This study will evaluate the safety, pharmacokinetics (how the body absorbs, distributes, metabolizes, and eliminates the drug), and pharmacodynamics (how the drug affects the body) of resmetirom in children and adolescents with metabolic dysfunction-associated steatohepatitis (MASH) and liver fibrosis. Participants will receive oral resmetirom once daily for approximately 14 days at one of several dose levels. The information from this study will help determine appropriate dosing and further evaluate the safety and biological effects of resmetirom in pediatric participants with MASH.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
61
Resmetirom (MGL-3196) : Oral resmetirom administered once daily. Participants receive one of several protocol-defined dose levels assigned according to the sequential multiple ascending-dose study design. Dose escalation proceeds following review of available safety, pharmacokinetic, and pharmacodynamic data.
Riley Hospital for Children at IU Health
Indianapolis, Indiana, United States
RECRUITINGSafety and tolerability of multiple ascending doses of resmetirom
Safety and tolerability will be assessed by the incidence and severity of adverse events, serious adverse events, clinical laboratory evaluations, vital signs, 12-lead electrocardiograms, physical examinations, and other protocol-defined safety assessments.
Time frame: Baseline through approximately Day 21 (or the protocol-defined safety follow-up period)
Pharmacokinetic parameters of resmetirom (MGL-3196) and its metabolite (MGL-3623)
Pharmacokinetic parameters of resmetirom and MGL-3623 following single and repeated dosing, including CL/F (parent only), Vz/F (parent only), Cmax, AUC0-24, and AUC0-∞ after the first dose, and CL/F (parent only), Cmax, and AUC0-24 following repeated dosing, as applicable.
Time frame: Day 1 through Day 14
Pharmacodynamic biomarkers associated with resmetirom exposure
Changes in pharmacodynamic biomarkers, including thyroid axis markers, sex hormone-binding globulin, and lipid parameters and their relationship to dose and/or plasma concentrations of resmetirom.
Time frame: Baseline through Day 21
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.