Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
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.
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 int…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis * Participant prescribed Livmarli Exclusion Criteria: * Refusal to provide informed consent/assent (if required by the local IRB) * Previously or currently on Livmarli through participation in a clinical study or expanded access program * Participants who have previously received an SBD or LT * Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study * Participants who have received an investigational dru…
Interventions
- DrugLivmarli
The recommended dosage is 380 mcg/kg once daily.
- DrugLivmarli
The recommended dosage us 570 mcg/kg twice daily.
Locations (8)
- Children's Hospital Los Angeles CHLALos Angeles, California
- Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of ColoradoAurora, Colorado
- Children's Healthcare of Atlanta - Emory University School of MedicineAtlanta, Georgia
- Children's Mercy Kansas City, Department of Gastroenterology, Section of HepatologyKansas City, Missouri
- Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of PediatricsPortland, Oregon
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania