Study of INT 747 in Combination With URSO in Patients With Primay Biliary Cirrhosis (PBC)

A Study of INT 747 (6α-ethyl Chenodeoxycholic Acid (6-ECDCA)) in Combination With Ursodeoxycholic Acid (URSO®, UDCA) in Patients With Primary Biliary Cirrhosis

The primary hypothesis is that INT-747 will cause a reduction in alkaline phosphatase levels in Primary Biliary Cirrhosis patients, over a 12 week treatment period, as compared to placebo.

Study Overview

• Status: Terminated
• Conditions: Liver Cirrhosis, Biliary
• Intervention / Treatment: Drug: Placebo; Drug: INT-747; Drug: Ursodeoxycholic Acid (URSO)

Detailed Description

None provided

• Study Type: Interventional
• Enrollment (Actual): 165
• Phase: Phase 2

Contacts and Locations

Study Locations

• Austria
  • Graz, Austria, A8036
    • Karls-Franzens University
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 4N1
      • University of Calgary
    • Edmonton, Alberta, Canada, T5G 2X8
      • University of Alberta
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3E 3P4
      • University of Manitoba
  • Ontario
    • Toronto, Ontario, Canada, M5T2S8
      • University of Toronto Western Hospital
  • Quebec
    • Montreal, Quebec, Canada, H2X 1P1
      • Centre de Recherche du CHUM / University of Montreal
• France
  • Lyon, France, 69288
    • Hôpital de l'Hôtel Dieu
• Germany
  • Frankfurt, Germany, 60590
    • Johann Wolfgang Goethe University
  • Hamburg, Germany, D20246
    • University Medical Centre Hamburg-Eppendorf
  • Hannover, Germany, 30623
    • Medical School of Hannover
  • Munich, Germany, D81377
    • University of Munich
• Netherlands
  • Amsterdam, Netherlands, NL-1100
    • AMC University of Amsterdam
  • Rotterdam, Netherlands, 3000
    • Erasmus Medical Centre
• Spain
  • Barcelona, Spain, 08036
    • Hospital Clinic i Provincial
• United Kingdom
  • Edinburgh, United Kingdom, EH16 4SA
    • Royal Infirmary
  • Newcastle Upon Tyne, United Kingdom, NE2 4HH
    • University Upon Tyne/Newcastle
  • Birmingham
    • Edgbaston, Birmingham, United Kingdom, B15 2TH
      • Queen Elizabeth Medical Center
  • London
    • Hampstead, London, United Kingdom, NW3 2QG
      • Royal Free Hospital
  • Oxford
    • Headington, Oxford, United Kingdom, OX3 9DU
      • John Radcliffe Hospital
• United States
  • Florida
    • Gainesville, Florida, United States, 32610
      • U Florida Hepatology
    • Miami, Florida, United States, 33136
      • University of Miami - Center for Liver Diseases
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Tufts Medical Center
  • Michigan
    • Novi, Michigan, United States, 48377
      • Henry Ford Health Center Columbus
  • Minnesota
    • Rochester, Minnesota, United States, 555905
      • Mayo Clinic
  • Missouri
    • Saint Louis, Missouri, United States, 63104
      • Saint Louis University
  • New York
    • New York, New York, United States, 10003
      • Beth Israel Medical Center
    • New York, New York, United States, 10029
      • Mt. Sinai School of Medicine
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Texas
    • Dallas, Texas, United States, 75390
      • UT Southwestern Medical Center
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine
  • Virginia
    • Richmond, Virginia, United States, 23249
      • McGuire DVAMC
  • Washington
    • Seattle, Washington, United States, 98101
      • Virginia Mason Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male or female age 18 to 70 years.
• Stable dose of ursodeoxycholic acid (URSO, UDCA) for at least 6 months prior to screening.
• Female patients must be postmenopausal, surgically sterile, or prepared to use 2 methods of contraception with all sexual partners during the study and for 14 days after the end of dosing.
• Male patients must be prepared to use 2 methods of contraception with all sexual partners during the study and for 14 days after the end of the dosing.

• Proven or likely PBC, as demonstrated by the patient presenting with at least 2 of the following 3 diagnostic factors:

  1. History of increased AP levels for at least 6 months prior to Day 0
  2. Positive AMA titer (>1:40 titer on immunofluorescence or M2 positive by ELISA) or PBC-specific antinuclear antibodies (antinuclear dot and nuclear rim positive)
  3. Liver biopsy consistent with PBC.
• Screening AP value between 1.5 and 10 × ULN.

Exclusion Criteria:

• Administration of the following drugs at any time during the 3 months prior to screening for the study: colchicine, methotrexate, azathioprine, or systemic corticosteroids.
• Screening conjugated (direct) bilirubin >2 × ULN.
• Screening ALT or AST >5 × ULN.
• Screening serum creatinine >1.5 mg/dL (133 mol/L).
• History or presence of hepatic decompensation (e.g., variceal bleeds, encephalopathy, or poorly controlled ascites).
• History or presence of other concomitant liver diseases including hepatitis due to hepatitis B or C virus (HCV, HBV) infection, primary sclerosing cholangitis (PSC), alcoholic liver disease, definite autoimmune liver disease or biopsy proven nonalcoholic steatohepatitis (NASH).
• Pregnancy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: INT-747 10 mg; INT-747 10 mg once daily in combination with URSO for 12 weeks.	Drug: INT-747; Once a day (QD) by mouth (PO); Other Names: Obeticholic acid (OCA), 6-ECDCA; Drug: Ursodeoxycholic Acid (URSO); Stable dose for at least 6 months prior to screening. Dose as prescribed by physician.; Other Names: URSO, UDCA
Experimental: INT-747 25 mg; INT-747 25 mg once daily in combination with URSO for 12 weeks.	Drug: INT-747; Once a day (QD) by mouth (PO); Other Names: Obeticholic acid (OCA), 6-ECDCA; Drug: Ursodeoxycholic Acid (URSO); Stable dose for at least 6 months prior to screening. Dose as prescribed by physician.; Other Names: URSO, UDCA
Experimental: INT-747 50 mg; INT-747 50 mg once daily in combination with URSO for 12 weeks.	Drug: INT-747; Once a day (QD) by mouth (PO); Other Names: Obeticholic acid (OCA), 6-ECDCA; Drug: Ursodeoxycholic Acid (URSO); Stable dose for at least 6 months prior to screening. Dose as prescribed by physician.; Other Names: URSO, UDCA
Placebo Comparator: Placebo; Placebo once daily in combination with URSO for 12 weeks.	Drug: Placebo; Placebo; Drug: INT-747; Once a day (QD) by mouth (PO); Other Names: Obeticholic acid (OCA), 6-ECDCA; Drug: Ursodeoxycholic Acid (URSO); Stable dose for at least 6 months prior to screening. Dose as prescribed by physician.; Other Names: URSO, UDCA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Baseline and Up to Day 85

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	Baseline and at Days 15, 29, 57 and 85
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.	Baseline and at Days 15, 29, 57 and 85
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.	Baseline and at 3, 6, 9 and 12 Months
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).	Baseline and at Days 15, 29, 57 and 85
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.	Baseline and at Days 15, 29, 57 and 85
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).	Baseline and at Days 15, 29, 57 and 85
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.	Baseline and at Days 15, 29, 57 and 85
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.	Baseline and at 3, 6, 9 and 12 Months
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.	Baseline and up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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).	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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.	Baseline and Up to Day 85
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	Baseline and at 3, 6, 9 and 12 Months
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.	Baseline and at Days 29, 57 and 85
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.	Baseline and at 6 and 12 months
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	Baseline and at Days 29, 57 and 85
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.	Up to Day 85
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.	Baseline and at Days 29, 57 and 85
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.	Up to 12 Months

Collaborators and Investigators

• Sponsor: Intercept Pharmaceuticals
• Investigators: Study Director: David A Shapiro, MD, Intercept Pharmaceuticals - Chief Medical Officer

Publications and helpful links

General Publications

• Hirschfield GM, Mason A, Luketic V, Lindor K, Gordon SC, Mayo M, Kowdley KV, Vincent C, Bodhenheimer HC Jr, Pares A, Trauner M, Marschall HU, Adorini L, Sciacca C, Beecher-Jones T, Castelloe E, Bohm O, Shapiro D. Efficacy of obeticholic acid in patients with primary biliary cirrhosis and inadequate response to ursodeoxycholic acid. Gastroenterology. 2015 Apr;148(4):751-61.e8. doi: 10.1053/j.gastro.2014.12.005. Epub 2014 Dec 11.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2007
• Primary Completion (Actual): August 1, 2009
• Study Completion (Actual): December 1, 2010

Study Registration Dates

• First Submitted: October 27, 2007
• First Submitted That Met QC Criteria: October 27, 2007
• First Posted (Estimated): October 30, 2007

Study Record Updates

• Last Update Posted (Estimated): February 6, 2024
• Last Update Submitted That Met QC Criteria: January 11, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: PBC, Primary Biliary Cirrhosis, Liver,
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Liver Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis, Intrahepatic; Cholestasis; Fibrosis; Liver Cirrhosis; Liver Cirrhosis, Biliary; Gastrointestinal Agents; Cholagogues and Choleretics; Ursodeoxycholic Acid
• Other Study ID Numbers: 747-202