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