Clinical trial NCT04956328: Study of Obeticholic Acid（OCA） Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primay Biliary Cirrhosis (PBC)
Official title: A Multi-center, Randomized, Double-blind Trail Evaluating the Efficacy and Safety of Combination of Obecholic Acid and UDCA Compared With UDCA Monotherapy in PBC Patients With an Inadequate Response to UDCA .
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Primary Biliary Cirrhosis
Lead sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2021-07-09
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: 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 )
timeFrame: up to 48 weeks
Secondary outcomes:
measure: 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 )
timeFrame: up to 36 weeks
measure: Rate of change of liver function indicators from baseline
timeFrame: up to 48 weeks
Other outcomes:
Locations:
city: Changchun
country: China
facility: The first hospital of Jilin University
state: Jilin
Study officials:
References: