Actively Recruiting

Phase 3
Age: 18Years - 80Years
All Genders
NCT06443606

Efficacy and Safety of Bezafibrate 400 mg and Bezafibrate 200 mg as Adjunctive Treatments in Patients With Primary Biliary Cholangitis and Non-optimal Biochemical Response to Ursodeoxycholic Acid Therapy

Led by Assistance Publique - Hôpitaux de Paris · Updated on 2025-08-27

108

Participants Needed

1

Research Sites

218 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Primary biliary cholangitis (PBC) is a rare chronic, progressive, cholestatic liver disease that leads to cirrhosis and its life-threatening complications if undertreated. Ursodeoxycholic acid (UDCA) is the standard-of-care therapy for PBC. However, patients with an inadequate biochemical response to UDCA according to the Paris-2 criteria are still at high-risk of poor clinical outcome. In this situation of biochemical resistance to UDCA, bezafibrate 400 mg/d given in association with UDCA has been shown to improve the symptoms, biochemical response (BEZURSO study), histologic features, and possibly long-term clinical outcome. However, it has been shown that even patients with an adequate response to UDCA but persistent elevation in biochemical markers of cholestasis or liver inflammation, including alkaline phosphatases (ALP), gamma-glutamyl transpeptidase (GGT), transaminases, or total bilirubin (i.e., non-optimal biochemical response) have still an increased risk of death or liver transplantation in the long term, thus defining the complete normalization of these markers as the new clinically-relevant target for PBC treatment. In parallel to these findings, bezafibrate 400 mg/d as a second-line therapy for PBC could be associated with potentially dose-related, muscle, kidney, or liver toxic effects, and whether bezafibrate 200 mg/d could have a better benefit/risk ratio in this disease-setting remains to be determined. Therefore, our aim is to evaluate the efficacy and safety of bezafibrate 400 mg and bezafibrate 200 mg as adjunctive treatments in PBC patients with non-optimal biochemical response to UDCA.

CONDITIONS

Official Title

Efficacy and Safety of Bezafibrate 400 mg and Bezafibrate 200 mg as Adjunctive Treatments in Patients With Primary Biliary Cholangitis and Non-optimal Biochemical Response to Ursodeoxycholic Acid Therapy

Who Can Participate

Age: 18Years - 80Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 to less than 80 years
  • Diagnosis of primary biliary cholangitis (PBC) based on at least two criteria: elevated alkaline phosphatase (ALP), positive antimitochondrial or specific antinuclear antibodies, or liver biopsy features compatible with PBC
  • Stable ursodeoxycholic acid (UDCA) therapy for at least 12 months with a dose of 12 mg/kg/day or higher for at least 3 months
  • Non-optimal biochemical response to UDCA defined by at least one of the following twice at least 4 weeks apart in the last 3 months including at inclusion: ALP > 1.0 x upper limit, GGT > 3.0 x upper limit, ALT or AST > 1.0 x upper limit, total or conjugated bilirubin > 1.0 x upper limit
  • Women of childbearing potential must use at least one barrier contraceptive during the study and for 90 days after last dose
  • Affiliation to a social security system (except AME)
  • Signed informed consent
Not Eligible

You will not qualify if you...

  • Signs of advanced chronic liver disease such as total bilirubin > 2.0 x upper limit, serum albumin < 32 g/l, platelet count < 100,000/mm3, INR > 1.3 or prothrombin index < 60%, Child-Pugh score B or C, MELD score ≥ 14, history or presence of cirrhotic decompensation within 24 months, or on liver transplant waiting list
  • Estimated glomerular filtration rate (GFR) < 60 mL/min
  • Creatine phosphokinase (CPK) > 5.0 x upper limit
  • AST or ALT > 3.0 x upper limit
  • History of liver transplantation
  • Autoimmune hepatitis overlap syndrome diagnosed by at least two criteria including histology
  • Other chronic liver diseases such as hepatitis C, hepatitis B, non-alcoholic steatohepatitis, alcoholic liver disease, hemochromatosis, Wilson's disease, alpha-1 antitrypsin deficiency, or celiac disease
  • Untreated thyroid disorders such as Hashimoto or Graves disease
  • Conditions causing non-liver increases in ALP (Paget's disease, osteodystrophy, hyperparathyroidism, dysglobulinemia)
  • Gilbert's syndrome or chronic hemolysis
  • History or suspicion of hepatocellular carcinoma
  • Malignancy diagnosed or treated within 2 years (except recent localized skin cancers)
  • Severe comorbidity reducing life expectancy to 2 years or less
  • Pregnancy or breastfeeding
  • Known intolerance or hypersensitivity to bezafibrate or related drugs
  • Photosensitivity or photoallergy to fibrates
  • Congenital galactosemia, glucose malabsorption, or lactase deficiency due to lactose in the study tablets
  • Participation in another interventional study within 6 months
  • Use of certain medications including fibrates, obeticholic acid, corticosteroids, immunosuppressants, or statins within specified recent periods

AI-Screening

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Trial Site Locations

Total: 1 location

1

Hepatology department - Hospital Saint Antoine

Paris, France, 75012

Actively Recruiting

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Research Team

C

Christophe Corpechot, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

DOUBLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

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