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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Primary Biliary Cholangitis

In brief

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).

Key facts

Study ID

NCT07282353

Run by

Cascade Pharmaceuticals, Inc

People needed

135

Starts

2025-12-01

Expected to finish

2028-01-06

Last updated by the study team

2026-07-08

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

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;
• 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.

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)
• 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.

Where it is running

• Taizhou Hospital of Zhejiang Province — Taizhou, Zhejiang, China (enrolling)
• The First Affiliated Hospital of Anhui Medical University — Hefei, Anhui, China (enrolling)
• The Second Affiliated Hospital of Anhui Medical University — Hefei, Anhui, China (enrolling)
• Beijing Friendship Hospital, Capital Medical University — Beijing, Beijing Municipality, China (enrolling)
• Beijing Youan Hospital, Capital Medical University — Beijing, Beijing Municipality, China (enrolling)
• China-Japan Friendship Hospital — Beijing, Beijing Municipality, China (enrolling)
• Chongqing University Three Gorges Hospital — Chongqing, Chongqing Municipality, China (enrolling)
• The Second Affiliated Hospital of Chongqing Medical University — Chongqing, Chongqing Municipality, China (enrolling)
• Mengchao Hepatobiliary Hospital Of Fujian Medical University — Fuzhou, Fujian, China (enrolling)
• Xiamen Hospital of T.C,M. — Xiamen, Fujian, China (enrolling)
• Peking University Shenzhen Hospital — Shenzhen, Guangdong, China (enrolling)
• Shenzhen Third People's Hospital — Shenzhen, Guangdong, China (enrolling)
• Affiliated Hospital of Guangdong Medical University — Zhanjiang, Guangdong, China (enrolling)
• The First Affiliated Hospital of Guangxi Medical University — Nanning, Guangxi, China (enrolling)
• The Fourth Hospital Of Harbin Medical University — Harbin, Heilongjiang, China (enrolling)
• Henan Provincial People's Hospital — Zhengzhou, Henan, China (enrolling)
• Taihe Hospital of Shiyan — Shiyan, Hubei, China (enrolling)
• Tianjin Third Central Hospital — Tianjin, Tianjin Municipality, China (enrolling)
• Hangzhou Xixi Hospital — Hangzhou, Zhejiang, China (enrolling)
• The First Affiliated Hospital of USTC Anhui Provincial Hospital — Hefei, Anhui, China (enrolling)
• Xiangya Hospital Central South University — Changsha, Hunan, China (enrolling)
• Jiangsu Province Hospital — Nanjing, Jiangsu, China (enrolling)
• The Second Hospital of Nanjing — Nanjing, Jiangsu, China (enrolling)
• The Fifth People's Hospital of Suzhou — Suzhou, Jiangsu, China (enrolling)
• The Fifth People's Hospital Of Wuxi — Wuxi, Jiangsu, China (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.