Congenital Adrenal HyperplasiaPrenatal DexNCT02795871
Prenatal Dex Study
A study testing Neuropsychological and cognitive assessment in people with Congenital Adrenal Hyperplasia, run by Hospices Civils de Lyon at 10 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 15 yearsHealthy volunteers can take part
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- What is being tested?
- Neuropsychological and cognitive assessment
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 354 participants
- Where
- 10 sitesFrance
- Study dates
- 4 Oct 2016to 13 Jun 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 15 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- 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
Show all 12Show fewer
- 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
Who cannot join
- 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 locations and contacts
10 sites in 1 country.
10 sites in 1 country
France10 sites
Service d'endocrinologie pédiatrique, CHU de Besançon
Besançon
Service d'endocrinologie pédiatrique, CHU de Bordeaux
Bordeaux
Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est. 59 boulevard Pinel
Bron, 69677
Service d'endocrinologie pédiatrique, CHU de Grenoble
Grenoble
Service d'endocrinologie pédiatrique, CHRU de Lille
Lille
Service d'endocrinologie pédiatrique, Hopital de la Timone, APHM
Marseille
Service d'endocrinologie pédiatrique, CHU de Nantes
Nantes
Service d'endocrinologie pédiatrique, CHU de Nice
Nice
Service d'endocrinologie pédiatrique, Hôpital Armand Trousseau, AP-HP
Paris
Service d'endocrinologie pédiatrique, CHU de Reims
Reims
About this study
Official title: Multicentric Evaluation of in Utero Dexamethasone (DEX) on the Cognitive Development of Children at Risk of Congenital Adrenal Hyperplasia
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.
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- Neuropsychological and cognitive assessment
- Keywords
- Congenital Adrenal Hyperplasia, Prenatal treatment, Dexamethasone, Neuropsychological assessment, Cognitive development
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neuropsychological and cognitive assessmentBehavioural
| Study group | Role | What they receive |
|---|---|---|
| Group D+ 1 | Experimental | Girls and boys at risk of CAH treated in utero by Dexamethasone but unaffected. Receives: Behavioral: Neuropsychological and cognitive assessment |
| Group D+ 2 | Experimental | Girls and boys affected by CAH and treated in utero by Dexamethasone. Receives: Behavioral: Neuropsychological and cognitive assessment |
| : Group D - 1 | Active comparator | Girls and boys not affected by CAH and not treated in utero by Dexamethasone. Receives: Behavioral: Neuropsychological and cognitive assessment |
| Group D - 2 | Active comparator | Girls and boys affected by CAH and not treated in utero by Dexamethasone. Receives: Behavioral: Neuropsychological and cognitive assessment |
| Group D - 3 | Other | Girls and boys enrolled in school closed to Lyon Receives: Behavioral: Neuropsychological and cognitive assessment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Intelligence quotient
Wechsler Intelligence Scale for Children (WISC) is designed to measure human intelligence. WISC-IV includes:
- Verbal Comprehension Index: measure of verbal concept formation.
- Perceptual Reasoning Index: measure of non-verbal and fluid reasoning.
- Working Memory Index: measure of working memory.
- Processing Speed Index: measure of processing speed. Data measured: raw score (0 to 150), standard score (1 to 19), index (40 to 160).
The four indexes of the intelligence quotient will be considered hierarchically as follows: first the Working Memory Index, then the Processing Speed Index, third the Perceptual Reasoning Index, fourth the Verbal Comprehension Index.
Measured: 3 months
Secondary outcomes (6)
Children Memory Scale (CMS)
Neurocognitive profile of children assessed by Children Memory Scale (CMS) at Visit 2.
Memory tests: Children Memory Scale (CMS), the following composites will be considered in our study:
- Immediate and delayed visual memory : face recognition and dot location
- Immediate and delayed verbal memory, recognition : stories, word pairs
- Learning: dot location and word pairs
- Attention, concentration: numbers, sequences
Measured: 3 months
Rey figure test
Neurocognitive profile of children assessed by visuoperceptual test (Rey Figure test) at Visit 2
Measured: 3 months
Revised - Child Measure of Anxiety Scale (R- CMAS) score
Emotional profile assessed by Revised - Child Measure of Anxiety Scale (R- CMAS) score
Measured: 15 months
Mood Depressive Inventory for Children (MDI- C) score
Emotional profile assessed by Mood Depressive Inventory for Children (MDI- C) score
Measured: 15 months
Child Behavior Checklist (CBCL) score
Emotional profile assessed by Child Behavior Checklist (CBCL) score
Measured: 15 months
Evaluation of the benefits of in utero DEX in preventing or reducing the virilization of external genitalia in CAH girls
The anatomical evaluation (phenotyping) of girls' external genitalia was performed at the time of the surgical correction and included the measurement of the genital tubercule (GT), the distance between the perineum and the urethra-vaginal confluence, the degree of fusion of the genital folds (labia majora), their pigmentation and creases. The measurement of the ano-genital distance was not routine practice in the past.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2016 (estimated)
- Study start4 Oct 2016
- Primary completion13 Jun 2025
- Study completion13 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 354 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Véronique TARDY-GUIDOLLET, MD PHD, Principal investigator, Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02795871
- Sponsor's protocol ID
- 69HCL14_0447
- Acronym
- Prenatal Dex
- Status last verified
- Dec 2025
- First submitted
- 25 May 2016
- Last updated
- 11 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentric Evaluation of in Utero Dexamethasone (DEX) on the Cognitive Development of Children at Risk of Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT02795871
- Acronym / sponsor ID
- Prenatal Dex / 69HCL14_0447
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- Congenital Adrenal Hyperplasia, Prenatal treatment, Dexamethasone, Neuropsychological assessment, Cognitive development
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 354 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 4 Oct 2016 (actual)
- Primary completion
- 13 Jun 2025 (actual)
- Study completion
- 13 Jun 2025 (actual)
- First posted
- 10 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Group D+ 1 | Experimental | Behavioral: Neuropsychological and cognitive assessmentGirls and boys at risk of CAH treated in utero by Dexamethasone but unaffected. |
| Group D+ 2 | Experimental | Behavioral: Neuropsychological and cognitive assessmentGirls and boys affected by CAH and treated in utero by Dexamethasone. |
| : Group D - 1 | Active comparator | Behavioral: Neuropsychological and cognitive assessmentGirls and boys not affected by CAH and not treated in utero by Dexamethasone. |
| Group D - 2 | Active comparator | Behavioral: Neuropsychological and cognitive assessmentGirls and boys affected by CAH and not treated in utero by Dexamethasone. |
| Group D - 3 | Other | Behavioral: Neuropsychological and cognitive assessmentGirls and boys enrolled in school closed to Lyon |
Who may qualify
- Ages
- 6 to 15 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Neuropsychological and cognitive assessment | Behavioural | : Group D - 1, Group D - 2, Group D - 3, Group D+ 1, Group D+ 2 |
Primary outcome
Intelligence quotient
Time frame: 3 months
Wechsler Intelligence Scale for Children (WISC) is designed to measure human intelligence. WISC-IV includes: * Verbal Comprehension Index: measure of verbal concept formation. * Perceptual Reasoning Index: measure of non-verbal and fluid reasoning. * Working Memory Index: measure of working memory. * Processing Speed Index: measure of processing speed. Data measured: raw score (0 to 150), standard score (1 to 19), index (40 to 160). The four indexes of the intelligence quotient will be considered hierarchically as follows: first the Working Memory Index, then the Processing Speed Index, third the Perceptual Reasoning Index, fourth the Verbal Comprehension Index.
6 secondary outcomes
Children Memory Scale (CMS)
Time frame: 3 months
Rey figure test
Time frame: 3 months
Revised - Child Measure of Anxiety Scale (R- CMAS) score
Time frame: 15 months
Mood Depressive Inventory for Children (MDI- C) score
Time frame: 15 months
Child Behavior Checklist (CBCL) score
Time frame: 15 months
Evaluation of the benefits of in utero DEX in preventing or reducing the virilization of external genitalia in CAH girls
Time frame: Day 1
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Véronique TARDY-GUIDOLLET, MD PHD principal investigator
Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Neuropsychological and cognitive assessment in people with Congenital Adrenal Hyperplasia, run by Hospices Civils de Lyon at 10 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Intelligence quotient (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Dec 2025.