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Recruiting NCT06193928

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

— Observational Alagille Syndrome Progressive Familial Intrahepatic Cholestasis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Livmarli, Livmarli.
Who it may be relevant to
Registry conditions: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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.

Detailed description

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

Interventions

  • Drug Livmarli
    The recommended dosage is 380 mcg/kg once daily.
  • Drug Livmarli
    The recommended dosage us 570 mcg/kg twice daily.

Primary outcome measures

  • 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.]
  • Liver Transplant Indication and Waitlist Status [Time frame: LT waitlist status will be collected at enrollment and every 6 months for 5 years.]
  • Assessment of Growth and Development [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 [Time frame: The incidence of events will be assessed and reported every year for 5 years.]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 8 centers
  • Children's Hospital Los Angeles CHLA — Los Angeles
  • Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Di — Aurora
  • Children's Healthcare of Atlanta - Emory University School of Medicine — Atlanta
  • Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology — Kansas City
  • Oregon Health and Science University, Division of Pediatric Gastroenterology, Department o — Portland
  • Children's Hospital of Philadelphia — Philadelphia
  • Children Hospital of Pittsburgh — Pittsburgh
  • University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition — Salt Lake City

Identifiers

NCT: NCT06193928 · MRX-310

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗