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Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis (POISE)

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ClinicalTrials.gov Identifier: NCT01473524
Recruitment Status : Active, not recruiting
First Posted : November 17, 2011
Results First Posted : February 13, 2017
Last Update Posted : April 3, 2018
Sponsor:
Information provided by (Responsible Party):
Intercept Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Primary Biliary Cirrhosis
Interventions Drug: Obeticholic Acid (OCA)
Drug: Placebo
Enrollment 217

Recruitment Details Recruitment into hospitals and physicians' clinics started JAN 2012 and completed DEC 2012.
Pre-assignment Details Screening interim allowed for pre-randomization eligibility assessment of 1 to 8 weeks.
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated. One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Period Title: Overall Study
Started 71 73 73
Completed 64 64 70
Not Completed 7 9 3
Reason Not Completed
Adverse Event             4             8             2
Death             1             0             0
Withdrawal by Subject             2             1             1
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo Total
Hide Arm/Group Description

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated. One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated. Total of all reporting groups
Overall Number of Baseline Participants 70 73 73 216
Hide Baseline Analysis Population Description
71 subjects were randomized in the OCA 5-10 mg group, however 1 subject never received treatment. Therefore, the Baseline Analysis Population of 70 subjects is the Intent-to-Treat Population
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 70 participants 73 participants 73 participants 216 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
60
  85.7%
56
  76.7%
60
  82.2%
176
  81.5%
>=65 years
10
  14.3%
17
  23.3%
13
  17.8%
40
  18.5%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 70 participants 73 participants 73 participants 216 participants
55.8  (10.53) 56.2  (11.00) 55.5  (10.03) 55.8  (10.48)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 70 participants 73 participants 73 participants 216 participants
Female
65
  92.9%
63
  86.3%
68
  93.2%
196
  90.7%
Male
5
   7.1%
10
  13.7%
5
   6.8%
20
   9.3%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 70 participants 73 participants 73 participants 216 participants
Asian 1 1 1 3
Black or African American 1 1 1 3
Other 1 1 5 7
White 67 70 66 203
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 70 participants 73 participants 73 participants 216 participants
United States 18 19 17 54
United Kingdom 8 5 9 22
Spain 4 2 3 9
Canada 2 2 4 8
Austria 2 0 1 3
Netherlands 3 7 6 16
Sweden 3 1 0 4
Belgium 2 9 5 16
Poland 4 6 4 14
Italy 11 10 11 32
Australia 5 1 3 9
France 0 0 1 1
Germany 8 11 9 28
Alkaline Phosphatase (U/L)  
Mean (Standard Deviation)
Unit of measure:  U/L
Number Analyzed 70 participants 73 participants 73 participants 216 participants
325.87  (116.238) 316.34  (103.881) 327.49  (115.014) 323.19  (111.375)
Total Bilirubin (umol/L)  
Mean (Standard Deviation)
Unit of measure:  umol/L
Number Analyzed 70 participants 73 participants 73 participants 216 participants
10.192  (5.549) 11.278  (6.634) 11.757  (7.227) 11.088  (6.522)
Direct Bilirubin (umol/L)  
Mean (Standard Deviation)
Unit of measure:  umol/L
Number Analyzed 70 participants 73 participants 73 participants 216 participants
4.398  (4.528) 4.868  (4.473) 5.469  (6.214) 4.919  (5.138)
Alanine Aminotransferase (ALT) (U/L)  
Mean (Standard Deviation)
Unit of measure:  U/L
Number Analyzed 70 participants 73 participants 73 participants 216 participants
61.56  (39.037) 56.31  (39.741) 55.99  (30.312) 57.9  (36.498)
Aspartate Aminotransferase (AST) (U/L)  
Mean (Standard Deviation)
Unit of measure:  U/L
Number Analyzed 70 participants 73 participants 73 participants 216 participants
52.25  (25.289) 50.49  (31.100) 48.79  (22.449) 50.49  (26.456)
Gamma-Glutamyltransferase (GGT) (U/L)  
Mean (Standard Deviation)
Unit of measure:  U/L
Number Analyzed 70 participants 73 participants 73 participants 216 participants
252.83  (167.038) 261.07  (207.396) 309.58  (449.356) 274.79  (302.673)
1.Primary Outcome
Title Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 10 mg OCA vs. Placebo
Hide Description Proportion of subjects at Month 12 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 10 mg Placebo
Hide Arm/Group Description:
OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 73 73
Measure Type: Number
Unit of Measure: percentage of participants
47 10
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments H0: The response rates are equal between placebo and 10 mg OCA. H1: The response rates are different between placebo and 10 mg OCA.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments Cochran–Mantel–Haenszel (CMH) General Association test stratified by randomization strata factor.
2.Secondary Outcome
Title Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 10 mg vs. Placebo
Hide Description Proportion of subjects at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 10 mg Placebo
Hide Arm/Group Description:
OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 73 73
Measure Type: Number
Unit of Measure: percentage of participants
51 7
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments H0: The response rates are equal between placebo and 10 mg OCA. H1: The response rates are different between placebo and 10 mg OCA.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments Cochran–Mantel–Haenszel (CMH) General Association test stratified by randomization strata factor.
3.Secondary Outcome
Title Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 5-10 mg vs. Placebo
Hide Description Proportion of subjects at Month 12 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame 12 Months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 70 73
Measure Type: Number
Unit of Measure: percentage of participants
46 10
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments H0: The response rates are equal between placebo and 5-10 mg OCA. H1: The response rates are different between placebo and 5-10 mg OCA.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments Cochran–Mantel–Haenszel (CMH) General Association test stratified by randomization strata factor.
4.Secondary Outcome
Title Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 5-10 mg vs. Placebo
Hide Description Proportion of subjects at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame 6 Months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 70 73
Measure Type: Number
Unit of Measure: percentage of participants
34 7
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments H0: The response rates are equal between placebo and 5-10 mg OCA. H1: The response rates are different between placebo and 5-10 mg OCA.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments Cochran–Mantel–Haenszel (CMH) General Association test stratified by randomization strata factor.
5.Secondary Outcome
Title Alkaline Phosphatase Absolute Change From Baseline to Month 12
Hide Description Alkaline Phosphatase Absolute Change from Baseline to Month 12
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 64 62 70
Least Squares Mean (Standard Error)
Unit of Measure: U/L
-112.51  (14.36) -129.90  (14.60) -14.42  (14.74)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
6.Secondary Outcome
Title Total Bilirubin Absolute Change From Baseline to Month 12
Hide Description Total Bilirubin Absolute Change from Baseline to Month 12
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 64 62 70
Least Squares Mean (Standard Error)
Unit of Measure: umol/L
-0.33  (0.68) -0.90  (0.71) 1.98  (0.70)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0004
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
7.Secondary Outcome
Title Direct Bilirubin Absolute Change From Baseline to Month 12
Hide Description Direct Bilirubin Absolute Change from Baseline to Month 12
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 64 62 70
Least Squares Mean (Standard Error)
Unit of Measure: umol/L
-0.13  (0.52) -0.49  (0.54) 1.89  (0.53)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
8.Secondary Outcome
Title Alanine Aminotransferase (ALT) Absolute Change From Baseline to Month 12
Hide Description Alanine Aminotransferase (ALT) Absolute Change from Baseline to Month 12
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 64 62 70
Least Squares Mean (Standard Error)
Unit of Measure: U/L
-21.26  (3.27) -25.31  (3.35) -4.95  (3.32)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
9.Secondary Outcome
Title Aspartate Aminotransferase (AST) Absolute Change From Baseline to Month 12
Hide Description Aspartate Aminotransferase (AST) Absolute Change from Baseline to Month 12
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double blind-period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 64 62 70
Least Squares Mean (Standard Error)
Unit of Measure: U/L
-13.03  (4.17) -15.00  (4.28) 1.04  (4.22)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor
10.Secondary Outcome
Title Gamma-glutamyltransferase (GGT) Absolute Change From Baseline to Month 12
Hide Description Gamma-glutamyltransferase (GGT) Absolute Change from Baseline to Month 12
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description:

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
One tablet daily for double-blind period. After completion of the 1-year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
Overall Number of Participants Analyzed 64 62 70
Least Squares Mean (Standard Error)
Unit of Measure: U/L
-140.83  (24.70) -176.66  (25.58) 6.70  (25.56)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA 5-10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection OCA 10 mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA model with baseline value as a covariate and fixed effects for treatment and randomization strata factor.
Time Frame From informed consent to end of study participation
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title OCA 5-10 mg OCA 10 mg Placebo
Hide Arm/Group Description

OCA 5 mg for 6 months and then titrating up to 10 mg based on tolerability and response for remainder of double blind-period.

After completion of the 1 year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.

OCA 10 mg for double-blind period. After completion of the 1 year double-blind period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated. One tablet daily for double-blind period. After completion of the 1 year double-blind period period subjects will be offered the opportunity to enter an open label long term safety extension for up to 5 years beginning at 5 mg OCA. Doses up to 25 mg daily will be evaluated.
All-Cause Mortality
OCA 5-10 mg OCA 10 mg Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
OCA 5-10 mg OCA 10 mg Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   11/70 (15.71%)      8/73 (10.96%)      3/73 (4.11%)    
Blood and lymphatic system disorders       
Anaemia  1  0/70 (0.00%)  0 1/73 (1.37%)  1 0/73 (0.00%)  0
Cardiac disorders       
Cardiac failure  1  1/70 (1.43%)  2 0/73 (0.00%)  0 0/73 (0.00%)  0
Sick sinus syndrome  1  0/70 (0.00%)  0 0/73 (0.00%)  0 1/73 (1.37%)  1
Gastrointestinal disorders       
Upper gastrointestinal haemorrhage  1  1/70 (1.43%)  1 0/73 (0.00%)  0 1/73 (1.37%)  1
Varices oesophageal  1  0/70 (0.00%)  0 0/73 (0.00%)  0 1/73 (1.37%)  2
Abdominal wall haematoma  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
Ascites  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
Splenic artery aneurysm  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
General disorders       
Chest pain  1  0/70 (0.00%)  0 0/73 (0.00%)  0 1/73 (1.37%)  1
Non-cardiac chest pain  1  0/70 (0.00%)  0 0/73 (0.00%)  0 1/73 (1.37%)  1
Oedema  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
Infections and infestations       
Erysipelas  1  0/70 (0.00%)  0 1/73 (1.37%)  1 0/73 (0.00%)  0
Parotitis  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
Pneumonia  1  0/70 (0.00%)  0 1/73 (1.37%)  1 0/73 (0.00%)  0
Injury, poisoning and procedural complications       
Radius fracture  1  0/70 (0.00%)  0 1/73 (1.37%)  2 0/73 (0.00%)  0
Clavicle fracture  1  0/70 (0.00%)  0 1/73 (1.37%)  1 0/73 (0.00%)  0
Post procedural haemorrhage  1  0/70 (0.00%)  0 1/73 (1.37%)  1 0/73 (0.00%)  0
Tibia fracture  1  0/70 (0.00%)  0 0/73 (0.00%)  0 1/73 (1.37%)  1
Wrist fracture  1  0/70 (0.00%)  0 1/73 (1.37%)  1 0/73 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Osteoarthritis  1  0/70 (0.00%)  0 2/73 (2.74%)  2 0/73 (0.00%)  0
Intervertebral disc protrusion  1  0/70 (0.00%)  0 1/73 (1.37%)  1 0/73 (0.00%)  0
Rotator cuff syndrome  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
Nervous system disorders       
Hepatic encephalopathy  1  1/70 (1.43%)  2 0/73 (0.00%)  0 0/73 (0.00%)  0
Syncope  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Dyspnoea  1  0/70 (0.00%)  0 0/73 (0.00%)  0 1/73 (1.37%)  1
Interstitial lung disease  1  1/70 (1.43%)  1 0/73 (0.00%)  0 0/73 (0.00%)  0
Vascular disorders       
Varicose vein  1  2/70 (2.86%)  2 0/73 (0.00%)  0 0/73 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
OCA 5-10 mg OCA 10 mg Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   63/70 (90.00%)      63/73 (86.30%)      58/73 (79.45%)    
Blood and lymphatic system disorders       
Anaemia  1  2/70 (2.86%)  2 3/73 (4.11%)  3 4/73 (5.48%)  5
Cardiac disorders       
Palpitations  1  2/70 (2.86%)  3 5/73 (6.85%)  5 1/73 (1.37%)  1
Endocrine disorders       
Hypothyroidism  1  4/70 (5.71%)  4 1/73 (1.37%)  1 1/73 (1.37%)  1
Eye disorders       
Dry eye  1  2/70 (2.86%)  3 4/73 (5.48%)  4 4/73 (5.48%)  4
Gastrointestinal disorders       
Nausea  1  4/70 (5.71%)  5 8/73 (10.96%)  10 9/73 (12.33%)  18
Diarrhoea  1  2/70 (2.86%)  3 8/73 (10.96%)  10 8/73 (10.96%)  13
Abdominal pain upper  1  5/70 (7.14%)  5 4/73 (5.48%)  5 5/73 (6.85%)  8
Constipation  1  5/70 (7.14%)  5 5/73 (6.85%)  5 4/73 (5.48%)  7
Abdominal distension  1  3/70 (4.29%)  3 3/73 (4.11%)  3 7/73 (9.59%)  7
Dyspepsia  1  4/70 (5.71%)  4 0/73 (0.00%)  0 8/73 (10.96%)  11
Vomiting  1  3/70 (4.29%)  3 3/73 (4.11%)  3 5/73 (6.85%)  8
Gastrooesophageal reflux disease  1  2/70 (2.86%)  2 4/73 (5.48%)  5 4/73 (5.48%)  6
Abdominal pain  1  3/70 (4.29%)  3 1/73 (1.37%)  1 6/73 (8.22%)  6
Abdominal discomfort  1  5/70 (7.14%)  5 0/73 (0.00%)  0 1/73 (1.37%)  5
General disorders       
Fatigue  1  11/70 (15.71%)  13 17/73 (23.29%)  25 10/73 (13.70%)  12
Oedema peripheral  1  2/70 (2.86%)  2 5/73 (6.85%)  7 2/73 (2.74%)  3
Pyrexia  1  0/70 (0.00%)  0 5/73 (6.85%)  6 1/73 (1.37%)  1
Infections and infestations       
Nasopharyngitis  1  17/70 (24.29%)  27 13/73 (17.81%)  15 13/73 (17.81%)  19
Urinary tract infection  1  4/70 (5.71%)  6 4/73 (5.48%)  6 8/73 (10.96%)  15
Upper respiratory tract infection  1  4/70 (5.71%)  4 4/73 (5.48%)  4 8/73 (10.96%)  8
Influenza  1  5/70 (7.14%)  5 4/73 (5.48%)  4 4/73 (5.48%)  5
Sinusitis  1  1/70 (1.43%)  1 4/73 (5.48%)  5 0/73 (0.00%)  0
Bronchitis  1  4/70 (5.71%)  4 1/73 (1.37%)  1 0/73 (0.00%)  0
Injury, poisoning and procedural complications       
Procedural pain  1  4/70 (5.71%)  4 1/73 (1.37%)  1 1/73 (1.37%)  3
Musculoskeletal and connective tissue disorders       
Back pain  1  4/70 (5.71%)  4 4/73 (5.48%)  5 8/73 (10.96%)  8
Arthralgia  1  4/70 (5.71%)  5 7/73 (9.59%)  7 3/73 (4.11%)  3
Muscle spasms  1  2/70 (2.86%)  2 2/73 (2.74%)  2 4/73 (5.48%)  5
Nervous system disorders       
Headache  1  12/70 (17.14%)  26 6/73 (8.22%)  7 13/73 (17.81%)  16
Psychiatric disorders       
Insomnia  1  2/70 (2.86%)  2 3/73 (4.11%)  3 7/73 (9.59%)  8
Respiratory, thoracic and mediastinal disorders       
Cough  1  4/70 (5.71%)  5 6/73 (8.22%)  6 5/73 (6.85%)  9
Oropharyngeal pain  1  5/70 (7.14%)  5 6/73 (8.22%)  8 1/73 (1.37%)  1
Skin and subcutaneous tissue disorders       
Pruritus  1  39/70 (55.71%)  93 50/73 (68.49%)  99 28/73 (38.36%)  49
Rash  1  3/70 (4.29%)  3 4/73 (5.48%)  4 3/73 (4.11%)  3
Eczema  1  4/70 (5.71%)  4 2/73 (2.74%)  2 0/73 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Principal Investigators must wait 18 months after the study ends to publish their results and a multi-center publication must come first. The sponsor has a 45 day review period with the option to extend to an additional 90 days.
Results Point of Contact
Name/Title: Medical Information
Organization: Intercept Pharmaceuticals, Inc.
Phone: 844-782-4278
Responsible Party: Intercept Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01473524     History of Changes
Other Study ID Numbers: 747-301
First Submitted: November 14, 2011
First Posted: November 17, 2011
Results First Submitted: December 20, 2016
Results First Posted: February 13, 2017
Last Update Posted: April 3, 2018