Overview

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

Status:
Completed

Trial end date:
2020-06-17

Target enrollment:

Participant gender:

Summary

The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001) in children with ALGS who have completed participation in a core LUM001 treatment protocol. Efficacy will be assessed by evaluating the effect of LUM001 on pruritus, biochemical markers of pruritus, as well as biochemical markers of cholestasis and liver disease.

Phase:

Phase 2

Details

Lead Sponsor:

Mirum Pharmaceuticals, Inc.
Shire

Collaborator:

Lumena Pharmaceuticals, Inc.