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.

Phase 1

• Conditions: Biliary atresia; MedDRA version: 20.0Level: LLTClassification code 10004653Term: Biliary atresiaSystem Organ Class: 100000004850; MedDRA version: 20.1Level: LLTClassification code 10004654Term: Biliary atresia, congenitalSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-12-23
• Last Update Posted: 6 October 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 72

Inclusion Criteria

1. Male or female pediatric subjects, aged <18 years
2. Diagnosis of biliary atresia
3. Demonstrated 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.
4. Able to take medications orally (ie, tablet or mini-tablet formulations)
Are the trial subjects under 18? yes
Number of subjects for this age range: 72
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

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. Recent (within the previous 6 months prior to Screening) bleeding from gastroesophageal varices
e. Recent (within the previous 6 months prior to Screening) uncontrolled ascites (diuretic resistant)
f. Hepatic encephalopathy
g. Prior placement of portosystemic shunt
h. Diagnosis of hepatopulmonary syndrome or portopulmonary hypertension
3. Current intractable pruritus or those requiring systemic treatment for pruritus within 3 months of Screening (eg, with bile acid sequestrants or rifampicin)
4. Subjects aged <2 years
5. Subjects weighing <11 kg.

Study & Design

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