Clinical trial NCT02795871: Prenatal Dex Study
Official title: Multicentric Evaluation of in Utero Dexamethasone (DEX) on the Cognitive Development of Children at Risk of Congenital Adrenal Hyperplasia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Congenital Adrenal Hyperplasia
Keywords: Congenital Adrenal Hyperplasia
Prenatal treatment
Dexamethasone
Neuropsychological assessment
Cognitive development
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2016-06-10
Enrollment: 500
Sex: ALL
Minimum age (years): 6
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: 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.


Eligibility criteria: 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

Exclusion Criteria:

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

Primary outcomes:
measure: Intelligence quotient
timeFrame: 3 months
Secondary outcomes:
measure: Children Memory Scale (CMS)
timeFrame: 3 months
measure: Rey figure test
timeFrame: 3 months
measure: Revised - Child Measure of Anxiety Scale (R- CMAS) score
timeFrame: 15 months
measure: Mood Depressive Inventory for Children (MDI- C) score
timeFrame: 15 months
measure: Child Behavior Checklist (CBCL) score
timeFrame: 15 months
measure: Evaluation of the benefits of in utero DEX in preventing or reducing the virilization of external genitalia in CAH girls
timeFrame: Day 1
Other outcomes:
Locations:
city: Bron
country: France
facility: Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est. 59 boulevard Pinel
Study officials:
affiliation: Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est.
name: Véronique TARDY-GUIDOLLET, MD PHD
role: PRINCIPAL_INVESTIGATOR
References: