OptiThyDose for children with congenital hypothyroidism and Graves' disease
Prospective Evaluation of OptiThyDose, a Mathematical Model for Calculating Appropriate Dose Ranges in Children With Thyroid Diseases
This trial will test whether the OptiThyDose math model helps doctors choose the right thyroid medicine dose for newborns with congenital hypothyroidism and children with Graves' disease.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 150 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | University Children's Hospital Basel Academic / other |
| Locations | 2 sites (Paris and 1 other locations) |
| Trial ID | NCT06864351 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are newborns with confirmed congenital hypothyroidism and children up to 18 years with a new, recurrent, or insufficiently controlled diagnosis of Graves' disease who can attend scheduled visits and whose legal guardians provide informed consent.
Not a fit: Patients outside the enrolled age groups (adults), those with other thyroid conditions not specified in the inclusion criteria, those unable to attend follow-up visits, or those with contraindications to standard thyroid medications are unlikely to benefit from this trial.
Why it matters
Potential benefit: If successful, OptiThyDose could lead to more accurate medication dosing, steadier thyroid hormone control, fewer side effects, and better growth and development outcomes for affected children.
How similar studies have performed: Mathematical dosing algorithms have been applied in other pediatric and adult settings with mixed results, and OptiThyDose represents a relatively novel, unproven approach specifically for pediatric thyroid dosing.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Paris and 1 other locations
- Department of Paediatric Endocrinology, Diabetology and Gynaecology, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris — Paris, France (Not_yet_recruiting)
- Paediatric Endocrinology and Diabetology, University Children's Hospital Basel (UKBB) — Basel, Canton of Basel-City, Switzerland (Recruiting)
Study contacts
- Principal investigator: Gabor Szinnai, Prof. MD, PhD — Paediatric Endocrinology, UKBB
- Study coordinator: Gabor Szinnai, Prof. MD, PhD
- Email: Gabor.Szinnai@ukbb.ch
- Phone: +41 61 704 29 22
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.