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Trial of Obeticholic Acid in Patients With Moderately Severe Alcoholic Hepatitis (AH) (TREAT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02039219
Recruitment Status : Terminated (Post-marketing reports of hepatotoxicity associated with obeticholic acid emerged in June 2017, investigators temporarily halted patient recruitment June 2017.)
First Posted : January 17, 2014
Results First Posted : January 28, 2020
Last Update Posted : January 28, 2020
Sponsor:
Collaborators:
National Institute on Alcohol Abuse and Alcoholism (NIAAA)
Intercept Pharmaceuticals
Information provided by (Responsible Party):
Naga P. Chalasani, Indiana University School of Medicine

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Alcoholic Hepatitis
Interventions Drug: Placebo
Drug: 10 mg Obeticholic Acid (OCA)
Enrollment 19
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Period Title: Treatment
Started 11 8
Completed 7 6
Not Completed 4 2
Reason Not Completed
Withdrawal by Subject             1             0
Adverse Event             0             1
Physician Decision             1             0
Lost to Follow-up             0             1
Protocol Violation             1             0
Death             1             0
Period Title: Follow up
Started 7 6
Completed 7 5
Not Completed 0 1
Reason Not Completed
Adverse Event             0             1
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA) Total
Hide Arm/Group Description

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Total of all reporting groups
Overall Number of Baseline Participants 11 8 19
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 8 participants 19 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
11
 100.0%
8
 100.0%
19
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants 8 participants 19 participants
48.45  (9.61) 50.375  (14.83) 49.26  (11.74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 8 participants 19 participants
Female
2
  18.2%
4
  50.0%
6
  31.6%
Male
9
  81.8%
4
  50.0%
13
  68.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 8 participants 19 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
  18.2%
0
   0.0%
2
  10.5%
White
9
  81.8%
8
 100.0%
17
  89.5%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 11 participants 8 participants 19 participants
11
 100.0%
8
 100.0%
19
 100.0%
1.Primary Outcome
Title MELD Score Mean(SD)
Hide Description The Model for End-Stage Liver Disease (MELD) is a numerical scale, ranging from 6 (less ill) to 40 (gravely ill), used for liver transplant candidates age 12 and older. It gives each person a 'score' (number) based on how urgently he or she needs a liver transplant within the next three months.
Time Frame Baseline to 6 weeks (Day 42)
Hide Outcome Measure Data
Hide Analysis Population Description
The decrease in MELD score from baseline to Day 42 was -3.4 in the OCA arm, and -2.2 in the placebo arm with an overall P-Value of 0.6170.
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 16.4  (2.2) 14.9  (2.4)
Day 42 13.9  (4.6) 11.5  (6.8)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 10 mg Obeticholic Acid (OCA)
Comments Baseline
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value 0.1758
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.5
Parameter Dispersion
Type: Standard Deviation
Value: 2.4
Estimation Comments Two-sample t-test compared MELD score between OCA and placebo arms at baseline OCA arms had a mean (SD) of 14.9(2.4), Placebo arms had a mean (SD) of 16.4 (2.2).
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 10 mg Obeticholic Acid (OCA)
Comments Day 42
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value 0.3839
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.4
Parameter Dispersion
Type: Standard Deviation
Value: 6.8
Estimation Comments Two-sample t-test compared MELD score between OCA and placebo arms at day 42 OCA arms had a mean (SD) of 11.5 (6.8), Placebo arms had a mean (SD) of 13.9(4.6).
2.Primary Outcome
Title Incidence of Serious Adverse Events (SAEs) During the Treatment Phase
Hide Description Number of subjects with one or more SAE are reported in relation to study medication (not related, unlikely, possible, probable, definite).
Time Frame Baseline to 6 weeks (Day 42)
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects with SAEs not related to OCA
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Measure Type: Number
Unit of Measure: Events
Not Related 5 1
Unlikely 6 3
Possible 0 0
Probable 0 0
Definite 0 0
3.Primary Outcome
Title MELD Score Change From Baseline Mean(SD)
Hide Description The Model for End-Stage Liver Disease (MELD) is a numerical scale, ranging from 6 (less ill) to 40 (gravely ill), used for liver transplant candidates age 12 and older. It gives each person a 'score' (number) based on how urgently he or she needs a liver transplant within the next three months.
Time Frame Baseline to 6 weeks (Day 42)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
-2.2  (3.9) -3.4  (5.9)
4.Secondary Outcome
Title Any SAEs During the Follow-up Phase
Hide Description Number of subjects with one or more SAE are reported in relation to study medication (not related, unlikely, possible, probable, definite).
Time Frame Days 42 to 180
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Measure Type: Number
Unit of Measure: Events
Not Related 5 5
Unlikely 3 1
Possible 0 0
Probable 0 0
Definite 0 0
5.Secondary Outcome
Title SAEs Attributable to the Study Medicine During the Treatment and Follow-up Phases
Hide Description Number of subjects with one or more SAE are reported in relation to study medication (not related, unlikely, possible, probable, definite).
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Measure Type: Number
Unit of Measure: Events
Not Related 10 6
Unlikely 9 4
Possible 0 0
Probable 0 0
Definite 0 0
6.Secondary Outcome
Title Adverse Events (AEs) During the Treatment and Follow-up Phases
Hide Description Number of subjects with one or more AEs are reported in relation to study medication (not related, unlikely, possible, probable, definite).
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Measure Type: Number
Unit of Measure: Event
Not Related 30 31
Unlikely 17 25
Possible 3 3
Probable 0 1
Definite 0 0
7.Secondary Outcome
Title Change in MELD Score at 90 and 180 Days
Hide Description The Model for End-Stage Liver Disease (MELD) is a numerical scale, ranging from 6 (less ill) to 40 (gravely ill), used for liver transplant candidates age 12 and older. It gives each person a 'score' (number) based on how urgently he or she needs a liver transplant within the next three months.
Time Frame Days 90 and 180
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
Day 90 -4.4  (2.8) -6.0  (4.0)
Day 180 -4.0  (5.9) -4.5  (2.3)
8.Secondary Outcome
Title Change in Child-Pugh Score at Day 42, 90 and 180 Days
Hide Description The Child-Pugh score is a system for assessing the prognosis - including the required strength of treatment and necessity of liver transplant - of chronic liver disease, primarily cirrhosis. It provides a forecast of the increasing severity of your liver disease and your expected survival rate. The Child-Pugh score is determined by scoring five clinical measures of liver disease. A score of 1, 2, or 3 is given to each measure, with 3 being the most severe. The total Child-Pugh range is 5-15, with 15 being the most severe.
Time Frame Days 42, 90 and 180
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
Day 42 -1.6  (1.9) -2.8  (1.7)
Day 90 -3.0  (1.2) -3.0  (0.6)
Day 180 -2.3  (3.0) -3.5  (0.8)
9.Secondary Outcome
Title Percentage of Participants Deceased at Day 42, 90 and 180
Hide Description Number of subjects deceased at day 42, 90, and 180.
Time Frame Days 42, 90 and 180
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Measure Type: Number
Unit of Measure: percentage of mortality
Day 42 9.09 0
Day 90 9.09 0
Day 180 9.09 0
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo
Comments Day 42
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value .3938
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Survival Anaylsis
Estimated Value 0
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 10 mg Obeticholic Acid (OCA)
Comments Day 90
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value .3938
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Survival Anaylsis
Estimated Value 0
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection 10 mg Obeticholic Acid (OCA)
Comments Day 180
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value .3938
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Survival Anaylsis
Estimated Value 0
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo
Comments Day 42
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value .3938
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Survival Analysis
Estimated Value 0.09
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Placebo
Comments Day 90
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value .3938
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Survival Analysis
Estimated Value 0.09
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Placebo
Comments Day 180
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value .3938
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Survival Analysis
Estimated Value 0.09
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Rates of Hospitalization
Hide Description Number of subjects with one or more hospitalization are reported in relation to study medication (not related, unlikely, possible, probable, definite).
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Measure Type: Number
Unit of Measure: Events
Not Related 4 6
Unlikely 0 0
Possible 0 0
Probable 0 0
Definite 0 0
11.Secondary Outcome
Title Changes in Intestinal Inflammation
Hide Description Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Time Frame Baseline to Day 180
Hide Outcome Measure Data
Hide Analysis Population Description
Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
12.Secondary Outcome
Title Changes in Serum Oxidative Stress.
Hide Description Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
13.Secondary Outcome
Title Length of Hospital Stays
Hide Description [Not Specified]
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Mean (Standard Deviation)
Unit of Measure: Days
1.9  (3.3) 2.3  (2.5)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 10 mg Obeticholic Acid (OCA)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments The type of statistical test is superiority.
Statistical Test of Hypothesis P-Value 0.8094
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value .4
Parameter Dispersion
Type: Standard Deviation
Value: 2.5
Estimation Comments [Not Specified]
14.Secondary Outcome
Title Changes in Bacterial Translocation
Hide Description Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
15.Secondary Outcome
Title Changes in Cytokines
Hide Description Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
16.Secondary Outcome
Title Changes in Activation of Innate Immunity
Hide Description Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
Early termination of the study resulted in insufficient numbers of participants in each arm to allow meaningful assessment of obeticholic acid effects on these secondary outcomes. Therefore these endpoints were not measured and no statistical analysis for these endpoints was done as they endpoints were not measured.
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
17.Secondary Outcome
Title Discontinuation Rate During the Treatment and Follow-up Phases
Hide Description [Not Specified]
Time Frame Baseline to 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description:

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

Overall Number of Participants Analyzed 11 8
Measure Type: Number
Unit of Measure: Events
4 2
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo 10 mg Obeticholic Acid (OCA)
Hide Arm/Group Description

Placebo

Placebo: 1 tablet of placebo, taken orally daily with water, approximately 30 minutes prior to breakfast for 6 weeks.

10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily for 6 weeks.

10 mg Obeticholic Acid (OCA): 10 mg Obeticholic Acid (OCA) Study medication will be administered orally, once daily, approximately 30 minutes prior to breakfast for 6 weeks.

All-Cause Mortality
Placebo 10 mg Obeticholic Acid (OCA)
Affected / at Risk (%) Affected / at Risk (%)
Total   1/11 (9.09%)      0/8 (0.00%)    
Hide Serious Adverse Events
Placebo 10 mg Obeticholic Acid (OCA)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/11 (45.45%)      6/8 (75.00%)    
Gastrointestinal disorders     
Abdominal pain   2/11 (18.18%)  2 0/8 (0.00%)  0
Ascites   2/11 (18.18%)  2 0/8 (0.00%)  0
Chronic gastrointestinal bleeding   1/11 (9.09%)  1 0/8 (0.00%)  0
Gastritis   0/11 (0.00%)  0 1/8 (12.50%)  1
Gastrointestinal haemorrhage   1/11 (9.09%)  1 0/8 (0.00%)  0
Haematemesis   1/11 (9.09%)  1 0/8 (0.00%)  0
Pancreatitis   1/11 (9.09%)  1 0/8 (0.00%)  0
Pancreatitis acute   0/11 (0.00%)  0 2/8 (25.00%)  2
Upper gastrointestinal haemorrhage   1/11 (9.09%)  1 0/8 (0.00%)  0
Vomiting   0/11 (0.00%)  0 2/8 (25.00%)  2
Hepatobiliary disorders     
Acute hepatic failure   1/11 (9.09%)  1 0/8 (0.00%)  0
Hyperbilirubinaemia   0/11 (0.00%)  0 1/8 (12.50%)  1
Infections and infestations     
Peritonitis bacterial   1/11 (9.09%)  1 0/8 (0.00%)  0
Investigations     
Blood electrolytes abnormal   0/11 (0.00%)  0 1/8 (12.50%)  1
Kidney injury molecule-1   1/11 (9.09%)  1 0/8 (0.00%)  0
Metabolism and nutrition disorders     
Hyperglycaemia   1/11 (9.09%)  1 0/8 (0.00%)  0
Hyponatraemia   2/11 (18.18%)  2 0/8 (0.00%)  0
Nervous system disorders     
Headache   0/11 (0.00%)  0 1/8 (12.50%)  1
Psychiatric disorders     
Hallucination   1/11 (9.09%)  1 0/8 (0.00%)  0
Major depression   0/11 (0.00%)  0 1/8 (12.50%)  1
Suicidal ideation   1/11 (9.09%)  2 0/8 (0.00%)  0
Renal and urinary disorders     
Renal failure acute   0/11 (0.00%)  0 1/8 (12.50%)  1
Respiratory, thoracic and mediastinal disorders     
Hydrothorax   1/11 (9.09%)  1 0/8 (0.00%)  0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo 10 mg Obeticholic Acid (OCA)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/11 (81.82%)      8/8 (100.00%)    
Blood and lymphatic system disorders     
Anaemia   1/11 (9.09%)  1 0/8 (0.00%)  0
Coagulopathy   1/11 (9.09%)  1 0/8 (0.00%)  0
Increased tendency to bruise   0/11 (0.00%)  0 1/8 (12.50%)  1
Leukocytosis   1/11 (9.09%)  1 0/8 (0.00%)  0
Thrombocytopenia   1/11 (9.09%)  1 0/8 (0.00%)  0
Cardiac disorders     
Arrhythmia   0/11 (0.00%)  0 1/8 (12.50%)  1
Tachycardia   0/11 (0.00%)  0 2/8 (25.00%)  2
Ear and labyrinth disorders     
Vertigo   0/11 (0.00%)  0 1/8 (12.50%)  1
Endocrine disorders     
Hypothyroidism   1/11 (9.09%)  1 0/8 (0.00%)  0
Gastrointestinal disorders     
Abdominal distension   0/11 (0.00%)  0 1/8 (12.50%)  1
Abdominal pain   1/11 (9.09%)  1 2/8 (25.00%)  2
Ascites   1/11 (9.09%)  1 0/8 (0.00%)  0
Constipation   3/11 (27.27%)  3 2/8 (25.00%)  2
Faeces pale   0/11 (0.00%)  0 1/8 (12.50%)  1
Flatulence   0/11 (0.00%)  0 1/8 (12.50%)  1
Gastric Ulcer   1/11 (9.09%)  1 0/8 (0.00%)  0
Gingival bleeding   1/11 (9.09%)  1 0/8 (0.00%)  0
Nausea   1/11 (9.09%)  1 4/8 (50.00%)  4
Vomiting   1/11 (9.09%)  1 2/8 (25.00%)  2
General disorders     
Fatigue   1/11 (9.09%)  1 1/8 (12.50%)  1
Jittery   1/11 (9.09%)  1 0/8 (0.00%)  0
Oedema peripheral   4/11 (36.36%)  4 4/8 (50.00%)  5
Hepatobiliary disorders     
Portal hypertension   1/11 (9.09%)  1 0/8 (0.00%)  0
Infections and infestations     
Clostridium difficile infection   0/11 (0.00%)  0 1/8 (12.50%)  1
Electrocardiogram QT prolonged   1/11 (9.09%)  1 1/8 (12.50%)  1
Escherichia urinary tract infection   0/11 (0.00%)  0 1/8 (12.50%)  1
Infection   0/11 (0.00%)  0 1/8 (12.50%)  1
Nasopharyngitis   1/11 (9.09%)  2 0/8 (0.00%)  0
Upper respiratory tract infection   1/11 (9.09%)  1 0/8 (0.00%)  0
Injury, poisoning and procedural complications     
Accidental overdose   0/11 (0.00%)  0 1/8 (12.50%)  1
Investigations     
Neutrophil count increased   0/11 (0.00%)  0 1/8 (12.50%)  1
Pulmonary physical examination abnormal   1/11 (9.09%)  1 0/8 (0.00%)  0
Metabolism and nutrition disorders     
Weigh Loss   1/11 (9.09%)  1 0/8 (0.00%)  0
Decreased Appetite   1/11 (9.09%)  1 0/8 (0.00%)  0
Electrolyte imbalance   0/11 (0.00%)  0 1/8 (12.50%)  1
Hypokalaemia   1/11 (9.09%)  1 0/8 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Arthralgia   0/11 (0.00%)  0 1/8 (12.50%)  1
Flank pain   0/11 (0.00%)  0 1/8 (12.50%)  1
Muscular weakness   0/11 (0.00%)  0 1/8 (12.50%)  1
Musculoskeletal pain   0/11 (0.00%)  0 1/8 (12.50%)  2
Musculoskeletal stiffness   1/11 (9.09%)  1 0/8 (0.00%)  0
Soft Tissue Mass   0/11 (0.00%)  0 1/8 (12.50%)  1
Swelling   1/11 (9.09%)  1 0/8 (0.00%)  0
Nervous system disorders     
Dizziness   3/11 (27.27%)  3 2/8 (25.00%)  5
Headache   2/11 (18.18%)  2 2/8 (25.00%)  3
Migraine   0/11 (0.00%)  0 1/8 (12.50%)  1
Psychiatric disorders     
Confusional state   0/11 (0.00%)  0 1/8 (12.50%)  1
Insomnia   1/11 (9.09%)  1 2/8 (25.00%)  4
Renal and urinary disorders     
Lower urinary tract symptoms   1/11 (9.09%)  1 0/8 (0.00%)  0
Metabolic acidosis   1/11 (9.09%)  1 0/8 (0.00%)  0
Micturition urgency   1/11 (9.09%)  1 0/8 (0.00%)  0
Reproductive system and breast disorders     
Penile swelling   1/11 (9.09%)  1 0/8 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Epistaxis   3/11 (27.27%)  3 0/8 (0.00%)  0
Hyperventilation   0/11 (0.00%)  0 1/8 (12.50%)  1
Respiration abnormal   0/11 (0.00%)  0 1/8 (12.50%)  1
Skin and subcutaneous tissue disorders     
Cholestatic pruritus   0/11 (0.00%)  0 1/8 (12.50%)  1
Pruritus   3/11 (27.27%)  3 1/8 (12.50%)  1
Rash   2/11 (18.18%)  2 0/8 (0.00%)  0
Skin Lesion   0/11 (0.00%)  0 1/8 (12.50%)  1
Skin Ulcer   0/11 (0.00%)  0 1/8 (12.50%)  1
Vascular disorders     
Hypotension   0/11 (0.00%)  0 1/8 (12.50%)  1
Varices oesophageal   0/11 (0.00%)  0 1/8 (12.50%)  1
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Naga Chalasani, MD
Organization: Indiana University School of Medicine
Phone: (317) 278-0414
EMail: nchalasa@iu.edu
Layout table for additonal information
Responsible Party: Naga P. Chalasani, Indiana University School of Medicine
ClinicalTrials.gov Identifier: NCT02039219    
Other Study ID Numbers: TREAT 002
1U01AA021840-01 ( U.S. NIH Grant/Contract )
U01AA021883 ( U.S. NIH Grant/Contract )
U01AA021891 ( U.S. NIH Grant/Contract )
U01AA021788 ( U.S. NIH Grant/Contract )
First Submitted: January 15, 2014
First Posted: January 17, 2014
Results First Submitted: June 14, 2019
Results First Posted: January 28, 2020
Last Update Posted: January 28, 2020