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.
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
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
60
Matching placebo tablets were administered orally once daily.
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.
Henry Ford
Detroit, Michigan, United States
Baylor College of Medicine
Houston, Texas, United States
DB Phase: Mean Percent Change In Serum Alkaline Phosphatase (ALP) From Baseline To 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.
Time frame: Baseline, Day 85
DB Phase: Mean Percent Change In Gamma-glutamyl Transferase (GGT) From Baseline To Day 85
As a marker of hepatocellular injury and liver function, the percent change in GGT from baseline to Day 85 is reported.
Time frame: Baseline, Day 85
DB Phase: Mean Percent Change In Alanine Transaminase (ALT) From Baseline To Day 85
As a marker of hepatocellular injury and liver function, the percent change in ALT from baseline to Day 85 is reported.
Time frame: Baseline, Day 85
DB: Plasma Trough Concentrations Of OCA And Its Major, Known Metabolites
Time frame: 12 weeks
DB Phase: Mean Percent Change In Conjugated Bilirubin From Baseline To Day 85
As a marker of hepatocellular injury and liver function, the percent change in conjugated bilirubin from baseline to Day 85 is reported.
Time frame: Baseline, Day 85
LTSE Phase: Median Percent Change In Serum ALP From Baseline To Month 24, Month 48, Month 72, And Last Available Visit
The percent change in serum ALP from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Time frame: 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
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Virginia Commonwealth University
Richmond, Virginia, United States
Virginia Mason Medical Center
Seattle, Washington, United States
Karls-Franzens University
Graz, Austria
University of Alberta
Edmonton, Alberta, Canada
University of Toronto
Toronto, Ontario, Canada
Centre de Recherche du CHUM / University of Montreal
Montreal, Quebec, Canada
Hopital de l'Hotel Dieu
Lyon, France
Hopital Saint-Antoine
Paris, France
...and 11 more locations
The percent change in serum ALP from baseline to the last available visit is reported. The DB baseline value was used as the baseline.
Time frame: 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
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In GGT From Baseline To Last Available Visit
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Median Percent Change In ALT From Baseline To Last Available Visit
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In ALT From Baseline To Last Available Visit
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Median Percent Change In Conjugated Bilirubin From Baseline To Last Available Visit
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In Conjugated Bilirubin From Baseline To Last Available Visit
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Median Percent Change In Total Bilirubin From Baseline To Last Available Visit
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)
LTSE: Mean Percent Change In Total Bilirubin From Baseline To Last Available Visit
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.
Time frame: Baseline (DB), Last Available Visit (up to 96 months)