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PFIC - Progressive Familial Intrahepatic CholestasisOvEC-PFICNCT07497724

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

An observational study of PFIC - Progressive Familial Intrahepatic Cholestasis, run by Ipsen at 1 site in France.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 3 months to 100 years
How many people
200 participants
Where
1 siteFrance
Study dates
26 Apr 2026to 30 Nov 2026
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 months to 100 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

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.

Trial locations and contacts

1 site in 1 country.

France1 site

  • Not applicable - retrospective secondary use of data

    Paris

Other trials in France

About this study

Official title: Analysis of the Effect of Odevixibat (A4250) Versus External Controls on Clinical Outcomes in Patients With PFIC (Odevixibat Versus External Control [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.

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.

Medical subject headings
Cholestasis, progressive familial intrahepatic 1

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT07497724
Study groupRoleWhat they receive
Odevixibat CohortParticipants 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 CohortParticipants 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.

What is being measured

The primary outcome is the main question the study is designed to answer.

  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)

    Measured: 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

    Measured: 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)

  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)

    Measured: 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)

    Measured: 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)

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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)

    Measured: 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)

    Measured: 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)

    Measured: 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)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Mar 2026
  2. Study start26 Apr 2026
  3. Primary completion30 Nov 2026 (estimated)
  4. Study completion30 Nov 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Other
Time perspective
Retrospective
Enrolment
200 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07497724
Sponsor's protocol ID
CLIN-60240-458
Acronym
OvEC-PFIC
Status last verified
Oct 2026
First submitted
23 Mar 2026
Last updated
23 Oct 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
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.

Questions about this trial

What is this clinical trial studying?

An observational study of PFIC - Progressive Familial Intrahepatic Cholestasis, run by Ipsen at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07497724.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2026.

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