Primary Biliary Cirrhosis
Conditions
Brief summary
Obecholic acid is a modified bile acid and Farnesoid X receptor (FXR) agonist. FXR is a key regulator of bile acid synthesis and transport. Bile acids are used by the body to help with digestion. Conventional therapy with obecholic acid will improve liver function of patients with PBC.
Interventions
DRUGObeticholic Acid Tablets
Obeticholic Acid：Once a day (QD) by mouth (PO).
DRUGUDCA
UDCA：continue prestudy dose
DRUGPlacebo
Placebo
Sponsors
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. Male or female aged 18 to 75 years. 2. Meet at least 2 of the following 3 PBC diagnoses: 1. Patients had elevated alkaline phosphatase for at least 3 months before enrolment. 2. AMA positive (titer ≥1:40), or if AMA negative, PBC specific antibodies (anti- GP210 and/or anti-SP100 and/or AMA-M2) are required. 3. Liver biopsy suggested PBC 48 weeks before enrollment. 3. ALP \> 1.67× ULN before enrollment. 4. Taking UDCA with stable dose for at least 3 months before enrollment.
Exclusion criteria
1. Merging with other virus infected. 2. With other existing liver disease or a history of liver disease. 3. With clinical complications of PBC or clinically significant hepatic decompensation. 4. Child-pugh grade B or C. 5. Creatinine (Cr) ≥ 1.5×ULN and serum creatinine clearance rate \< 60mL/min; \[Calculation formula: Cr:(140-age)×weight(kg) /0.818 × Scr (μmol/L)，female Cr=Cr × 0.85\]. 6. ALT or AST\>5×ULN;Tbil \> 2×ULN. 7. Patients with a history of severe pruritus 2 months before enrollment. 8. The presence of clinically relevant arrhythmias or associated history that may affect survival during the study period. 9. With diseases that may cause nonhepatic ALP increases (e.g., Paget's disease) or which may diminish life expectancy to \< 2 years.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of PBC patients reaching the compound endpoint after 48 weeks of treatment (Compound endpoint: alkaline phosphatase (ALP) < 1.67× Upper Limit of Normal(ULN), ALP decrease by at least 15% , and total bilirubin ≤ ULN ) | up to 48 weeks | Compound endpoint: ALP \< 1.67× ULN, ALP decrease by at least 15% , and total bilirubin ≤ ULN |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of PBC patients reaching the compound endpoint after 4 weeks, 12 weeks, 24 weeks and 36 weeks of treatment (Compound endpoint: ALP < 1.67× ULN, ALP decrease by at least 15% , and total bilirubin ≤ ULN ) | up to 36 weeks | Compound endpoint: ALP \< 1.67× ULN, ALP decrease by at least 15% , and total bilirubin ≤ ULN |
| Rate of change of liver function indicators from baseline | up to 48 weeks | Liver function: ALP (alkaline phosphatase ), ALT (Alaninetransaminase), AST(aspartate transaminase), GGT( γ-glutamyl transpeptadase ), TBA (total bile acid) and Tbil (total bilirubin) |
Countries
China
Contacts
Primary ContactJunqi Niu, Professor
Outcome results
None listed