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Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

Who can join

All ages · All sexes

Inclusion Criteria:

* A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
* Participant prescribed Livmarli

Exclusion Criteria:

* 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

About the 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.

What is being tested

• Livmarli (drug)
• Livmarli (drug)

Sponsor: Mirum Pharmaceuticals, Inc. · Participants: 70 · Started: Sep 21, 2023

Contact the study team

• Clinical Trials Mirum · Phone: +16506674085

Official record on ClinicalTrials.gov — NCT06193928

Locations in the U.S.

Conditions

• Liver Diseases

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.