Comparison of Levothyroxine Formulations in the Treatment of Congenital Hypothyroidism

Recruiting

• Conditions: Congenital Hypothyroidism

• Registration Number: NCT06724224
• Lead Sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2024-04-08
• Status Verified: 2024-09
• Study First Submitted: 2024-09-24
• Study First Submitted QC: 2024-12-06
• Last Update Submitted: 2024-12-06
• Study First Posted: 2024-12-09
• Last Update Posted: 2024-12-09
• Primary Completion: 2031-04-08
• Study Completion: 2031-10-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

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 informed consent 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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Mean and median values of TSH	at 7-15 days, at 1-3-6-12 months and thereafter annually until 3 years after the start of therapy	microU/mL
Mean and median values of FT4	at 7-15 days, at 1-3-6-12 months and thereafter annually until 3 years after the start of therapy	pg/mL
Neuromotor-Neurocognitive development	at 1-3 years of age	Griffiths Scale for patients aged 0-2 years; WPPSI-III Scale for patients aged 2.6-7.3 years
Proportion of patients with adverse effects	at 7-15 days, at 1-3-6-12 months and thereafter annually until 3 years after the start of therapy	hypersensitivity reactions, tachycardia, irritability, headache, sweating, diarrhea, vomiting, heat intolerance

Trial Locations

Locations (1)

IRCCS Azienda Ospedaliero-Universitaria di Bologna; 🇮🇹 Bologna, Italy