Official Title
Odevixibat (A4250) for the Treatment of Progressive Familial Intrahepatic Cholestasis (Expanded Access Program)
Brief Summary

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

Detailed Description

Eligible patients will be enrolled into this expanded-access program and treated with an
oral dose of 120 μg/kg/day of odevixibat and evaluated on an ongoing basis.

Approved for marketing
Treatment IND/Protocol
Progressive Familial Intrahepatic Cholestasis

Drug: Odevixibat

A4250 is a small molecule and selective inhibitor of IBAT
Other Name: A4250

Eligibility Criteria

Inclusion Criteria:

1. A male or female patient of any age, with a clinical diagnosis of PFIC, and with a
body weight ≥5 kg at Screening visit

2. Patient must have a clinical diagnosis of PFIC

3. Patient must have clinically confirmed pruritus

4. Patient must have elevated serum bile acid levels, specifically measured to be ≥2 ×
the upper limit of normal (ULN) prior to start of medication

5. 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

Exclusion Criteria:

1. Patient is expected to have a liver transplant within 6 months of Screening

2. Decompensated liver disease, coagulopathy, history or presence of clinically
significant ascites, variceal hemorrhage, and/or encephalopathy

3. 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)

4. Serum ALT >10 × ULN at Screening

5. Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate
etiology was confirmed for the elevation

6. Total bilirubin >10 × ULN at Screening

7. Any patient who is pregnant, lactating, or planning to get pregnant

8. Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to
support marketing approval in PFIC

Eligibility Gender
All
Eligibility Age
Minimum: N/A ~ Maximum: N/A
Countries
United States
Locations

Albireo Pharma Inc.
Boston, Massachusetts, United States

Ipsen Medical Director, Study Director
Ipsen

NCT Number
Keywords
PFIC
Pediatric
Cholestasis
MeSH Terms
Cholestasis
Cholestasis, Intrahepatic