Obeticholic Acid in Pediatric Subjects With Biliary Atresia

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.

Timeline

Start

2015-07-01

Primary completion

2023-03-09

Completion

2023-03-09

Outcome

Outcome not reported

Stopped (Enrollment): “After extensive efforts to improve recruitment, it is deemed not feasible to enroll the requisite number of subjects to generate data needed to meet the study objectives. EMA Paediatric Committee agreed with the Sponsor to terminate this study.”

Drugs

Indications