Ignite Creation Date:
2024-06-16 @ 11:51 AM
Last Modification Date:
2024-10-26 @ 3:31 PM
Study NCT ID:
NCT06443606
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-06-05
First Post:
2024-02-21
Brief 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
Sponsor:
Assistance Publique - Hôpitaux de Paris
Organization:
Assistance Publique - Hôpitaux de Paris
Study Overview
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 a 12-month Double-blind Randomized Placebo-controlled Trial With a 12-month Double-blind Placebo-free Extension Phase
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
BEZURSO 2
Brief Summary:
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 mgd 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 ie 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 mgd as a second-line therapy for PBC could be associated with potentially dose-related muscle kidney or liver toxic effects and whether bezafibrate 200 mgd could have a better benefitrisk 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
Detailed Description:
The study is a phase-3 multicenter randomized parallel-group 111 placebo-controlled trial with a 12-month double-blind placebo-free extension phase
It evaluates the efficacy and safety of bezafibrate 400 mg and bezafibrate 200 mg as adjunctive treatments in patients with PBC with an non-optimal biochemical response to UDCA
Treatments groups
Arm 1 Bezafibrate 400 mg and Placebo of Bezafibrate 200 mg until 96 weeks in double blind
Arm 2 Bezafibrate 200 mg and Placebo of Bezafibrate 400 mg until 96 weeks in double blind
Arm 3 Placebo of Bezafibrate 400 mg and Placebo of Bezafibrate 200 mg until 48 weeks in double blind Then follow-up extension phase of bezafibrate 400 mg or bezafibrate 200 mg second randomization until 48 weeks in double blind
Assessement Study visits at Inclusion Randomisation M0 and then every 3 months until W48 and extension until W96 In accordance with routine care an additional follow-up is added between 108 and 120 weeks
32 sites within the French network of reference and competence centres for rare liver diseases FILFOIE will participate
No interim analysis planned Analysis will be performed at the end of the study after data reviewed and data base locked according to the intent to treat principle
It evaluates the efficacy and safety of bezafibrate 400 mg and bezafibrate 200 mg as adjunctive treatments in patients with PBC with an non-optimal biochemical response to UDCA
Treatments groups
Arm 1 Bezafibrate 400 mg and Placebo of Bezafibrate 200 mg until 96 weeks in double blind
Arm 2 Bezafibrate 200 mg and Placebo of Bezafibrate 400 mg until 96 weeks in double blind
Arm 3 Placebo of Bezafibrate 400 mg and Placebo of Bezafibrate 200 mg until 48 weeks in double blind Then follow-up extension phase of bezafibrate 400 mg or bezafibrate 200 mg second randomization until 48 weeks in double blind
Assessement Study visits at Inclusion Randomisation M0 and then every 3 months until W48 and extension until W96 In accordance with routine care an additional follow-up is added between 108 and 120 weeks
32 sites within the French network of reference and competence centres for rare liver diseases FILFOIE will participate
No interim analysis planned Analysis will be performed at the end of the study after data reviewed and data base locked according to the intent to treat principle
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None