An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

Phase 2 Completed 34 enrolled Mirum Pharmaceuticals, Inc. Childhood Liver Disease Research and Education Network · collab Lumena Pharmaceuticals, Inc. · collab

NaSingle-groupDouble-blindTreatment

Summary

This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.

Timeline

Start: 2015-03-16
Primary completion: 2020-06-01
Completion: 2020-06-01

Publications

Hoskins BJ, Mogul DB, Chen C, Karnsakul W. Maralixibat Improves Xanthomas and Hypercholesterolemia in Children with Alagille Syndrome: A Post Hoc Integrated Analysis From Two Clinical Trials. J Pediatr. 2026 Jul;294:115108. doi: 10.1016/j.jpeds.2026.115108. Epub 2026 Apr 17.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Maralixibat	Small molecule	280 ug/kg	Oral

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

Summary

Timeline

Publications

Drugs

Indications