New hope for rare liver disease: phase 3 trial of CS0159 launches

NCT ID NCT07282353

Disease control Sponsor: Cascade Pharmaceuticals, Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 09, 2026 · Updated 2 times

Summary

This study tests a new drug called CS0159 in 135 adults with primary biliary cholangitis (PBC), a chronic liver disease. Participants must have had an inadequate response or intolerance to the standard treatment UDCA. The trial compares CS0159 to a placebo over 52 weeks to see if it improves liver function markers like ALP and bilirubin. The goal is to better control the disease, not cure it.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 135 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

Jan 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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. \-

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Conditions

The condition(s) this trial relates to.

primary biliary cholangitis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Primary biliary cholangitis

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

42 sites. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

Affiliated Hospital of Guangdong Medical University
RECRUITING

Zhanjiang, Guangdong, China
Baoji People's Hospital
RECRUITING

Baoji, Shaanxi, China
Beijing Friendship Hospital, Capital Medical University
RECRUITING

Beijing, Beijing Municipality, 100050, China
Beijing Youan Hospital, Capital Medical University
RECRUITING

Beijing, Beijing Municipality, 100069, China
China-Japan Friendship Hospital
RECRUITING

Beijing, Beijing Municipality, China
Chongqing University Three Gorges Hospital
RECRUITING

Chongqing, Chongqing Municipality, China
Hangzhou Xixi Hospital
RECRUITING

Hangzhou, Zhejiang, China
Henan Provincial People's Hospital
RECRUITING

Zhengzhou, Henan, China
Jiangsu Province Hospital
RECRUITING

Nanjing, Jiangsu, China
Linyi People's Hospital
RECRUITING

Linyi, Shandong, China
Mengchao Hepatobiliary Hospital Of Fujian Medical University
RECRUITING

Fuzhou, Fujian, China
Ningbo Medical Center Li Huili hospital
NOT_YET_RECRUITING

Ningbo, Zhejiang, China
Peking University Shenzhen Hospital
RECRUITING

Shenzhen, Guangdong, China
Renji Hospital, School of Medicine, Shanghai Jiao Tong University
RECRUITING

Shanghai, Shanghai Municipality, 200001, China
Shanghai Jiao Tong University School of Medicine Ruijin Hospital
RECRUITING

Shanghai, Shanghai Municipality, China
Shenzhen Third People's Hospital
RECRUITING

Shenzhen, Guangdong, China
Taihe Hospital of Shiyan
RECRUITING

Shiyan, Hubei, China
Taizhou Hospital of Zhejiang Province
RECRUITING

Taizhou, Zhejiang, China
The Affiliated Hospital of Qingdao University
RECRUITING

Qingdao, Shandong, China
The Fifth People's Hospital Of Wuxi
RECRUITING

Wuxi, Jiangsu, China
The Fifth People's Hospital of Suzhou
RECRUITING

Suzhou, Jiangsu, China
The First Affiliated Hospital of Anhui Medical University
RECRUITING

Hefei, Anhui, China
The First Affiliated Hospital of Guangxi Medical University
RECRUITING

Nanning, Guangxi, China
The First Affiliated Hospital of Nanchang University
RECRUITING

Nanchang, Jiangxi, China
The First Affiliated Hospital of USTC Anhui Provincial Hospital
RECRUITING

Hefei, Anhui, 230001, China
The First Affiliated Hospital of Xi'an Jiaotong University
RECRUITING

Xi'an, Shaanxi, China
The First Bethune Hospital of Jilin University
RECRUITING

Changchun, Jilin, China
The First Hospital of China Medical University
RECRUITING

Shenyang, Liaoning, China
The Fourth Hospital Of Harbin Medical University
RECRUITING

Harbin, Heilongjiang, China
The Fourth People's Hospital of Qinghai Province
NOT_YET_RECRUITING

Xining, Qinghai, China
The Second Affiliated Hospital of Anhui Medical University
RECRUITING

Hefei, Anhui, China
The Second Affiliated Hospital of Chongqing Medical University
RECRUITING

Chongqing, Chongqing Municipality, China
The Second Hospital of Nanjing
RECRUITING

Nanjing, Jiangsu, China
The Third People's Hospital of Zhenjiang
RECRUITING

Zhenjiang, Jiangsu, China
Tianjin First Central Hospital
RECRUITING

Tianjin, Tianjin Municipality, China
Tianjin Medical University General Hospital
RECRUITING

Tianjin, Tianjin Municipality, China
Tianjin Third Central Hospital
RECRUITING

Tianjin, Tianjin Municipality, China
West China Hospital, Sichuan University
RECRUITING

Chengdu, Sichuan, China
Xiamen Hospital of T.C,M.
RECRUITING

Xiamen, Fujian, China
Xiangya Hospital Central South University
RECRUITING

Changsha, Hunan, China
Xin Hua Hospital Affiliated To Shanghai Jiao Tong University School of Medicine
RECRUITING

Shanghai, Shanghai Municipality, China
Zhongnan Hospital of Wuhan University
NOT_YET_RECRUITING

Wuhan, Hubei, China

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