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

A Post-Authorisation Non-Interventional Observational, Multi-Site Study of Patients in France With Primary Biliary Cholangitis Treated With Obeticholic Acid in Real Life (OCARELIFE)

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

Study Overview

• Status: Unknown
• Conditions: Primary Biliary Cholangitis

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 Type: Observational
• Enrollment (Anticipated): 150

Contacts and Locations

• Study Contact: Name: Francois BOER, MD; Phone Number: +33 07 62 69 31 02; Email: francois.boer@interceptpharma.com
• Study Contact Backup: Name: Benedicte SAMEY; Phone Number: +33 06 87 97 14 27; Email: bsamey@axonal.com

Study Locations

• France
  • Grenoble, France, 38700
    • Recruiting
    • Chu Grenoble
    • Contact: Vincent LEROY, MD; Phone Number: +33 0476765450; Email: vleroy@chu-grenoble.fr
    • Contact: Laure Bordy; Email: lbordy@chu-grenoble.fr
  • Paris, France, 75571
    • Recruiting
    • Hôpital Saint Antoine
    • Contact: Christophe Corpechot, MD; Phone Number: +33 01 49 28 28 36; Email: christophe.corpechot@aphp.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

1. Patients with PBC treated between October 1st, 2016 and December 31, 2017 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).

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response rate using the Paris 2 criteria at the end of the study	Response rate using the Paris 2 criteria at the end of the study, 18 month after treatment initiation	18 month

Collaborators and Investigators

• Sponsor: SAMEY
• Collaborators: Axonal-Biostatem

Study record dates

Study Major Dates

• Study Start (Actual): April 10, 2018
• Primary Completion (Anticipated): April 1, 2019
• Study Completion (Anticipated): April 1, 2019

Study Registration Dates

• First Submitted: October 9, 2018
• First Submitted That Met QC Criteria: October 9, 2018
• First Posted (Actual): October 11, 2018

Study Record Updates

• Last Update Posted (Actual): October 11, 2018
• Last Update Submitted That Met QC Criteria: October 9, 2018
• Last Verified: October 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Liver Diseases; Fibrosis; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis, Intrahepatic; Cholestasis; Liver Cirrhosis; Cholangitis; Liver Cirrhosis, Biliary
• Other Study ID Numbers: 2018-A00075-50

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No