Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
NCT06193928 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 70
Last updated 2025-05-25
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.
Conditions
- Alagille Syndrome
- Progressive Familial Intrahepatic Cholestasis
Interventions
- DRUG
-
Livmarli
The recommended dosage is 380 mcg/kg once daily.
- DRUG
-
Livmarli
The recommended dosage us 570 mcg/kg twice daily.
Sponsors & Collaborators
-
Mirum Pharmaceuticals, Inc.
lead INDUSTRY
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2023-09-21
- Primary Completion
- 2028-09-20
- Completion
- 2030-09-20
- FDA Drug
- Yes
Countries
- United States
Study Locations
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