Informations générales (source: ClinicalTrials.gov)
Multicentric Evaluation of in Utero Dexamethasone (DEX) on the Cognitive Development of Children at Risk of Congenital Adrenal Hyperplasia (Prenatal Dex)
Interventional
N/A
Hospices Civils de Lyon (Voir sur ClinicalTrials)
octobre 2016
décembre 2019
29 juin 2024
The classic form of 21-hydroxylase deficiency (prevalence 1/15,000) is the most common
cause of congenital adrenal hyperplasia (CAH). This autosomic recessive disease is
responsible for virilization of the external genitalia in girls through androgen
hypersecretion during fetal life. Since 1984, the Lyon Pediatric Endocrinology group has
proposed prenatal dexamethasone (DEX) for all fetuses at risk of CAH With the aim of
preventing fetal androgen hypersecretion in affected girls and avoiding poor long-term
results from reconstructive surgery. Prenatal DEX was used in Europe and the USA but its
use was recently suspended: in 2007, a Swedish study conducted on 26 children treated
with DEX in utero for a short period of time reported cognitive impairments. These data
were not confirmed by an American study on the short-term DEX use, which showed potential
cognitive impairments in CAH children exposed to DEX for long periods of time. These
confusing and controversial results have caused the scientific community to question its
position and have resulted in the suspension of the use of prenatal DEX with drastic
consequences for CAH girls (virilization; genital surgery etc.). In this context, an
evaluation of neuropsychological development under in utero DEX is essential to validate
its indication for use during the prenatal period. This study will evaluate outcomes
using prospective cognitive and emotional assessments. It will first focus on the
unaffected children previously treated in utero in order to assess the adverse effects of
the drug. The study will then assess the children with CAH for whom DEX could have
beneficial effects.
Etablissements
| Les établissements hors Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est. 59 boulevard Pinel - 69677 - Bron - France | Véronique TARDY-GUIDOLLET, MD PHD | Contact (sur clinicalTrials) | |||
Critères
Tous
Inclusion Criteria:
Groups D+1, D+2, D-1 and D-2
- Male or female
- Patient with Congenital Adrenal Hyperplasia or sibling of a CAH patient
- Age: 6 to 15 years (at the time of inclusion)
- The subject's legal representatives have understood the information note/informed
consent form, obtained answers to all their questions and have given signed,
written, informed consent
- Subject with health insurance
Group D-3 (Schoolchildren)
- Male or female
- With no connection with Congenital Adrenal Hyperplasia
- Age: 6 to 15 years (at the time of inclusion)
- The subject's legal representatives have understood the information note/informed
consent form, obtained answers to all their questions and have given signed,
written, informed consent
- Subject with health insurance
Groups D+1, D+2, D-1 and D-2
- Male or female
- Patient with Congenital Adrenal Hyperplasia or sibling of a CAH patient
- Age: 6 to 15 years (at the time of inclusion)
- The subject's legal representatives have understood the information note/informed
consent form, obtained answers to all their questions and have given signed,
written, informed consent
- Subject with health insurance
Group D-3 (Schoolchildren)
- Male or female
- With no connection with Congenital Adrenal Hyperplasia
- Age: 6 to 15 years (at the time of inclusion)
- The subject's legal representatives have understood the information note/informed
consent form, obtained answers to all their questions and have given signed,
written, informed consent
- Subject with health insurance
For all groups :
- Patient/Subject with another genetic disease
- Patient/Subject with known neuropsychology disease(s)
- Patient/Subject whose mother has received another treatment during her pregnancy
with possible known adverse events on the neuropsychological development of the
child