Status:

TERMINATED

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

Lead Sponsor:

IBSA Institut Biochimique SA

Collaborating Sponsors:

Cromsource

Conditions:

Congenital Hypothyroidism

Eligibility:

All Genders

1-9 years

Phase:

PHASE4

Brief 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...

Detailed Description

Newly diagnosed neonates will be randomly assigned to start therapy with LT4 at the initial dose recommended by the Standard of Care (SOC). Infants already on LT4 therapy will continue at the same dai...

Eligibility Criteria

Inclusion

  • Male and female patient aged 0 to 9 months
  • Primary CH diagnosis with elevated TSH and low or normal FT4, 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 3 weeks;
  • Provide and comply with the informed consent.

Exclusion

  • Preterm neonates with a gestational age \< 37 weeks;
  • Low birth weight (LBW) or very low birth weight (VLBW) neonates (weight \< 2.5 kg) or VLBW infants (weight \< 1.5 kg);
  • Neonates in neonatal intensive care units or requiring admission to NICU or neonates/infants hospitalized or requiring hospitalization or in fragile health conditions (e.g. with serious health problems or complications);
  • Neonates with CH diagnosis \> 4 weeks after delivery;
  • 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.

Key Trial Info

Start Date :

January 21 2022

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

March 27 2025

Estimated Enrollment :

34 Patients enrolled

Trial Details

Trial ID

NCT05228184

Start Date

January 21 2022

End Date

March 27 2025

Last Update

October 23 2025

Active Locations (10)

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Page 1 of 3 (10 locations)

1

Children's Hospital of Los Angeles

Los Angeles, California, United States, 90027

2

CHOC Children's Hospital

Orange, California, United States, 92868

3

University of California San Francisco

San Francisco, California, United States, 94143

4

Yale University

New Haven, Connecticut, United States, 06511

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