An observational study in Congenital Hypothyroidism, sponsored by IRCCS Azienda Ospedaliero-Universitaria di Bologna. Recruiting at 1 site in Italy. Open to participants aged Up to 11 Years. Per ClinicalTrials.gov, last updated 2026-03-12.
Sponsored by IRCCS Azienda Ospedaliero-Universitaria di Bologna · Observational
Observational, retrospective, prospective, pharmacological, single-centre, non-profit study.
The aim is to evaluate, in newborns diagnosed with Congenital Hypothyroidism, the medium- and long-term efficacy and safety of the new formulation of levothyroxine in oral solution, compared to the liquid drop formulation and the tablet formulation.
The study will involve male and female patients referred to the Neonatal Screening Centre at the Centre for Endocrine-Metabolic Diseases of the Paediatrics Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico di S. Orsola, Italy, who tested positive for Congenital Hypothyroidism.
The prospective cohort consists of patients with Congenital Hypothyroidism identified through Neonatal Screening, who come for observation at the Centre for Endocrine-Metabolic Diseases of the Pediatrics Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico di S. Orsola, Italy, from the approval of the study until the number of patients stipulated in the protocol is reached, which is expected to be achieved in about 4 years. The recruited patients will be assigned, based on the therapy taken in normal clinical practice, to a pharmacological group (group A: drops, group B: oral solution, group C: tablets), and their medical history, clinical and biochemical data will be collected at diagnosis.
The retrospective cohort includes children born from January 1, 2019 to the date of study approval, who came for observation at the Centre for Endocrine-Metabolic Diseases of the Pediatrics Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico di S. Orsola, Italy, for Congenital Hypothyroidism identified by Neonatal Screening. These patients had levothyroxine therapy in one of the three formulations (solid, drops, oral solution) assigned according to normal clinical practice and will therefore be divided into 3 groups as for the prospective cohort. For these patients, retrospective collection and analysis of anamnestic, clinical, biochemical and radiological data and clinical, biochemical and neurocognitive follow-up is planned with the same timelines defined for the prospective cohort.
27 studies on the registry are indexed under Congenital Hypothyroidism; 9 are open to participants now.
This study's planned enrollment of 120 is below the median of 241 across 14 observational studies indexed under Congenital Hypothyroidism.
Browse Congenital Hypothyroidism studies →IRCCS Azienda Ospedaliero-Universitaria di Bologna is the lead sponsor of 511 studies on the registry; 287 are open to participants now.
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Patients of both sex refer to the Neonatal Screening Center at the Center for Endocrine-Metabolic Diseases, Pediatrics Unit of the IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico di S.Orsola, Italy, who test positive for Neonatal Screening for Congenital Hypothyroidism.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Newborn screening test positivity for Congenital Hypothyroidism; Subjects born in Emilia-Romagna region, Italy, and undergoing diagnostic confirmation and initiation of replacement therapy; Diagnosis-confirmed subjects who underwent L-T4 replacement therapy in the first month of life at Center for Endocrine-Metabolic Diseases, Pediatrics Units of the IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico di S.Orsola, Italy; Age ≤11 years old; Obtaining from parents and/or legal guardians and assent from minors included in the study.
Exclusion Criteria:
Known chromosomal abnormalities or complex syndromes; Patients transferred to another center before completion of at least one year of follow-up from the start of therapy; Patients who started therapy at another center.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Mean and median values of TSH
microU/mL
Time frame: at 7-15 days, at 1-3-6-12 months and thereafter annually until 3 years after the start of therapy
Mean and median values of FT4
pg/mL
Time frame: at 7-15 days, at 1-3-6-12 months and thereafter annually until 3 years after the start of therapy
Neuromotor-Neurocognitive development
Griffiths Scale for patients aged 0-2 years; WPPSI-III Scale for patients aged 2.6-7.3 years
Time frame: at 1-3 years of age
Proportion of patients with adverse effects
hypersensitivity reactions, tachycardia, irritability, headache, sweating, diarrhea, vomiting, heat intolerance
Time frame: at 7-15 days, at 1-3-6-12 months and thereafter annually until 3 years after the start of therapy
Plan to share: No
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