To find out whether T4 treatment of Down syndrome newborns until the age of 2 years compared with placebo treatment results in better cognitive or motor development at the age of 10 years and 6-8 months, and whether it is safe with regard to nerve…
ID
Source
Brief title
Condition
- Chromosomal abnormalities, gene alterations and gene variants
- Thyroid gland disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Cognitive development determined with the *Snijders-Oomen Non-Verbale
Intelligentietest-Revisie* or with the mental scale of the Dutch second version
of the *Bayley Scales of Infant Development* (BSID-II, in children with a
severe delay).
Secondary outcome
Gross Motor development determined with the *Movement Assessment Battery for
Children* or with the psychomotor scale of the BSID-II (in children with a
developmental age lower than 3 years). Fine Motor Development determined with
the *Beery test of Visuo-Motor Integration*. Processing Speed measured with the
baseline speed computer task of the *Amsterdamse Neuropsychologische
Testbatterij*. Behavioural development determined with the *Vragenlijst omtrent
ontwikkeling en gedrag bij kinderen*. Nerve function (conduction velocity)
determined with a somatosensory evoked potentials test. Somatic growth and
(pubertal) development determined with physical examination. (Spontaneous)
thyroid function determined with thyroid ultrasonography and laboratory tests.
Background summary
Thyroid hormone is indispensable for normal (pre- and) postnatal brain growth
and development. As a group, young Down syndrome children have a mild form of
(congenital) hypothyroidism that is not detected by neonatal screening and that
may harm their brain development. To find out whether these children might
benefit from thyroxine (T4) treatment, we conducted a randomized clinical trial
comparing T4 and placebo treatment started in the neonatal period with
cognitive and motor development at the age of 2 years as primary outcome (trial
period: June 1999 to October 2003). In this trial we found that the T4 treated
Down syndrome children had a smaller motor developmental age delay and, in a
subgroup analysis, a smaller mental developmental age delay. Until now, data on
the long-term efficacy (with regard to the development outcome) and safety of
T4 treatment of Down syndrome children during their first 2 years of life are
lacking.
Study objective
To find out whether T4 treatment of Down syndrome newborns until the age of 2
years compared with placebo treatment results in better cognitive or motor
development at the age of 10 years and 6-8 months, and whether it is safe with
regard to nerve function, somatic growth and development, and (spontaneous)
thyroid function at that age.
Study design
Observational study.
Study burden and risks
If the T4-treated Down syndrome children have a better developmental outcome at
the age of 10 years and 6-8 months compared with the placebo-treated Down
syndrome children, and the T4 treatment proves to be safe, it will be a strong
argument in favour of adopting and implementing this treatment *standard care*
for all (new) Down syndrome newborns. The risks of the current study are small
and mainly concern pain associated with the blood collection and *discomfort*
associated with the evoked potentials test. The pain will be minimized by the
application of anaesthetic cream (Emla®) at the blood collection site. If the
discomfort is too great, the sensory evoked potentials test will be ended and
skipped in accordance with the codes of conduct described in paragraph 7.3.
Postbus 22660
1100 DD Amsterdam
NL
Postbus 22660
1100 DD Amsterdam
NL
Listed location countries
Age
Inclusion criteria
See item D3.
Exclusion criteria
None
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL29139.018.09 |