A Study to Assess Efficacy and Safety of Bezafibrate in Patients With Primary Biliary Cholangitis

Study Overview
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Brief Summary
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Up to 40% of patients with PBC have an inadequate response to standard treatment with Ursodeoxycholic Acid (UDCA), those patients represent the group in need for additional therapies, having increased risk of disease progression and decreased survival free of liver transplantation. The main objective of the study is to evaluate safety and efficacy of bezafibrate plus ursodesoxicolic acid in patients with PBC and inadequate response to UDCA.

Official Title
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Safety and Efficacy of Bezafibrate Plus Ursodesoxicolic Acid in Patients With Primary Biliary Cholangitis Without Response

Conditions
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Primary Biliary Cirrhosis

Intervention / Treatment
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* Drug: Bezafibrate 200 MG Oral Tablet
* Drug: Placebo
* Drug: Ursodeoxycholic Acid


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: PBC diagnosis (consistent with American Association for the Study of Liver Disease [AASLD]: History of elevated alkaline phosphatase levels. Anti-mitochondrial antibodies positivity Histopathologic evidence of nonsuppurative cholangitis and destruction of small or medium- sized bile ducts. Use of ursodeoxycholic acid (UDCA) for at least 12 months at enrollment at a therapeutic dose (13 to 15 mg per Kg per day). Evidence of a suboptimal biochemical response to UDCA, defined by the presence of one of the Paris II criteria. Written informed consent. Age ≥ 18 years. Exclusion Criteria: Hepatic decompensation (ascitis, variceal upper gastrointestinal bleeding, hepatic encephalopathy). Coexistence autoimmune hepatitis. Bilirrubin >3mg/dl. For females, pregnancy or breast-feeding. Hepatocellular carcinoma. History or presence of spontaneous bacterial peritonitis.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Bezafibrate and Ursodeoxycholic acid<br>Bezafibrate 200 mg every 12 hours and ursodeoxycholic acid at a dose of 13 to 15 mg per Kg per day, for 6 months. | Drug: Bezafibrate 200 MG Oral Tablet<br>* Bezafibrate one tablet every 12 hours for six months.<br>Drug: Ursodeoxycholic Acid<br>* At a dose of 13 to 15 mg per Kg per day.<br>|
| Placebo Comparator: Placebo and Ursodeoxycholic acid<br>Placebo tablet every 12 hours and ursodeoxycholic acid at a dose of 13 to 15 mg per Kg per day, for 6 months. | Drug: Placebo<br>* Placebo one tablet every 12 hours for six months.<br>Drug: Ursodeoxycholic Acid<br>* At a dose of 13 to 15 mg per Kg per day.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Biochemical response | Biochemical response is defined as the reduction of alkaline phosphatase lower than 1.5 times the upper normal limit, reduction of aspartate transaminase lower than 1.5 times the upper normal limit and bilirubin lower than 1 mg/dl. | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Quality of life | Evaluation of quality of life with the Primary Biliary Cholangitis 40 questionnaire | Baseline and 6 months later | 
| Pruritus intensity | Evaluation made by the use of visual analogue scales. | Baseline and 6 months later | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ursodeoxycholic acid, Bezafibrate