Prospective Evaluation of OptiThyDose

Launched by UNIVERSITY CHILDREN'S HOSPITAL BASEL · Mar 3, 2025

Keywords

ClinConnect Summary

This clinical trial, called the Prospective Evaluation of OptiThyDose, is looking to study a new treatment approach for two thyroid conditions: Congenital Hypothyroidism (CH) and Graves' Disease (GD). Congenital Hypothyroidism is a condition present at birth where the thyroid gland doesn't produce enough hormones, while Graves' Disease is an autoimmune condition that causes the thyroid to become overactive. The trial aims to evaluate the effectiveness of a treatment called OptiThyDose in managing these conditions.

To participate, individuals must meet certain criteria. For Congenital Hypothyroidism, newborns must have a positive screening test and high levels of a hormone called Thyrotropin (TSH) confirmed by blood tests. For Graves' Disease, children up to 18 years old who are newly diagnosed or have ongoing issues with their condition may be eligible. Participants will need to attend regular visits for treatment and follow-up, and their parent or guardian must sign a consent form to agree to participate in the study. It's important to note that some individuals, like newborns born to mothers with Graves' Disease or those with certain other thyroid-related conditions, will not be eligible. The trial is set to begin soon, and it offers a chance for better management of these thyroid diseases.

Gender

ALL

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