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Active, not recruitingNCT07497724OvEC-PFICUpdated Oct 1, 2026

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

An observational study in PFIC - Progressive Familial Intrahepatic Cholestasis, sponsored by Ipsen. Active, not recruiting at 1 site in France. Open to participants aged 3 Months to 100 Years. Per ClinicalTrials.gov, last updated 2026-10-01.

Sponsored by Ipsen · Observational

Updated Oct 1, 2026Primary completion movedStudy completion movedGo to Updates ↓
Study type
Observational
Model
Other
Time perspective
Retrospective
Enrollment
200
Ages
3 Months to 100 Years
Sex
All
01

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.

Read the detailed description

This study analysis consists of two parts: Part A will evaluate the effect of odevixibat on clinical outcomes in patients without prior SBD (i.e., SBD-naïve) who were treated with odevixibat versus SBD-naïve external controls who were not treated with odevixibat; Part B will evaluate the effect of odevixibat on clinical outcomes by comparing outcomes in patients without prior SBD who were treated with odevixibat versus external controls who underwent SBD and were not treated with odevixibat.

02

Conditions studied

  • PFIC - Progressive Familial Intrahepatic Cholestasis
03

In context

Lead sponsor

Ipsen is the lead sponsor of 282 studies on the registry; 16 are open to participants now.

Of its 24 completed or terminated interventional studies of FDA-regulated products, 19 (79%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Months to 100 Years
Sexes eligible
All
Accepts healthy volunteers
No
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).

Eligibility criteria

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.
05

Study design

Observational model
Other
Time perspective
Retrospective
Enrollment
200 participants (estimated)
Patient registry
No

Groups and cohorts

  • 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.

06

What researchers measure

Primary outcomes

  1. 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)

    Time frame: 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)

  2. Part B: Liver transplant-free survival (LTFS)

    As defined for Part A

    Time frame: 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

  1. Part A: Event-free survival (EFS)

    Defined as the time from Day 1 to the first occurrence of the following: LT; Death; Surgical Biliary Diversion (SBD, a type of surgery that reroutes the flow of bile so that less of it returns to the liver)

    Time frame: 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)

  2. Part A: Surgical biliary diversion-free survival (DFS)

    Defined as the time from Day 1 to the first occurrence of the following: SBD; Death (any cause)

    Time frame: 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)

  3. Part A: Overall survival (OS)

    Defined as time from Day 1 to death (any cause)

    Time frame: 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)

  4. Part B: Overall survival (OS)

    As defined for Part A

    Time frame: 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

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

    Defined as the time from Day 1 to the first occurrence of a platelet count below 150,000/mm³ at any time prior to LT

    Time frame: 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)

  6. Part B: Time to progression to End-stage Liver Disease (ESLD)

    As defined for Part A

    Time frame: From the date of first odevixibat treatment (Odevixibat Cohort) or from the from the date of SBD (External control cohort) until ESLD (2017-2025)

  7. Part A: Proportion of patients who died

    Time frame: 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)

  8. Part B: Proportion of patients who died

    Time frame: 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)]

  9. Part A: Proportion of patients who experience Liver Transplant (LT)

    Time frame: 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 LT (2017-2025)

  10. Part B: Proportion of patients who experience Liver Transplant (LT)

    Time frame: From the date of first odevixibat treatment (Odevixibat Cohort) or from the from the date of SBD (External control cohort) until LT (2017-2025)

  11. Part A: Proportion of patients who experience surgical biliary diversion (SBD)

    Time frame: 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 SBD (2017-2025)

07

Study locations

1 site
  • Not applicable - retrospective secondary use of data
    Paris, France
08

References and documents

Individual participant data

Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

No publications or documents are linked to this record.

09

Updates

1 registry update since Sep 25, 2026
Primary completion
Sep 21, 2026→Nov 30, 2026
Oct 1, 2026
Study completion
Sep 30, 2026→Nov 30, 2026
Oct 1, 2026
Show all 1 update
  1. Oct 1, 2026
    Primary completion Sep 21, 2026→Nov 30, 2026
    Study completion Sep 30, 2026→Nov 30, 2026
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07497724
Lead sponsor
Ipsen
Collaborators
Erasmus Medical Center, University Medical Center Groningen
Responsible party
Sponsor
First posted
Mar 27, 2026
Start date
Apr 26, 2026
Primary completion
Nov 30, 2026 (estimated)
Completion
Nov 30, 2026 (estimated)
Last update
Oct 1, 2026

Study contacts

Ipsen Medical Director
study director · Ipsen

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.

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