Congenital Hypothyroidism Clinical Trial
Official title:
A Randomized Comparative Study Between Liquid (Tirosint®-SOL) and Tablet Formulations of Levothyroxine in Neonates and Infants With Congenital Hypothyroidism
Verified date | April 2024 |
Source | IBSA Institut Biochimique SA |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
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. |
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 |
Lead Sponsor | Collaborator |
---|---|
IBSA Institut Biochimique SA | Cromsource |
United States,
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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Withdrawn |
NCT01223638 -
The Prevalence of Hearing Loss Among Children With Congenital Hypothyroidism
|
N/A | |
Completed |
NCT01916018 -
Clinical and Genetic Analysis in Congenital Hypothyroidism Due to Thyroid Dysgenesis.
|
N/A | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Completed |
NCT02307175 -
A Study of 99m Tc Pertechnetate Produced in High Energy Cyclotron in Patients With Thyroid Scan Indication
|
N/A | |
Completed |
NCT00497575 -
Diagnosis and Follow-up of Patients With Subclinical Hypothyroidism
|
N/A | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Completed |
NCT00403390 -
Generic vs. Name-Brand Levothyroxine
|
N/A | |
Not yet recruiting |
NCT04734457 -
Final Height in Patients With CH Diagnosed by the Screening
|
||
Completed |
NCT05371262 -
Influence of Initial Levothyroxine Dose on Neurodevelopmental and Growth Outcomes in Congenital Hypothyroidism
|
Phase 4 | |
Recruiting |
NCT00505479 -
Iodine Status in Pregnant Women and Their Newborns: is Congenital Hypothyroidism Related to Iodine Deficiency in Pregnancy?
|
N/A | |
Completed |
NCT02374593 -
Targeted Levothyroxine Dosing in Infants With Congenital Hypothyroidism
|
N/A | |
Completed |
NCT00493103 -
TG Gene Mutations and Congenital Hypothyroidism
|
N/A | |
Recruiting |
NCT04712760 -
Congenital Hypothyroidism in Children With Eutopic Gland or Thyroid Hemiagenesis: Predictive Factors for Transient vs Permanent Hypothyroidism.
|
||
Completed |
NCT01488721 -
Clinical Evaluation of NeoPlex4 Assay and NeoPlex System
|
N/A |