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Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP) — LEAP  

Alagille Syndrome Clinical Trial

Official title:
Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)

Clinical Trial Summary

The goal of this observational study is to evaluate the long-term safety and clinical outcomes of Livmarli prescribed to patients with Alagille Syndrome (ALGS).

Clinical Trial Description

Livmarli® is a novel, minimally absorbed, pharmacological treatment for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS), a rare, genetic, autosomal dominant, complex multisystem disorder characterized by the presence of cholestasis (typically presenting as jaundice in the neonatal period) due to intrahepatic bile duct paucity. Livmarli inhibits the ileal bile acid transporter (IBAT) in the terminal ileum, leading to reduced levels of bile acids, thereby improving clinical manifestations and symptoms of cholestasis. Livmarli has been developed by Mirum Pharmaceuticals and approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients with ALGS who are 3 months of age and older. To be eligible for the study, participants must meet the following criteria: - A clinically and/or genetically confirmed ALGS diagnosis - Participant prescribed Livmarli ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06193928
Study type Observational [Patient Registry]
Source Mirum Pharmaceuticals, Inc.
Contact Clinical Trials Mirum
Phone +16506674085
Email Clinical Trials <clinicaltrials@mirumpharma.com>;
Status Recruiting
Phase
Start date September 21, 2023
Completion date September 20, 2028
View Details
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