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Trial record 22 of 34 for:    Biliary Cirrhosis, Primary, 4

Phase 2 Study on Effects of Obeticholic Acid (OCA) on Lipoprotein Metabolism in Subjects With Primary Biliary Cirrhosis

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ClinicalTrials.gov Identifier: NCT01865812
Recruitment Status : Completed
First Posted : May 31, 2013
Results First Posted : October 19, 2016
Last Update Posted : March 14, 2018
Sponsor:
Information provided by (Responsible Party):
Intercept Pharmaceuticals

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Basic Science
Condition Primary Biliary Cirrhosis
Intervention Drug: obeticholic acid (OCA)
Enrollment 26
Recruitment Details Recruitment started 19 Nov 2013 and completed 16 May 2014 in the primary treatment phase. Thirty-three subjects were screened and twenty-six enrolled.
Pre-assignment Details Screening window was up to 20 days in duration to assess eligibility. Stable dose of ursodeoxycholic acid for 3 months prior to Day 0 required. 28 day washout period for Bile Acid Sequestrants, and no serum-lipid modifying agents for 3 months prior to Day 0. Subjects may participate in an open label Long Term Safety Extension (LTSE) study.
Arm/Group Title OCA: 10 mg
Hide Arm/Group Description

obeticholic acid, oral administration, 10 mg, 8 weeks

obeticholic acid (OCA): All subjects will be treated with OCA (oral administration, 10 mg, once daily) for 8 weeks and should continue their prestudy dose of ursodeoxycholic acid (UDCA). After completion of the 8 week Primary Treatment Phase of the study and the 4 week follow up period, during which time subjects do not take OCA, all eligible subjects will be offered the opportunity to enter an open label long term safety extension phase, during which they will receive 10 mg OCA daily for up to 2 years.

Period Title: Overall Study
Started 26
Completed 24
Not Completed 2
Reason Not Completed
Adverse Event             2
Arm/Group Title OCA: 10 mg
Hide Arm/Group Description

obeticholic acid, oral administration, 10 mg, 8 weeks

obeticholic acid (OCA): All subjects will be treated with OCA (oral administration, 10 mg, once daily) for 8 weeks and should continue their prestudy dose of ursodeoxycholic acid (UDCA). After completion of the 8 week Primary Treatment Phase of the study and the 4 week follow up period, during which time subjects do not take OCA, all eligible subjects will be offered the opportunity to enter an open label long term safety extension phase, during which they will receive 10 mg OCA daily for up to 2 years.

Overall Number of Baseline Participants 26
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants
<=18 years
0
   0.0%
Between 18 and 65 years
22
  84.6%
>=65 years
4
  15.4%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 26 participants
56.5  (9.15)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants
Female
25
  96.2%
Male
1
   3.8%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 26 participants
White 25
Non-White/Hispanic 1
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 26 participants
26
HDL Cholesterol Concentration (mmol/L)  
Mean (Standard Deviation)
Unit of measure:  mmol/L
Number Analyzed 26 participants
1.86  (0.51798)
HDL Particle Size (nm)  
Mean (Standard Deviation)
Unit of measure:  Nm
Number Analyzed 26 participants
10.19  (0.734)
HDL Particle Concentration (total) (µmol/L)  
Mean (Standard Deviation)
Unit of measure:  µmol/L
Number Analyzed 26 participants
30.08  (7.228)
1.Primary Outcome
Title Absolute Change From Baseline in High-density Lipoprotein (HDL) Cholesterol Concentration
Hide Description [Not Specified]
Time Frame Baseline, Week 8
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title OCA: 10 mg
Hide Arm/Group Description:

obeticholic acid, oral administration, 10 mg, 8 weeks

obeticholic acid (OCA): All subjects will be treated with OCA (oral administration, 10 mg, once daily) for 8 weeks and should continue their prestudy dose of ursodeoxycholic acid (UDCA). After completion of the 8 week Primary Treatment Phase of the study and the 4 week follow up period, during which time subjects do not take OCA, all eligible subjects will be offered the opportunity to enter an open label long term safety extension phase, during which they will receive 10 mg OCA daily for up to 2 years.

Overall Number of Participants Analyzed 23
Least Squares Mean (95% Confidence Interval)
Unit of Measure: mmol/L
-0.38
(-0.51 to -0.24)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA: 10 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.38
Confidence Interval (2-Sided) 95%
-0.51 to -0.24
Estimation Comments [Not Specified]
2.Primary Outcome
Title Absolute Change From Baseline in High-density Lipoprotein (HDL) Particle Size
Hide Description [Not Specified]
Time Frame Baseline, Week 8
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title OCA: 10 mg
Hide Arm/Group Description:

obeticholic acid, oral administration, 10 mg, 8 weeks

obeticholic acid (OCA): All subjects will be treated with OCA (oral administration, 10 mg, once daily) for 8 weeks and should continue their prestudy dose of ursodeoxycholic acid (UDCA). After completion of the 8 week Primary Treatment Phase of the study and the 4 week follow up period, during which time subjects do not take OCA, all eligible subjects will be offered the opportunity to enter an open label long term safety extension phase, during which they will receive 10 mg OCA daily for up to 2 years.

Overall Number of Participants Analyzed 23
Least Squares Mean (95% Confidence Interval)
Unit of Measure: nm
-0.44
(-0.63 to -0.25)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA: 10 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.44
Confidence Interval (2-Sided) 95%
-0.63 to -0.25
Estimation Comments [Not Specified]
3.Primary Outcome
Title Absolute Change From Baseline in High-density Lipoprotein (HDL) Particle Concentration
Hide Description [Not Specified]
Time Frame Baseline, Week 8
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title OCA: 10 mg
Hide Arm/Group Description:

obeticholic acid, oral administration, 10 mg, 8 weeks

obeticholic acid (OCA): All subjects will be treated with OCA (oral administration, 10 mg, once daily) for 8 weeks and should continue their prestudy dose of ursodeoxycholic acid (UDCA). After completion of the 8 week Primary Treatment Phase of the study and the 4 week follow up period, during which time subjects do not take OCA, all eligible subjects will be offered the opportunity to enter an open label long term safety extension phase, during which they will receive 10 mg OCA daily for up to 2 years.

Overall Number of Participants Analyzed 23
Least Squares Mean (95% Confidence Interval)
Unit of Measure: umol/L
-0.06
(-1.58 to 1.46)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OCA: 10 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.06
Confidence Interval (2-Sided) 95%
-1.58 to 1.46
Estimation Comments [Not Specified]
Time Frame Adverse Event data were collected from the time of the first dose of investigational product until the subject fully completed their participation in the Primary Treatment Phase (PTP) of the study, up to 12 weeks.
Adverse Event Reporting Description Adverse Event data was collected through the Long Term Safety Extension (LTSE) phase, up to two years, which completed September 2016. Adverse Event data from the LTSE phase will be available on the registry once the data are final.
 
Arm/Group Title OCA: 10 mg
Hide Arm/Group Description

obeticholic acid, oral administration, 10 mg, 8 weeks

obeticholic acid (OCA): All subjects will be treated with OCA (oral administration, 10 mg, once daily) for 8 weeks and should continue their prestudy dose of ursodeoxycholic acid (UDCA). After completion of the 8 week Primary Treatment Phase of the study and the 4 week follow up period, during which time subjects do not take OCA, all eligible subjects will be offered the opportunity to enter an open label long term safety extension phase, during which they will receive 10 mg OCA daily for up to 2 years.

All-Cause Mortality
OCA: 10 mg
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
OCA: 10 mg
Affected / at Risk (%) # Events
Total   1/26 (3.85%)    
Gastrointestinal disorders   
Ascites  1  1/26 (3.85%)  1
Hepatobiliary disorders   
Jaundice  1  1/26 (3.85%)  1
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: 10 mg
Affected / at Risk (%) # Events
Total   18/26 (69.23%)    
Gastrointestinal disorders   
Diarrhoea  1  2/26 (7.69%)  4
Constipation  1  2/26 (7.69%)  2
Nausea  1  2/26 (7.69%)  2
General disorders   
Fatigue  1  4/26 (15.38%)  4
Oedema peripheral  1  2/26 (7.69%)  3
Pyrexia  1  2/26 (7.69%)  2
Infections and infestations   
Sinusitis  1  2/26 (7.69%)  2
Musculoskeletal and connective tissue disorders   
Back pain  1  2/26 (7.69%)  2
Nervous system disorders   
Dizziness  1  3/26 (11.54%)  3
Headache  1  2/26 (7.69%)  2
Skin and subcutaneous tissue disorders   
Pruritus  1  13/26 (50.00%)  17
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
Layout table for Results Point of Contact information
Name/Title: Medical Information
Organization: Intercept Pharmaceuticals, Inc.
Phone: 844-782-4278
EMail: medinfo@interceptpharma.com
Layout table for additonal information
Responsible Party: Intercept Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01865812     History of Changes
Other Study ID Numbers: 747-205
First Submitted: May 23, 2013
First Posted: May 31, 2013
Results First Submitted: August 25, 2016
Results First Posted: October 19, 2016
Last Update Posted: March 14, 2018