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

N/ARecruitingNCT06193928

Mirum Pharmaceuticals, Inc.70 enrolled

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.

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

Study Type

OBSERVATIONAL

Enrollment

Conditions

Alagille Syndrome Progressive Familial Intrahepatic Cholestasis

Interventions

LivmarliDRUG

The recommended dosage is 380 mcg/kg once daily.

LivmarliDRUG

The recommended dosage us 570 mcg/kg twice daily.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (8)

Children's Hospital Los Angeles CHLA

Los Angeles, California, United States

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, Colorado, United States

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: The incidence of events will be assessed and reported every year for 5 years.

Central Contacts

Clinical Trials Mirum

CONTACT

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

Data from ClinicalTrials.gov

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