Odevixibat for PFIC

Recruiting at 40 trial locations

Overseen By Albireo Pharma

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Albireo, an Ipsen Company

Must not be taking: Bile acid resins

Disqualifiers: Decompensated liver disease, Liver cancer, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 72 weeks

116 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing A4250, a medication, to see if it is safe and effective for children with a rare liver disease called PFIC. The goal is to improve liver function and reduce symptoms over time.

Research Team

Ipsen Medical Director

Principal Investigator

Ipsen

Eligibility Criteria

This trial is for children with PFIC, a liver disorder causing severe itching and jaundice. Participants need genetic confirmation of PFIC, have had significant pruritus, weigh at least 5 kg, and can use an eDiary. They must have completed or partially completed a prior A4250 study. Those with episodic PFIC should be experiencing a flare-up.

Inclusion Criteria

I have PFIC and have experienced significant itching, noted by myself or my caregiver.

I have PFIC with bile acid levels ≥100 μmol/L, confirmed by 2 tests.

I (or my guardian) have signed the consent form, and I will sign again if I turn 18 during the study.

Exclusion Criteria

Cohort 1: Patients not compliant with treatment in study A4250-005

Cohort 2: Total bilirubin >10 × ULN at Screening

I have a condition that affects how my body processes medication.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive A4250 capsules for oral administration once daily

72 weeks

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive A4250 to evaluate long-term safety and efficacy

Long-term

Treatment Details

Interventions

• A4250 (odevixibat)

Trial Overview The trial tests the long-term safety and effectiveness of A4250 (odevixibat) in kids with PFIC. It's an open-label extension study meaning all participants receive the drug and are monitored over time to see how well it works and what side effects occur.

Participant Groups

Group I: A4250 Experimental Treatment 1 Intervention

Capsules for oral administration (40 or 120 µg/kg) once daily for 72 weeks, or 40 µg/kg/day for the first 12 weeks followed by 120 µg/kg/day for the remaining 60 weeks"

Who Is Running the Clinical Trial?

Albireo, an Ipsen Company

Lead Sponsor

Trials

5

Recruited

640+

Albireo

Lead Sponsor

Trials

17

Recruited

1,200+