A Study of CS0159 in Patients With PBC With Inadequate Response or Intolerance to UDCA

NCT07282353 | Recruiting Phase 3 DMC

• 1 other identifier: CS0159-PBC-CN-III-07
• Study Type: interventional
• Target: 135
• Locations: 1 country
• Sites: 41

Brief Summary

A Randomized, Double-Blind, Placebo-controlled, Phase III Study to Evaluate the Efficacy and Safety of CS0159 in Patients with Primary Biliary Cholangitis (PBC) with inadequate response or intolerance to ursodeoxycholic acid (UDCA).

Conditions

Primary Biliary Cholangitis

Outcome Measures

Primary Outcomes (1)

• The proportion of patients who achieve the composite response criteria at Week 52

  1. ALP \< 1.67×ULN 2. and ALP decrease of ≥15% 3. and TB ≤1.0× ULN

  Baseline to week 52 .

Secondary Outcomes (7)

• Response to treatment based on γ-glutamyl transpeptidase (GGT) normalization in patients with baseline GGT>1.0× ULN at Week 26 and Week 52

  Basline to week 26 and week 52 .

• Response to treatment based on alanine aminotransferase (ALT) normalization in patients with baseline ALT >1.0× ULN at Week 26 and Week 52

  Basline to week 26 and week 52 .

• Proportion of patients with ALP< 1.67× ULN and TB ≤1.0× ULN and ALP decrease of ≥15% from baseline at Week 26

  Baseline to week 26 .

• Response to treatment based on AST normalization in patients with baseline AST>1.0× ULN at Week 26 and Week 52

  Baseline to week 26 and week 52 .

• Proportion of patients with ALP≤ 3× ULN and AST ≤ 2 × ULN and TB≤ 1.0×ULN at Week 52

  Baseline to week 52 .

Other Outcomes (1)

• Absolute changes of liver stiffness measured by iLivTouch

  week 26, week 52

Study Arms (2)

4 mg CS0159

EXPERIMENTAL

Take 4mg of CS0159 QD for 52 weeks.

Drug: 2mg CS0159

placebo

PLACEBO COMPARATOR

Take placebo QD for 52 weeks.

Drug: Placebo

Interventions

2mg CS0159 DRUG

Oral QD

4 mg CS0159

Placebo DRUG

Oral QD

placebo

Eligibility Criteria

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

You may qualify if:

• Must have given written informed consent (signed and dated) and any authorizations required by local law;
• When signing ICF age ≥18 years ≤75 years, male or female;
• Meets the diagnostic criteria of PBC, based on any two of the following criteria:
• History of ALP above 1.0× ULN for at least 6 months
• Positive antimitochondrial antibody (AMA) titer (\>1:40 on immunofluorescence or M2 positive by ELISA) or positive PBC- specific antinuclear antibody (ANA) (either SP100 or GP210 positive)
• Documented liver biopsy results consistent with PBC;
• Central laboratory parameters measured at screening period meet the following criteria:
• ALP ≥1.67× ULN
• ALT≤5× ULN
• AST ≤5× ULN
• TB \<2× ULN
• Estimated glomerular filtration rate (eGFR) \> 60mL/min/1.73m2 (calculated by CKD-EPI equation)
• INR ≤ 1.0× ULN. For participants on anticoagulation therapy, INR must be maintained in the range required for prophylaxis for their specific disease
• Platelet count ≥ 150× 109/μL (No thrombocytopenia-related treatment within the past two weeks)
• Albumin\> 35g/L

You may not qualify if:

• Previous exposure to CS0159;
• History of allergy to the CS0159 or its excipients or drugs of similar chemical classes;
• Advanced PBC as defined by the Rotterdam criteria (albumin\<1.0×LLN AND TB \>1.0× ULN);
• Patients who have had clinically significant complications of hepatic cirrhosis with clinically significant portal hypertension (CSPH), including the following:
• History of liver transplantation, current placement on a liver transplant list, current MELD -Na score ≥ 12;
• History of confirmed esophagogastric variceal bleeding;
• Clinically significant ascites requiring intervention, such as sodium restriction, diuretic therapy, or therapeutic paracentesis;
• Any secondary complications resulting from clinically significant ascites, such as spontaneous bacterial peritonitis, hepatorenal syndrome, or hepatic hydrothorax;
• Hepatic encephalopathy requiring drug therapy;
• Portopulmonary hypertension and/or hepatopulmonary syndrome;
• Hepatocellular carcinoma;
• Other concomitant liver disease including:
• Autoimmune hepatitis (AIH) (simplified AIH diagnostic score \>6), PBC-AIH overlap syndrome, or overlap with other autoimmune liver diseases
• Positive HBsAg or positive HCV RNA (tested for in case of known cured HCV infection or positive HCV Ab at screening)
• Primary sclerosing cholangitis (PSC)

Sponsors & Collaborators

• Cascade Pharmaceuticals, Inc: lead

Study Sites (41)

The First Affiliated Hospital of USTC Anhui Provincial Hospital

Hefei, Anhui, 230001, China

NOT YET RECRUITING

The Second Affiliated Hospital of Anhui Medical University

Hefei, Anhui, China

NOT YET RECRUITING

Beijing Friendship Hospital, Capital Medical University

Beijing, Beijing Municipality, 100050, China

NOT YET RECRUITING

Beijing Youan Hospital, Capital Medical University

Beijing, Beijing Municipality, 100069, China

NOT YET RECRUITING

China-Japan Friendship Hospital

Beijing, Beijing Municipality, China

NOT YET RECRUITING

Chongqing University Three Gorges Hospital

Chongqing, Chongqing Municipality, China

NOT YET RECRUITING

The Second Affiliated Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, China

NOT YET RECRUITING

Mengchao Hepatobiliary Hospital Of Fujian Medical University

Fuzhou, Fujian, China

NOT YET RECRUITING

Peking University Shenzhen Hospital

Shenzhen, Guangdong, China

NOT YET RECRUITING

Shenzhen Third People's Hospital

Shenzhen, Guangdong, China

NOT YET RECRUITING

Affiliated Hospital of Guangdong Medical University

Zhanjiang, Guangdong, China

NOT YET RECRUITING

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

NOT YET RECRUITING

Hebei Medical University Third Hospital

Shijiazhuang, Hebei, China

NOT YET RECRUITING

The Fourth Hospital Of Harbin Medical University

Harbin, Heilongjiang, China

NOT YET RECRUITING

Henan Provincial People's Hospital

Zhengzhou, Henan, China

NOT YET RECRUITING

Taihe Hospital of Shiyan

Shiyan, Hubei, China

NOT YET RECRUITING

Xiangya Hospital Central South University

Changsha, Hunan, China

NOT YET RECRUITING

Jiangsu Province Hospital

Nanjing, Jiangsu, China

NOT YET RECRUITING

The Second Hospital of Nanjing

Nanjing, Jiangsu, China

NOT YET RECRUITING

The Fifth People's Hospital of Suzhou

Suzhou, Jiangsu, China

NOT YET RECRUITING

The Fifth People's Hospital Of Wuxi

Wuxi, Jiangsu, China

NOT YET RECRUITING

The Third People's Hospital of Zhenjiang

Zhenjiang, Jiangsu, China

NOT YET RECRUITING

The First Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, China

NOT YET RECRUITING

The Second Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, China

NOT YET RECRUITING

The First Bethune Hospital of Jilin University

Changchun, Jilin, China

NOT YET RECRUITING

The First Hospital of China Medical University

Shenyang, Liaoning, China

NOT YET RECRUITING

The Sixth People's Hospital of Shenyang

Shenyang, Liaoning, China

NOT YET RECRUITING

Baoji People's Hospital

Baoji, Shaanxi, China

NOT YET RECRUITING

The First Affiliated Hospital of Xi'an Jiaotong University

Xi'an, Shaanxi, China

NOT YET RECRUITING

Linyi People's Hospital

Linyi, Shandong, China

NOT YET RECRUITING

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, China

NOT YET RECRUITING

Renji Hospital, School of Medicine, Shanghai Jiao Tong University

Shanghai, Shanghai Municipality, 200001, China

RECRUITING

Shanghai Jiao Tong University School of Medicine Ruijin Hospital

Shanghai, Shanghai Municipality, China

NOT YET RECRUITING

Xin Hua Hospital Affiliated To Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, China

NOT YET RECRUITING

West China Hospital, Sichuan University

Chengdu, Sichuan, China

NOT YET RECRUITING

Tianjin First Central Hospital

Tianjin, Tianjin Municipality, China

NOT YET RECRUITING

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, China

NOT YET RECRUITING

Tianjin Third Central Hospital

Tianjin, Tianjin Municipality, China

NOT YET RECRUITING

Hangzhou Xixi Hospital

Hangzhou, Zhejiang, China

NOT YET RECRUITING

Taizhou Hospital of Zhejiang Province

Taizhou, Zhejiang, China

NOT YET RECRUITING

The First Affiliated Hospital of Wenzhou Medical University

Wenzhou, Zhejiang, China

NOT YET RECRUITING

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

• Ma Xiong, MD

  Renji Hospital, School of Medicine, Shanghai Jiao Tong University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ma Xiong, MD

CONTACT

15900984550; maxiongmd@163.com

Xiao Xiao, MD

CONTACT

15921514136; xxiao_sh@163.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 21, 2025
• First Posted: December 15, 2025
• Study Start: December 1, 2025
• Primary Completion (Estimated): December 9, 2027
• Study Completion (Estimated): January 6, 2028
• Last Updated: December 30, 2025

Record last verified: 2025-12

Locations

CN (41)