Clinical trial NCT03703076: A Post-Authorisation Non-Interventional Observational of Patients in France With Primary Biliary Cholangitis Treated With Obeticholic Acid in Real Life
Official title: 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)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Primary Biliary Cholangitis
Lead sponsor: Intercept Pharma France SAS
Sponsor class: INDUSTRY
Collaborators: name: Axonal-Biostatem
First posted: 2018-10-11
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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).

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Response to Ocaliva® after 12 months of treatment
timeFrame: 12 month
Secondary outcomes:
measure: Response rate using the Paris 2 criteria at the end of the study
timeFrame: 18 month
Other outcomes:
Locations:
city: Grenoble
country: France
facility: Chu Grenoble
city: Paris
country: France
facility: Hopital Saint Antoine
Study officials:
References: