Prospective Evaluation of OptiThyDose
Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases
University Children's Hospital Basel
150 participants
Aug 28, 2025
INTERVENTIONAL
Conditions
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).
Eligibility
Inclusion Criteria9
- Congenital hypothyroidism (CH)
- Newborns with pathological neonatal screening and confirmation of an increased Thyrotropin (TSH) level in an independent venous blood sample
- Graves' disease (GD)
- Children until 18 years with new diagnosis of GD, recurrence of GD, or insufficiently controlled GD under CMZ/MMZ during follow-up according to:
- Pathological lab values (suppressed TSH, increased thyroid hormone levels, positive Anti-TSH-receptor antibodies)
- Typical clinical picture, if present (goitre, tachycardia, palpitations, weight loss, hyperphagia, altered mood)
- CH and GD
- The study participant must be accessible for scheduled visits, treatment and follow-up.
- Signed Informed Consent form (ICF) obtained prior to any study related procedure. Written IC for study participation must be signed and dated by the patient and/or his/her legal representative(s) in accordance with national legal requirements
Exclusion Criteria9
- CH and GD
- Exclusion of newborns from mothers with GD
- Exclusion of patients in case of a life-threatening event
- GD
- Exclusion of children with known other aetiologies of hyperthyroidism than GD without elevated Anti-TSH-receptor antibodies e.g.:
- known toxic thyroid nodules proven by ultrasound/scintigraphy
- known amiodarone induced hyperthyroidism
- known McCune Albright syndrome (based on clinical, laboratory, and genetic diagnosis) associated hyperthyroidism
- known genetically proven hyperthyroidism caused by activating mutations of the TSH receptor gene
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Interventions
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.
Locations(2)
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NCT06864351