Metabolic Risk Assessment in Prepubertal Children With Congenital Hypothyroidism

NCT ID: NCT07126353

Last Updated: 2025-08-17

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Total Enrollment: 170 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2025-09-30
• Study Completion Date: 2026-10-31

Brief Summary

We propose a multicenter prospective study to define the prevalence and severity score of metabolic syndrome in a prepubertal pediatric cohort with congenital hypothyroidism, compared to a healthy and normal-weight pediatric population. These data will help to define whether hypothyroidism can be considered a risk factor for the metabolic health of the pediatric population. The possible identification of an at-risk metabolic profile will provide useful information to optimize the diagnostic and monitoring pathway for affected children.

Detailed Description

Primary congenital hypothyroidism is the most common endocrine disorder in childhood and can present in either a permanent or transient form. Prolonged thyroid hormone deficiency can cause widespread damage affecting multiple organs and systems. Exposure to thyroid hormone deficiency during fetal and/or neonatal life has particularly severe consequences on the central nervous system, leading to neurocognitive delay. Appropriate and timely hormone replacement therapy (L-thyroxine) can prevent such outcomes, provided it is initiated early.

Thyroid hormones play a crucial role not only in growth and organ development but also in metabolic homeostasis. Thyroid function lies at the crossroads of multiple metabolic pathways. They have multiple effects on glucose and lipid metabolism, specifically by increasing glucose levels, fatty acid oxidation in muscle and liver, and lipolysis in adipose tissue. They also contribute to blood pressure regulation, thereby influencing the prevalence of metabolic syndrome, which is itself a key predictor of type 2 diabetes, cardiovascular diseases, and neurodegenerative conditions.Thyroid function plays a central regulatory role at the intersection of key metabolic pathways. Although, the role of thyroid hormones in metabolic processes is well established, and a bidirectional relationship between metabolic dysfunction and thyroid hypofunction has been reported in the adult population, data on metabolic risk in pediatric patients with congenital hypothyroidism are currently lacking.

The primary aim of this multicenter project is to assess the prevalence of metabolic syndrome in patients with congenital hypothyroidism and to determine whether this population presents a higher metabolic risk profile compared to the general population. As secondary objectives, this prospective study aims to:

1. Define the prevalence and severity score of metabolic syndrome in a prepubertal pediatric cohort with congenital hypothyroidism, compared to a pediatric population with obesity.

2. Evaluate the correlations between individual metabolic dysfunction parameters and the clinical and hormonal profile (including thyroid hormone levels and thyroid hormone sensitivity indices).

3. Assess the correlations between the metabolic profile and renal function.

4. Assess the correlations between the hormonal profile and renal function. To achieve these objectives, auxological parameters, vital signs (including blood pressure and heart rate), and metabolic profile data (glucose and lipid profiles, renal function, and hormonal status - FT3, FT4, TSH and thyroid hormone resistence indices TSHI, TT4RI, TT3RI, TFQI, PTFQI) will be collected for each enrolled subject. These parameters will be evaluated on peripheral blood samples collected during routine blood monitoring already scheduled according to established follow up. The potential presence of metabolic syndrome will be evaluated in each enrolled patient. The severity of the metabolic disorder will be evaluated using the Metabolic Score (MetS).

Data analysis will be performed using the statistical packages R 4.0.5 (R Core Team, 2021) and STATA (version 15.1, 2017, Stata Corporation, College Station, Texas, USA).

Conditions

Congenital Hypothyroidism; Metabolic Syndrome

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Permanent congenital hypothyroidism

170 patients in prepubertal age.

prospective observational study (clinical, hormonal and auxological data)

Intervention Type: OTHER

We collect clinical, hormonal and auxological data and compare them between the two groups.

Healthy childrens normal weight

170 childrens in prepubertal age.

prospective observational study (clinical, hormonal and auxological data)

Intervention Type: OTHER

We collect clinical, hormonal and auxological data and compare them between the two groups.

Healthy childrens overweight/obese

170 patients in prepubertal age

prospective observational study (clinical, hormonal and auxological data)

Intervention Type: OTHER

We collect clinical, hormonal and auxological data and compare them between the two groups.

Interventions

prospective observational study (clinical, hormonal and auxological data)

We collect clinical, hormonal and auxological data and compare them between the two groups.

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Pubertal stage Tanner 1
• Permanent congenital hypothyroidism
• All ethnic groups
• Informed consent signature

Exclusion Criteria

• Age< 5 years
• Pubertal stage Tanner 2-5
• Transient congenital hypothyroidism or other type of hypothyroidism

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Federico II University

OTHER

Sponsor Role: collaborator

IRCCS Ospedale San Raffaele

OTHER

Sponsor Role: collaborator

Policlinico di Bari Giovanni XXIII

UNKNOWN

Sponsor Role: collaborator

Azienda Ospedaliero Universitaria Policlinico Modena

OTHER

Sponsor Role: collaborator

Azienda Ospedaliero-Universitaria di Parma

OTHER

Sponsor Role: collaborator

Azienda Ospedaliera Ospedale Infantile Regina Margherita Sant'Anna

OTHER

Sponsor Role: collaborator

Azienda Ospedaliera Sant'Anna

OTHER

Sponsor Role: collaborator

University Hospital Perugia

UNKNOWN

Sponsor Role: collaborator

University of L'Aquila

OTHER

Sponsor Role: collaborator

Clinica Pediatrica Università di Novara

UNKNOWN

Sponsor Role: collaborator

Santobono-Pausilpon Hospital

UNKNOWN

Sponsor Role: collaborator

Policlinico G . Martino, Messina Italy

UNKNOWN

Sponsor Role: collaborator

IRCCS Azienda Ospedaliero-Universitaria di Bologna

OTHER

Sponsor Role: collaborator

University Hospital of Ancona

UNKNOWN

Sponsor Role: collaborator

Buzzi Children's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Valeria Calcaterra

Prof

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Buzzi Children's Hospital

Milan, , Italy

Countries

Italy

Central Contacts

Valeria Calcaterra, MD

Role: CONTACT

valeria.calcaterra@unipv.it

+39 0263631

Facility Contacts

Valeria Calcaterra, Prof, MD

Role: primary

valeria.calcaterra@unipv.it

00390263635321

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Other Identifiers

CET 503-2024

Identifier Type: -

Identifier Source: org_study_id