NCT04524390

Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai

Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Biliary Atresia After Hepatoportoenterostomy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 21 Days – 111 Days
• Healthy volunteers: Not accepted

Summary

A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).

Detailed description

This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will receive maralixibat to Week 104.

Conditions

• Biliary Atresia

Interventions

Type	Name	Description
DRUG	Maralixibat	A small molecule inhibitor of the ileal bile acid transporter (IBAT)
OTHER	Placebo	Identical to maralixibat except for the active drug substance

Timeline

• Start date: 2021-07-08
• Primary completion: 2023-11-07
• Completion: 2024-02-07
• First posted: 2020-08-24
• Last updated: 2025-03-19
• Results posted: 2025-02-17

Locations

22 sites across 8 countries: United States, China, Germany, Poland, Singapore, Taiwan, United Kingdom, Vietnam

Regulatory

• FDA-regulated drug study