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Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

RECRUITINGSponsored by Mirum Pharmaceuticals, Inc.
Actively Recruiting
SponsorMirum Pharmaceuticals, Inc.
Started2023-09-21
Est. completion2028-09-20
Eligibility
Healthy vol.Accepted
Locations8 sites
View on ClinicalTrials.gov →

NCT06193928

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

Healthy volunteers accepted
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

Conditions4

Alagille SyndromeHeart DiseaseLiver DiseaseProgressive Familial Intrahepatic Cholestasis

Locations8 sites

Children's Hospital Los Angeles CHLA
Los Angeles, California, 90027
Chuan-Hao Lin, MDChLin@chla.usc.edu
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, 80045
Shikha S. SundaramShikha.Sundaram@childrenscolorado.org
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, 30322
Angela Stallworth404-785-3279Angela.Stallworth@choa.org
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouri, 64108
Ryan T. Fischerrtfischer@cmh.edu
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
Portland, Oregon, 97239
Henry C. Linlinhe@ohsu.edu

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