Prospective Evaluation of OptiThyDose

Официальное название

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

Обзор

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).

Подробное описание

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).

Вмешательства

• Другое 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, wit

Первичные конечные точки

• Serum Free Thyroxine (FT4) value [Срок оценки: 90 days post treatment start]

Вторичные конечные точки (5)

• Proportion of Thyroid Hormone Levels Within Target Range [Срок оценки: 90 days post treatment start and up to 1 year post treatment start]
• Deviations from Local Laboratory Reference Ranges for Thyroid Hormones [Срок оценки: Up to 1 year post treatment start]
• Number of clinical visits [Срок оценки: Up to 1 year post treatment start]
• Disease-related adverse events [Срок оценки: Up to 1 year post treatment start]
• Average daily dose of administered drugs per kg [Срок оценки: Up to 1 year post treatment start]

Критерии участия

Критерии включения

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

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Простое слепое

Основная цель

Лечение

Центры проведения

Франция · 1 центр

• Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfant — Paris

Швейцария · 1 центр

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

Идентификаторы

NCT: NCT06864351 · 2025-00046; ks21Szinnai

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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