Prenatal Dex Study

Civil Hospices of Lyon

Status: Completed

Conditions: Congenital Adrenal Hyperplasia

Treatments: Behavioral: Neuropsychological and cognitive assessment

Study type: Interventional

Funder types: Other

Identifiers

NCT02795871

69HCL14_0447

Details and patient eligibility

About

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.

Enrollment: 354 patients

Sex: All

Ages: 6 to 15 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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354 participants in 5 patient groups

Group D+ 1

Experimental group

Description:

Girls and boys at risk of CAH treated in utero by Dexamethasone but unaffected.

Treatment:

Behavioral: Neuropsychological and cognitive assessment

Group D+ 2

Experimental group

Description:

Girls and boys affected by CAH and treated in utero by Dexamethasone.

Treatment:

Behavioral: Neuropsychological and cognitive assessment

: Group D - 1

Active Comparator group

Description:

Girls and boys not affected by CAH and not treated in utero by Dexamethasone.

Treatment:

Behavioral: Neuropsychological and cognitive assessment

Group D - 2

Active Comparator group

Description:

Girls and boys affected by CAH and not treated in utero by Dexamethasone.

Treatment:

Behavioral: Neuropsychological and cognitive assessment

Group D - 3

Other group

Description:

Girls and boys enrolled in school closed to Lyon

Treatment:

Behavioral: Neuropsychological and cognitive assessment