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

Progressive Familial Intrahepatic Cholestasis Alagille Syndrome

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

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Livmarli and a drug called Livmarli for people with alagille syndrome and progressive familial intrahepatic cholestasis. The study is currently recruiting participants at 8 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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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)

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

View Full Details on ClinicalTrials.gov

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NCT06193928

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