Actively Recruiting
Long-Term Safety and Clinical Outcomes of Livmarli in Patients with Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the United States
Led by Mirum Pharmaceuticals, Inc. · Updated on 2025-05-25
70
Participants Needed
8
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) who are treated with Livmarli. The study is a 5-year, prospective, observational cohort trial designed to monitor these patients in the United States. Livmarli is a novel drug that reduces bile acid levels by inhibiting the ileal bile acid transporter and has been approved for treating cholestatic pruritus in ALGS and PFIC patients of certain ages. Participants prescribed Livmarli and diagnosed with ALGS or PFIC will be observed over five years. Two dosing schedules are noted: 380 mcg/kg once daily and 570 mcg/kg twice daily. The study includes regular assessments of liver transplant waitlist status every six months and annual evaluations of growth and development. The treatment is given as prescribed by healthcare providers, and the study collects data without altering the prescribed therapy. During the study, participants will have their long-term clinical outcomes monitored, including events up to 180 days after stopping Livmarli. Data on liver transplant indications, growth measurements like weight and height z-scores, and events related to fat-soluble vitamin deficiencies will be collected at specified intervals. The trial aims to provide comprehensive safety and outcome information over the full 5-year period.
CONDITIONS
Brief 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)
- Currently prescribed Livmarli
You will not qualify if you...
- Refusal to provide informed consent or assent if required by local regulations
- Currently or previously receiving Livmarli through a clinical study or expanded access program
- History of surgical biliary diversion or liver transplant
- Any condition that may interfere with participation or study completion as judged by the investigator
- Received an investigational drug within 30 days before starting Livmarli
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 5 years
Participants who are prescribed Livmarli are observed for clinical outcomes and health status over time.
Visits every 6 months for up to 5 years, with yearly assessments for growth, development, and clinical events
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
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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