Study of Obeticholic Acid（OCA） Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primay Biliary Cirrhosis (PBC)

CTTQ

Status and phase: Unknown; Phase 3

Conditions: Primary Biliary Cirrhosis

Treatments: Drug: UDCA; Drug: Placebo; Drug: Obeticholic Acid Tablets

Study type: Interventional

Funder types: Industry

Identifiers

NCT04956328

ABDS-CS-01

Details and patient eligibility

About

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.

Enrollment: 120 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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120 participants in 2 patient groups, including a placebo group

OCA Tablets 5-10 mg

Experimental group

Description:

OCA 5 mg once daily in combination with UDCA for 24 weeks and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.

Treatment:

Drug: UDCA

Drug: Obeticholic Acid Tablets

Placebo

Placebo Comparator group

Description:

Placebo once daily in combination with UDCA for 48 weeks.

Treatment:

Drug: Placebo

Drug: UDCA