Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)
- Registration Number
- NCT06193928
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Brief Summary
The goal of this observational study is to evaluate the long-term safety and clinical outcomes of Livmarli prescribed to patients with Alagille Syndrome (ALGS).
- Detailed Description
Livmarli® is a novel, minimally absorbed, pharmacological treatment for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS), a rare, genetic, autosomal dominant, complex multisystem disorder characterized by the presence of cholestasis (typically presenting as jaundice in the neonatal period) due to intrahepatic bile duct paucity. Livmarli inhibits the ileal bile acid transporter (IBAT) in the terminal ileum, leading to reduced levels of bile acids, thereby improving clinical manifestations and symptoms of cholestasis. Livmarli has been developed by Mirum Pharmaceuticals and approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients with ALGS who are 3 months of age and older.
To be eligible for the study, participants must meet the following criteria:
* A clinically and/or genetically confirmed ALGS diagnosis
* Participant prescribed Livmarli
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 50
- A clinically and/or genetically confirmed ALGS diagnosis
- Participant prescribed Livmarli
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Alagille syndrome (ALGS) Maralixibat * A clinically and/or genetically confirmed ALGS diagnosis * Participant prescribed Livmarli
- Primary Outcome Measures
Name Time Method Long-term clinical outcome events 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 will be captured during each interval event assessments.
Liver Transplant Waitlist Status 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 an LT waitlist.
Assessment of Height Height z-score (centimeters) will be collected every year for 5 years. Height will be collected both at the time the participant started Livmarli and at the time of enrollment in the study. Subsequent height will be collected for up to 5 years.
Height z-score (centimeters) will be assessed and reported every year for 5 years.Assessment of Weight Weight z-score (kilograms) will be collected every year for 5 years. 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) will be assessed and reported every year for 5 years.Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies 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.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (8)
Children's Hospital Los Angeles CHLA
🇺🇸Los Angeles, California, United States
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
Children's Healthcare of Atlanta - Emory University School of Medicine
🇺🇸Atlanta, Georgia, United States
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
🇺🇸Kansas City, Missouri, United States
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
🇺🇸Portland, Oregon, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
🇺🇸Salt Lake City, Utah, United States