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

• Status: Recruiting
• Trial end date: 2023-06-15
• Target enrollment: 0
• Participant gender: All

Summary

This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed with CH.

Subjects will be randomized in a 2:1 ratio to Treatment (Tirosint®-SOL) or Control (conventional therapy with levothyroxine sodium crushed tablets).

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: IBSA Institut Biochimique SA
• Collaborator: Cromsource

Criteria

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.