NCT06864351

Prospective Evaluation of OptiThyDose

Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: University Children's Hospital Basel · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The aim of this multicentric, randomised, two-arms and single-blinded clinical trial is to prospectively evaluate OptiThyDose for Congenital hypothyroidism (CH) and Graves' disease (GD).

Detailed description

Thyroid diseases can affect people from birth to adulthood, ith some being present at birth (congenital) and others developing later in life (acquired). These diseases need to be treated quickly and properly because if left untreated, they can impact brain development, thinking abilities, growth, puberty, and other important body functions. However, treating thyroid diseases in children can be challenging, as it's important to avoid both under- and overdosing. Algorithms that help determine the best individual dose for children with thyroid diseases could reduce the risk of long-term problems, like impaired thinking and growth. This is especially important because cases of thyroid diseases in children are increasing worldwide. OptiThyDose is a new mathematical model developed to help doctors find the right dose for children with thyroid diseases. The primary goal of this multicentric, randomised, two-arms and single-blinded study is to test how well OptiThyDose works for children with two types of thyroid diseases: Congenital Hypothyroidism (CH) and Graves' Disease (GD). If proven effective, OptiThyDose could help ensure more accurate dosing of thyroid medications, leading to better hormone control, fewer side effects, and improved health outcomes in children with Congenital Hypothyroidism (CH) and Graves' Disease (GD).

Conditions

• Thyroid Diseases
• Congenital Hypothyroidism
• Graves Disease

Interventions

Type	Name	Description
OTHER	OptiThyDose	OptiThyDose is an iterative mathematical model applied at each patient visit, consisting of three components: (i) a disease-specific pharmacometrics (PMX) model, (ii) an empirical Bayesian estimation (EBE) component, and (iii) an optimal control theory (OCT) component. It calculates the optimal LT4 or CMZ/MMZ dose to maintain Free Thyroxine (FT4) levels within the upper half of the age-specific reference range, integrating past clinical and lab data. Dosing follows international guidelines, with physicians able to consult OptiThyDose for individualized dosing within recommended ranges. At each outpatient visit, the physician can either (A) prescribe a dose within OptiThyDose's suggested range or (B) choose a dose based on personal experience.

Timeline

• Start date: 2025-08-28
• Primary completion: 2028-05-01
• Completion: 2029-02-01
• First posted: 2025-03-07
• Last updated: 2025-09-03

Locations

2 sites across 2 countries: France, Switzerland