Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorMirum Pharmaceuticals, Inc.
About this trial
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.
Eligibility criteria
Qualifiers
A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
Participant prescribed Livmarli
Disqualifiers
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
Trial design
Treatments tested in this trial
- Livmarli
- Livmarli
Treatment groups
70 Participants
are divided into 2 treatment groupsLocations
8California
Children's Hospital Los Angeles CHLA90027, Los Angeles United States
Colorado
Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado80045, Aurora United States
Georgia
Children's Healthcare of Atlanta - Emory University School of Medicine30322, Atlanta United States
Missouri
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology64108, Kansas City United States
Sponsors and collaborators
Mirum Pharmaceuticals, Inc.
Lead sponsor
Source ClinicalTrials.gov