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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

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT06193928). StuddyBuddy aggregates publicly available trial information.