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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05228184
Other study ID # 20US-T414
Secondary ID
Status Active, not recruiting
Phase Phase 4
First received
Last updated
Start date January 21, 2022
Est. completion date April 30, 2025

Study information

Verified date April 2024
Source IBSA Institut Biochimique SA
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


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 daily dose within the randomly assigned treatment group (dose adjustments are allowed, if needed based on laboratory parameters and clinical response). Once enrolled, subjects will be treated and followed for 12 months (±1.5 months), participating in 7-8 study visits, consisting of 6-7 inclinic and 1-2 (or more if follow-up visits are required) telemedicine (TM) visits. The total number of visits depends on the age at inclusion.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 126
Est. completion date April 30, 2025
Est. primary completion date April 30, 2025
Accepts healthy volunteers No
Gender All
Age group 1 Day to 9 Months
Eligibility Inclusion Criteria: - 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 Criteria: - 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Tirosint®-SOL
Dosage will be according to the USPI and Standard of Care.
Levothyroxine Sodium
Tablets will be crushed and dissolved in solution. Dosage will be according to the USPI and Standard of Care.

Locations

Country Name City State
United States Children's Hospital of Atlanta Atlanta Georgia
United States Boston Children's Hospital Boston Massachusetts
United States Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
United States Children's Hospital Medical Center Cincinnati Ohio
United States Cook Children's Health Care Systems Fort Worth Texas
United States Children's Mercy Hospital and Clinics Kansas City Missouri
United States Children's Hospital of Los Angeles Los Angeles California
United States Yale University New Haven Connecticut
United States Icahn School of Medicine at Mount Sinai New York New York
United States CHOC Children's Hospital Orange California
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States University of California San Francisco San Francisco California

Sponsors (2)

Lead Sponsor Collaborator
IBSA Institut Biochimique SA Cromsource

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Other Time to normalize TSH in neonates Time to normalize TSH into reference range (unit: days) Up to 28 days
Other Time to normalize FT4 in neonates Time to normalize FT4 into the upper half of the laboratory normal FT4 range (unit: days) Up to 28 days
Primary 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 Hormonal profile for TSH TSH (unit mU/L) Up to 22 months based on age group
Secondary Hormonal profile for FT4 FT4 (unit ng/dL) Up to 22 months based on age group
Secondary Frequency of dose adjustments Percent number of subjects (%) who need a dose adjustment in the long-term follow up phase Up to 22 months based on age group
Secondary Number of events of TSH values above 4.5 mU/L TSH value above 4.5 mU/L Up to 22 months based on age group
Secondary Number of events of FT4 values below the middle of the laboratory normal range FT4 value below the middle of the laboratory normal range Up to 22 months based on age group
Secondary Growth pattern for length of body Subject growth measurements in Length (cm) of body Up to 22 months based on age group
Secondary Growth pattern for body weight Subject growth measurements in Body weight (kg) Up to 22 months based on age group
Secondary Growth pattern for head circumference Subject growth measurements in Head circumference (cm) Up to 22 months based on age group
Secondary Parent/caregiver reports of satisfaction and ease of administration Parents Satisfaction Questionnaire is a study-specific tool measuring coping and mental well-being and satisfaction
Score: 1-Strongly disagree, 2-Disagree, 3-Neutral, 4-Agree, 5-Strongly agree.
Up to 22 months based on age group
Secondary Subject acceptance of the treatment (CareCAT) Caregiver Administered Children's Acceptance Tool (CareCAT) is a 5-point nominal scale used to assess the acceptance of oral medicines in infants and toddlers who are unable to verbally give their opinion about a medicine Up to 22 months based on age group
Secondary Signs and symptoms of hypothyroidism Total number of subjects experiencing hypothyroidism's signs and symptoms Up to 22 months based on age group
Secondary Signs and symptoms of hyperthyroidism Total number of subjects experiencing hyperthyroidism's signs and symptoms Up to 22 months based on age group
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