Testing OptiThyDose, a New Model to Find the Best Medicine Dose for Children With Congenital Hypothyroidism and Graves Disease

What this Trial Is About

This research aims to evaluate the OptiThyDose mathematical model for children with thyroid diseases, specifically Congenital Hypothyroidism CH and Graves Disease GD. These thyroid diseases can affect individuals from birth through adolescence and require careful treatment to avoid impacts on brain development, growth, and other vital functions. The study is designed as a multicenter, randomized, two-arm, single-blinded clinical trial to assess how well OptiThyDose helps in dosing thyroid medications for children. Participants are assigned to one of four groups children with CH receiving routine levothyroxine LT4 treatment either guided by OptiThyDose or standard care, and children with GD receiving routine carbimazole CMZ or methimazole MMZ treatment either with OptiThyDose dosing or standard care. OptiThyDose uses a mathematical model incorporating clinical and laboratory data to suggest optimal dosing aimed at maintaining Free Thyroxine FT4 levels within the upper half of age-specific reference ranges. Physicians can choose to follow the models recommended dose or use their own clinical judgment at each visit. During the study, participants undergo regular outpatient visits with monitoring of thyroid hormone levels, clinical assessments, and medication dosing adjustments over a period of up to one year. The primary outcome is the serum Free Thyroxine FT4 value at 90 days after starting treatment. Secondary outcomes include the proportion of hormone levels within target ranges, deviations from lab reference values, number of clinical visits, disease-related adverse events, and average daily drug dose per kilogram of body weight. The study includes signed informed consent and requires participants to be accessible for scheduled visits and follow-up.

Prospective Evaluation of OptiThyDose