NCT05321524

Obeticholic Acid in Pediatric Subjects With Biliary Atresia

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Intercept Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, multicenter, open-label, single dose and multi-dose, dose-finding study with an optional open-label extension (OLE) to assess the safety, tolerability, and pharmacokinetics of obeticholic acid (OCA) in pediatric subjects with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterosomy). The OLE will continue to evaluate safety, tolerability, pharmacodynamics, and efficacy of OCA. In addition, a change in vitamin A and D levels, and where possible the degree of change in liver stiffness, will be assessed during the OLE.

Conditions

• Biliary Atresia

Interventions

Type	Name	Description
DRUG	OCA 0.1mg	Tablets administered orally once daily.
DRUG	OCA 1.5mg	Tablets administered orally once daily.
DRUG	OCA 5mg	Tablets administered orally once daily.

Timeline

• Start date: 2015-07-01
• Primary completion: 2023-03-09
• Completion: 2023-03-09
• First posted: 2022-04-11
• Last updated: 2023-03-28

Locations

17 sites across 9 countries: Belgium, France, Germany, Israel, Italy, Netherlands, Poland, Spain, United Kingdom