Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
LEAP
1 other identifier
observational
70
1 country
8
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Sep 2023
Longer than P75 for all trials
8 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 21, 2023
CompletedFirst Submitted
Initial submission to the registry
December 11, 2023
CompletedFirst Posted
Study publicly available on registry
January 5, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 20, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 20, 2030
May 25, 2025
May 1, 2025
5 years
December 11, 2023
May 21, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
Incidence of Long-Term Clinical Outcomes
The dates, and reasons for the following first events (of this first endpoint) will be collected: Surgical Biliary Diversion (SBD), Liver Transplant (LT), and all-cause mortality. In addition, manifestations of clinically evident portal hypertension (CEPH) will be captured during each interval event assessments.
Long-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded.
Liver Transplant Indication and Waitlist Status
LT waitlist status will be collected, including when placed on or removed from LT waitlist.
LT waitlist status will be collected at enrollment and every 6 months for 5 years.
Assessment of Growth and Development
Height and weight will be collected both at the time the participant started Livmarli and at the time of enrollment in the study. Subsequent weight will be collected for up to 5 years. Weight z-score (kilograms) and height z-score (centimeters) will be assessed and reported every year for 5 years.
Weight (kilograms) and height (centimeters) z-scores will be collected every year for 5 years.
Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies and Their Long-Term Sequelae
Bleeding events (including all gastrointestinal \[GI\] or non-GI bleeding requiring hospitalization, emergency department care, or transfusion) and fracture events will be reported.
The incidence of events will be assessed and reported every year for 5 years.
Study Arms (2)
Alagille syndrome (ALGS)
* A clinically and/or genetically confirmed ALGS diagnosis * Participant prescribed Livmarli
Progressive familial intrahepatic cholestasis (PFIC)
* A clinically and/or genetically confirmed PFIC diagnosis * Prescribed Livmarli
Interventions
The recommended dosage is 380 mcg/kg once daily.
Eligibility Criteria
All patients with ALGS and PFIC prescribed Livmarli at a participating study center and who consent/assent to participation (if required by the local IRB/IEC) will be included in the study at the discretion of the center investigator.
You may qualify if:
- A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
- Participant prescribed Livmarli
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (8)
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
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 5 Years
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 11, 2023
First Posted
January 5, 2024
Study Start
September 21, 2023
Primary Completion (Estimated)
September 20, 2028
Study Completion (Estimated)
September 20, 2030
Last Updated
May 25, 2025
Record last verified: 2025-05