Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)
- Conditions
- Congenital Hypothyroidism
- Interventions
- Drug: Tirosint®-SOL
- Registration Number
- NCT05228184
- Lead Sponsor
- IBSA Institut Biochimique SA
- 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).
- 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 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 & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 34
-
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.
-
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;
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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
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Treatment Tirosint®-SOL 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. Control Levothyroxine Sodium Crushed levothyroxine sodium tablets
- Primary Outcome Measures
Name Time Method LT4 dose required to maintain TSH in target range (unit: mcg/kg/day) Up to 22 months based on age group 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.
- Secondary Outcome Measures
Name Time Method Growth pattern for length of body Up to 22 months based on age group Subject growth measurements in Length (cm) of body
Frequency of dose adjustments Up to 22 months based on age group Percent number of subjects (%) who need a dose adjustment in the long-term follow up phase
Number of events of FT4 values below the middle of the laboratory normal range Up to 22 months based on age group FT4 value below the middle of the laboratory normal range
Hormonal profile for FT4 Up to 22 months based on age group FT4 (unit ng/dL)
Hormonal profile for TSH Up to 22 months based on age group TSH (unit mU/L)
Parent/caregiver reports of satisfaction and ease of administration Up to 22 months based on age group 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.Number of events of TSH values above 4.5 mU/L Up to 22 months based on age group TSH value above 4.5 mU/L
Subject acceptance of the treatment (CareCAT) Up to 22 months based on age group 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
Signs and symptoms of hypothyroidism Up to 22 months based on age group Total number of subjects experiencing hypothyroidism's signs and symptoms
Growth pattern for body weight Up to 22 months based on age group Subject growth measurements in Body weight (kg)
Growth pattern for head circumference Up to 22 months based on age group Subject growth measurements in Head circumference (cm)
Signs and symptoms of hyperthyroidism Up to 22 months based on age group Total number of subjects experiencing hyperthyroidism's signs and symptoms
Trial Locations
- Locations (12)
CHOC Children's Hospital
🇺🇸Orange, California, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Children's Hospital of Atlanta
🇺🇸Atlanta, Georgia, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Cook Children's Health Care Systems
🇺🇸Fort Worth, Texas, United States
University of California San Francisco
🇺🇸San Francisco, California, United States
Yale University
🇺🇸New Haven, Connecticut, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Mercy Hospital and Clinics
🇺🇸Kansas City, Missouri, United States
Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States