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Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)

NCT07497724 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 200

Last updated 2026-05-06

No results posted yet for this study

Summary

Progressive familial intrahepatic cholestasis (PFIC) is a rare inherited liver disease that causes a build-up of bile acids in the liver. This can lead to severe itching (pruritus), poor sleep, impaired growth, liver damage, and in some cases the need for surgery or liver transplantation.

The purpose of this non-interventional, retrospective study is to compare long-term health outcomes in patients with PFIC. The comparison is between patients who received odevixibat in two odevixibat clinical trials (Studies A4250-005 and A4250-008) and an aligned, balanced external control cohort of patients with PFIC from the Natural course and Prognosis of PFIC and Effect of biliary Diversion (NAPPED) registry who were not treated with odevixibat (or other ileal bile acid transporter \[IBAT\] inhibitors). Outcomes such as liver transplantation, death, and surgical biliary diversion (SBD) will be examined to better understand how treatment with odevixibat compares to the natural course of PFIC. This study aims to provide a robust comparative evaluation of long-term clinical outcomes with odevixibat.

Conditions

  • PFIC - Progressive Familial Intrahepatic Cholestasis

Sponsors & Collaborators

Principal Investigators

  • Ipsen Medical Director · Ipsen

Eligibility

Min Age
3 Months
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-26
Primary Completion
2026-08-01
Completion
2026-09-30

Countries

  • France

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07497724 on ClinicalTrials.gov