Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

Status:

APPROVED_FOR_MARKETING

Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

Lead Sponsor:

Albireo

Conditions:

Progressive Familial Intrahepatic Cholestasis

Eligibility:

All Genders

Brief Summary

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 m...

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.

Eligibility Criteria

Inclusion

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

Exclusion

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

Key Trial Info

Start Date :

Trial Type :

EXPANDED_ACCESS

End Date :

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT04483531

Last Update

March 7 2024

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