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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01473524
First Posted: November 17, 2011
Last Update Posted: June 12, 2017
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.
Information provided by (Responsible Party):
Intercept Pharmaceuticals
Results First Submitted: December 20, 2016  
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

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Recruitment into hospitals and physicians' clinics started JAN 2012 and completed DEC 2012.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Screening interim allowed for pre-randomization eligibility assessment of 1 to 8 weeks.

Reporting Groups
  Description
OCA 5-10 mg

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 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.
Placebo 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.

Participant Flow:   Overall Study
    OCA 5-10 mg   OCA 10 mg   Placebo
STARTED   71   73   73 
COMPLETED   64   64   70 
NOT COMPLETED   7   9   3 
Adverse Event                4                8                2 
Death                1                0                0 
Withdrawal by Subject                2                1                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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

Reporting Groups
  Description
OCA 5-10 mg

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 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.
Placebo 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 Total of all reporting groups

Baseline Measures
   OCA 5-10 mg   OCA 10 mg   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 70   73   73   216 
Age 
[Units: Participants]
Count of 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 
[Units: Years]
Mean (Standard Deviation)
 55.8  (10.53)   56.2  (11.00)   55.5  (10.03)   55.8  (10.48) 
Sex: Female, Male 
[Units: Participants]
Count of 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 
[Units: 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 
[Units: 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) 
[Units: U/L]
Mean (Standard Deviation)
 325.87  (116.238)   316.34  (103.881)   327.49  (115.014)   323.19  (111.375) 
Total Bilirubin (umol/L) 
[Units: umol/L]
Mean (Standard Deviation)
 10.192  (5.549)   11.278  (6.634)   11.757  (7.227)   11.088  (6.522) 
Direct Bilirubin (umol/L) 
[Units: umol/L]
Mean (Standard Deviation)
 4.398  (4.528)   4.868  (4.473)   5.469  (6.214)   4.919  (5.138) 
Alanine Aminotransferase (ALT) (U/L) 
[Units: U/L]
Mean (Standard Deviation)
 61.56  (39.037)   56.31  (39.741)   55.99  (30.312)   57.9  (36.498) 
Aspartate Aminotransferase (AST) (U/L) 
[Units: U/L]
Mean (Standard Deviation)
 52.25  (25.289)   50.49  (31.100)   48.79  (22.449)   50.49  (26.456) 
Gamma-Glutamyltransferase (GGT) (U/L) 
[Units: U/L]
Mean (Standard Deviation)
 252.83  (167.038)   261.07  (207.396)   309.58  (449.356)   274.79  (302.673) 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 10 mg OCA vs. Placebo   [ Time Frame: 12 months ]

2.  Secondary:   Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 10 mg vs. Placebo   [ Time Frame: 6 months ]

3.  Secondary:   Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 5-10 mg vs. Placebo   [ Time Frame: 12 Months ]

4.  Secondary:   Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 5-10 mg vs. Placebo   [ Time Frame: 6 Months ]

5.  Secondary:   Alkaline Phosphatase Absolute Change From Baseline to Month 12   [ Time Frame: 12 months ]

6.  Secondary:   Total Bilirubin Absolute Change From Baseline to Month 12   [ Time Frame: 12 months ]

7.  Secondary:   Direct Bilirubin Absolute Change From Baseline to Month 12   [ Time Frame: 12 months ]

8.  Secondary:   Alanine Aminotransferase (ALT) Absolute Change From Baseline to Month 12   [ Time Frame: 12 months ]

9.  Secondary:   Aspartate Aminotransferase (AST) Absolute Change From Baseline to Month 12   [ Time Frame: 12 months ]

10.  Secondary:   Gamma-glutamyltransferase (GGT) Absolute Change From Baseline to Month 12   [ Time Frame: 12 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Medical Information
Organization: Intercept Pharmaceuticals, Inc.
phone: 844-782-4278
e-mail: medinfo@interceptpharma.com


Publications of Results:
Other Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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: June 12, 2017