RecruitingNCT06307990

Understanding, Diagnosis and Monitoring of Thyroid Hormone Action Defects

Advancing Understanding, Diagnosis and Monitoring of Thyroid Hormone Action Defects (ADAM-THAD)


Sponsor

Istituto Auxologico Italiano

Enrollment

150 participants

Start Date

Jan 1, 2023

Study Type

OBSERVATIONAL

Conditions

Summary

The goal of this observational study is to learn about the neurological and cardiological phenotype of patients with resistance to thyroid hormone (RTH) syndromes beta and alpha (RTHß and RTHa) due to dominant negative variants in the genes encoding the thyroid hormone receptors alpha (THRA) and beta (THRB). The main question\[s\] it aims to answer are: * Define frequency and improve early diagnosis for RTH syndromes * Developing tools to accelerate diagnosis of RTH syndromes * Development and validation of monitoring tools Participants, recruited at neonatal screening or from cohorts of patients with unexplained specific neuro-cognitive or cardiovascular phenotypes will be submitted to biochemical and genetic investigations. In addition pluripotent stem cells will be generated from peripheral blood cells of RTHs patients and studied in vitro to understand the molecular mechanisms underlying neurological and cardiovascular consequences. In vitro and clinical data, will be correlated to identify biomarkers for monitoring treatment.


Eligibility

Inclusion Criteria1

  • \- biochemical signature suggestive of RTHs syndromes at birth (a) or symptoms suggestive of RTHs syndromes (b) or known diagnosis of RTHs syndromes (c)

Exclusion Criteria1

  • none

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Interventions

GENETICNGS sequencing

analysis of candidate genes for RTHs syndromes, transporters defects or gene involved in thyroid hormone metabolism. Whole exome sequencing (WES) in a minority of cases

DIAGNOSTIC_TESTserological tests

assessment of T4, T3 and other TH metabolites (LC-MS) in serum and dried blood spots


Locations(2)

Istituto Auxologico Italiano IRCCS

Milan, Italy

Department of Endocrine & Metabolic Diseases, San Luca Hospital

Milan, Italy

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NCT06307990