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: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Mirum Pharmaceuticals, Inc.; Shire
• Collaborator: Lumena Pharmaceuticals, Inc.

Criteria

Participation for an individual patient is expected to be approximately 72 weeks.

Patients who complete 72 weeks of treatment may be eligible to receive treatment for up to 52 weeks during the follow-up treatment period. Planned participation for each patient enrolled in the optional follow-up treatment study is 128 weeks, inclusive of a 4-week follow-up phone call.