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Recruiting
NCT06193928
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Conditions: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Sex: All
Healthy volunteers: No
Enrollment: 70
Sponsor: Mirum Pharmaceuticals, Inc.
Location: Children's Hospital Los Angeles CHLA Los Angeles California
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 intrahepatic cholestasis (PFIC) treated with Livmarli.
Eligibility Criteria
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
Source: ClinicalTrials.gov (NCT06193928). StuddyBuddy aggregates publicly available trial information.