A Multicenter, Randomized, Open Label, Single- and Multiple-Dose, Dose Finding Study and Open-Label Extension to Assess the Effects of Obeticholic Acid in Pediatric Subjects with Biliary Atresia

Intercept Pharmaceuticals, Inc.0 个研究点目标入组 60 人开始时间: 2015年9月9日最近更新: 2016年1月18日

试验速览

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•1.Male or female pediatric subjects, aged <18 years
•2.Diagnosis of biliary atresia, with successful HPE (also known as Kasai portoenterostomy) as defined by total bilirubin <2 mg/dL (34.2 µmol/L) at least 3 months post-HPE procedure.
•3.Gamma-glutamyl transferase (GGT) =2x ULN
•4.Able to take medications orally (ie, prior to the availability of the suspension formulation, able to swallow tablet(s) whole)
•5.Prior to the availability of the suspension formulation, this study will initially restrict subject enrollment by weight into the 3 OCA dose groups as follows (ie, subjects must meet these restrictions at Screening and Day 0):
•- Low Dose Group: =42 kg
•- Medium Dose Group: =20 kg
•- High Dose Group: =21 kg
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 60
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Subjects with or experiencing:
•1. Prior liver transplant or active status on transplant list
•2.Complications of decompensated cirrhosis including:
•a. Total bilirubin =2 mg/dL (34.2 µmol/L)
•b. Platelets <40,000/µL
•d. Presence of known or large esophageal varices
•e. Uncontrolled ascites (diuretic resistant)
•f. Prior placement of portosystemic shunt
•g. Diagnosis of hepatopulmonary syndrome or portopulmonary hypertension
•3.Current intractable pruritus or those requiring systemic treatment for pruritus within 3 months (eg, with bile acid sequestrants or rifampicin)

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