the study |
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| Secondary | Double-blind Phase: Change From Baseline in Quality of Life as Determined by 5-Dimension (5-D) Domain Scale | 5-D questionnaire has 5 domains: Duration, degree, direction, disability, distribution, and total score. Single item domain scores (duration, degree, direction)=range:1-5.Disability domain has 4 items=assess impact of itching on daily activities: sleep, leisure/social activities, housework/errands, work/school;score calculated as highest score on any of 4 items; disability domain range=1-5.For distribution domain only section "Mark whether itching has been present in following parts of your body over the last 2 weeks" was used. Number of affected body parts('present') is tallied(potential sum 0-16);sum was sorted into 5 scoring bins: sum 0-2= score 1,sum 3-5=score 2,sum 6-10=score 3, sum 11-13=score 4,sum 14-16=score 5. Distribution score range reported=1-5. For all domains, higher scores=worse outcomes. Total 5D score=summing domain scores; ranges:5(no pruritus) to25 (most severe pruritus); Higher scores=worse outcomes. Baseline=Day 0.Change from Baseline=post Baseline minus Baseline | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=163). | Posted | | Mean | Standard Deviation | Scores on a scale | | Baseline and at Days 29, 57 and 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). |
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| Secondary | Double-blind Phase: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a participant administered a medicinal product without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. TEAEs were defined as any AE that started or worsened in severity on or after the first dose of study treatment. | | Posted | | Count of Participants | | Participants | | Up to Day 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | Double-blind Phase: Change From Baseline in Pruritus Visual Analog Scale (VAS) | A VAS questionnaire was used to assess participant's pruritus. The pruritus VAS measures participant's perception of itch on a continuous scale with score ranged from 0 = no itching and 10 = worst possible itching; higher score indicates worse outcomes. Baseline was defined as the last observed value before the first dose of investigational product (Day 0). Change from Baseline was defined as value of post Baseline minus Baseline value. | ITT Population (N=165). Only those participants with data available at specified time points has been presented (n=152). | Posted | | Mean | Standard Deviation | Scores on a scale | | Baseline and at Days 29, 57 and 85 | | | | ID | Title | Description |
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| OG000 | Obeticholic Acid (OCA) 10 Milligrams (mg) | Participants were randomized to receive OCA 10 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until end of study (EOS) (Day 85). | | OG001 | OCA 25 mg | Participants were randomized to receive OCA 25 mg orally as a capsule once daily from Day 1 to Day 85 approximately 30 minutes before breakfast with water. Participants were instructed to swallow the capsule whole and not to chew, divide, or crush the capsule. Per the titration strategy, the participants were administered investigational product once every 3 days in the first week, once every 2 days in the second week, and daily from the third week onwards until EOS (Day 85). |
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| Secondary | LTSE Phase: Number of Participants With TEAEs and SAEs | An AE was any untoward medical occurrence in a participant administered a medicinal product without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. TEAEs were defined as any AE that started or worsened in severity on or after the first dose of study treatment. | | Posted | | Count of Participants | | Participants | | Up to 12 Months | | | | ID | Title | Description |
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| OG000 | LTSE OCA Total | A total of 78 participants from the double-blind phase entered the open-label LTSE phase of the study. Seven participants entered the LTSE within 2 weeks of completing the double-blind phase. Participants initially started dosing at less than or equal to (<=) 10 mg daily OCA and doses up to 50 mg daily were evaluated. Entry of 71 participants from double-blind phase to LTSE phase was delayed up to 10 months due to administrative reasons. All participants received open-label OCA during the LTSE phase of the study |
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