Assessing Obeticholic Acid for Pediatric 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
This study is testing if Obeticholic Acid can safely help children with biliary atresia feel better after their surgery.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 144 (estimated) |
| Ages | 1 Day to 18 Years |
| Sex | All |
| Sponsor | Intercept Pharmaceuticals Industry-sponsored |
| Locations | 9 sites (South Brisbane, Queensland and 8 other locations) |
| Trial ID | NCT06121375 on ClinicalTrials.gov |
What this trial studies
This study evaluates the efficacy, safety, and tolerability of Obeticholic Acid (OCA) in pediatric patients diagnosed with biliary atresia who have undergone successful hepatoportoenterostomy. It includes a double-blind design with two phases: a dose titration phase and an age expansion treatment phase. The study aims to gather pharmacokinetic and pharmacodynamic data to better understand how OCA affects this patient population.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients from birth to under 18 years old with non-syndromic biliary atresia and successful hepatoportoenterostomy.
Not a fit: Patients with prior liver transplants, active transplant status, or specific complications related to biliary atresia may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve liver function and overall health outcomes for children with biliary atresia.
How similar studies have performed: Other studies have explored treatments for biliary atresia, but the specific use of OCA in this context is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion 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: 1. Gastroesophageal varices and/or variceal bleeding 2. Clinically evident ascites related to portal hypertension 3. Hepatic encephalopathy 4. Prior placement of portosystemic shunt 5. Hepatopulmonary syndrome or portopulmonary hypertension 6. Hepatorenal syndrome 7. Any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.) 8. Hepatocellular carcinoma 9. 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).
Where this trial is running
South Brisbane, Queensland and 8 other locations
- Queensland Childrens Hospital — South Brisbane, Queensland, Australia (Recruiting)
- Women's and Children's Hospital — North Adelaide, South Australia, Australia (Recruiting)
- Royal Childrens Hospital — Parkville, Victoria, Australia (Recruiting)
- Stollery Children's Hospital — Edmonton, Alberta, Canada (Recruiting)
- Queen Mary Hospital — Hong Kong, Hong Kong (Recruiting)
- Hospital Raja Perempuan Azinab II — Kota Bharu, Kelantan, Malaysia (Recruiting)
- University Malaya Medical Center — Kuala Lumpur, Malaysia (Recruiting)
- Starship Child Health — Auckland, New Zealand (Recruiting)
- KK Women's and Children's Hospital — Singapore, Singapore (Recruiting)
Study contacts
- Study coordinator: Scott Birnbaum
- Email: scott.birnbaum@interceptpharma.com
- Phone: +1 (646) 757-2331
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.