Clinical trial NCT06193928: Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Official title: Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
Keywords: ALGS
Liver Diseases
PFIC
Livmarli
Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2024-01-05
Enrollment: 70
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 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.

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.

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

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

Primary outcomes:
measure: Incidence of Long-Term Clinical Outcomes
timeFrame: Long-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded.
measure: Liver Transplant Indication and Waitlist Status
timeFrame: LT waitlist status will be collected at enrollment and every 6 months for 5 years.
measure: Assessment of Growth and Development
timeFrame: Weight (kilograms) and height (centimeters) z-scores will be collected every year for 5 years.
measure: Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies and Their Long-Term Sequelae
timeFrame: The incidence of events will be assessed and reported every year for 5 years.
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles CHLA
state: California
city: Aurora
country: United States
facility: Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado
state: Colorado
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Emory University School of Medicine
state: Georgia
city: Kansas City
country: United States
facility: Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
state: Missouri
city: Portland
country: United States
facility: Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children Hospital of Pittsburgh
state: Pennsylvania
city: Salt Lake City
country: United States
facility: University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
state: Utah
Study officials:
References: