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

NCT07497724 | Active Not Recruiting

• 1 other identifier: CLIN-60240-458
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief 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

Outcome Measures

Primary Outcomes (2)

• Part A: Liver transplant-free survival (LTFS)

  Defined as time from Day 1 to the first occurrence of any of the following clinical events: Death (any cause); Liver Transplant (LT)

  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)

  As defined for Part A

  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)

Secondary Outcomes (11)

• Part A: Event-free survival (EFS)

  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 A: Surgical biliary diversion-free survival (DFS)

  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 A: Overall survival (OS)

  From the date of first odevixibat treatment (Odevixibat Cohort) or from the date of first registry entry into NAPPED group where OvEC cohort eligibility criteria were met (External control Cohort), until death (any cause) (2017-2025)

• Part B: Overall survival (OS)

  From the date of first odevixibat treatment (Odevixibat Cohort) or from the from the date of SBD (External control cohort) until death (any cause) (2017-2025

• Part A: Time to progression to End-stage Liver Disease (ESLD)

  From the date of first odevixibat treatment (Odevixibat Cohort) or from the date of first registry entry into NAPPED group where OvEC cohort eligibility criteria were met (External control Cohort), until ESLD (2017-2025)

Study Arms (2)

Odevixibat Cohort

Participants who were treated with odevixibat from two odevixibat clinical trials (Studies A4250-005 and A4250-008) Eligibility criteria for Part A and Part B analyses are described in the Eligibility section.

External Control Cohort

Participants who were not treated with odevixibat or any other IBAT inhibitor from the NAtural course and Prognosis of PFIC and Effect of biliary Diversion (NAPPED) registry Eligibility criteria for Part A and Part B analyses are described in the Eligibility section.

Eligibility Criteria

• Age: 3 Months - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population source comprises patients of any age (paediatric and adults) with genetically confirmed PFIC who have either participated in odevixibat interventional trials (A4250-005 and A4250-008) or are included in the retrospective natural history registry (NAPPED).

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 A4250-008. * The patient must be IBAT inhibitor-naïve (has not received prior treatment with odevixibat, maralixibat, or other IBAT inhibitors). * Patient 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 underwent SBD (the date of surgery becomes the Day 1 visit for this cohort) but did not previously undergo a LT. * The patient meets the additional eligibility criteria. * Patients must be in the regions that participated in A4250-008.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Ipsen: lead
• Erasmus Medical Center: collaborator
• University Medical Center Groningen: collaborator

Study Sites (1)

Not applicable - retrospective secondary use of data

Paris, France

Location

MeSH Terms

Conditions

Cholestasis, progressive familial intrahepatic 1

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2026
• First Posted: March 27, 2026
• Study Start: April 26, 2026
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: May 28, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share
• Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
• Access Criteria: Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Locations

FR (1)