Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

NO_LONGER_AVAILABLE

Conditions studied: Progressive Familial Intrahepatic Cholestasis

In brief

To provide treatment access to patients with PFIC in the US who have pruritus and elevated serum bile acids and who are not able to enroll in A4250-008 (PEDFIC2) for the following reasons: 1) Do not meet eligibility criteria for PEDFIC 2; 2) Are not able to get to a PEDFIC 2 site for geographical reasons, and 3) Do meet the eligibility criteria for PEDFIC 2 after recruitment has been completed

Key facts

Study ID: NCT04483531
Run by: Albireo
Last updated by the study team: 2026-07-23

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

A male or female patient of any age, with a clinical diagnosis of PFIC, and with a body weight ≥5 kg at Screening visit
Patient must have a clinical diagnosis of PFIC
Patient must have clinically confirmed pruritus
Patient must have elevated serum bile acid levels, specifically measured to be ≥2 × the upper limit of normal (ULN) prior to start of medication
Patient and/or legal guardian must sign informed consent (and assent) as appropriate. Patients who turn 18 years of age (or legal age per country) during

You may not qualify if…

Patient is expected to have a liver transplant within 6 months of Screening
Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
International normalized ratio (INR) >1.4 (the patient may be treated with Vitamin K intravenously, and if INR is ≤1.4 at resampling the patient may be started on program medication)
Serum ALT >10 × ULN at Screening
Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
Total bilirubin >10 × ULN at Screening
Any patient who is pregnant, lactating, or planning to get pregnant
Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to support marketing approval in PFIC

Where it is running

Albireo Pharma Inc. — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

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