Authorised Therapeutic confirmatory (Phase III) Subjects with PBC with a non-optimal response to UDCA.

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: a 12-month, double-blind, randomized, placebo-controlled trial with a 12-month, double-blind, placebo-free extension phase - BEZURSO 2

EU CTIS ID: 2023-504086-23-00

What this study is testing

To assess the effect of bezafibrate 200 mg and bezafibrate 400 mg versus placebo as adjunctive treatments in patients with PBC and a non-optimal response to UDCA.

Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1. Age ≥ 18 and < 80 years
7. Signed informed consent
2. Diagnosis of PBC based on at least 2 of the following criteria (EASL clinical practice guidelines 2017:
a. Elevated ALP level
b. Presence of antimitochondrial antibody (immunofluorescence titer ≥ 1:40 or positive antigen-specific test), specific antinuclear immunofluorescence (nuclear dots or perinuclear rims) or positive antigen-specific test for anti-gp210 or anti-Sp100 antibodies
c. Records of histologic features suggestive of, or compatible with PBC

You likely can't join if

1. Any of the following signs of advanced chronic liver disease: Total bilirubin > 2.0 xULN; Serum albumin < 32 g/l; Platelet count < 100,000/mm3; INR > 1.3 or prothrombin index < 60%; in the last 6 months; Child-Pugh score B or C; MELD score ≥ 14; History ≤ 24 months or presence of cirrhotic decompensation; Patients on the waiting list for LT
10. Gilbert’s syndrome or chronic hemolysis (hyperbilirubinemia with an unconjugated to total bilirubin ratio ≥ 75%)
11. History of or established or suspected hepatocellular carcinoma
12. History of malignancy diagnosed or treated within 2
13. Any severe comorbidity that may reduce life expectancy ≤ 2 years
14. Pregnancy or lactating

See the full eligibility criteria

Who can join

1. Age ≥ 18 and < 80 years
7. Signed informed consent
2. Diagnosis of PBC based on at least 2 of the following criteria (EASL clinical practice guidelines 2017:
a. Elevated ALP level
b. Presence of antimitochondrial antibody (immunofluorescence titer ≥ 1:40 or positive antigen-specific test), specific antinuclear immunofluorescence (nuclear dots or perinuclear rims) or positive antigen-specific test for anti-gp210 or anti-Sp100 antibodies
c. Records of histologic features suggestive of, or compatible with PBC
3. UDCA therapy for the past 12 months (stable dose ≥ 12 mg/kg/d for ≥ 3 months prior to inclusion)
4. Biochemical evidence of non-optimal response to UCDA (i.e. ALP > 1.0 xULN, or GGT > 3.0 xULN, or ALT or AST > 1.0 xULN, or total and conjugated bilirubin > 1.0 xULN) between 6 months and 2 weeks before inclusion visit. If total bilirubin is within the normal range, measurement of conjugated bilirubin is not mandatory.
5. Women of child-bearing potential (WOCBP), i.e., fertile, following menarche and until becoming post-menopaused unless permanently sterile, who are sexually active have to apply an effective method of birth control*, throughout the study period and for 90 days following the last dose of study treatment. *the list of acceptable method of contraception is in addenda 18.1
6. Affiliation to a social security system (AME excepted)

What rules you out

1. Any of the following signs of advanced chronic liver disease: Total bilirubin > 2.0 xULN; Serum albumin < 32 g/l; Platelet count < 100,000/mm3; INR > 1.3 or prothrombin index < 60%; in the last 6 months; Child-Pugh score B or C; MELD score ≥ 14; History ≤ 24 months or presence of cirrhotic decompensation; Patients on the waiting list for LT
10. Gilbert’s syndrome or chronic hemolysis (hyperbilirubinemia with an unconjugated to total bilirubin ratio ≥ 75%)
11. History of or established or suspected hepatocellular carcinoma
12. History of malignancy diagnosed or treated within 2
13. Any severe comorbidity that may reduce life expectancy ≤ 2 years
14. Pregnancy or lactating
15. Known intolerance to bezafibrate
16. Known hypersensitivity to bezafibrate, any of the components of Befizal© or other fibrates
17. Known photosensitivity reactions or photoallergic reactions to fibrates
18. Patient with congenital galactosemia, glucose malabsorption, or lactase deficiency because of presence of lactose in LP tablets of bezafibrate
19. Participation in any other interventional study in the past 6 months (RIPH1, clinical investigation or clinical trial)
2. GFR estimated by CKI-EPI equation < 60 mL/min in the last 6 months
20. Any of the following medications used in the past 3 months before inclusion: bezafibrate, fenofibrate, ciprofibrate, gemfibrozil, obeticholic acid, budesonide, any other systemic corticosteroids, azathioprine, mycophenolate mofetil, cyclosporine, tacrolimus, sirolimus, everolimus, methotrexate.
21. Use of statins in the month before inclusion
3. CPK > 5.0 xULN in the last 6 months
4. AST or ALT > 3.0 xULN in the last 6 months
5. History of LT
6. Features of autoimmune hepatitis (AIH) overlap syndrome (either past or newly diagnosed during follow up)
7. Any other chronic hepatic comorbidities (HIV, HCV, HBV, NASH, alcoholic liver disease, hemochromatosis, Wilson's disease, α1-antitrypsin deficiency, celiac disease)
8. Untreated hypo or hyperthyroidism (Hashimoto or Graves autoimmune thyroiditis)
9. Conditions that may cause non-hepatic increases in ALP (Paget’s disease, osteodystrophy, hyperparathyroidism, dysglobulinemia)

The study team makes the final eligibility decision.

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.