A Post-Authorisation Non-Interventional Observational of Patients in France With Primary Biliary Cholangitis Treated With Obeticholic Acid in Real Life

• Conditions: Primary Biliary Cholangitis

• Registration Number: NCT03703076
• Lead Sponsor: SAMEY

Brief Summary

Post-authorisation non-interventional observational study of patients with Primary Biliary Cholangitis who started Ocaliva® treatment between October 1st, 2016 and December 31, 2017.

Detailed Description

This is a non-interventional, multi-site, ambispective, national follow-up study of patients with Primary Biliary Cholangitis who started Ocaliva® treatment between October 1st, 2016 and December 31, 2017. This represents approximately 150 patients to date, who are therefore the first patients to have received treatment with Ocaliva® in France outside of pre-approval clinical trials.

All patients who received treatment within the context of the ATU (conditional marketing authorization in France) or after EU marketing authorization approval for the product will be followed up for 18 months (this follow-up will be partly retrospective and partly prospective). The study therefore involves retrospective recruitment with retrospective and prospective data collection.

Study Timeline

• Study Start: 2018-04-10
• Status Verified: 2018-10
• Study First Submitted: 2018-10-09
• Study First Submitted QC: 2018-10-09
• Last Update Submitted: 2018-10-09
• Study First Posted: 2018-10-11
• Last Update Posted: 2018-10-11
• Primary Completion: 2019-04
• Study Completion: 2019-04

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

• Patients with PBC treated with Ocaliva® 5 mg or 10 mg according to the terms of Ocaliva® approved SmPC: in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA (over the age of 18 years at the time of treatment introduction).
• Patients with PBC with a suboptimal response to UDCA who received at least one dose of Ocaliva®.
• Patients with or without an overlap syndrome (Nguyen-Khac E, 2004)
• Prior to any follow-up and inclusion in this cohort, the attending physician must give the patient or his or her legal representative or designated healthcare proxy information on the drug, a side effect report and notice of his or her inclusion in the cohort (patient information sheet in writing).
• The patient (or his or her legal representative or designated healthcare proxy) must have read the information sheet and agreed verbally to take part, and must show the sheet to any doctor who is consulted.

Exclusion Criteria

• Diseases triggering a non-hepatic elevation of alkaline phosphatases (ALP) (for example Paget's disease or fractures within the previous three months).
• Participation in another investigational study on a product, biological material or medical device within the 30 days prior to selection.
• Inability to comply with the study follow-up procedures.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Response to Ocaliva® after 12 months of treatment	12 month	Response to Ocaliva® after 12 months of treatment as monotherapy or in combination, using the published Paris 2 response criteria

Secondary Outcome Measures

Name	Time	Method
Response rate using the Paris 2 criteria at the end of the study	18 month	Response rate using the Paris 2 criteria at the end of the study, 18 month after treatment initiation

Trial Locations

Locations (2)

Hopital Saint Antoine; 🇫🇷 Paris, France

Chu Grenoble; 🇫🇷 Grenoble, France