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

January 11, 2024 updated by: Intercept Pharmaceuticals

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

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

None provided

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
165

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Double-blind Phase: Percent Change From Baseline in Serum Alkaline Phosphatase (ALP)
Time Frame: Baseline and Up to Day 85
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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
Time Frame: Baseline and at Days 15, 29, 57 and 85
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
Time Frame: Baseline and at Days 15, 29, 57 and 85
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
Time Frame: Baseline and at 3, 6, 9 and 12 Months
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
Time Frame: Baseline and at Days 15, 29, 57 and 85
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
Time Frame: Baseline and at Days 15, 29, 57 and 85
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
Time Frame: Baseline and at Days 15, 29, 57 and 85
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
Time Frame: Baseline and at Days 15, 29, 57 and 85
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
Time Frame: Baseline and at 3, 6, 9 and 12 Months
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
Time Frame: Baseline and up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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)
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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)
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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)
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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)
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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)
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and Up to Day 85
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
Time Frame: Baseline and at 3, 6, 9 and 12 Months
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
Time Frame: Baseline and at Days 29, 57 and 85
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
Time Frame: Baseline and at 6 and 12 months
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
Time Frame: Baseline and at Days 29, 57 and 85
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)
Time Frame: Up to Day 85
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)
Time Frame: Baseline and at Days 29, 57 and 85
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
Time Frame: Up to 12 Months
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

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Intercept Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: David A Shapiro, MD, Intercept Pharmaceuticals - Chief Medical Officer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 1, 2007

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 1, 2009

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2010

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 27, 2007

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 27, 2007

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 30, 2007

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 6, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 11, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 747-202

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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