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NCT Number: NCT07497724

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

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.

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Key information

Conditions

PFIC - Progressive Familial Intrahepatic Cholestasis Cholestasis, progressive familial intrahepatic 1

Age range

3 month–100 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Not applicable - retrospective secondary use of data

Paris, France

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Part A: Liver transplant-free survival (LTFS)

    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)

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

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

    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)

    As defined for Part A

Secondary outcomes

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

    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)

    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)

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

    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)

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

  3. Part A: Overall survival (OS)

    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)

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

  4. Part B: Overall survival (OS)

    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

    As defined for Part A

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

    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)

    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

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

    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)

    As defined for Part A

  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)

Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Collaborators

  • Erasmus Medical Center
  • University Medical Center Groningen

Registry information

Official study title

Analysis of the Effect of Odevixibat (A4250) Versus External Controls on Clinical Outcomes in Patients With PFIC (Odevixibat Versus External Control [OvEC]-PFIC)

Acronym: OvEC-PFIC

Important dates

Study start
2026
Primary completion
2026
Study completion
2026
First posted
Mar 27, 2026
Registry last updated
Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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