Status:
RECRUITING
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Lead Sponsor:
Mirum Pharmaceuticals, Inc.
Conditions:
Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
Eligibility:
All Genders
Brief 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 intrah...
Detailed 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 (maralix...
Eligibility Criteria
Inclusion
- A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
- Participant prescribed Livmarli
Exclusion
- 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
Key Trial Info
Start Date :
September 21 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 20 2030
Estimated Enrollment :
70 Patients enrolled
Trial Details
Trial ID
NCT06193928
Start Date
September 21 2023
End Date
September 20 2030
Last Update
May 25 2025
Active Locations (8)
Enter a location and click search to find clinical trials sorted by distance.
1
Children's Hospital Los Angeles CHLA
Los Angeles, California, United States, 90027
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
3
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States, 30322
4
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouri, United States, 64108