A Multicenter, Open-Label, Single- and Multiple-Dose, Dose-Finding Study, with an Optional Open-Label Extension to Assess the Safety, Tolerability, and Pharmacokinetics of Obeticholic Acid in Pediatric Subjects with Biliary Atresia.;Trial Acronym: ObetiCholic Acid in Pediatric Subjects with BiliaRy AtrEsia (CARE)
- Conditions
- Biliary atresia1001980610004606
- Registration Number
- NL-OMON53035
- Lead Sponsor
- Intercept Pharmaceuticals Inc.
- Brief Summary
Trial never started
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 5
1. Male or female pediatric subjects >=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 (i.e., tablet or mini-tablet formulations)
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 availble: 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 requires systemic treatment for pruritus
within 3 months of Screening (e.g., 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. Gamma-glutamyl transferase (GGT) >500 U/L
13. Anticoagulation therapy
14. Albumin <3.5 g/dL
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>- Safety and tolerability: Treatment-emergent AEs (TEAEs) including serious AEs<br /><br>(SAEs), electrocardiogram (ECG), physical exam, clinical laboratory results,<br /><br>vital signs.<br /><br><br /><br>- PK: Plasma concentrations of unconjugated OCA, its conjugates (glycine<br /><br>conjugate of OCA [glyco-OCA] and taurine conjugate of OCA [tauro-OCA]), and<br /><br>total OCA in the SD and MD Phases</p><br>
- Secondary Outcome Measures
Name Time Method