Study to Evaluate the Safety and Efficacy of ASC42 Tablets in Subjects With Primary Biliary Cholangitis

Gannex Pharma

Status and phase: Completed; Phase 2

Conditions: Primary Biliary Cholangitis

Treatments: Drug: ASC42 5 mg; Drug: ASC42 15 mg; Drug: ASC42 10 mg; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05190523

ASC42-202

Details and patient eligibility

About

This study is a phase II, multicenter, randomized, double-blind, placebo-controlled, seamless adaptive design clinical study, aiming to evaluate the safety and effectiveness of three doses of ASC42 matched placebo in subjects with primary biliary cholangitis.

Enrollment: 98 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥ 18 and ≤75 years old at screening.

• Definite or probable PBC diagnosis, as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors:

  1. Biochemical evidence of cholestasis based on ALP elevation.
  2. Presence of AMA, or other PBC-specific autoantibodies, including sp100 or gp210, if AMA is negative.
  3. Liver biopsy consistent with PBC.

• Screening ALP ≥ 1.67× ULN

• Taking UDCA for at least 6 months (stable dose for ≥ 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ≥ 3 months) prior to Day 0.

Exclusion criteria

• ALT or AST > 5× ULN; ALP >10× ULN
• History or presence of other concomitant liver diseases
• Child-Pugh grade B or C

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

98 participants in 4 patient groups, including a placebo group

ASC42 tablets of 5mg

Experimental group

Description:

ASC42 tablets 5mg for 12 weeks

Treatment:

Drug: ASC42 5 mg

ASC42 tablets of 10mg

Experimental group

Description:

ASC42 tablets 10mg for 12 weeks

Treatment:

Drug: ASC42 10 mg

ASC42 tablets of 15mg

Experimental group

Description:

ASC42 tablets 15mg for 12 weeks

Treatment:

Drug: ASC42 15 mg

Placebo

Placebo Comparator group

Description:

Placebo for 12 weeks

Treatment:

Drug: Placebo