Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

NCT02057692 ↗

Phase 2 Completed 37 enrolled Mirum Pharmaceuticals, Inc. Childhood Liver Disease Research and Education Network · collab

RandomizedParallel-groupTriple-blindTreatment

Summary

The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.

Timeline

Start: 2014-11-24
Primary completion: 2016-11-16
Completion: 2016-11-16

Outcome

Met primary endpoint

registry analysis (superiority test); Maralixibat 140 mcg/kg/Day vs Placebo; p = 0.0145; LS mean difference -0.906 (95% CI -1.620 to -0.192); ANCOVA NCT02057692 ↗

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	—	Oral