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Recruiting Phase 3

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

NCT07282353 · tracked via the Priya Life Science Japan tracker
Phase
Phase 3
Started
2025-12-01
Last updated
2026-07-08

Condition(s) studied

Primary Biliary Cholangitis

Investigational drug(s) / intervention(s)

2mg CS0159 →Placebo

2mg CS0159: Oral QD

Placebo: Oral QD

Study 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).

Eligibility

Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
Inclusion Criteria: 1. Must have given written informed consent (signed and dated) and any authorizations required by local law; 2. When signing ICF age ≥18 years ≤75 years, male or female; 3. Meets the diagnostic criteria of PBC, based on any two of the following criteria: 1. History of ALP above 1.0× ULN for at least 6 months 2. 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) 3. Documented liver biopsy results consistent with PBC; 4. UDCA≥6 months before randomization and a stable dose ≥3 months after the efficacy was poor \[meeting inclusion criteria (5)a\], or UDCA was not tolerated, and stop taking UDCA (no UDCA use for ≥3 months before randomization); 5. Laboratory parameters measured at screening period meet the following criteria: 1. ALP ≥1.67× ULN 2. ALT≤5× ULN 3. AST ≤5× ULN 4. TB \<2× ULN 5. Estimated glomerular filtration rate (eGFR) \> 60mL/min/1.73m2 (calculated by CKD-EPI equation) 6. Platelet count ≥ 1.0× LLN (No thrombocytopenia-related treatment within the past two weeks) 7. Albumin\> 35g/L 8. White blood cells count (WBC) \>3×109/L 9. Absolute neutrophil count (ANC) \>1.5×109/L 10. Hemoglobin A1c (HbA1c) ≤9.0%; 6. INR ≤ 1.0× ULN. For participants on anticoagulation therapy, INR must be maintained in the range required for prophylaxis for their specific disease; 7. Females of reproductive potential must use at least 1 barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male participants who are sexually active with female partners of reproductive potential must use barrier contraception, and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose. \- Exclusion Criteria: 1. Previous exposure to CS0159; 2. History of allergy to the CS0159 or its excipients or drugs of similar chemical classes; 3. Advanced PBC as defined by the Rotterdam criteria (albumin\<1.0×LLN AND TB \>1.0× ULN); 4. Patients who have had clinically significant complications of hepatic cirrhosis with clinically significant portal hypertension (CSPH), including the following: 1. History of liver transplantation, current placement on a liver transplant list, current MELD -Na score ≥ 12; 2. History of confirmed esophagogastric variceal bleeding; 3. Clinically significant ascites requiring intervention, such as sodium restriction, diuretic therapy, or therapeutic paracentesis; 4. Any secondary complications resulting from clinically significant ascites, such as spontaneous bacterial peritonitis, hepatorenal syndrome, or hepatic hydrothorax; 5. Hepatic encephalopathy requiring drug therapy; 6. Portopulmonary hypertension and/or hepatopulmonary syndrome; 7. Hepatocellular carcinoma; 5. Other concomitant liver disease including: 1. Autoimmune hepatitis (AIH) (simplified AIH diagnostic score \>6), PBC-AIH overlap syndrome, or overlap with other autoimmune liver diseases 2. Positive HBsAg or positive HCV RNA (tested for in case of known cured HCV infection or positive HCV Ab at screening) 3. Primary sclerosing cholangitis (PSC) 4. History or clinical evidence of Alcoholic liver disease (ALD) 5. Biopsy confirmed Non-alcoholic steatohepatitis (NASH) 6. Gilbert's Syndrome 7. History or evidence of alpha-1 antitrypsin deficiency 8. Liver stiffness measured by transient elastography (TE) \> 16.9 Kpa; 6. Patient has a positive test for HIV at screening, or active syphilis \[defined as positive Treponema pallidum antibody (TP Ab) and a rapid plasma reagin (RPR) card test titer ≥1:8; for low titers (e.g., 1:1 or 1:2), clinical judgment is required to determine if it is active syphilis\]; 7. Administration of the following medications are prohibited as specified below: 1. Use of medications, food, and drinks (e.g., grapefruit juice) that are strong or moderate CYP3A4 inhibitors or inducers within 14 days before randomization; 2. Use of P-glycoprotein (P-gp) substrate drugs within 14 days before randomization; 3. 2 months prior to randomization: fibrates, glitazones, seladelpar and elafibranor. 4. 3 months prior to randomization: obeticholic acid (OCA), azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, systemic corticosteroids and budesonide (˃2 weeks); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid isoniazid, or nitrofurantoin). 5. Patients with systemic treatment for pruritus (e.g., with bile acid sequestrants \[BAS\]) within 3 months prior to randomization. 6. 12 months prior to randomization: antibodies or immunotherapy directed against ILs or other cytokines or chemokines; 8. Medical conditions that may cause non-hepatic increases in ALP (e.g., paget's disease); 9. Patients with severe arrhythmia, or a QTcF interval corrected by Fridericia's formula ≥450 ms (males) or ≥470 ms (females) at screening \[Fridericia's formula: QTcF=QT/(RR\^0.33)\]; 10. 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); 11. History of malignancy (except for those with a disease-free survival of ≥5 years) or currently under evaluation for malignancy. Except for cases with long-term stable disease and assessed by the investigator as having no significant impact on the trial-such as cured squamous or non-invasive basal cell skin carcinoma, cervical carcinoma in situ, mild to moderate cervical dysplasia, or incidental prostate cancer stage T1a. 12. Drug abuse or heavy alcohol use from 12 months prior to randomization throughout the entire clinical study period. Heavy alcohol use is defined as an average weekly alcohol consumption of more than approximately 7 standard drinks for females and more than approximately 14 standard drinks for males. One standard drink is defined as any beverage containing 14g of pure alcohol, such as 12 oz/360 mL of beer (5% alcohol), 8 oz/240 mL of malt liquor (7% alcohol), 5 oz/150 mL of wine (12% alcohol), or 1.5 oz/45 mL of distilled spirits (40% alcohol); 13. Poor blood pressure control is indicated after treatment by a systolic pressure greater than 160 mmHg or diastolic pressure greater than 100 mmHg during screening; 14. Pregnancy, planned pregnancy, lactation; 15. Treatment with any other investigational therapy or device within 30 days or within 5 half-lives, whichever is longer, prior to screening; 16. Mental instability or incompetence that may compromise the validity of informed consent or ability to adhere to study requirements; 17. Any other condition(s) that would compromise the safety of the patient or compromise the quality of the clinical study, as judged by the investigator. \-

Primary outcome measure(s)

  • The proportion of patients who achieve the composite response criteria at Week 52 — Baseline to week 52 .
    1. ALP \< 1.67×ULN 2. and ALP decrease of ≥15% 3. and TB ≤1.0× ULN

Trial sites (42)

FacilityCityRegionStatus
The First Affiliated Hospital of USTC Anhui Provincial Hospital Hefei Anhui Recruiting
The First Affiliated Hospital of Anhui Medical University Hefei Anhui Recruiting
The Second Affiliated Hospital of Anhui Medical University Hefei Anhui Recruiting
Beijing Friendship Hospital, Capital Medical University Beijing Beijing Municipality Recruiting
Beijing Youan Hospital, Capital Medical University Beijing Beijing Municipality Recruiting
China-Japan Friendship Hospital Beijing Beijing Municipality Recruiting
Chongqing University Three Gorges Hospital Chongqing Chongqing Municipality Recruiting
The Second Affiliated Hospital of Chongqing Medical University Chongqing Chongqing Municipality Recruiting
Mengchao Hepatobiliary Hospital Of Fujian Medical University Fuzhou Fujian Recruiting
Xiamen Hospital of T.C,M. Xiamen Fujian Recruiting
Peking University Shenzhen Hospital Shenzhen Guangdong Recruiting
Shenzhen Third People's Hospital Shenzhen Guangdong Recruiting
Affiliated Hospital of Guangdong Medical University Zhanjiang Guangdong Recruiting
The First Affiliated Hospital of Guangxi Medical University Nanning Guangxi Recruiting
The Fourth Hospital Of Harbin Medical University Harbin Heilongjiang Recruiting
Henan Provincial People's Hospital Zhengzhou Henan Recruiting
Taihe Hospital of Shiyan Shiyan Hubei Recruiting
Zhongnan Hospital of Wuhan University Wuhan Hubei Not Yet Recruiting
Xiangya Hospital Central South University Changsha Hunan Recruiting
Jiangsu Province Hospital Nanjing Jiangsu Recruiting
The Second Hospital of Nanjing Nanjing Jiangsu Recruiting
The Fifth People's Hospital of Suzhou Suzhou Jiangsu Recruiting
The Fifth People's Hospital Of Wuxi Wuxi Jiangsu Recruiting
The Third People's Hospital of Zhenjiang Zhenjiang Jiangsu Recruiting
The First Affiliated Hospital of Nanchang University Nanchang Jiangxi Recruiting
The First Bethune Hospital of Jilin University Changchun Jilin Recruiting
The First Hospital of China Medical University Shenyang Liaoning Recruiting
The Fourth People's Hospital of Qinghai Province Xining Qinghai Not Yet Recruiting
Baoji People's Hospital Baoji Shaanxi Recruiting
The First Affiliated Hospital of Xi'an Jiaotong University Xi'an Shaanxi Recruiting
Linyi People's Hospital Linyi Shandong Recruiting
The Affiliated Hospital of Qingdao University Qingdao Shandong Recruiting
Renji Hospital, School of Medicine, Shanghai Jiao Tong University Shanghai Shanghai Municipality Recruiting
Shanghai Jiao Tong University School of Medicine Ruijin Hospital Shanghai Shanghai Municipality Recruiting
Xin Hua Hospital Affiliated To Shanghai Jiao Tong University School of Medicine Shanghai Shanghai Municipality Recruiting
West China Hospital, Sichuan University Chengdu Sichuan Recruiting
Tianjin First Central Hospital Tianjin Tianjin Municipality Recruiting
Tianjin Medical University General Hospital Tianjin Tianjin Municipality Recruiting
Tianjin Third Central Hospital Tianjin Tianjin Municipality Recruiting
Hangzhou Xixi Hospital Hangzhou Zhejiang Recruiting

+ 2 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07282353 on ClinicalTrials.gov ↗ ← All trials in Japan