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Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Sponsor: Mirum Pharmaceuticals, Inc.
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.
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
70
Start Date
2023-09-21
Completion Date
2030-09-20
Last Updated
2025-05-25
Healthy Volunteers
No
Interventions
Livmarli
The recommended dosage is 380 mcg/kg once daily.
Livmarli
The recommended dosage us 570 mcg/kg twice daily.
Locations (8)
Children's Hospital Los Angeles CHLA
Los Angeles, California, United States
Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado
Aurora, Colorado, United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouri, United States
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
Salt Lake City, Utah, United States