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