Prospective Evaluation of OptiThyDose
1 other identifier
interventional
150
2 countries
2
Brief Summary
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for not_applicable
Started Aug 2025
Longer than P75 for not_applicable
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 3, 2025
CompletedFirst Posted
Study publicly available on registry
March 7, 2025
CompletedStudy Start
First participant enrolled
August 28, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 1, 2029
September 3, 2025
September 1, 2025
2.7 years
March 3, 2025
September 1, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
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)
90 days post treatment start
Secondary Outcomes (5)
Proportion of Thyroid Hormone Levels Within Target Range
90 days post treatment start and up to 1 year post treatment start
Deviations from Local Laboratory Reference Ranges for Thyroid Hormones
Up to 1 year post treatment start
Number of clinical visits
Up to 1 year post treatment start
Disease-related adverse events
Up to 1 year post treatment start
Average daily dose of administered drugs per kg
Up to 1 year post treatment start
Other Outcomes (2)
Heart Rate
Up to 1 year post treatment start
Overall Treatment Costs
Up to 1 year post treatment start
Study Arms (4)
Control group w/ Congenital Hypothyroidism
NO INTERVENTIONPatients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
OptiThyDose w/ Congenital Hypothyroidism
EXPERIMENTALPatients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
Control group w/ Graves' Disease
NO INTERVENTIONPatients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
OptiThyDose w/ Graves' Disease
EXPERIMENTALPatients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- CH and GD
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris
Paris, 75015, France
Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB)
Basel, Canton of Basel-City, 4031, Switzerland
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Gabor Szinnai, Prof. MD, PhD
Paediatric Endocrinology, UKBB
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- SINGLE
- Who Masked
- PARTICIPANT
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 3, 2025
First Posted
March 7, 2025
Study Start
August 28, 2025
Primary Completion (Estimated)
May 1, 2028
Study Completion (Estimated)
February 1, 2029
Last Updated
September 3, 2025
Record last verified: 2025-09