Primary Biliary Cholangitis
Conditions
Keywords
Primary Biliary Cholangitis, Obeticholic Acid, Bezafibrate, Ursodeoxycholic acid
Brief summary
An Open Label Long-Term Study to Evaluate the Safety and Tolerability of the Fixed-Dose Combination (FDC) of Obeticholic Acid (OCA) and Bezafibrate (BZF) tablet in Subjects with Primary Biliary Cholangitis (PBC).
Interventions
DRUGFDC tablet (OCA 5 mg + BZF 400 mg SR)
Participants will be administered with FDC tablets once daily.
Sponsors
Intercept Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* All subjects with PBC who participated and are actively taking investigational product in Study 747-213 or Study 747-214 are eligible to enroll in this study (977-311).
Exclusion criteria
* History or presence of other concomitant liver diseases * Clinical complications of PBC * History or presence of hepatic decompensating events * Current or history of gallbladder disease * If female, known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating. Note: Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Discontinuation | Early Termination (Up to Month 12) | A TEAE was defined as any event not present before the initiation of the investigational product in this study or any event already present, which worsened in either severity or frequency following exposure to the investigational product in this study. A serious TEAE was defined as any untoward medical occurrence that at any dose resulted in death, life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality or birth defect, an important medical event that may jeopardize the participant or may require medical intervention. |
| Number of Participants Reporting Severe TEAEs | Early Termination (Up to Month 12) | A severe (Grade 3) TEAE is defined as an AE that causes inability to carry out usual activities; the subject may experience intolerable discomfort or pain. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Reporting All-Cause Mortality | Early Termination (Up to Month 12) | All-cause mortality included all reported deaths of participants during the study due to any cause. |
| Number of Participants With Clinically Significant Changes in Baseline in Clinical Hematology Parameters | Early Termination (Up to Month 12) | Clinically notable shift from baseline in blood hematology parameters data was presented for low and high numbers of participants at early termination visit (up to Month 12). The number of participants with clinically notable shift from baseline was presented. |
| Number of Participants With Clinically Significant Changes in Baseline in Blood Serum Chemistry Parameters | Early Termination (Up to Month 12) | Clinically notable shift from baseline in blood serum chemistry data were presented for low and high numbers of participants at early termination visit (up to Month 12). Change from baseline was calculated as post baseline value minus baseline value. Baseline was defined as the last assessment performed before the first dose of investigational product in Study 977-311. |
Countries
Argentina, Australia, Belgium, Canada, Croatia, Czechia, Estonia, France, Germany, Greece, Hungary, Israel, Italy, Lithuania, Netherlands, Norway, South Korea, Spain, Turkey (Türkiye), United Kingdom, United States
Contacts
STUDY_DIRECTORLynda Szczech, PhD
Intercept Pharmaceuticals Inc.
Participant flow
Recruitment details
This was a Phase 3, open-label, long-term safety extension (LTSE) that evaluated the safety and tolerability of the fixed-dose combination (FDC) of obeticholic acid (OCA) and bezafibrate (BZF) in participants with primary biliary cholangitis (PBD) for up to 60 months. All participants from Studies 747-213 (NCT04594694) or 747-214 (NCT05239468) who met respective protocol requirements were transitioned into Study 977-311 at their respective sites.
Pre-assignment details
62 total participants were enrolled.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 55.7 years STANDARD_DEVIATION 9.93 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 20 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 41 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 61 Participants |
| Sex/Gender, Customized Female | 54 participants |
| Sex/Gender, Customized Male | 5 participants |
| Sex/Gender, Customized Unknown | 3 participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 62 |
| other Total, other adverse events | 29 / 62 |
| serious Total, serious adverse events | 3 / 62 |
Outcome results
None listed