Primary Biliary Cholangitis
Conditions
Keywords
Primary Biliary Cholangitis (PBC), PBC
Brief summary
The goal of this clinical study is to learn more about the study drug, seladelpar, and its effects on clinical outcomes in participants with primary biliary cholangitis (PBC) and compensated cirrhosis. The primary objective of this study is to evaluate the effect of seladelpar compared to placebo on EFS. EFS is defined as the time from start of treatment to the first occurrence of any of the following adjudicated events up to Week 156.
Interventions
DRUGSeladelpar
Administered orally
DRUGPlacebo
Administered orally
Sponsors
Gilead Sciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Individuals must meet the following criteria to be eligible for study participation: 1. Must be at least 18 years old. 2. Must have a confirmed prior diagnosis of primary biliary cholangitis (PBC) 3. Evidence of cirrhosis 4. CP Score A or B 5. Females of reproductive potential must use at least 1 barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male individuals who are sexually active with female partners of reproductive potential must use barrier contraception, and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose 6. Individuals must be able to comply with the instructions for study drug administration and be able to complete the study schedule of assessments (SOA)
Exclusion criteria
Individuals must not meet any of the following criteria to be eligible for study participation: 1. Prior exposure to seladelpar 2. A medical condition other than PBC that, in the Investigator's opinion, would preclude full participation in the study 3. History of liver transplantation or actively listed for cadaveric or planned living donor transplant. 4. Decompensated cirrhosis 5. Evidence of portal vein thrombosis based on imaging at time of Screening by Doppler ultrasound or prior evidence by CT or MRI 6. Hospitalization for liver-related complication within 12 weeks of Screening 7. Laboratory parameters at Screening: 1. Alkaline phosphatase (ALP) \< 1.5× Upper limit of normal (ULN) or ≥ 10×ULN 2. Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≥5×ULN 3. Total bilirubin (TB) ≤ 0.6 × ULN or ≥ 5 × ULN 4. Platelet count ≤50×10\^3/µL 5. Albumin ≤2.8 g/dL 6. Estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73 m\^2 7. MELD score \>12. For individuals on anticoagulation medication, baseline International normalized ratio (INR) determination for MELD score calculation should take anticoagulant use into account, in consultation with the Medical Monitor. 8. Serum alpha-fetoprotein (AFP) \>20 ng/mL 9. INR \>1.7 8. CP-C cirrhosis 9. History or presence of other concomitant liver diseases
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Event Free Survival (EFS) | 36 months | EFS is defined as measured by the time from start of treatment to the first occurrence of any of the following clinical events: 1. Death by any cause 2. Liver transplantation 3. MELD score ≥15 4. Ascites requiring treatment 5. Hospitalization for any of the following 1. Esophageal or gastric variceal bleeding 2. Hepatic encephalopathy 3. Spontaneous bacterial peritonitis |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival (OS) | 36 months | Time from start of treatment to death from any cause. |
| Liver Transplant-Free Survival (LTFS) | 36 months | Time from start of treatment to the first occurrence of liver-related death or liver transplantation. |
| Time to Hospitalization | 36 months | Time from start of treatment to the first occurrence of hospitalization for qualifying Primary biliary cholangitis (PBC) clinical events |
| Time to Each Type of Adjudicated PBC Clinical Event: Model for End-Stage Liver Disease (MELD) score ≥ 15 | 36 months | — |
| Time to Each Type of Adjudicated PBC Clinical Event: Ascites Requiring Treatment | 36 months | — |
Countries
Argentina, Australia, Austria, Belgium, Canada, Chile, China, Czechia, Denmark, France, Germany, Greece, Hong Kong, Hungary, Israel, Italy, Mexico, Poland, Portugal, Puerto Rico, Romania, Singapore, South Korea, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom, United States
Contacts
CONTACTGilead Clinical Study Information Center
STUDY_DIRECTORGilead Study Director
Gilead Sciences
Outcome results
None listed