APPROVED_FOR_MARKETINGNot Applicable

Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

United States

Plain-language 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 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

Who can participate

SexALL

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Inclusion criteria

✓. 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 criteria

✕. 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

Trial details

NCT IDNCT04483531

SponsorAlbireo

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Progressive Familial Intrahepatic Cholestasis trials