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 including an optional long-term extension.

Phase 1

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

• Registration Number: EUCTR2014-004693-42-IT
• Lead Sponsor: INTERCEPT PHARMACEUTICALS INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-09-14
• Last Update Posted: 21 August 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

1. Male or female pediatric subjects, aged =2 to <18 years old
2. Diagnosis of biliary atresia
3. Demonstrated successful HPE (also known as a Kasai
portoenterostomy) as defined by total bilirubin <2 mg/dL (34.2 µmol/L)
at least 3 months post-HPE procedure.
4. Able to swallow tablets (ie, tablet or mini-tablet formulation)
Please refer to the protocol for the entire list of inclusion criteria.
Are the trial subjects under 18? yes
Number of subjects for this age range:
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.Conjugated (direct) bilirubin =ULN of site specific reference range
3.If conjugated bilirubin is not available: total bilirubin =2 mg/dL (34.2
µmol/L)
4.Platelets <150,000/µL
5.INR =1.5
6.Current or history of complications of decompensated chronic liver
disease including:
a)High-risk gastroesophageal varices and/or varcieal bleeding
b)Clinically evident ascites related to portal hypertension
c)Hepatic encephalopathy
d)Prior placement of portosystemic shunt
e)Hepatopulmonary syndrome or portopulmonary hypertension
f)Hepatorenal syndrome
7.Current intractable pruritus or those requires systemic treatment for
pruritus within 3 months of Screening (eg, with bile acid sequestrants or
rifampicin)
8.Height and weight Z-score <-2 per site specific reference ranges
9.Acholic (pale) stools
10.AST >4x ULN
11.ALT > 4X ULN
12.GGT > 500 U/L
13.Anticoagulation therapy
14.Albumin < 3.5 g/Dl
15.Ongoing current cholangitis
16.Choledochal cystic disease
17.Renal disease defined as serum creatinine >ULN for subject's age,
prior to enrollment

Please refer to the protocol for the entire list of exclusion criteria.

Study & Design

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