An interventional study of OptiThyDose in Thyroid Diseases, Congenital Hypothyroidism and Graves Disease, sponsored by University Children's Hospital Basel. Recruiting at 7 sites in 3 countries. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2026-10-02.
Sponsored by University Children's Hospital Basel · Not applicable, Interventional, and Treatment
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).
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).
365 studies on the registry are indexed under Thyroid Diseases; 80 are open to participants now.
This study's planned enrollment of 150 is above the median of 88 across 198 interventional studies indexed under Thyroid Diseases.
Browse Thyroid Diseases studies →University Children's Hospital Basel is the lead sponsor of 28 studies on the registry; 9 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria:
Congenital hypothyroidism (CH)
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:
CH and GD
Exclusion Criteria:
CH and GD
GD
Exclusion of children with known other aetiologies of hyperthyroidism than GD without elevated Anti-TSH-receptor antibodies e.g.:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
Other: OptiThyDose
Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
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.
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
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: Up to 1 year post treatment start
Number of clinical visits
The number of routine clinical visits as required.
Time frame: Up to 1 year post treatment start
Disease-related adverse events
Assessment of Disease-related adverse events (number and type) occurring during the study period.
Time frame: Up to 1 year post treatment start
Average daily dose of administered drugs per kg
Assessment of the average daily dose per kilogram of administered drugs (LT4 or CMZ/MMZ) throughout the study period.
Time frame: Up to 1 year post treatment start
Heart Rate
Assessment of heart rate measured both on-site and with a wearable device, at or between clinical visits, during the study period
Time frame: Up to 1 year post treatment start
Overall Treatment Costs
Evaluation of overall treatment costs (e.g. expenses based on the number of consultations and costs for laboratory measurements) from a health economic perspective during the study period.
Time frame: Up to 1 year post treatment start
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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University Children's Hospital Basel