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.
- Conditions
- Biliary atresiaMedDRA version: 20.0Level: LLTClassification code 10004653Term: Biliary atresiaSystem Organ Class: 100000004850MedDRA version: 20.1Level: LLTClassification code 10004654Term: Biliary atresia, congenitalSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
- Registration Number
- EUCTR2014-004693-42-BE
- Lead Sponsor
- Intercept Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 8
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: 32
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
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 WHO criteria
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
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method