Actively Recruiting
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Led by Mirum Pharmaceuticals, Inc. · Updated on 2025-05-25
70
Participants Needed
8
Research Sites
365 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinically and/or genetically confirmed diagnosis of Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)
- Participant has been prescribed Livmarli
You will not qualify if you...
- Refusal to provide informed consent or assent, if required by local regulations
- Current or previous participation in a Livmarli clinical study or expanded access program
- Participants who have previously received surgical biliary diversion or liver transplant
- Any condition or abnormality that may interfere with participation or completion of the study, as judged by the investigator
- Use of any investigational drug within 30 days prior to the first dose of Livmarli
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
Children's Hospital Los Angeles CHLA
Los Angeles, California, United States, 90027
Actively Recruiting
2
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, 80045
Actively Recruiting
3
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouri, United States, 64108
Actively Recruiting
5
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
Portland, Oregon, United States, 97239
Actively Recruiting
6
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
7
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15260
Actively Recruiting
8
University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
Salt Lake City, Utah, United States, 84112
Actively Recruiting
Research Team
C
Clinical Trials Mirum
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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