Prospective Evaluation of OptiThyDose

NCT ID: NCT06864351

Last Updated: 2025-09-03

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 150 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-08-28
• Study Completion Date: 2029-02-28

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).

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).

Conditions

Thyroid Diseases; Congenital Hypothyroidism; Graves Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

Control group w/ Congenital Hypothyroidism

Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment

Group Type: NO_INTERVENTION

No interventions assigned to this group

OptiThyDose w/ Congenital Hypothyroidism

Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment

Group Type: EXPERIMENTAL

OptiThyDose

Intervention Type: 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.

Control group w/ Graves' Disease

Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment

Group Type: NO_INTERVENTION

No interventions assigned to this group

OptiThyDose w/ Graves' Disease

Patients with Graves' Disease receiving routine carbimazole (CMZ) or methimazole (MMZ) treatment

Group Type: EXPERIMENTAL

OptiThyDose

Intervention Type: 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.

Interventions

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, 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.

Intervention Type: OTHER

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

• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Children's Hospital Basel

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gabor Szinnai, Prof. MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Paediatric Endocrinology, UKBB

Locations

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

Paris, , France

Site Status: NOT_YET_RECRUITING

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

Basel, Canton of Basel-City, Switzerland

Site Status: RECRUITING

Countries

France; Switzerland

Central Contacts

Gabor Szinnai, Prof. MD, PhD

Role: CONTACT

Gabor.Szinnai@ukbb.ch

+41 61 704 29 22

Facility Contacts

Gabor Szinnai, Prof. MD, PhD

Role: primary

Gabor.Szinnai@ukbb.ch

+41 61 704 29 22

Other Identifiers

2025-00046; ks21Szinnai

Identifier Type: -

Identifier Source: org_study_id