Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
NCT ID: NCT06193928
Last Updated: 2025-05-25
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
70 participants
OBSERVATIONAL
2023-09-21
2030-09-20
Brief Summary
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Detailed Description
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* A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
* Prescribed Livmarli
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Alagille syndrome (ALGS)
* A clinically and/or genetically confirmed ALGS diagnosis
* Participant prescribed Livmarli
Livmarli
The recommended dosage is 380 mcg/kg once daily.
Progressive familial intrahepatic cholestasis (PFIC)
* A clinically and/or genetically confirmed PFIC diagnosis
* Prescribed Livmarli
Livmarli
The recommended dosage us 570 mcg/kg twice daily.
Interventions
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Livmarli
The recommended dosage is 380 mcg/kg once daily.
Livmarli
The recommended dosage us 570 mcg/kg twice daily.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participant prescribed Livmarli
Exclusion Criteria
* 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
ALL
No
Sponsors
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Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Children's Hospital Los Angeles CHLA
Los Angeles, California, United States
Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado
Aurora, Colorado, United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouri, United States
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
Salt Lake City, Utah, United States
Countries
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Central Contacts
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Facility Contacts
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George Yanni
Role: backup
James E. Squires
Role: backup
Related Links
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Mirum Pharmaceuticals homepage
Genetics Home Reference - ALGS
US FDA Resources
Other Identifiers
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MRX-310
Identifier Type: -
Identifier Source: org_study_id
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