LEAP-US Study for Alagille Syndrome and PFIC Patients on Livmarli
This 5-year study, called LEAP-US, is looking at the long-term safety and effects of Livmarli in patients with Alagille syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC). Livmarli is a medication that helps reduce bile acid levels. You may be able to join if you have a confirmed diagnosis of ALGS or PFIC and are currently prescribed Livmarli. The study will track important health outcomes like the need for a liver transplant, changes in growth and development, and any long-term health issues for up to 180 days after stopping Livmarli. This study aims to understand how Livmarli impacts patients over time.
- Study design
- This is an observational study, meaning researchers will watch and collect information about 70 participants over time. It is not a randomized or blinded study.
- What's involved
- You will have your liver transplant waitlist status checked every 6 months for 5 years. Your weight and height will be measured annually for 5 years.
- Compensation
- Not stated in the trial record.
- Follow-up
- Long-term health outcomes will be recorded for up to 180 days after stopping Livmarli. Liver transplant waitlist status will be collected for 5 years, and growth will be assessed for 5 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
At a glance
Conditions
NCT06193928
Where you'd take part
This study runs at 8 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvaniastudy coordinator listed
Recruiting
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgiastudy coordinator listed
Recruiting
Children's Hospital Los Angeles CHLA
Los Angeles, Californiastudy coordinator listed
Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvaniastudy coordinator listed
Recruiting
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouristudy coordinator listed
Recruiting
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
Portland, Oregonstudy coordinator listed
Recruiting
Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado
Aurora, Coloradostudy coordinator listed
Recruiting
University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
Salt Lake City, Utahstudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
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Inclusion
Exclusion
What this trial measures
- Incidence of Long-Term Clinical OutcomesLong-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded.
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.
- Liver Transplant Indication and Waitlist StatusLT waitlist status will be collected at enrollment and every 6 months for 5 years.
LT waitlist status will be collected, including when placed on or removed from LT waitlist.
- Assessment of Growth and DevelopmentWeight (kilograms) and height (centimeters) z-scores will be collected every year for 5 years.
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.
- Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies and Their Long-Term SequelaeThe incidence of events will be assessed and reported every year for 5 years.
Bleeding events (including all gastrointestinal \[GI\] or non-GI bleeding requiring hospitalization, emergency department care, or transfusion) and fracture events will be reported.