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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Alagille Syndrome Abnormalities, Multiple Bile Duct Diseases Biliary Tract Diseases Cardiovascular Abnormalities Cardiovascular Diseases Cholestasis Cholestasis, Intrahepatic Cholestasis, progressive familial intrahepatic 1 Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Heart Defects, Congenital Liver Diseases Progressive Familial Intrahepatic Cholestasis

Sex eligibility

All sexes

Study type

Observational

Primary location

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

About this study

Livmarli® is a novel, minimally absorbed, pharmacological product that inhibits the ileal bile acid transporter (IBAT) in the terminal ileum, leading to reduced levels of bile acids. Livmarli (maralixibat) has been developed by Mirum Pharmaceuticals and was the first treatment approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients 3 months of age and older with Alagille syndrome (ALGS). Subsequently, Livmarli was approved by the FDA for the treatment of cholestatic pruritus in patients 12 months of age and older with Progressive familial intrahepatic cholestasis (PFIC). To be eligible for the study, participants must meet the following criteria:

A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
Prescribed Livmarli

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

Livmarli

Drug

The recommended dosage is 380 mcg/kg once daily.

Other names: Maralixibat

Primary outcomes

Incidence of Long-Term Clinical Outcomes

Time frame: Long-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 Status

Time frame: LT 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 Development

Time frame: Weight (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 Sequelae

Time frame: The 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trials Mirum

CONTACT

Clinical Trials <[email protected]>;

+16506674085

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Acronym: LEAP

Important dates

Study start: 2023
Primary completion: 2028
Study completion: 2030
First posted: Jan 5, 2024
Registry last updated: May 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Recruiting