Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
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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
Condition or disease
Progressive Familial Intrahepatic Cholestasis
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.
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Layout table for eligibility information
Ages Eligible for Study:
Child, Adult, Older Adult
Sexes Eligible for Study:
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
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