Prospective Evaluation of OptiThyDose

ConditionsThyroid Diseases Congenital Hypothyroidism Graves Disease

Trial statusRecruiting

Trial phaseNot applicable

Trial typeInterventional

Biological sexAll

AgeUp to 18

SponsorUniversity Children's Hospital Basel

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

Newborns with pathological neonatal screening and confirmation of an increased Thyrotropin (TSH) level in an independent venous blood sample

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)

The study participant must be accessible for scheduled visits, treatment and follow-up.

Disqualifiers

Exclusion of newborns from mothers with GD

Exclusion of patients in case of a life-threatening event

known toxic thyroid nodules proven by ultrasound/scintigraphy

known amiodarone induced hyperthyroidism

Trial design

Design model

Parallel

Treatments tested in this trial

OptiThyDose
Other intervention
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.

Treatment groups

150 Participants

are divided into 4 treatment groups

Group A: Control group w/ Congenital HypothyroidismNo intervention 0 interventions

Group B: OptiThyDose w/ Congenital HypothyroidismExperimental treatment 1 intervention

Group C: Control group w/ Graves' DiseaseNo intervention 0 interventions

Group D: OptiThyDose w/ Graves' DiseaseExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

Serum Free Thyroxine (FT4) value

The serum Free Thyroxine (FT4) values is evaluated. FT4, interpreted according to the age of patients, is used in clinical routine as marker of the adequacy of: * Thyroid hormone substitution with LT4 of insufficient thyroid function in patients with CH (low FT4 levels in case of under-dosing of LT4, high FT4 levels in case of over-dosing of LT4) * Suppression of overactive thyroid function with CMZ/MMZ in patients with GD (low FT4 levels in case of over-dosing of CMZ/MMZ, high FT4 levels in case of under-dosing of CMZ/MMZ)

Time frame

90 days post treatment start

Secondary outcomes

Proportion of Thyroid Hormone Levels Within Target Range

The proportion of serum Thyroid Hormone Levels (FT4, TSH, FT3, T3, and T4) that fall within the upper half of the local laboratory reference range at the time point closest to 90 days after treatment initiation.

Time frame

90 days post treatment start and up to 1 year post treatment start

Deviations from Local Laboratory Reference Ranges for Thyroid Hormones

Assessment of deviations in serum thyroid hormone levels (time point, magnitude of elevation, area under the curve (AUC), and fold change) when exceeding the upper or falling below the lower limit of the respective local laboratory reference range.

Time frame