Prospective Evaluation of OptiThyDose

Not Applicable

Not yet recruiting

• Conditions: Thyroid Diseases; Congenital Hypothyroidism; Graves Disease

• Registration Number: NCT06864351
• Lead Sponsor: University Children's Hospital Basel

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).

Study Timeline

• Status Verified: 2025-03
• Study Start: 2025-03
• Study First Submitted: 2025-03-03
• Study First Submitted QC: 2025-03-03
• Last Update Submitted: 2025-03-03
• Study First Posted: 2025-03-07
• Last Update Posted: 2025-03-07
• Primary Completion: 2028-05
• Study Completion: 2029-02

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 150

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Serum Free Thyroxine (FT4) value	90 days post treatment start	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)

Secondary Outcome Measures

Name	Time	Method
Deviations from Local Laboratory Reference Ranges for Thyroid Hormones	Up to 1 year post treatment start	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.
Disease-related adverse events	Up to 1 year post treatment start	Assessment of Disease-related adverse events (number and type) occurring during the study period.
Average daily dose of administered drugs per kg	Up to 1 year post treatment start	Assessment of the average daily dose per kilogram of administered drugs (LT4 or CMZ/MMZ) throughout the study period.
Proportion of Thyroid Hormone Levels Within Target Range	90 days post treatment start and up to 1 year post treatment start	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.
Number of clinical visits	Up to 1 year post treatment start	The number of routine clinical visits as required.

Trial Locations

Locations (2)

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

Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB); 🇨🇭 Basel, BS, Switzerland