Active — Not RecruitingNot Applicable

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

France200 participantsStarted 2026-04-26

Plain-language 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.

Who can participate

Age range3 Months – 100 Years

SexALL

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Part A Eligibility: Comparisons to Evaluate the Effect of Odevixibat Versus SBD-naïve Odevixibat Cohort: * Patients treated with odevixibat in A4250-005 with at least one post-odevixibat assessment and who did not have prior LT or SBD. OR * Patients treated with placebo in A4250-005, first received odevixibat in Cohort 1 of A4250-008 with at least one post-odevixibat assessment, and meet the additional eligibility criteria OR * Patients treated with odevixibat in Cohort 2 of A4250-008 with at least one post-odevixibat assessment and who meet the additional eligibility criteria External Control Cohort: * A male or female patient in NAPPED registry not enrolled in A4250-005 or A4250-008. * The patient must be IBAT inhibitor-naïve (has not received prior treatment with odevixibat, maralixibat, or other IBAT inhibitors). * Patients must have clinical genetic confirmation of PFIC (any type), excluding known pathologic variations of the ABCB11 gene that predict complete absence of the BSEP protein. * The patient has at least one visit in NAPPED (the first of which becomes the Day 1 visit for this cohort) where they meet the eligibility criteria * Patients must be in the regions that participated in A4250-008. Part B Eligibility: Comparisons to Evaluate the Effect of Odevixibat Versus SBD Odevixibat Cohort * Same as Part A External Control Cohort: * A male or female patient in NAPPED registry with clinical diagnosis of PFIC (any type) and not enrolled in A4250-005 or A42…

What they're measuring

Part A: Liver transplant-free survival (LTFS)

Timeframe: From the date of first odevixibat treatment or from the date of first registry entry into NAPPED group where OvEC cohort eligibility criteria were met, until the first occurrence of the defined event or last available follow-up (2017-2025)

Part B: Liver transplant-free survival (LTFS)

Timeframe: From the date of first odevixibat treatment (Odevixibat Cohort) or from the from the date of SBD (External control cohort) until the first occurrence of the defined event or last available follow-up (2017-2025)

Trial details

NCT IDNCT07497724

SponsorIpsen

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2026-08-01

View on ClinicalTrials.gov More PFIC - Progressive Familial Intrahepatic Cholestasis trials