A study to look at how the study drug Obeticholic Acid (OCA), when given as a single dose and multiple doses, is tolerated in children who have biliary atresia and to look at the effects that the study drug has on the body.

• Conditions: Biliary atresia; MedDRA version: 18.0Level: LLTClassification code 10004653Term: Biliary atresiaSystem Organ Class: 100000004850; MedDRA version: 18.0Level: LLTClassification code 10004654Term: Biliary atresia, congenitalSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

• Registration Number: EUCTR2014-004693-42-NL
• Lead Sponsor: Intercept Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-09-09
• Last Update Posted: 18 January 2016

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

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

Exclusion Criteria

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
c. INR =2
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)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified