Comparison of Levothyroxine Formulations in the Treatment of Congenital Hypothyroidism

NCT06724224 | Recruiting

• 2 other identifiers: TreatCHPed012/2024/Oss/AOUBo
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 1

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.

Conditions

Congenital Hypothyroidism

Outcome Measures

Primary Outcomes (4)

• Mean and median values of TSH

  microU/mL

  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

  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

  at 1-3 years of age

• Proportion of patients with adverse effects

  hypersensitivity reactions, tachycardia, irritability, headache, sweating, diarrhea, vomiting, heat intolerance

  at 7-15 days, at 1-3-6-12 months and thereafter annually until 3 years after the start of therapy

Eligibility Criteria

• Age: Up to 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• IRCCS Azienda Ospedaliero-Universitaria di Bologna: lead

Study Sites (1)

IRCCS Azienda Ospedaliero-Universitaria di Bologna

Bologna, Bologna, 40138, Italy

RECRUITING

MeSH Terms

Conditions

Congenital Hypothyroidism

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Endocrine System Diseases; Hypothyroidism; Thyroid Diseases

Study Officials

• Rita Ortolano, MD

  IRCCS Azienda Ospedaliero-Universitaria di Bologna

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rita Ortolano, MD

CONTACT

0512144816; rita.ortolano@aosp.bo.it

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: September 24, 2024
• First Posted: December 9, 2024
• Study Start: April 8, 2024
• Primary Completion (Estimated): April 8, 2031
• Study Completion (Estimated): October 8, 2031
• Last Updated: March 12, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)