Clinical Trials List
2024-06-01 - 2025-09-17
Phase II/III
Not yet recruiting2
Recruiting2
ICD-10Q44.2
Atresia of bile ducts
ICD-10Q44.3
Congenital stenosis and stricture of bile ducts
ICD-9751.61
Biliary atresia
A Randomized, Double-blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Obeticholic Acid Compared to Placebo in Pediatric Subjects With Biliary Atresia, Post-hepatoportoenterostomy
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Trial Applicant
Syneos Health
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Sponsor
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Trial scale
Multi-Regional Multi-Center
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Update
2026/06/18
Investigators and Locations
Co-Principal Investigator
- SHENG-YANG HUANG Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- 吳嘉峯 Division of Pediatrics
- 戴季珊 Division of Pediatrics
- YEN-HSUAN NI Division of Pediatrics
- 張凱琪 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- 陳建彰 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- 沈靜芬 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Objectives
Test Drug
Active Ingredient
Dosage Form
Dosage
1.5 mg/tab
Endpoints
• Death (all causes)
• Liver transplantation
• Pediatric end-stage liver disease (PELD) score >= 17 / Model for end-stage liver disease (MELD) >= 15
• Hospitalization for any of the following new or recurrent conditions (defined as hospitalization for 24 hours or more):
- Variceal bleeding
- Hepatic encephalopathy (defined as West Haven score >= 2)
- Spontaneous bacterial peritonitis (confirmed by diagnostic endovascular aspiration)
• Clinically significant ascites associated with portal hypertension (diuretic-resistant ascites requiring at least two therapeutic endovascular aspirations per month)
Inclution Criteria
Male or female pediatric participants from birth to <18 years old. Note: Participants aged <2 years old will not be enrolled until after review of safety data during the planned interim analysis and agreement from the Data Safety Monitoring Board (DSMB) that there is sufficient safety data to enroll this age group.
Diagnosis of non-syndromic biliary atresia.
Demonstrated successful HPE as defined by total bilirubin <2 milligrams per deciliter (mg/dL) (34.2 micromoles per liter [μmol/L]) at least 3 months post-HPE procedure.
Exclusion Criteria
Prior liver transplant or active status on transplant list.
Participants diagnosed with biliary atresia splenic malformation (BASM).
Conjugated (direct) bilirubin ≥ upper limit of normal (ULN) of site-specific reference range. If conjugated bilirubin is not available: total bilirubin ≥2 mg/dL (34.2 mol/L).
Platelets <120,000/μL
International normalized ratio (INR) ≥1.5.
Current or history of complications of decompensated chronic liver disease including:
Gastroesophageal varices and/or variceal bleeding
Clinically evident ascites related to portal hypertension
Hepatic encephalopathy
Prior placement of portosystemic shunt
Hepatopulmonary syndrome or portopulmonary hypertension
Hepatorenal syndrome
Any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.)
Hepatocellular carcinoma
Childs-Pugh B or C
Height and weight Z-score <-2 per site-specific reference ranges.
Acholic (pale) stools.
Aspartate aminotransferase (AST) >4x ULN.
Alanine aminotransferase >4x ULN
GGT >500 Units per Liter (U/L)
On anticoagulation therapy
Albumin <3.5 grams per deciliter (g/dL).
Inability to swallow tablets (i.e., tablet or mini-tablet formulations).
The Estimated Number of Participants
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Taiwan
8 participants
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Global
144 participants
