RecruitingNCT06193928
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Sponsor
Mirum Pharmaceuticals, Inc.
Enrollment
70 participants
Start Date
Sep 21, 2023
Study Type
OBSERVATIONAL
Conditions
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
Inclusion Criteria2
- A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
- Participant prescribed Livmarli
Exclusion Criteria5
- 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
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Interventions
DRUGLivmarli
The recommended dosage is 380 mcg/kg once daily.
DRUGLivmarli
The recommended dosage us 570 mcg/kg twice daily.
Locations(8)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06193928
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