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Thyroid DiseasesNCT06864351

Prospective Evaluation of OptiThyDose

A study testing OptiThyDose in people with Thyroid Diseases and Congenital Hypothyroidism, run by University Children's Hospital Basel at 7 sites in 3 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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). Read more about this study

Who is it for?
Ages Up to 18 years
What is being tested?
OptiThyDose
Could I receive a placebo?
No, everyone receives an active treatment
How many people
150 participants
Where
7 sitesin 3 countries
Study dates
28 Aug 2025to Feb 2029
Phase
Interventional
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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)
Show all 9Show fewer
  • 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

Who cannot join

  • CH and GD
  • 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

Trial locations and contacts

7 sites in 3 countries. Contact the study team or a site near you to ask about taking part.

Gabor Szinnai, Prof. MD, PhD contact

+41 61 704 12 12

optithydose@ukbb.ch

7 sites in 3 countries

Switzerland5 sites, 5 recruiting

  • Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB)

    Basel, Canton of Basel-City, 4031

    Gabor Szinnai, Prof. MD, PhD, +41 61 704 12 12, optithydose@ukbb.ch

    Gabor Szinnai, Prof. MD, PhD

    Recruiting

  • Centre hospitalier universitaire vaudois (CHUV)

    Lausanne, Suisse, 1011

    Michael Hauschild, PD Dr., 0041 (0)21 314 87 73, michael.hauschild@chuv.ch

    Michael Hauschild, PD Dr.

    Recruiting

  • Kinderspital Zentralschweiz (LUKS)

    Lucerne, 6000

    Inge Ruiz Arana, Dr., 0041 (0)41 205 31 66, ambulatorium.kinderspital@luks.ch

    Inge Ruiz Arana, Dr.

    Recruiting

  • Ostschweizer Kinderspital (OKS)

    Sankt Gallen, 9000

    Julia Rohayem, PD Dr., 0041 (0)71 243 14 67, info.endokrinologie@oks.ch

    Julia Rohayem, PD Dr.

    Recruiting

  • Universitäts-Kinderspital Zürich - Eleonorenstiftung

    Zurich, 8008

    Daniel Konrad, Prof. Dr., 0041 (0)44 249 65 21, endokrinologie.sekretariat@kispi.uzh.ch

    Daniel Konrad, Prof. Dr.

    Recruiting

Other trials in Switzerland

Armenia1 site, 1 recruiting

  • Arabkir Medical Cente & Institute of child and adolescent Health LLC

    Yerevan, 0014

    Lusine Navasardyan, Prof. Dr., 00374 94 94 25 44, lusinevnavasardyan@gmail.com

    Lusine Navasardyan, Prof. Dr.

    Recruiting

Other trials in Armenia

France1 site, 1 recruiting

  • Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris

    Paris, 75015

    Not yet recruiting

Other trials in France

About this study

Official title: Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases

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).

Read the 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).

Being tested
OptiThyDose
Medical subject headings
Thyroid Diseases, Congenital Hypothyroidism, Graves Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • OptiThyDoseOther

    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.

Study groups in NCT06864351
Study groupRoleWhat they receive
Control group w/ Congenital HypothyroidismNo interventionPatients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
OptiThyDose w/ Congenital HypothyroidismExperimentalPatients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment
Receives: Other: OptiThyDose
Control group w/ Graves' DiseaseNo interventionPatients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
OptiThyDose w/ Graves' DiseaseExperimentalPatients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment
Receives: Other: OptiThyDose

What is being measured

The primary outcome is the main question the study is designed to answer.

  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)

    Measured: 90 days post treatment start

Secondary outcomes (5)

  1. 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.

    Measured: 90 days post treatment start and up to 1 year post treatment start

  2. 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.

    Measured: Up to 1 year post treatment start

  3. Number of clinical visits

    The number of routine clinical visits as required.

    Measured: Up to 1 year post treatment start

  4. Disease-related adverse events

    Assessment of Disease-related adverse events (number and type) occurring during the study period.

    Measured: Up to 1 year post treatment start

  5. 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.

    Measured: Up to 1 year post treatment start

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Mar 2025
  2. Study start28 Aug 2025
  3. Primary completionMay 2028 (estimated)
  4. Study completionFeb 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Participant
Enrolment
150 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06864351
Sponsor's protocol ID
2025-00046; ks21Szinnai
Other IDs
2025
Status last verified
Oct 2026
First submitted
3 Mar 2025
Last updated
2 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A study testing OptiThyDose in people with Thyroid Diseases and Congenital Hypothyroidism, run by University Children's Hospital Basel at 7 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in Switzerland, Armenia, France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Gabor Szinnai, Prof. MD, PhD (+41 61 704 12 12, optithydose@ukbb.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Serum Free Thyroxine (FT4) value (time frame: 90 days post treatment start). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06864351.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026.

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