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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Control group w/ Congenital Hypothyroidism | No Intervention | Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment | |
| OptiThyDose w/ Congenital Hypothyroidism | Experimental | Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment |
|
| Control group w/ Graves' Disease | No Intervention | Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment | |
| OptiThyDose w/ Graves' Disease | Experimental | Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OptiThyDose | Other | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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:
| 90 days post treatment start |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 90 days post treatment start and up to 1 year post treatment start |
| Measure | Description | Time Frame |
|---|---|---|
| Heart Rate | Assessment of heart rate measured both on-site and with a wearable device, at or between clinical visits, during the study period | Up to 1 year post treatment start |
| Overall Treatment Costs |
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.:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Gabor Szinnai, Prof. MD, PhD | Contact | +41 61 704 29 22 | Gabor.Szinnai@ukbb.ch |
| Name | Affiliation | Role |
|---|---|---|
| Gabor Szinnai, Prof. MD, PhD | Paediatric Endocrinology, UKBB | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris | Not yet recruiting | Paris | 75015 | France |
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| ID | Term |
|---|---|
| D013959 | Thyroid Diseases |
| D003409 | Congenital Hypothyroidism |
| D006111 | Graves Disease |
| ID | Term |
|---|---|
| D004700 | Endocrine System Diseases |
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
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|
| 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. |
| Up to 1 year post treatment start |
| Number of clinical visits | The number of routine clinical visits as required. | 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. | 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. | Up to 1 year post treatment start |
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.
| Up to 1 year post treatment start |
| Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB) | Recruiting | Basel | Canton of Basel-City | 4031 | Switzerland |
|
| D009140 |
| Musculoskeletal Diseases |
| D001849 | Bone Diseases, Endocrine |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007037 | Hypothyroidism |
| D005094 | Exophthalmos |
| D009916 | Orbital Diseases |
| D005128 | Eye Diseases |
| D006042 | Goiter |
| D006980 | Hyperthyroidism |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |