Actively Recruiting
Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases
Led by University Children's Hospital Basel · Updated on 2025-09-03
150
Participants Needed
2
Research Sites
39 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the OptiThyDose mathematical model for children with thyroid diseases, specifically Congenital Hypothyroidism (CH) and Graves' Disease (GD). These thyroid diseases can affect individuals from birth through adolescence and require careful treatment to avoid impacts on brain development, growth, and other vital functions. The study is designed as a multicenter, randomized, two-arm, single-blinded clinical trial to assess how well OptiThyDose helps in dosing thyroid medications for children. Participants are assigned to one of four groups: children with CH receiving routine levothyroxine (LT4) treatment either guided by OptiThyDose or standard care, and children with GD receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment either with OptiThyDose dosing or standard care. OptiThyDose uses a mathematical model incorporating clinical and laboratory data to suggest optimal dosing aimed at maintaining Free Thyroxine (FT4) levels within the upper half of age-specific reference ranges. Physicians can choose to follow the model's recommended dose or use their own clinical judgment at each visit. During the study, participants undergo regular outpatient visits with monitoring of thyroid hormone levels, clinical assessments, and medication dosing adjustments over a period of up to one year. The primary outcome is the serum Free Thyroxine (FT4) value at 90 days after starting treatment. Secondary outcomes include the proportion of hormone levels within target ranges, deviations from lab reference values, number of clinical visits, disease-related adverse events, and average daily drug dose per kilogram of body weight. The study includes signed informed consent and requires participants to be accessible for scheduled visits and follow-up.
CONDITIONS
Brief Title
Prospective Evaluation of OptiThyDose
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Congenital Hypothyroidism confirmed by abnormal neonatal screening and elevated Thyrotropin (TSH) in blood
- Children up to 18 years old with new, recurring, or uncontrolled Graves' Disease under treatment
- Presence of pathological lab values (suppressed TSH, increased thyroid hormones, positive Anti-TSH-receptor antibodies) or typical clinical signs of Graves' Disease
- Ability to attend scheduled visits, follow treatment, and participate in follow-up
- Signed informed consent form by patient or legal representative according to legal requirements
You will not qualify if you...
- Newborns from mothers with Graves' Disease
- Patients experiencing a life-threatening event
- Children with other causes of hyperthyroidism besides Graves' Disease, such as toxic thyroid nodules, amiodarone-induced hyperthyroidism, McCune Albright syndrome, or genetically proven activating mutations of the TSH receptor gene
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 year post treatment start
Participants receive routine thyroid disease treatment and dosing is guided either by the OptiThyDose mathematical model or by physician experience at outpatient visits.
Regular outpatient visits for dosing and monitoring throughout treatment
Trial Site Locations
Total: 2 locations
1
Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris
Paris, France, 75015
Not Yet Recruiting
2
Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB)
Basel, Canton of Basel-City, Switzerland, 4031
Actively Recruiting
Research Team
G
Gabor Szinnai, Prof. MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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