RECRUITING

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

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Conditions

Alagille SyndromeProgressive Familial Intrahepatic CholestasisALGSLiver DiseasesPFICLivmarli

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2023-09-21
Study Completion:2030-09-20
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06193928

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
  2. * Participant prescribed Livmarli
  1. * Refusal to provide informed consent/assent (if required by the local IRB)
  2. * Previously or currently on Livmarli through participation in a clinical study or expanded access program
  3. * Participants who have previously received an SBD or LT
  4. * Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study
  5. * Participants who have received an investigational drug within 30 days of the first dose of Livmarli

Contacts and Locations

Study Contact

Clinical Trials Mirum
CONTACT
+16506674085
Clinical Trials <clinicaltrials@mirumpharma.com>;

Study Locations (Sites)

Children's Hospital Los Angeles CHLA
Los Angeles, California, 90027
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, 80045
United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, 30322
United States
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouri, 64108
United States
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
Portland, Oregon, 97239
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15260
United States
University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
Salt Lake City, Utah, 84112
United States

Collaborators and Investigators

Sponsor: Mirum Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-09-21
Study Completion Date2030-09-20

Study Record Updates

Study Start Date2023-09-21
Study Completion Date2030-09-20

Terms related to this study

Keywords Provided by Researchers

  • ALGS
  • Liver Diseases
  • PFIC
  • Livmarli
  • Alagille Syndrome
  • Progressive Familial Intrahepatic Cholestasis

Additional Relevant MeSH Terms

  • Alagille Syndrome
  • Progressive Familial Intrahepatic Cholestasis