Clinical trial NCT06864351: Prospective Evaluation of OptiThyDose
Official title: Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Thyroid Diseases
Congenital Hypothyroidism
Graves Disease
Lead sponsor: University Children's Hospital Basel
Sponsor class: OTHER
First posted: 2025-03-07
Enrollment: 150
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

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

Primary outcomes:
measure: Serum Free Thyroxine (FT4) value
timeFrame: 90 days post treatment start
Secondary outcomes:
measure: Proportion of Thyroid Hormone Levels Within Target Range
timeFrame: 90 days post treatment start and up to 1 year post treatment start
measure: Deviations from Local Laboratory Reference Ranges for Thyroid Hormones
timeFrame: Up to 1 year post treatment start
measure: Number of clinical visits
timeFrame: Up to 1 year post treatment start
measure: Disease-related adverse events
timeFrame: Up to 1 year post treatment start
measure: Average daily dose of administered drugs per kg
timeFrame: Up to 1 year post treatment start
Other outcomes:
measure: Heart Rate
timeFrame: Up to 1 year post treatment start
measure: Overall Treatment Costs
timeFrame: Up to 1 year post treatment start
Locations:
city: Paris
country: France
facility: Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris
city: Basel
country: Switzerland
facility: Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB)
state: BS
Study officials:
affiliation: Paediatric Endocrinology, UKBB
name: Gabor Szinnai, Prof. MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: