Overview

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

Status:
Completed

Trial end date:
2020-06-01

Target enrollment:

Participant gender:

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.

Phase:

Phase 2

Details

Lead Sponsor:

Mirum Pharmaceuticals, Inc.
Shire

Collaborators:

Childhood Liver Disease Research and Education Network
Lumena Pharmaceuticals, Inc.