Influence of Initial Levothyroxine Dose on Neurodevelopmental and Growth Outcomes in Congenital Hypothyroidism

Federico II University

Status and phase: Completed; Phase 4

Conditions: Congenital Hypothyroidism

Treatments: Drug: Levothyroxin

Study type: Interventional

Funder types: Other

Identifiers

NCT05371262

FARM8A8FHP

Details and patient eligibility

About

The primary objective of this study is to evaluate the risk-benefit profile of long-term treatment of two different initials treatment schemes with L-T4 on the neurodevelopmental and auxological outcomes in children with congenital hypothyroidism, diagnosed by neonatal screening in order to find the best dose of initial thyroid hormone replacement to assure the best long-term developmental outcome without any adverse effects on auxological, cardiovascular and skeletal outcomes. The secondary objective of the study is to evaluate the role of other factors that, in addition to the initial L-T4 therapy,can influence long-term neurodevelopmental and auxological outcomes as well as the cardiovascular system and bone metabolism outcomes.

Full description

Seventy-two neonates detected by the neonatal screening program for congenital hypothyroidism were randomly assigned to receive one of the two initial L-T4 replacement dose: Group A received an initial replacement dose of 10-12.5 mcg/kg/die and Group B received an initial replacement dose of 12.6-15 mcg/kg/die. The adequacy of treatment were monitored closely by clinical evaluation and regular measurement of FT4 and TSH. Cognitive development were evaluated through the Griffiths Mental Development Scales at the the age of 2 yeas. Cognitive and behavioural assessment at the age of 4 years were performed using the Wechsler Preschool and Primary scale of Intelligence. Growth were evaluate at the enrolment and at each visit. Skeletal maturation were evaluated at diagnosis and at the age of 1 and 4 years. At the age of 4 years a quantitative ultrasound measurements and cardiovascular evaluation were performed.

Enrollment: 72 patients

Sex: All

Ages: 3 to 29 days old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Congenital Hypothyroidism diagnosed by neonatal screening program
• Age less than 30 days at diagnosis
• TSH value at confirmatory diagnosis above 30 mU/l
• Caucasian ethnicity

Exclusion criteria

• Prematurity
• Major congenital malformations
• Neonatal diseases
• Chromosomopathies
• Known maternal thyroid diseases

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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72 participants in 2 patient groups

10-12.5 μg/kg/day of L-T4

Active Comparator group

Description:

Children with congenital hypothyroidism who received an initial L-T4 dose of 10-12.5 μg/kg/day.

Treatment:

Drug: Levothyroxin

12.6-15 μg/kg/day of L-T4

Active Comparator group

Description:

Children with congenital hypothyroidism who received an initial L-T4 dose of 12.6-15 μg/kg/day.

Treatment:

Drug: Levothyroxin