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

Description

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

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Alagille Syndrome
Intervention / Treatment

-

Contacts and Locations

Los Angeles

Children's Hospital Los Angeles CHLA, Los Angeles, California, United States, 90027

Aurora

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, 80045

Atlanta

Children's Healthcare of Atlanta - Emory University School of Medicine, Atlanta, Georgia, United States, 30322

Kansas City

Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology, Kansas City, Missouri, United States, 64108

Portland

Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics, Portland, Oregon, United States, 97239

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Pittsburgh

Children Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States, 15260

Salt Lake City

University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Salt Lake City, Utah, United States, 84112

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
  • * Participant prescribed Livmarli
  • * 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

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Mirum Pharmaceuticals, Inc.,

Study Record Dates

2030-09-20