Actively Recruiting
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of CS0159 in Patients With Primary Biliary Cholangitis With Inadequate Response or Intolerance to Ursodeoxycholic Acid
Led by Cascade Pharmaceuticals, Inc · Updated on 2025-12-30
135
Participants Needed
41
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effectiveness and safety of a drug called CS0159 in adults with Primary Biliary Cholangitis (PBC) who have an inadequate response or cannot tolerate ursodeoxycholic acid (UDCA). This Phase III trial is randomized, double-blind, and placebo-controlled, aiming to improve treatment options for PBC patients with limited success from existing therapies. Participants will be randomly assigned in a 2:1 ratio to receive either 4 mg of CS0159 or a placebo once daily for up to 52 weeks. Those already taking UDCA may continue their stable dose if applicable. The study compares CS0159 to placebo to assess its impact over this one-year period. During the trial, participants will undergo regular monitoring including laboratory tests to measure liver enzymes and other health markers. Researchers will track response to treatment, adverse events, and clinical outcomes up to 56 weeks. The main goal is to measure how many patients meet specific response criteria at week 52, with ongoing safety assessments throughout the study period.
CONDITIONS
Brief Title
A Study of CS0159 in Patients With PBC With Inadequate Response or Intolerance to UDCA
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must provide written informed consent
- Age between 18 and 75 years, male or female
- Diagnosis of Primary Biliary Cholangitis based on at least two criteria: ALP above 1.0× ULN for 6+ months, positive antimitochondrial antibody or PBC-specific antinuclear antibody, or liver biopsy consistent with PBC
- UDCA treatment for 6+ months before randomization with stable dose for 3+ months if response was poor, or intolerance to UDCA with no use for 3+ months prior
- Screening lab values meeting criteria: ALP ≥1.67× ULN, ALT ≤5× ULN, AST ≤5× ULN, TB <2× ULN, eGFR >60 mL/min/1.73m2, INR ≤1.0× ULN (or maintained therapeutic range if on anticoagulation), platelet count ≥150×10^9/L, albumin >35 g/L, WBC >3×10^9/L, ANC >1.5×10^9/L, HbA1c ≤9.0%
- Females of reproductive potential must use two forms of effective contraception during the study and for 90 days after last dose; males with partners of reproductive potential must use barrier contraception and partners must use a second method during study and 90 days after
You will not qualify if you...
- Previous exposure to CS0159
- Allergy to CS0159, its components, or similar drugs
- Advanced PBC by Rotterdam criteria (albumin <1.0×LLN and TB >1.0×ULN)
- Significant liver cirrhosis complications including liver transplant history, MELD-Na score ≥12, variceal bleeding, ascites requiring treatment, spontaneous bacterial peritonitis, hepatorenal syndrome, hepatic hydrothorax, hepatic encephalopathy, portopulmonary hypertension, hepatopulmonary syndrome, or hepatocellular carcinoma
- Other liver diseases such as autoimmune hepatitis, overlap syndromes, hepatitis B or C infection, primary sclerosing cholangitis, alcoholic liver disease, non-alcoholic steatohepatitis, Gilbert's Syndrome, alpha-1 antitrypsin deficiency, or liver stiffness >16.9 Kpa
- Positive HIV or active syphilis infection
- Use of prohibited medications including CYP3A4 inhibitors/inducers, P-gp substrates, certain fibrates, glitazones, immunotherapies, or hepatotoxic drugs within specified timeframes
- Medical conditions causing non-liver related ALP increase
- Severe arrhythmia or prolonged QTc interval
- Diseases affecting drug absorption or metabolism such as inflammatory bowel disease or bariatric surgery
- History of malignancy within 5 years except certain skin and cervical cancers
- Drug abuse or heavy alcohol use within 12 months
- Poor blood pressure control during screening
- Pregnancy, planned pregnancy, or lactation
- Use of other investigational therapies within 30 days or 5 half-lives before screening
- Mental instability affecting consent or adherence
- Any condition judged unsafe or compromising study quality by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive daily oral doses of either 4 mg CS0159 or placebo for up to 52 weeks while continuing their pre-study dose of UDCA if applicable.
Visits occur periodically during treatment for assessments and safety monitoring
Trial Site Locations
Total: 41 locations
1
The First Affiliated Hospital of USTC Anhui Provincial Hospital
Hefei, Anhui, China, 230001
Not Yet Recruiting
2
The Second Affiliated Hospital of Anhui Medical University
Hefei, Anhui, China
Not Yet Recruiting
3
Beijing Friendship Hospital, Capital Medical University
Beijing, Beijing Municipality, China, 100050
Not Yet Recruiting
4
Beijing Youan Hospital, Capital Medical University
Beijing, Beijing Municipality, China, 100069
Not Yet Recruiting
5
China-Japan Friendship Hospital
Beijing, Beijing Municipality, China
Not Yet Recruiting
6
Chongqing University Three Gorges Hospital
Chongqing, Chongqing Municipality, China
Not Yet Recruiting
7
The Second Affiliated Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, China
Not Yet Recruiting
8
Mengchao Hepatobiliary Hospital Of Fujian Medical University
Fuzhou, Fujian, China
Not Yet Recruiting
9
Peking University Shenzhen Hospital
Shenzhen, Guangdong, China
Not Yet Recruiting
10
Shenzhen Third People's Hospital
Shenzhen, Guangdong, China
Not Yet Recruiting
11
Affiliated Hospital of Guangdong Medical University
Zhanjiang, Guangdong, China
Not Yet Recruiting
12
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
Not Yet Recruiting
13
Hebei Medical University Third Hospital
Shijiazhuang, Hebei, China
Not Yet Recruiting
14
The Fourth Hospital Of Harbin Medical University
Harbin, Heilongjiang, China
Not Yet Recruiting
15
Henan Provincial People's Hospital
Zhengzhou, Henan, China
Not Yet Recruiting
16
Taihe Hospital of Shiyan
Shiyan, Hubei, China
Not Yet Recruiting
17
Xiangya Hospital Central South University
Changsha, Hunan, China
Not Yet Recruiting
18
Jiangsu Province Hospital
Nanjing, Jiangsu, China
Not Yet Recruiting
19
The Second Hospital of Nanjing
Nanjing, Jiangsu, China
Not Yet Recruiting
20
The Fifth People's Hospital of Suzhou
Suzhou, Jiangsu, China
Not Yet Recruiting
21
The Fifth People's Hospital Of Wuxi
Wuxi, Jiangsu, China
Not Yet Recruiting
22
The Third People's Hospital of Zhenjiang
Zhenjiang, Jiangsu, China
Not Yet Recruiting
23
The First Affiliated Hospital of Nanchang University
Nanchang, Jiangxi, China
Not Yet Recruiting
24
The Second Affiliated Hospital of Nanchang University
Nanchang, Jiangxi, China
Not Yet Recruiting
25
The First Bethune Hospital of Jilin University
Changchun, Jilin, China
Not Yet Recruiting
26
The First Hospital of China Medical University
Shenyang, Liaoning, China
Not Yet Recruiting
27
The Sixth People's Hospital of Shenyang
Shenyang, Liaoning, China
Not Yet Recruiting
28
Baoji People's Hospital
Baoji, Shaanxi, China
Not Yet Recruiting
29
The First Affiliated Hospital of Xi'an Jiaotong University
Xi'an, Shaanxi, China
Not Yet Recruiting
30
Linyi People's Hospital
Linyi, Shandong, China
Not Yet Recruiting
31
The Affiliated Hospital of Qingdao University
Qingdao, Shandong, China
Not Yet Recruiting
32
Renji Hospital, School of Medicine, Shanghai Jiao Tong University
Shanghai, Shanghai Municipality, China, 200001
Actively Recruiting
33
Shanghai Jiao Tong University School of Medicine Ruijin Hospital
Shanghai, Shanghai Municipality, China
Not Yet Recruiting
34
Xin Hua Hospital Affiliated To Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
Not Yet Recruiting
35
West China Hospital, Sichuan University
Chengdu, Sichuan, China
Not Yet Recruiting
36
Tianjin First Central Hospital
Tianjin, Tianjin Municipality, China
Not Yet Recruiting
37
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, China
Not Yet Recruiting
38
Tianjin Third Central Hospital
Tianjin, Tianjin Municipality, China
Not Yet Recruiting
39
Hangzhou Xixi Hospital
Hangzhou, Zhejiang, China
Not Yet Recruiting
40
Taizhou Hospital of Zhejiang Province
Taizhou, Zhejiang, China
Not Yet Recruiting
41
The First Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang, China
Not Yet Recruiting
Research Team
M
Ma Xiong, MD
X
Xiao Xiao, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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