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

Recruiting now

Conditions studied: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis

In brief

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 facts

Study ID

NCT06193928

Run by

Mirum Pharmaceuticals, Inc.

People needed

70

Starts

2023-09-21

Expected to finish

2030-09-20

Last updated by the study team

2025-05-25

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
• Participant prescribed Livmarli

You may not qualify if…

• 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 drug within 30 days of the first dose of Livmarli

Where it is running

• Children's Hospital Los Angeles CHLA — Los Angeles, California, United States (enrolling)
• 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 (enrolling)
• Children's Healthcare of Atlanta - Emory University School of Medicine — Atlanta, Georgia, United States (enrolling)
• Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology — Kansas City, Missouri, United States (enrolling)
• Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics — Portland, Oregon, United States (enrolling)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Children Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
• University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition — Salt Lake City, Utah, United States (enrolling)

Full record on ClinicalTrials.gov

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