Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)

This is an interventional treatment trial for Congenital Hypothyroidism Read More

Inclusion Criteria:

• Male and female patient aged 0 to 9 months

• Primary CH diagnosis with elevated TSH and low T4, requiring treatment with LT4, under either of the following conditions:

  • Neonates newly diagnosed with primary CH and needing to initiate LT4 therapy, or
  • Infants previously diagnosed with primary CH and who are already on LT4 therapy for at least 4 weeks;
• Provide and comply with the informed consent.

Exclusion Criteria:

• Preterm neonates with a gestational age < 37 weeks;
• Low birth weight and very low birth weight neonates (weight < 2.5 kg);
• Neonates in neonatal intensive care units or requiring admission to NICU or neonates/infants hospitalized or requiring hospitalization;
• CH diagnosis > 4 weeks after delivery (for newly diagnosed neonates only);

• Diagnosis of primary gastrointestinal disease:

  • Gastroesophageal reflux requiring medical therapy (beyond thickening of formula or position);
  • Anatomic defects (e.g. intestinal atresia, malrotation, tracheoesophageal fistula, pyloric stenosis, Hirschsprung's disease, gastroschisis);
  • Dietary allergy (e.g. cow's milk protein allergy);
  • Malabsorption related to cystic fibrosis, celiac disease and others;
  • Necrotizing enterocolitis requiring surgical resection;
• Known or suspected adrenal insufficiency (e.g. congenital adrenal hyperplasia, hypopituitarism);
• Diagnosis of congenital cardiac disease, cardiac insufficiency or risk for cardiac failure;
• Diagnosis of chromosomopathy;
• Diagnosis of central hypothyroidism;
• Hypersensitivity to glycerol;
• Concomitant anticonvulsant medications, liothyronine, combination of LT4 and liothyronine, thyroid extracts and/or chronic or long-term use of systemic glucocorticoids
• History of nonadherence with medication or medical visit schedule; or
• Any condition for which, participation would not be in the best interest of the patient or that could limit protocol specified assessments.