NCT06193928

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 70 (estimated)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.

Detailed description

Livmarli® is a novel, minimally absorbed, pharmacological product that inhibits the ileal bile acid transporter (IBAT) in the terminal ileum, leading to reduced levels of bile acids. Livmarli (maralixibat) has been developed by Mirum Pharmaceuticals and was the first treatment approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients 3 months of age and older with Alagille syndrome (ALGS). Subsequently, Livmarli was approved by the FDA for the treatment of cholestatic pruritus in patients 12 months of age and older with Progressive familial intrahepatic cholestasis (PFIC). To be eligible for the study, participants must meet the following criteria: * A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis * Prescribed Livmarli

Conditions

• Alagille Syndrome
• Progressive Familial Intrahepatic Cholestasis

Interventions

Type	Name	Description
DRUG	Livmarli	The recommended dosage is 380 mcg/kg once daily.
DRUG	Livmarli	The recommended dosage us 570 mcg/kg twice daily.

Timeline

• Start date: 2023-09-21
• Primary completion: 2028-09-20
• Completion: 2030-09-20
• First posted: 2024-01-05
• Last updated: 2025-05-25

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study