NCT06121375

Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy

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

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: Intercept Pharmaceuticals · Industry
• Sex: All
• Age: 1 Day – 18 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the efficacy, safety and tolerability, as well as PK/PD of OCA in eligible pediatric participants with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterostomy). The double-blind period comprises of 2 phases: dose titration phase and age expansion treatment phase.

Conditions

• Biliary Atresia

Interventions

Type	Name	Description
DRUG	OCA	OCA will be administered.
DRUG	Matching Placebo	Matching Placebo will be administered.

Timeline

• Start date: 2024-09-02
• Primary completion: 2025-10-21
• Completion: 2025-10-21
• First posted: 2023-11-07
• Last updated: 2025-10-31

Locations

24 sites across 10 countries: Australia, Canada, China, Hong Kong, Israel, Malaysia, New Zealand, Singapore, Taiwan, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study