Prospective Evaluation of OptiThyDose

University Children's Hospital Basel

Status: Enrolling

Conditions: Thyroid Diseases; Graves Disease; Congenital Hypothyroidism

Treatments: Other: OptiThyDose

Study type: Interventional

Funder types: Other

Identifiers

NCT06864351

2025-00046 ks21Szinnai;

Details and patient eligibility

About

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).

Full 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).

Enrollment: 150 estimated patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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150 participants in 4 patient groups

Control group w/ Congenital Hypothyroidism

No Intervention group

Description:

Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment

OptiThyDose w/ Congenital Hypothyroidism

Experimental group

Description:

Patients with Congenital Hypothyroidism receiving routine levothyroxine (LT4) treatment

Treatment:

Other: OptiThyDose

Control group w/ Graves' Disease

No Intervention group

Description:

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

OptiThyDose w/ Graves' Disease

Experimental group

Description:

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

Treatment:

Other: OptiThyDose