Prospective Evaluation of OptiThyDose
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: OptiThyDose.
- Who it may be relevant to
- Registry conditions: Thyroid Diseases, Congenital Hypothyroidism, Graves Disease. Basic parameters: up to 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France, Switzerland
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases
Overview
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).
Interventions
- 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, wit
Primary outcome measures
- Serum Free Thyroxine (FT4) value [Time frame: 90 days post treatment start]
Secondary outcome measures (5)
- Proportion of Thyroid Hormone Levels Within Target Range [Time frame: 90 days post treatment start and up to 1 year post treatment start]
- Deviations from Local Laboratory Reference Ranges for Thyroid Hormones [Time frame: Up to 1 year post treatment start]
- Number of clinical visits [Time frame: Up to 1 year post treatment start]
- Disease-related adverse events [Time frame: Up to 1 year post treatment start]
- Average daily dose of administered drugs per kg [Time frame: Up to 1 year post treatment start]
Eligibility criteria
Inclusion criteria
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 criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Single blind
- Primary purpose
- Treatment
Study locations
France · 1 center
- Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfant — Paris
Switzerland · 1 center
- Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB) — Basel
Identifiers
NCT: NCT06864351 · 2025-00046; ks21Szinnai