Fenofibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cholangitis

The main objectives of the study were to assess the effects of fenofibrate on serum alkaline phosphatase, as a composite endpoint and on safety in participants with primary biliary cholangitis (PBC)

Study Overview

• Status: Active, not recruiting
• Conditions: Primary Biliary Cholangitis
• Intervention / Treatment: Drug: Placebo; Drug: UDCA; Drug: Fenofibrate

• Study Type: Interventional
• Enrollment (Estimated): 184
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Peking Union Medical College Hospital
  • Gansu
    • Lanzhou, Gansu, China
      • The Second Hospital of Lanzhou University
  • Guangdong
    • Guangzhou, Guangdong, China
      • Sun Yat-Sen Memorial Hospital
  • Jiangsu
    • Nanjing, Jiangsu, China
      • Jiangsu Province Hospital
    • Nanjing, Jiangsu, China
      • Nanjing Drum Tower Hospital
    • Nanjing, Jiangsu, China
      • Nanjing Second Hospital
  • Liaoning
    • Shenyang, Liaoning, China
      • Shengjing Hospital affiliated to China Medical University
  • Shaanxi
    • Xi'an, Shaanxi, China
      • Xijing Hospital
  • Shanxxi
    • Yan’an, Shanxxi, China
      • Yan'an University Affiliated Hospital
  • Yunnan
    • Kunming, Yunnan, China
      • The Second Affiliated Hospital of Kunming Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Must have provided written informed consent
• Age 18-75 years;
• BMI 17-28 kg/m2

• Male or female with a diagnosis of PBC, by at least two of the following criteria:

  1. History of alkaline phosphatase（ALP） above upper limit of normal（ULN） for at least six months;
  2. Positive Anti-mitochondrial antibody (AMA) titers (>1/40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies;
  3. Documented liver biopsy result consistent with PBC.
• Incomplete response to UDCA defined by 1 x ULN< ALP <= 1.67 x ULN
• Taking UDCA for at least 6 months (stable dose for ≥ 3 months) prior to Day 0

Exclusion Criteria:

• History or presence of other concomitant liver diseases.
• ALT or AST > 5×ULN, total bilirubin（TBIL） > 3×ULN.
• If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating.
• Allergic to fenofibrate or ursodeoxycholic acid.
• Taking hepatotoxic drugs (e.g., dapsone, erythromycin, fluconazole, ketoconazole, rifampicin) for more than 2 weeks within 6 months, and long-term hormonal users.

Recurrent variceal bleeding, poorly controlled hepatic encephalopathy or refractory ascites.

• Patients with a history of severe cardiac, cerebrovascular, renal, respiratory disease or functional failure, and psychiatric disorders (including those due to alcohol and drug abuse).
• Creatinine >1.5×ULN and creatinine clearance <60 ml/min.
• Currently using statins (such as pravastatin, fluvastatin, and simvastatin), other fibrates (such as gemfibrozil and bezafibrate), and drugs structurally similar to fenofibrate (like ketoprofen).
• Planned to receive an organ transplant or an organ transplant recipient.
• Needing Liver transplantation within 1 year according to the Mayo Rick score.
• Any other condition(s) that would compromise the safety of the subject or compromise

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo-UDCA; 1 tablet/ day and UDCA 13-15mg/kg/day for 12 months	Drug: Placebo; 1 tablet/ day; Drug: UDCA; UDCA 13-15mg/kg/day
Experimental: Fenofibrate-ursodesoxycholic acid（UDCA）; Fenofibrate 200 mg/day and UDCA 13-15mg/kg/day for 12 months	Drug: UDCA; UDCA 13-15mg/kg/day; Drug: Fenofibrate; Fenofibrate 200 mg/day

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of patients with biochemical response	The normalisation of Alkaline Phosphatase	48 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of patients having biochemical response	The normalisation of Alkaline Phosphatase at 4, 12, 24, and 36 weeks	4, 12, 24 and 36weeks
Survival without transplantation and hepatic impairment	Occurrence of ascites, variceal bleeding, hepatic encephalopathy, liver-transplantation, or death	48 weeks
Assessment of the pruritus	Change From Baseline in Pruritus as Assessed by Visual Analogue Scale (VAS) Total Score for Fatigue and Pruritus. (0-10, higher scores mean a worse outcome)	4, 12, 24, 36, and 48 weeks
Assessment of the fatigue	Change From Baseline in Fatigue as Assessed by Visual Analogue Scale (VAS) Total Score for Fatigue and Pruritus. (0-10, higher scores mean a worse outcome)	4, 12, 24, 36, and 48 weeks
Percentage of patients having biological or clinical adverse events	Increase of creatinine, blood urea nitrogen, alanine aminotransferase and aspartate aminotransferase	4, 12, 24, 36, and 48 weeks

Collaborators and Investigators

• Sponsor: Han Ying

Study record dates

Study Major Dates

• Study Start (Actual): August 21, 2023
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: December 8, 2023
• First Submitted That Met QC Criteria: December 8, 2023
• First Posted (Actual): December 18, 2023

Study Record Updates

• Last Update Posted (Actual): January 8, 2026
• Last Update Submitted That Met QC Criteria: January 6, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Digestive System Diseases; Biliary Tract Diseases; Liver Diseases; Bile Duct Diseases; Fibrosis; Cholestasis, Intrahepatic; Cholestasis; Liver Cirrhosis; Pathological Conditions, Signs and Symptoms; Liver Cirrhosis, Biliary; Organic Chemicals; Ethers; Hydrocarbons; Hydrocarbons, Cyclic; Acids, Acyclic; Carboxylic Acids; Hydrocarbons, Aromatic; Phenols; Benzene Derivatives; Butyrates; Phenyl Ethers; Ketones; Fibric Acids; Isobutyrates; Benzophenones; Fenofibrate
• Other Study ID Numbers: KY-20232219-C-1

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No