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

NCT05228184 | Terminated Phase 4 FDA Drug

• 1 other identifier: 20US-T414
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 10

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 2:1 ratio to Treatment (Tirosint®-SOL) or Control (conventional therapy with levothyroxine sodium crushed tablets).

Conditions

Congenital Hypothyroidism

Outcome Measures

Primary Outcomes (1)

• LT4 dose required to maintain TSH in target range (unit: mcg/kg/day)

  The LT4 dose is calculated based on the daily LT4 dose (mcg) used in the time period preceding the visit (or the average daily dose on a weekly basis if more than one strength is used over the course of the week) and the body weight (kg) measured during the visit.

  Up to 22 months based on age group

Secondary Outcomes (12)

• Hormonal profile for TSH

  Up to 22 months based on age group

• Hormonal profile for FT4

  Up to 22 months based on age group

• Frequency of dose adjustments

  Up to 22 months based on age group

• Number of events of TSH values above 4.5 mU/L

  Up to 22 months based on age group

• Number of events of FT4 values below the middle of the laboratory normal range

  Up to 22 months based on age group

Other Outcomes (2)

• Time to normalize TSH in neonates

  Up to 28 days

• Time to normalize FT4 in neonates

  Up to 28 days

Study Arms (2)

Treatment

EXPERIMENTAL

Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.

Drug: Tirosint®-SOL

Control

ACTIVE COMPARATOR

Crushed levothyroxine sodium tablets

Drug: Levothyroxine Sodium

Interventions

Tirosint®-SOL DRUG

Dosage will be according to the USPI and Standard of Care.

Also known as: levothyroxine sodium oral solution (liquid)

Treatment

Levothyroxine Sodium DRUG

Tablets will be crushed and dissolved in solution. Dosage will be according to the USPI and Standard of Care.

Also known as: levothyroxine sodium tablets

Control

Eligibility Criteria

• Age: 1 Day - 9 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• IBSA Institut Biochimique SA: lead
• Cromsource: collaborator

Study Sites (10)

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

CHOC Children's Hospital

Orange, California, 92868, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Yale University

New Haven, Connecticut, 06511, United States

Location

Children's Hospital of Atlanta

Atlanta, Georgia, 30329, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Mercy Hospital and Clinics

Kansas City, Missouri, 64111, United States

Location

Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Cook Children's Health Care Systems

Fort Worth, Texas, 76104, United States

Location

MeSH Terms

Conditions

Congenital Hypothyroidism

Interventions

Thyroxine; Fluid Therapy

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Endocrine System Diseases; Hypothyroidism; Thyroid Diseases

Intervention Hierarchy (Ancestors)

Thyroid Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Drug Therapy; Therapeutics

Study Officials

• Giuseppe Mautone

  IBSA Head of R&D Scientific Affairs

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2021
• First Posted: February 8, 2022
• Study Start: January 21, 2022
• Primary Completion: March 27, 2025
• Study Completion: March 27, 2025
• Last Updated: October 23, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)