RecruitingAlagille SyndromeProgressive Familial Intrahepatic Cholestasis
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Eligible age
All ages
Accepts
All genders
Locations
7 states
Healthy volunteers
No
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About this study
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.
Sponsor: Mirum Pharmaceuticals, Inc.
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's recruiting
California
Los Angeles
Colorado
Aurora
Georgia
Atlanta
Missouri
Kansas City
Oregon
Portland
Pennsylvania
Philadelphia · Pittsburgh
Utah
Salt Lake City
Source: ClinicalTrials.gov · NCT06193928 · last updated 2025-05-25