Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

• Status: Completed
• Trial end date: 2016-11-16
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Mirum Pharmaceuticals, Inc.; Shire
• Collaborator: Childhood Liver Disease Research and Education Network

Criteria

Inclusion Criteria:

1. Diagnosis of Alagille Syndrome

2. Evidence of cholestasis

3. Moderate to severe pruritus

4. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures

Exclusion Criteria:

1. Surgical disruption of the enterohepatic circulation

2. Liver transplant

3. History or presence of other concomitant liver disease

4. Females who are pregnant or lactating

5. Known HIV infection