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Primary Biliary CholangitisPhase 3NCT07282353

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

A Phase 3 study testing 2mg CS0159 in people with Primary Biliary Cholangitis, run by Cascade Pharmaceuticals, Inc at 42 sites in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Primary Biliary Cholangitis
What is being tested?
2mg CS0159
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
135 participants
Where
42 sitesChina
Study dates
1 Dec 2025to 6 Jan 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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;
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  • 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);
  • 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)
  • Platelet count ≥ 1.0× LLN (No thrombocytopenia-related treatment within the past two weeks)
  • Albumin> 35g/L
  • White blood cells count (WBC) >3×109/L
  • Absolute neutrophil count (ANC) >1.5×109/L
  • Hemoglobin A1c (HbA1c) ≤9.0%;
  • INR ≤ 1.0× ULN. For participants on anticoagulation therapy, INR must be maintained in the range required for prophylaxis for their specific disease;
  • 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.
  • -

Who cannot join

  • 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;
Show all 39Show fewer
  • 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)
  • History or clinical evidence of Alcoholic liver disease (ALD)
  • Biopsy confirmed Non-alcoholic steatohepatitis (NASH)
  • Gilbert's Syndrome
  • History or evidence of alpha-1 antitrypsin deficiency
  • Liver stiffness measured by transient elastography (TE) > 16.9 Kpa;
  • 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];
  • Administration of the following medications are prohibited as specified below:
  • Use of medications, food, and drinks (e.g., grapefruit juice) that are strong or moderate CYP3A4 inhibitors or inducers within 14 days before randomization;
  • Use of P-glycoprotein (P-gp) substrate drugs within 14 days before randomization;
  • 2 months prior to randomization: fibrates, glitazones, seladelpar and elafibranor.
  • 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).
  • Patients with systemic treatment for pruritus (e.g., with bile acid sequestrants [BAS]) within 3 months prior to randomization.
  • 12 months prior to randomization: antibodies or immunotherapy directed against ILs or other cytokines or chemokines;
  • Medical conditions that may cause non-hepatic increases in ALP (e.g., paget's disease);
  • 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)];
  • 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);
  • 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.
  • 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);
  • 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;
  • Pregnancy, planned pregnancy, lactation;
  • Treatment with any other investigational therapy or device within 30 days or within 5 half-lives, whichever is longer, prior to screening;
  • Mental instability or incompetence that may compromise the validity of informed consent or ability to adhere to study requirements;
  • 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.
  • -

Trial locations and contacts

42 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Ma Xiong, MD contact

15900984550

maxiongmd@163.com

Xiao Xiao, MD contact

15921514136

xxiao_sh@163.com

42 sites in 1 country

China42 sites, 42 recruiting

  • Baoji People's Hospital

    Baoji, Shaanxi

    Jing Fuchun

    Recruiting

  • Beijing Friendship Hospital, Capital Medical University

    Beijing, Beijing Municipality, 100050

    You Hong, MD

    Recruiting

  • Beijing Youan Hospital, Capital Medical University

    Beijing, Beijing Municipality, 100069

    Xu Bin

    Recruiting

  • China-Japan Friendship Hospital

    Beijing, Beijing Municipality

    Yang Song

    Recruiting

  • The First Bethune Hospital of Jilin University

    Changchun, Jilin

    Niu Junqi

    Recruiting

  • Xiangya Hospital Central South University

    Changsha, Hunan

    Zhang Xiaomei

    Huang Yan

    Recruiting

  • West China Hospital, Sichuan University

    Chengdu, Sichuan

    Yang Li

    Recruiting

  • Chongqing University Three Gorges Hospital

    Chongqing, Chongqing Municipality

    An Xuan

    Recruiting

  • The Second Affiliated Hospital of Chongqing Medical University

    Chongqing, Chongqing Municipality

    Cai Dachuan

    Recruiting

  • Mengchao Hepatobiliary Hospital Of Fujian Medical University

    Fuzhou, Fujian

    Huang Zuxiong

    Recruiting

  • Hangzhou Xixi Hospital

    Hangzhou, Zhejiang

    Jin Qiaofei

    Recruiting

  • The Fourth Hospital Of Harbin Medical University

    Harbin, Heilongjiang

    Zhong Lihua

    Recruiting

  • The First Affiliated Hospital of USTC Anhui Provincial Hospital

    Hefei, Anhui, 230001

    He Hongliang

    Recruiting

  • The First Affiliated Hospital of Anhui Medical University

    Hefei, Anhui

    Gao Yufeng

    Recruiting

  • The Second Affiliated Hospital of Anhui Medical University

    Hefei, Anhui

    Zhang Zhenhua

    Recruiting

  • Linyi People's Hospital

    Linyi, Shandong

    Gao Feng

    Recruiting

  • The First Affiliated Hospital of Nanchang University

    Nanchang, Jiangxi

    Wu Xiaoping

    Recruiting

  • Jiangsu Province Hospital

    Nanjing, Jiangsu

    Li Jun

    Recruiting

  • The Second Hospital of Nanjing

    Nanjing, Jiangsu

    Yang Yongfeng

    Recruiting

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, Guangxi

    Su Minghua

    Recruiting

  • Ningbo Medical Center Li Huili hospital

    Ningbo, Zhejiang

    Ye Hua

    Not yet recruiting

  • The Affiliated Hospital of Qingdao University

    Qingdao, Shandong

    Jiang Yueping

    Recruiting

  • Renji Hospital, School of Medicine, Shanghai Jiao Tong University

    Shanghai, Shanghai Municipality, 200001

    Ma Xiong, MD, 15900984550, maxiongmd@163.com

    Recruiting

  • Shanghai Jiao Tong University School of Medicine Ruijin Hospital

    Shanghai, Shanghai Municipality

    Xie Qing

    Recruiting

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

    Shanghai, Shanghai Municipality

    Fan Jiangao

    Recruiting

  • The First Hospital of China Medical University

    Shenyang, Liaoning

    Li Yiling

    Recruiting

  • Peking University Shenzhen Hospital

    Shenzhen, Guangdong

    Wu Jing

    Hu Guoxin

    Recruiting

  • Shenzhen Third People's Hospital

    Shenzhen, Guangdong

    Chen Jun

    Recruiting

  • Taihe Hospital of Shiyan

    Shiyan, Hubei

    Meng Zhongji

    Recruiting

  • The Fifth People's Hospital of Suzhou

    Suzhou, Jiangsu

    Zhu Chuanwu

    Recruiting

  • Taizhou Hospital of Zhejiang Province

    Taizhou, Zhejiang

    Chen Huazhong

    Recruiting

  • Tianjin First Central Hospital

    Tianjin, Tianjin Municipality

    Wang Fengmei

    Recruiting

  • Tianjin Medical University General Hospital

    Tianjin, Tianjin Municipality

    Wang Bangmao

    Recruiting

  • Tianjin Third Central Hospital

    Tianjin, Tianjin Municipality

    Xiang Huiling

    Recruiting

  • Zhongnan Hospital of Wuhan University

    Wuhan, Hubei

    Chen mingkai

    Not yet recruiting

  • The Fifth People's Hospital Of Wuxi

    Wuxi, Jiangsu

    Wang Zheng

    Recruiting

  • The First Affiliated Hospital of Xi'an Jiaotong University

    Xi'an, Shaanxi

    He Yinli

    Recruiting

  • Xiamen Hospital of T.C,M.

    Xiamen, Fujian

    Liang Huiqin

    Recruiting

  • The Fourth People's Hospital of Qinghai Province

    Xining, Qinghai

    Zu Hongmei

    Not yet recruiting

  • Affiliated Hospital of Guangdong Medical University

    Zhanjiang, Guangdong

    Han Huanqin

    Recruiting

  • Henan Provincial People's Hospital

    Zhengzhou, Henan

    Shang Jia

    Recruiting

  • The Third People's Hospital of Zhenjiang

    Zhenjiang, Jiangsu

    Tan Youwen, 0511-80578875

    Recruiting

Other trials in China

About this study

Official title: 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)

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).

Read the detailed description

This is an multicenter, randomized, double-blind, placebo-controlled, parallel-group study that evaluates the efficacy and safety of CS0159 in patients with PBC who have inadequate response or intolerance to ursodeoxycholic acid (UDCA). Approximately 135 participants will be randomized in a 2:1 ratio to receive CS0159 (4 mg) or placebo, once daily, for a maximum of 52 weeks. When applicable, study participants should continue their pre-study dose of UDCA.

Being tested
2mg CS0159, Placebo
Medical subject headings
Liver Cirrhosis, Biliary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 2mg CS0159Drug

    Oral QD

  • PlaceboDrug

    Oral QD

Study groups in NCT07282353
Study groupRoleWhat they receive
4 mg CS0159ExperimentalTake 4mg of CS0159 QD for 52 weeks.
Receives: Drug: 2mg CS0159
placeboPlacebo comparatorTake placebo QD for 52 weeks.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: Baseline to week 52 .

Secondary outcomes (7)

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

    Measured: Basline to week 26 and week 52 .

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

    Measured: Basline to week 26 and week 52 .

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

    Measured: Baseline to week 26 .

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

    Measured: Baseline to week 26 and week 52 .

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

    Measured: Baseline to week 52 .

  6. The first occurrence of clinical outcome events as defined

    Overall death Liver transplantation Model for End-Stage Liver Disease (MELD)-Na score >14 for at least 2 consecutive visits Ascites requiring treatment

    Hospitalization for new onset or recurrence of any of the following:

    1. Variceal bleeding ii. Hepatic encephalopathy (as defined by a West Haven score ≥2) iii. Spontaneous bacterial peritonitis (confirmed by culture from diagnostic paracentesis).

    Measured: up to 56 weeks

  7. Assessment of treatment-emergent AEs (TEAEs)

    Safety

    Measured: up to 56 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Dec 2025
  2. Study start1 Dec 2025
  3. Primary completion9 Dec 2027 (estimated)
  4. Study completion6 Jan 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
135 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07282353
Sponsor's protocol ID
CS0159-PBC-CN-III-07
Status last verified
Jul 2026
First submitted
21 Nov 2025
Last updated
8 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing 2mg CS0159 in people with Primary Biliary Cholangitis, run by Cascade Pharmaceuticals, Inc at 42 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 21 inclusion and 39 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 42 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Ma Xiong, MD (15900984550, maxiongmd@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The proportion of patients who achieve the composite response criteria at Week 52 (time frame: Baseline to week 52 .). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07282353.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2026.

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