Study of INT-747 as Monotherapy in Participants With Primary Biliary Cirrhosis (PBC)

May 28, 2021 updated by: Intercept Pharmaceuticals

A Study of INT-747 (6-ECDCA) Monotherapy in Patients With Primary Biliary Cirrhosis

The primary hypothesis was that obeticholic acid (OCA) will cause a reduction in alkaline phosphatase levels in PBC participants, over a 12-week treatment period, as compared to placebo.

Study Overview

Status

Status

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

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

The study included 2 phases: a 3-month randomized, double-blind (DB), placebo-controlled, parallel group phase, followed by a long-term safety extension (LTSE). The planned duration of the LTSE phase was country-specific, ranging from 108 months to indefinitely. On-site visits occurred at least every 6 months.

Following completion of the 3-month DB phase, participants who continued to meet protocol requirements were given the opportunity to enroll in the LTSE phase of the study at selected study sites. The participants who enrolled in the LTSE phase started OCA administration, from a starting dose (10 or 50 milligrams [mg]) based on the dose of OCA or placebo received in the DB phase or on the timing of entry into the LTSE phase.

Because the LTSE phase was not planned in the original study design, participants had varied gaps between the end of the DB phase and the start of the LTSE phase. Moreover, some participants initiated ursodeoxycholic acid during the course of that break.

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.
60

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, 8036
        • Karls-Franzens University
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 1K8
        • University of Alberta
    • Ontario
      • Toronto, Ontario, Canada, M5T 1S8
        • University of Toronto
    • Quebec
      • Montreal, Quebec, Canada, H2L 3R4
        • Centre de Recherche du CHUM / University of Montreal
  • France
      • Lyon, France, 69288
        • Hôpital de l'Hôtel Dieu
      • Paris, France, 75012
        • Hopital Saint-Antoine
  • Germany
      • Frankfurt, Germany, 60590
        • Johann Wolfgang Goethe University
      • Hamburg, Germany, 20246
        • University Medical Centre Hamburg-Eppendorf
      • Hannover, Germany, 30625
        • Medical School of Hannover
      • Munich, Germany, 81377
        • University of Munich
  • Spain
      • Barcelona, Spain, 08036
        • Hospital Clínic i Provincial
  • United Kingdom
      • Edinburgh, United Kingdom, EH16 4SA
        • Royal Infirmary
      • Jarrow, United Kingdom, NE32 3DT
        • Sunderland Research Ethics Committee
      • 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
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Henry Ford
    • Texas
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98105
        • 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

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Female participants must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use 1 effective method of contraception with all sexual partners during the study and for 14 days after the end of dosing.
  • Male participants must be prepared to use 1 effective method of contraception with all sexual partners during the study during the study unless they had a prior vasectomy.
  • Proven or likely PBC, as demonstrated by the participant presenting with at least 2 of the following 3 diagnostic factors:
  • History of increased alkaline phosphatase (ALP) levels for at least 6 months;
  • Positive antimitochondrial antibody titer (>1:40 titer on immunofluorescence or M2 positive by enzyme-linked immunosorbent assay) or PBC-specific antinuclear antibodies (antinuclear dot and nuclear rim positive);
  • Liver biopsy consistent with PBC
  • Screening ALP level between 1.5 and 10 × upper limit of normal (ULN).

Exclusion Criteria:

  • Administration of the following drugs at any time during the 3 months prior to screening for the study: ursodeoxycholic acid, colchicine, methotrexate, azathioprine, or systemic corticosteroids.
  • Screening conjugated (direct) bilirubin >2 × ULN.
  • Screening alanine aminotransferase or aspartate aminotransferase >5 × ULN.
  • Screening serum creatinine >133 micromoles/liter (1.5 mg/deciliter).

History or presence of hepatic decompensation (for example, variceal bleeds, encephalopathy, or poorly controlled ascites).

  • History or presence of other concomitant liver diseases including hepatitis due to hepatitis B or C virus infection, primary sclerosing cholangitis, alcoholic liver disease, definite autoimmune liver disease or biopsy proven nonalcoholic steatohepatitis.
  • 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: DB OCA 10 mg
OCA 10 mg for 3 months during the DB phase.
Drug: Obeticholic Acid (OCA)
Starting dose of 10 or 50 mg administered orally once daily, followed by dose titration planned from 10 mg to 25 mg to 50 mg once daily, which could be modified for safety and tolerability issues or to achieve adequate therapeutic response.
Other Names:
  • INT-747
  • 6α-ethyl-chenodeoxycholic acid (6-ECDCA)
Experimental: DB OCA 50 mg
OCA 50 mg for 3 months during the DB phase.
Drug: Obeticholic Acid (OCA)
Starting dose of 10 or 50 mg administered orally once daily, followed by dose titration planned from 10 mg to 25 mg to 50 mg once daily, which could be modified for safety and tolerability issues or to achieve adequate therapeutic response.
Other Names:
  • INT-747
  • 6α-ethyl-chenodeoxycholic acid (6-ECDCA)
Placebo Comparator: DB OCA Placebo
Matching placebo for 3 months during the DB phase.
Drug: Placebo
Matching placebo tablets were administered orally once daily.
Experimental: LTSE OCA Total
After completion of the 3-month DB phase, all eligible participants were offered the opportunity to enter an open-label LTSE for up to 96 months beginning at 10 mg OCA. Doses up to 50 mg daily were evaluated.
Drug: Obeticholic Acid (OCA)
Starting dose of 10 or 50 mg administered orally once daily, followed by dose titration planned from 10 mg to 25 mg to 50 mg once daily, which could be modified for safety and tolerability issues or to achieve adequate therapeutic response.
Other Names:
  • INT-747
  • 6α-ethyl-chenodeoxycholic acid (6-ECDCA)

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
DB Phase: Mean Percent Change In Serum Alkaline Phosphatase (ALP) From Baseline To Day 85
Time Frame: Baseline, Day 85
The percent change in serum ALP from baseline to Day 85 is reported. The baseline value used was the mean of the pretreatment Screening and Day 0 evaluations.
Baseline, 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
DB Phase: Mean Percent Change In Gamma-glutamyl Transferase (GGT) From Baseline To Day 85
Time Frame: Baseline, Day 85
As a marker of hepatocellular injury and liver function, the percent change in GGT from baseline to Day 85 is reported.
Baseline, Day 85
DB Phase: Mean Percent Change In Alanine Transaminase (ALT) From Baseline To Day 85
Time Frame: Baseline, Day 85
As a marker of hepatocellular injury and liver function, the percent change in ALT from baseline to Day 85 is reported.
Baseline, Day 85
DB: Plasma Trough Concentrations Of OCA And Its Major, Known Metabolites
Time Frame: 12 weeks
12 weeks
DB Phase: Mean Percent Change In Conjugated Bilirubin From Baseline To Day 85
Time Frame: Baseline, Day 85
As a marker of hepatocellular injury and liver function, the percent change in conjugated bilirubin from baseline to Day 85 is reported.
Baseline, Day 85
LTSE Phase: Median Percent Change In Serum ALP From Baseline To Month 24, Month 48, Month 72, And Last Available Visit
Time Frame: Baseline (DB), Month 24, Month 48, Month 72, Last Available Visit (up to 96 months)
The percent change in serum ALP from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Month 24, Month 48, Month 72, Last Available Visit (up to 96 months)
LTSE Phase: Mean Percent Change In Serum ALP From Baseline To Month 24, Month 48, Month 72, And Last Available Visit
Time Frame: Baseline (DB), Month 24, Month 48, Month 72, Last Available Visit (up to 96 months)
The percent change in serum ALP from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Month 24, Month 48, Month 72, Last Available Visit (up to 96 months)
LTSE: Median Percent Change In GGT From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in GGT from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In GGT From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in GGT from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Median Percent Change In ALT From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in ALT from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In ALT From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in ALT from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Median Percent Change In Conjugated Bilirubin From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in conjugated bilirubin from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In Conjugated Bilirubin From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in conjugated bilirubin from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Median Percent Change In Total Bilirubin From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in total bilirubin from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In Total Bilirubin From Baseline To Last Available Visit
Time Frame: Baseline (DB), Last Available Visit (up to 96 months)
As a marker of hepatocellular injury and liver function, the percent change in total bilirubin from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Baseline (DB), Last Available Visit (up to 96 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 Chair: Christian Weyer, MD, Intercept Pharmaceuticals, Inc.

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.
January 17, 2008

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.
September 21, 2010

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.
September 25, 2017

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).
December 7, 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.
December 7, 2007

First Posted (Estimate)

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.
December 11, 2007

Study Record Updates

Last Update Posted (Actual)

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.
June 22, 2021

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.
May 28, 2021

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.
May 1, 2021

More Information

Terms related to this study

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.
  • 747-201
  • 2007-001424-12 (EudraCT Number)

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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