Improving Drug Dosing and Outcomes for Children and Young Adults with Fontan Associated Liver Disease

What this Trial Is About

Researchers are studying how liver congestion and fibrosis affect the way children, adolescents, and young adults with Fontan circulation process certain drugs. This investigation focuses on measuring the impact of these liver conditions on the metabolism and transport of medications in this specific group. The study is a Phase 1, open-label, single-center trial designed to gather important information on drug dosing for patients with Fontan-associated liver disease. Participants in the study will receive a single oral dose of two drugs sildenafil and pravastatin. The dose of sildenafil varies by weight, with 10 mg given to those under 20 kg and 20 mg for those 20 kg or above. Pravastatin dosing is age-based, with 20 mg for children under 13 years and 40 mg for those 14 years or older. This dosing occurs during the assessment phase to evaluate drug concentrations and how the liver condition influences drug processing. Participants will be involved in fasting overnight before receiving the medications. Researchers will measure the concentration of sildenafil and pravastatin over a two-year period, focusing on the area under the curve AUC to understand drug metabolism. The study includes assessments to monitor liver function and ensure participant safety, with the total involvement spanning up to two years to capture the long-term effects on drug disposition in this population.

IMProving DRug Dosing and Outcomes for Single VEntricle Patients With Fontan Associated Liver Disease