CS0159 in Chinese Patients With PBC (Primary Biliary Cholangitis)

NCT05896124 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: PBC-CS0159-004
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 16

Brief Summary

A phase II study to evaluate safety, tolerability and efficacy of CS0159 in patients with PBC (Primary Biliary Cholangitis).

Conditions

Primary Biliary Cholangitis (PBC)

Outcome Measures

Primary Outcomes (2)

• AE incidence

  AE incidence in three arms

  baseline to 12 weeks

• relative changes from baseline in ALP at week 12

  Compared with placebo ,Percentage change of CS0159 to ALP relative to baseline

  baseline to 12 weeks

Secondary Outcomes (4)

• Absulute changes from baseline in ALP at week 12

  baseline to 12 weeks

• ALP and TBil

  baseline to 12 weeks

• Pruritus

  from basline to 40 weeks

• Liver function: ALT, AST, ALB, LDL-C, HDL-C, TBA, GGT, TC, TG

  from baseline to week 40.

Study Arms (3)

2mg CS0159

EXPERIMENTAL

QD for 12 weeks

Drug: 2mg CS0159; Drug: Placebo

4mg CS0159

EXPERIMENTAL

QD for 12 weeks

Drug: 2mg CS0159

Placebo

EXPERIMENTAL

QD for 12 weeks

Drug: Placebo

Interventions

Placebo DRUG

Oral QD

2mg CS0159; Placebo

2mg CS0159 DRUG

Oral QD

2mg CS0159; 4mg CS0159

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• When signing ICF age≥18 years≤75 years, male or female
• Meets the diagnostic criteria of PBC, such as elevation ALP, positive AMA or AMA-M2, If negative for AMA, positive for PBC specific antibody and Liver biopsy meeting PBC criteria six months before screening
• × ULN ≤ALP ≤ 10 × ULN and TBil≤ 3 × ULN
• Understand the study content, comply with the study protocol, and sign the ICF voluntarily

You may not qualify if:

• ALT or AST\>5×ULN；
• OCA(Obercholic acid) in the 3 months prior to randomization
• Known concomitant hepatobiliary disease or history
• Significant hepatic impairment as defined by Child-Pugh classification of B or C, history of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥15.
• Patients were screened for HBsAg positive, HCVAb positive, HIV Ab positive, or TPAb positive.
• (creatinine, Cr) ≥1.5×ULN and Cr clearance rate \<60 mL/min
• Platelet\<80×10\^9/L；
• INR\>1.3
• ALB\<3.5 g/dL
• Severe pruritus or systemic medication was required within 2 months prior to randomization
• Arrhythmia, Or during screening the QTc interval was ≥450 ms for male and 470 ms for female
• History or presence of any disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs including bile salt metabolism in the large intestine, eg, inflammatory bowel disease, prior or planned (during the study period) bariatric surgery (such as gastroplasty, roux-en-Y gastric bypass).
• Concomitant use of medications, food, and drinks that are strong or moderate CYP3A4 inhibitors or inducers within 14 days prior to the first dose of study drug and throughout the study duration.
• Diseases that may cause non-hepatic elevation of ALP (such as Paget's disease) or may result in a life expectancy of less than 2 years
• A history of malignant tumor within 5 years prior to randomization

Sponsors & Collaborators

• Cascade Pharmaceuticals, Inc: lead

Study Sites (16)

The First Affiliated Hospital of USTC Anhui Provincial Hospital

Hefei, Anhui, 230001, China

Location

Beijing Friendship Hospital, Capitai Medical University

Beijing, Beijing Municipality, 100050, China

Location

Beijing Youan Hospital, Capital Medical University

Beijing, Beijing Municipality, 100069, China

Location

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

The Third Affiliated Hospital, Sun Yat-Sen University

Guangzhou, Guangdong, 510630, China

Location

The Fifth Affiliated Hospital of Guangzhou Medical University

Guangzhou, Guangzhou, China

Location

Henan Provincial People's Hospital

Zhengzhou, Henan, China

Location

Wuhan Union Hospital of China

Wuhan, Hubei, 430022, China

Location

The Seconed Xiangya Hospital of Central South University

Changsha, Hunan, 410011, China

Location

The Third People's Hospital of Zhenjiang

Zhenjiang, Jiangsu, China

Location

The First Bethune Hospital of Jilin University

Changchun, Jilin, China

Location

Qilu Hospital of Shandong University

Jinan, Shandong, China

Location

Renji Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200120, China

Location

West China Hospital, Sichuan University

Chengdu, Sichuan, China

Location

Shaoyifu Hospital of Zhejiang University Medical

Hangzhou, Zhejiang, 310000, China

Location

The First Affiliated Hospital,Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Rong Deng

  Cascade Pharmaceuticals, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 17, 2023
• First Posted: June 9, 2023
• Study Start: August 7, 2023
• Primary Completion: December 31, 2025
• Study Completion: March 15, 2026
• Last Updated: March 13, 2026

Record last verified: 2026-03

Locations

CN (16)