OptiThyDose for children with congenital hypothyroidism and Graves' disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, randomized, two-arm, single-blinded trial will compare dosing guided by the OptiThyDose mathematical model versus standard care in pediatric patients with congenital hypothyroidism (newborns identified by screening) and children up to 18 years with new or recurrent/insufficiently controlled Graves' disease. OptiThyDose aims to individualize initial and follow-up levothyroxine or antithyroid medication dosing to avoid under- and overdosing. Participants will be followed with scheduled visits and laboratory monitoring to measure hormone control, need for dose adjustments, and adverse events. The study is conducted at pediatric endocrinology centers in Basel, Switzerland and Paris, France.

Why it matters

Who can join: the full rules

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

Where it's running

• Arabkir Medical Cente & Institute of child and adolescent Health LLC
  Yerevan, Armenia Recruiting
• Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB)
  Basel, Canton of Basel-City, Switzerland Recruiting
• Centre hospitalier universitaire vaudois (CHUV)
  Lausanne, Suisse, Switzerland Recruiting
• Kinderspital Zentralschweiz (LUKS)
  Lucerne, Switzerland Recruiting
• Ostschweizer Kinderspital (OKS)
  Sankt Gallen, Switzerland Recruiting
• Universitäts-Kinderspital Zürich - Eleonorenstiftung
  Zurich, Switzerland Recruiting

Study contacts

• Principal investigator: Gabor Szinnai, Prof. MD, PhD — Paediatric Endocrinology, UKBB
• Study coordinator: Gabor Szinnai, Prof. MD, PhD
• Email: optithydose@ukbb.ch
• Phone: +41 61 704 12 12

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.