search
Back to results

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

Primary Purpose

Primary Biliary Cirrhosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Obeticholic Acid (OCA)
Placebo
Sponsored by
Intercept Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Biliary Cirrhosis focused on measuring Primary Biliary Cholangitis, Primary Biliary Cirrhosis, PBC, Cirrhosis, Liver

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Definite or probable PBC diagnosis (consistent with American Association for the Study of Liver Disease [AASLD] and European Association for Study of the Liver [EASL] Practice Guidelines; [Lindor 2009; EASL 2009]), as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors:

    • History of elevated alkaline phosphatase (ALP) levels for at least 6 months
    • Positive antimitochondrial antibodies (AMA) titer or if AMA negative or in low titer (<1:80) PBC specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components (pyruvate dehydrogenase complex-E2 [PDC-E2], 2-oxo-glutaric acid dehydrogenase complex)
    • Liver biopsy consistent with PBC
  2. At least 1 of the following qualifying biochemistry values:

    • ALP ≥ 1.67x upper limit of normal (ULN)
    • Total bilirubin > ULN but < 2x ULN
  3. Age ≥ 18 years
  4. Taking ursodeoxycholic acid (UDCA) for at least 12 months (stable dose for ≥ 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ≥ 3 months) prior to Day 0.
  5. Contraception: Female participants must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use ≥ 1 effective (≤ 1% failure rate) method of contraception during the trial and for 30 days after the end of treatment (EOT) visit. Effective methods of contraception are considered to be:

    • Hormonal (for example, contraceptive pill, patch, intramuscular implant or injection); or
    • Double barrier method, that is, (a) condom (male or female) or (b) diaphragm, with spermicide; or
    • Intrauterine device (IUD); or
    • Vasectomy (partner); or
    • Sexual abstinence
  6. Must provide written informed consent and agree to comply with the trial protocol.

Exclusion Criteria:

  1. History or presence of other concomitant liver diseases including:

    • Hepatitis C virus (HCV) infection; participants with active hepatitis B (HBV) infection will be excluded, however, participants who have seroconverted (hepatitis B surface antigen [Hbs Ag] and hepatitis B e antigen [Hbe Ag] negative) may be included after consultation with the medical monitor.
    • Primary sclerosing cholangitis (PSC)
    • Alcoholic liver disease
    • Definite autoimmune liver disease or overlap hepatitis
    • Nonalcoholic steatohepatitis (NASH)
    • Gilbert's Syndrome (due to interpretability of bilirubin levels)
  2. Presence of clinical complications of PBC or clinically significant hepatic decompensation, including:

    • History of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥ 15
    • Portal hypertension with complications, including: known gastric or large esophageal varices, poorly controlled or diuretic resistant ascites, history of variceal bleeds or related therapeutic or prophylactic interventions (for example, beta blockers, insertion of variceal bands or transjugular intrahepatic portosystemic shunt [TIPS]), or hepatic encephalopathy
    • Cirrhosis with complications, including history or presence of: spontaneous bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2x ULN
    • Hepatorenal syndrome (type I or II) or Screening serum creatinine > 2 mg/deciliter dL) (178 micromole [µmol])/liter [L])
  3. Participants with severe pruritus or those requiring systemic treatment for pruritus (for example, with bile acid sequestrants [BAS] or rifampicin) within 2 months of Day 0 will be excluded
  4. Administration of the following medications is prohibited as specified below:

    • Prohibited 6 months prior to Day 0 and throughout the trial (that is, to last dose and/or EOT): azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates; budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazide, or nitrofurantoin)
    • Prohibited 12 months prior to Day 0 and throughout the trial (that is, to last dose and/or EOT): antibodies or immunotherapy directed against interleukins or other cytokines or chemokines
  5. Participants who have previously participated in a clinical trial of OCA will not be allowed to participate
  6. History or presence of clinically concerning cardiac arrhythmias likely to affect survival during the trial, or prolongation of Screening (pretreatment) QT or QTc interval of > 500 milliseconds (msec)
  7. If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
  8. Known history of human immunodeficiency virus (HIV) infection
  9. Presence of any other disease or condition that is interfering with the absorption, distribution, metabolism, or excretion of drugs including bile salt metabolism in the intestine. Participants with inflammatory bowel disease or who have undergone gastric bypass procedures will be excluded (gastric lap band is acceptable).
  10. Medical conditions that may cause nonhepatic increases in ALP (for example, Paget's disease) or which may diminish life expectancy to < 2 years, including known cancers (except carcinomas in situ or other stable, relatively benign conditions such as chronic lymphatic leukemia)
  11. Other clinically significant medical conditions that are not well controlled or for which medication needs are anticipated to change during the trial
  12. Anticipated changes to current concomitant medications during the course of the trial
  13. History of alcohol abuse, defined as consumption of more than 210 mL of alcohol per week (that is, the equivalent of fourteen 4-ounce (125 mL) glasses of wine or fourteen 12 ounce cans/bottles of beer), or other substance abuse within 1 year prior to Day 0
  14. Participation in another investigational drug, biologic, or medical device trial within 30 days prior to Screening
  15. History of noncompliance with medical regimens, or participants who are considered to be potentially unreliable
  16. Blood or plasma donation within 30 days prior to Day 0
  17. Mental instability or incompetence, such that the validity of informed consent or compliance with the trial is uncertain

Sites / Locations

  • UC Davis Medical Center
  • Scripps Clinic
  • University of Colorado, Denver
  • University of Chicago
  • Indiana University School of Medicine
  • Henry Ford Health System
  • St. Louis University
  • Beth Israel Medical Center
  • Duke University Medical Center
  • University of Texas Southwestern Medical Center
  • Baylor College of Medicine
  • Liver Institute of Virginia
  • Liver Institute of Virginia
  • Virginia Commonwealth University/McGuire DVAMC
  • Swedish Medical Center
  • Royal Prince Alfred Hospital
  • Austin Hospital
  • The Alfred Hospital
  • Medizinische Universität Innsbruck
  • Medizinische Universität Wien
  • UZ Leuven
  • Toronto Western Hospital Liver Centre
  • CHUM Hôpital St-Luc
  • Hopital Haut-Leveque
  • Universitätsklinikum Aachen
  • Friedrich-Alexander-Universität Erlangen
  • Klinikum der Johann-Wolfgang Goethe Universität Frankfurt am Main
  • Universitätsklinikum Hamburg Eppendorf
  • Medizinische Hochschule Hannover
  • Medizinische Universitätsklinik
  • Gastroenterologische Gemeinschaftspraxis, Dres. Felten / Hartmann / Hüppe
  • Universitätsklinikum des Saarlandes
  • Universitätsklinikum Leipzig
  • LMU Klinikum der Universität München
  • Dip. Medicina Clinica - Università di Bologna
  • Dip. Medicina Clinica- Università di Bologna
  • Azienda Ospedaliera di Padova - Gastroenterologia
  • Istituto Clinico Humanitas
  • VUmc Amsterdam
  • AMC Amsterdam
  • UMC St. Radboud, Nijmegen
  • UMC Utrecht
  • All-Medicus
  • Klinika Gastroenterologii i Hepatologii SP CSK im. prof. K. Gibinskiego SUM
  • Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie
  • Niepubliczny Zakład Opieki Zdrowotnej "SONOMED"
  • Centrum Onkologii - Instytut im. Marii Skłodowskiej - Curie, Klinika Gastroenterologii
  • Hospital Vall d'Hebron
  • Hospital Clinic de Barcelona
  • Sahlgrenska University Hospital
  • Queen Elizabeth Hospital
  • Bristol Royal Infirmary
  • Ninewells Hospital Dundee
  • Forth Valley Royal Hospital
  • The Royal Free Hospital
  • Manchester Royal Infirmary
  • Institute of Cellular Medicine, Newcastle University
  • Nottingham University Hospitals NHS Trust
  • Oxford University Hospitals Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Placebo Comparator

Experimental

Arm Label

DB OCA 5-10 mg

DB OCA 10 mg

DB Placebo

LTSE OCA

Arm Description

OCA 5 milligram (mg) for 6 months and then titrating up to 10 mg based on tolerability and response for remaining 6 months of the DB phase.

OCA 10 mg for 12 months during the DB phase.

Matching placebo for 12 months during the DB phase.

After completion of the 12-month DB phase all participants were offered the opportunity to enter an open-label LTSE for up to 5 years beginning at 5 mg OCA. Initially, participants were allowed to titrate to doses up to 25 mg, however, the maximum dose was then limited to 10 mg. Participants who were previously titrated above 10 mg OCA daily were down-titrated to ≤10 mg OCA daily.

Outcomes

Primary Outcome Measures

DB Phase: Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin, 10 mg OCA Versus Placebo
Percentage of participants at Month 12 with ALP < 1.67 x upper limit of normal (ULN) and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
LTSE Phase: Composite Endpoint ALP And Total Bilirubin
Percentage of participants at Months 24, 36, 48, and 60 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline. DB Month 12 is the baseline for the LTSE phase.

Secondary Outcome Measures

DB Phase: Composite Endpoint ALP And Total Bilirubin, 10 mg Versus Placebo
Percentage of participants at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
DB Phase: Composite Endpoint ALP And Total Bilirubin, 5-10 mg Versus Placebo
Percentage of participants at Month 12 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
DB Phase: Composite Endpoint ALP And Total Bilirubin, 5-10 mg Versus Placebo
Percentage of participants at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
DB Phase: ALP Absolute Change From Baseline To Month 12
Blood samples were evaluated for ALP levels. ALP Absolute Change From Baseline (ALP at Month 12 - ALP at Baseline) is presented.
DB Phase: Total Bilirubin Absolute Change From Baseline To Month 12
Blood samples were evaluated for bilirubin levels. Total bilirubin absolute change from baseline (total bilirubin at Month 12 - total bilirubin at Baseline) is presented.
DB Phase: Direct Bilirubin Absolute Change From Baseline To Month 12
Blood samples were evaluated for bilirubin levels. Direct bilirubin absolute change from baseline (direct bilirubin at Month 12 - direct bilirubin at Baseline) is presented.
DB Phase: Alanine Aminotransferase (ALT) Absolute Change From Baseline To Month 12
Blood samples were evaluated for ALT levels. ALT absolute change from baseline (ALT at Month 12 - ALT at Baseline) is presented.
DB Phase: Aspartate Aminotransferase (AST) Absolute Change From Baseline To Month 12
Blood samples were evaluated for AST levels. AST absolute change from baseline (AST at Month 12 - AST at Baseline) is presented.
DB Phase: Gamma-glutamyltransferase (GGT) Absolute Change From Baseline To Month 12
Blood samples were evaluated for GGT levels. GGT absolute change from baseline (GGT at Month 12 - GGT at Baseline) is presented.
LTSE Phase: ALP Levels
Blood samples were evaluated for ALP levels.
LTSE Phase: ALP Change From DB Baseline
Blood samples were evaluated for ALP levels. ALP Change From Baseline (ALP at LTSE Months 12, 24, 36, 48, and 60 - ALP at Baseline) is presented. DB baseline is the mean of all available evaluations prior to DB treatment.

Full Information

First Posted
November 14, 2011
Last Updated
April 9, 2021
Sponsor
Intercept Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT01473524
Brief Title
Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis
Acronym
POISE
Official Title
A Phase 3, Double-Blind, Placebo-Controlled Trial and Long-Term Safety Extension of Obeticholic Acid in Patients With Primary Biliary Cirrhosis
Study Type
Interventional

2. Study Status

Record Verification Date
April 2021
Overall Recruitment Status
Completed
Study Start Date
January 2012 (undefined)
Primary Completion Date
December 2013 (Actual)
Study Completion Date
December 17, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Intercept Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The main objectives of the study were to assess the effects of Obeticholic Acid (OCA) on serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint and on safety in participants with primary biliary cirrhosis (PBC).
Detailed Description
The study included 2 phases: a 12-month randomized, double-blind (DB), placebo-controlled, parallel group phase, followed by a long-term safety extension (LTSE) phase up to 5 years. Participants from the 12-month DB phase, including those who received placebo, were eligible to participate in the open-label LTSE phase. The Month 12 visit from the DB phase served as the Day 1 visit of the LTSE phase. After completion of the 12-month DB phase all participants were offered the opportunity to enter an open-label LTSE for up to 5 years beginning at 5 mg OCA. Data for the LTSE phase is reported by the randomized dose group assigned in the DB phase.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Biliary Cirrhosis
Keywords
Primary Biliary Cholangitis, Primary Biliary Cirrhosis, PBC, Cirrhosis, Liver

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
217 (Actual)

8. Arms, Groups, and Interventions

Arm Title
DB OCA 5-10 mg
Arm Type
Experimental
Arm Description
OCA 5 milligram (mg) for 6 months and then titrating up to 10 mg based on tolerability and response for remaining 6 months of the DB phase.
Arm Title
DB OCA 10 mg
Arm Type
Experimental
Arm Description
OCA 10 mg for 12 months during the DB phase.
Arm Title
DB Placebo
Arm Type
Placebo Comparator
Arm Description
Matching placebo for 12 months during the DB phase.
Arm Title
LTSE OCA
Arm Type
Experimental
Arm Description
After completion of the 12-month DB phase all participants were offered the opportunity to enter an open-label LTSE for up to 5 years beginning at 5 mg OCA. Initially, participants were allowed to titrate to doses up to 25 mg, however, the maximum dose was then limited to 10 mg. Participants who were previously titrated above 10 mg OCA daily were down-titrated to ≤10 mg OCA daily.
Intervention Type
Drug
Intervention Name(s)
Obeticholic Acid (OCA)
Other Intervention Name(s)
6α-ethyl chenodeoxycholic acid (6-ECDCA), INT-747
Intervention Description
OCA was administered orally once daily and provided in tablet form in 2 strengths: 5 mg and 10 mg.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Matching placebo tablets were administered orally once daily.
Primary Outcome Measure Information:
Title
DB Phase: Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin, 10 mg OCA Versus Placebo
Description
Percentage of participants at Month 12 with ALP < 1.67 x upper limit of normal (ULN) and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame
DB Month 12
Title
LTSE Phase: Composite Endpoint ALP And Total Bilirubin
Description
Percentage of participants at Months 24, 36, 48, and 60 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline. DB Month 12 is the baseline for the LTSE phase.
Time Frame
Baseline (DB Month 12), LTSE Months 24, 36, 48, and 60
Secondary Outcome Measure Information:
Title
DB Phase: Composite Endpoint ALP And Total Bilirubin, 10 mg Versus Placebo
Description
Percentage of participants at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame
DB Month 6
Title
DB Phase: Composite Endpoint ALP And Total Bilirubin, 5-10 mg Versus Placebo
Description
Percentage of participants at Month 12 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame
DB Month 12
Title
DB Phase: Composite Endpoint ALP And Total Bilirubin, 5-10 mg Versus Placebo
Description
Percentage of participants at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Time Frame
DB Month 6
Title
DB Phase: ALP Absolute Change From Baseline To Month 12
Description
Blood samples were evaluated for ALP levels. ALP Absolute Change From Baseline (ALP at Month 12 - ALP at Baseline) is presented.
Time Frame
Baseline, DB Month 12
Title
DB Phase: Total Bilirubin Absolute Change From Baseline To Month 12
Description
Blood samples were evaluated for bilirubin levels. Total bilirubin absolute change from baseline (total bilirubin at Month 12 - total bilirubin at Baseline) is presented.
Time Frame
Baseline, DB Month 12
Title
DB Phase: Direct Bilirubin Absolute Change From Baseline To Month 12
Description
Blood samples were evaluated for bilirubin levels. Direct bilirubin absolute change from baseline (direct bilirubin at Month 12 - direct bilirubin at Baseline) is presented.
Time Frame
Baseline, DB Month 12
Title
DB Phase: Alanine Aminotransferase (ALT) Absolute Change From Baseline To Month 12
Description
Blood samples were evaluated for ALT levels. ALT absolute change from baseline (ALT at Month 12 - ALT at Baseline) is presented.
Time Frame
Baseline, DB Month 12
Title
DB Phase: Aspartate Aminotransferase (AST) Absolute Change From Baseline To Month 12
Description
Blood samples were evaluated for AST levels. AST absolute change from baseline (AST at Month 12 - AST at Baseline) is presented.
Time Frame
Baseline, DB Month 12
Title
DB Phase: Gamma-glutamyltransferase (GGT) Absolute Change From Baseline To Month 12
Description
Blood samples were evaluated for GGT levels. GGT absolute change from baseline (GGT at Month 12 - GGT at Baseline) is presented.
Time Frame
Baseline, DB Month 12
Title
LTSE Phase: ALP Levels
Description
Blood samples were evaluated for ALP levels.
Time Frame
LTSE Day 0 and LTSE Months 12, 24, 36, 48, and 60
Title
LTSE Phase: ALP Change From DB Baseline
Description
Blood samples were evaluated for ALP levels. ALP Change From Baseline (ALP at LTSE Months 12, 24, 36, 48, and 60 - ALP at Baseline) is presented. DB baseline is the mean of all available evaluations prior to DB treatment.
Time Frame
DB Baseline, LTSE Months 12, 24, 36, 48, and 60

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Definite or probable PBC diagnosis (consistent with American Association for the Study of Liver Disease [AASLD] and European Association for Study of the Liver [EASL] Practice Guidelines; [Lindor 2009; EASL 2009]), as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors: History of elevated alkaline phosphatase (ALP) levels for at least 6 months Positive antimitochondrial antibodies (AMA) titer or if AMA negative or in low titer (<1:80) PBC specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components (pyruvate dehydrogenase complex-E2 [PDC-E2], 2-oxo-glutaric acid dehydrogenase complex) Liver biopsy consistent with PBC At least 1 of the following qualifying biochemistry values: ALP ≥ 1.67x upper limit of normal (ULN) Total bilirubin > ULN but < 2x ULN Age ≥ 18 years Taking ursodeoxycholic acid (UDCA) for at least 12 months (stable dose for ≥ 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ≥ 3 months) prior to Day 0. Contraception: Female participants must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use ≥ 1 effective (≤ 1% failure rate) method of contraception during the trial and for 30 days after the end of treatment (EOT) visit. Effective methods of contraception are considered to be: Hormonal (for example, contraceptive pill, patch, intramuscular implant or injection); or Double barrier method, that is, (a) condom (male or female) or (b) diaphragm, with spermicide; or Intrauterine device (IUD); or Vasectomy (partner); or Sexual abstinence Must provide written informed consent and agree to comply with the trial protocol. Exclusion Criteria: History or presence of other concomitant liver diseases including: Hepatitis C virus (HCV) infection; participants with active hepatitis B (HBV) infection will be excluded, however, participants who have seroconverted (hepatitis B surface antigen [Hbs Ag] and hepatitis B e antigen [Hbe Ag] negative) may be included after consultation with the medical monitor. Primary sclerosing cholangitis (PSC) Alcoholic liver disease Definite autoimmune liver disease or overlap hepatitis Nonalcoholic steatohepatitis (NASH) Gilbert's Syndrome (due to interpretability of bilirubin levels) Presence of clinical complications of PBC or clinically significant hepatic decompensation, including: History of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥ 15 Portal hypertension with complications, including: known gastric or large esophageal varices, poorly controlled or diuretic resistant ascites, history of variceal bleeds or related therapeutic or prophylactic interventions (for example, beta blockers, insertion of variceal bands or transjugular intrahepatic portosystemic shunt [TIPS]), or hepatic encephalopathy Cirrhosis with complications, including history or presence of: spontaneous bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2x ULN Hepatorenal syndrome (type I or II) or Screening serum creatinine > 2 mg/deciliter dL) (178 micromole [µmol])/liter [L]) Participants with severe pruritus or those requiring systemic treatment for pruritus (for example, with bile acid sequestrants [BAS] or rifampicin) within 2 months of Day 0 will be excluded Administration of the following medications is prohibited as specified below: Prohibited 6 months prior to Day 0 and throughout the trial (that is, to last dose and/or EOT): azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates; budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazide, or nitrofurantoin) Prohibited 12 months prior to Day 0 and throughout the trial (that is, to last dose and/or EOT): antibodies or immunotherapy directed against interleukins or other cytokines or chemokines Participants who have previously participated in a clinical trial of OCA will not be allowed to participate History or presence of clinically concerning cardiac arrhythmias likely to affect survival during the trial, or prolongation of Screening (pretreatment) QT or QTc interval of > 500 milliseconds (msec) If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating Known history of human immunodeficiency virus (HIV) infection Presence of any other disease or condition that is interfering with the absorption, distribution, metabolism, or excretion of drugs including bile salt metabolism in the intestine. Participants with inflammatory bowel disease or who have undergone gastric bypass procedures will be excluded (gastric lap band is acceptable). Medical conditions that may cause nonhepatic increases in ALP (for example, Paget's disease) or which may diminish life expectancy to < 2 years, including known cancers (except carcinomas in situ or other stable, relatively benign conditions such as chronic lymphatic leukemia) Other clinically significant medical conditions that are not well controlled or for which medication needs are anticipated to change during the trial Anticipated changes to current concomitant medications during the course of the trial History of alcohol abuse, defined as consumption of more than 210 mL of alcohol per week (that is, the equivalent of fourteen 4-ounce (125 mL) glasses of wine or fourteen 12 ounce cans/bottles of beer), or other substance abuse within 1 year prior to Day 0 Participation in another investigational drug, biologic, or medical device trial within 30 days prior to Screening History of noncompliance with medical regimens, or participants who are considered to be potentially unreliable Blood or plasma donation within 30 days prior to Day 0 Mental instability or incompetence, such that the validity of informed consent or compliance with the trial is uncertain
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Christian Weyer, MD
Organizational Affiliation
Intercept Pharmaceuticals, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Scripps Clinic
City
San Diego
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
University of Colorado, Denver
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Indiana University School of Medicine
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Henry Ford Health System
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48377
Country
United States
Facility Name
St. Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
Beth Israel Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10003
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Liver Institute of Virginia
City
Newport News
State/Province
Virginia
ZIP/Postal Code
23602
Country
United States
Facility Name
Liver Institute of Virginia
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23226
Country
United States
Facility Name
Virginia Commonwealth University/McGuire DVAMC
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23249
Country
United States
Facility Name
Swedish Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States
Facility Name
Royal Prince Alfred Hospital
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Austin Hospital
City
Heidelberg
State/Province
Victoria
ZIP/Postal Code
3084
Country
Australia
Facility Name
The Alfred Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Facility Name
Medizinische Universität Innsbruck
City
Innsbruck
ZIP/Postal Code
6020
Country
Austria
Facility Name
Medizinische Universität Wien
City
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
B-3000
Country
Belgium
Facility Name
Toronto Western Hospital Liver Centre
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5T 2S8
Country
Canada
Facility Name
CHUM Hôpital St-Luc
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H2X 3J4
Country
Canada
Facility Name
Hopital Haut-Leveque
City
Pessac
ZIP/Postal Code
33604
Country
France
Facility Name
Universitätsklinikum Aachen
City
Aachen
ZIP/Postal Code
D-52074
Country
Germany
Facility Name
Friedrich-Alexander-Universität Erlangen
City
Erlangen
ZIP/Postal Code
D-91054
Country
Germany
Facility Name
Klinikum der Johann-Wolfgang Goethe Universität Frankfurt am Main
City
Frankfurt am Main
ZIP/Postal Code
60590
Country
Germany
Facility Name
Universitätsklinikum Hamburg Eppendorf
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
ZIP/Postal Code
D-30625
Country
Germany
Facility Name
Medizinische Universitätsklinik
City
Heidelberg
ZIP/Postal Code
D-69120
Country
Germany
Facility Name
Gastroenterologische Gemeinschaftspraxis, Dres. Felten / Hartmann / Hüppe
City
Herne
ZIP/Postal Code
D-44623
Country
Germany
Facility Name
Universitätsklinikum des Saarlandes
City
Homburg
ZIP/Postal Code
66421
Country
Germany
Facility Name
Universitätsklinikum Leipzig
City
Leipzig
ZIP/Postal Code
04103
Country
Germany
Facility Name
LMU Klinikum der Universität München
City
München
ZIP/Postal Code
D-81377
Country
Germany
Facility Name
Dip. Medicina Clinica - Università di Bologna
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Dip. Medicina Clinica- Università di Bologna
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Azienda Ospedaliera di Padova - Gastroenterologia
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Istituto Clinico Humanitas
City
Rozzano (MI)
ZIP/Postal Code
20089
Country
Italy
Facility Name
VUmc Amsterdam
City
Amsterdam
ZIP/Postal Code
1081 HV
Country
Netherlands
Facility Name
AMC Amsterdam
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
UMC St. Radboud, Nijmegen
City
Nijmegen
ZIP/Postal Code
6525
Country
Netherlands
Facility Name
UMC Utrecht
City
Utrecht
ZIP/Postal Code
3508 GA
Country
Netherlands
Facility Name
All-Medicus
City
Katowice
ZIP/Postal Code
40-660
Country
Poland
Facility Name
Klinika Gastroenterologii i Hepatologii SP CSK im. prof. K. Gibinskiego SUM
City
Katowice
ZIP/Postal Code
40-752
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie
City
Lublin
ZIP/Postal Code
20-954
Country
Poland
Facility Name
Niepubliczny Zakład Opieki Zdrowotnej "SONOMED"
City
Szczecin
ZIP/Postal Code
70-361
Country
Poland
Facility Name
Centrum Onkologii - Instytut im. Marii Skłodowskiej - Curie, Klinika Gastroenterologii
City
Warszawa
ZIP/Postal Code
02-781
Country
Poland
Facility Name
Hospital Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Clinic de Barcelona
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Sahlgrenska University Hospital
City
Gothenburg
ZIP/Postal Code
SE-41345
Country
Sweden
Facility Name
Queen Elizabeth Hospital
City
Birmingham
ZIP/Postal Code
B15 2TH
Country
United Kingdom
Facility Name
Bristol Royal Infirmary
City
Bristol
ZIP/Postal Code
BS2 8HW
Country
United Kingdom
Facility Name
Ninewells Hospital Dundee
City
Dundee
ZIP/Postal Code
DD1 9SY
Country
United Kingdom
Facility Name
Forth Valley Royal Hospital
City
Larbert
ZIP/Postal Code
FK5 4WR
Country
United Kingdom
Facility Name
The Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Manchester Royal Infirmary
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
Institute of Cellular Medicine, Newcastle University
City
Newcastle Upon Tyne
ZIP/Postal Code
NE2 4 HH
Country
United Kingdom
Facility Name
Nottingham University Hospitals NHS Trust
City
Nottingham
ZIP/Postal Code
NG7 2UH
Country
United Kingdom
Facility Name
Oxford University Hospitals Trust
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
19554543
Citation
Lindor KD, Gershwin ME, Poupon R, Kaplan M, Bergasa NV, Heathcote EJ; American Association for Study of Liver Diseases. Primary biliary cirrhosis. Hepatology. 2009 Jul;50(1):291-308. doi: 10.1002/hep.22906. No abstract available.
Results Reference
background
PubMed Identifier
27532829
Citation
Nevens F, Andreone P, Mazzella G, Strasser SI, Bowlus C, Invernizzi P, Drenth JP, Pockros PJ, Regula J, Beuers U, Trauner M, Jones DE, Floreani A, Hohenester S, Luketic V, Shiffman M, van Erpecum KJ, Vargas V, Vincent C, Hirschfield GM, Shah H, Hansen B, Lindor KD, Marschall HU, Kowdley KV, Hooshmand-Rad R, Marmon T, Sheeron S, Pencek R, MacConell L, Pruzanski M, Shapiro D; POISE Study Group. A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis. N Engl J Med. 2016 Aug 18;375(7):631-43. doi: 10.1056/NEJMoa1509840.
Results Reference
result
PubMed Identifier
30922873
Citation
Trauner M, Nevens F, Shiffman ML, Drenth JPH, Bowlus CL, Vargas V, Andreone P, Hirschfield GM, Pencek R, Malecha ES, MacConell L, Shapiro D. Long-term efficacy and safety of obeticholic acid for patients with primary biliary cholangitis: 3-year results of an international open-label extension study. Lancet Gastroenterol Hepatol. 2019 Jun;4(6):445-453. doi: 10.1016/S2468-1253(19)30094-9. Epub 2019 Mar 26.
Results Reference
result
PubMed Identifier
33319187
Citation
Harms MH, Hirschfield GM, Floreani A, Mayo MJ, Pares A, Liberman A, Malecha ES, Pencek R, MacConell L, Hansen BE. Obeticholic acid is associated with improvements in AST-to-platelet ratio index and GLOBE score in patients with primary biliary cholangitis. JHEP Rep. 2020 Sep 29;3(1):100191. doi: 10.1016/j.jhepr.2020.100191. eCollection 2021 Feb.
Results Reference
derived
PubMed Identifier
31606455
Citation
Bowlus CL, Pockros PJ, Kremer AE, Pares A, Forman LM, Drenth JPH, Ryder SD, Terracciano L, Jin Y, Liberman A, Pencek R, Iloeje U, MacConell L, Bedossa P. Long-Term Obeticholic Acid Therapy Improves Histological Endpoints in Patients With Primary Biliary Cholangitis. Clin Gastroenterol Hepatol. 2020 May;18(5):1170-1178.e6. doi: 10.1016/j.cgh.2019.09.050. Epub 2019 Oct 10.
Results Reference
derived
PubMed Identifier
25543678
Citation
Mousa HS, Lleo A, Invernizzi P, Bowlus CL, Gershwin ME. Advances in pharmacotherapy for primary biliary cirrhosis. Expert Opin Pharmacother. 2015 Apr;16(5):633-43. doi: 10.1517/14656566.2015.998650. Epub 2014 Dec 29.
Results Reference
derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs