Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Conditions
Keywords
ALGS, Liver Diseases, PFIC, Livmarli, Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
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
Interventions
DRUGLivmarli
The recommended dosage is 380 mcg/kg once daily.
Sponsors
Mirum Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Long-Term Clinical Outcomes | 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 | 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 | 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 | 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. |
Countries
United States
Contacts
Primary ContactClinical Trials Mirum
Outcome results
None listed