116 Participants Needed

Odevixibat for PFIC

Recruiting at 40 trial locations
AP
Overseen ByAlbireo Pharma
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: Albireo, an Ipsen Company
No Placebo GroupAll 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 DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

IM

Ipsen Medical Director

Principal Investigator

Ipsen

Are You a Good Fit for This Trial?

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.
See 9 more

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.
See 20 more

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: A4250Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Albireo, an Ipsen Company

Lead Sponsor

Trials
5
Recruited
640+

Albireo

Lead Sponsor

Trials
17
Recruited
1,200+
Unbiased ResultsWe believe in providing patients with all the options.
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