search
Back to results

Prenatal Dex Study (Prenatal Dex)

Primary Purpose

Congenital Adrenal Hyperplasia

Status
Unknown status
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Neuropsychological and cognitive assessment
Sponsored by
Hospices Civils de Lyon
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Congenital Adrenal Hyperplasia focused on measuring Congenital Adrenal Hyperplasia, Prenatal treatment, Dexamethasone, Neuropsychological assessment, Cognitive development

Eligibility Criteria

6 Years - 15 Years (Child)All SexesAccepts 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

Sites / Locations

  • Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est. 59 boulevard PinelRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Active Comparator

Active Comparator

Other

Arm Label

Group D+ 1

Group D+ 2

: Group D - 1

Group D - 2

Group D - 3

Arm Description

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

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

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

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

Girls and boys enrolled in school closed to Lyon

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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
Rey figure test
Neurocognitive profile of children assessed by visuoperceptual test (Rey Figure test) at Visit 2
Revised - Child Measure of Anxiety Scale (R- CMAS) score
Emotional profile assessed by Revised - Child Measure of Anxiety Scale (R- CMAS) score
Mood Depressive Inventory for Children (MDI- C) score
Emotional profile assessed by Mood Depressive Inventory for Children (MDI- C) score
Child Behavior Checklist (CBCL) score
Emotional profile assessed by Child Behavior Checklist (CBCL) score
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.

Full Information

First Posted
May 25, 2016
Last Updated
February 21, 2017
Sponsor
Hospices Civils de Lyon
search

1. Study Identification

Unique Protocol Identification Number
NCT02795871
Brief Title
Prenatal Dex Study
Acronym
Prenatal Dex
Official Title
Multicentric Evaluation of in Utero Dexamethasone (DEX) on the Cognitive Development of Children at Risk of Congenital Adrenal Hyperplasia
Study Type
Interventional

2. Study Status

Record Verification Date
February 2017
Overall Recruitment Status
Unknown status
Study Start Date
October 2016 (Actual)
Primary Completion Date
December 2018 (Anticipated)
Study Completion Date
December 2019 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hospices Civils de Lyon

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia
Keywords
Congenital Adrenal Hyperplasia, Prenatal treatment, Dexamethasone, Neuropsychological assessment, Cognitive development

7. Study Design

Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
500 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Group D+ 1
Arm Type
Experimental
Arm Description
Girls and boys at risk of CAH treated in utero by Dexamethasone but unaffected.
Arm Title
Group D+ 2
Arm Type
Experimental
Arm Description
Girls and boys affected by CAH and treated in utero by Dexamethasone.
Arm Title
: Group D - 1
Arm Type
Active Comparator
Arm Description
Girls and boys not affected by CAH and not treated in utero by Dexamethasone.
Arm Title
Group D - 2
Arm Type
Active Comparator
Arm Description
Girls and boys affected by CAH and not treated in utero by Dexamethasone.
Arm Title
Group D - 3
Arm Type
Other
Arm Description
Girls and boys enrolled in school closed to Lyon
Intervention Type
Behavioral
Intervention Name(s)
Neuropsychological and cognitive assessment
Primary Outcome Measure Information:
Title
Intelligence quotient
Description
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.
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Children Memory Scale (CMS)
Description
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
Time Frame
3 months
Title
Rey figure test
Description
Neurocognitive profile of children assessed by visuoperceptual test (Rey Figure test) at Visit 2
Time Frame
3 months
Title
Revised - Child Measure of Anxiety Scale (R- CMAS) score
Description
Emotional profile assessed by Revised - Child Measure of Anxiety Scale (R- CMAS) score
Time Frame
15 months
Title
Mood Depressive Inventory for Children (MDI- C) score
Description
Emotional profile assessed by Mood Depressive Inventory for Children (MDI- C) score
Time Frame
15 months
Title
Child Behavior Checklist (CBCL) score
Description
Emotional profile assessed by Child Behavior Checklist (CBCL) score
Time Frame
15 months
Title
Evaluation of the benefits of in utero DEX in preventing or reducing the virilization of external genitalia in CAH girls
Description
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.
Time Frame
Day 1

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Véronique TARDY-GUIDOLLET, MD PHD
Phone
(0)4.72.12.96.35
Ext
+33
Email
veronique.tardy@chu-lyon.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Annie IUNG, PHD
Phone
(0)4 72 40 68 24
Email
annie.iung@chu-lyon.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Véronique TARDY-GUIDOLLET, MD PHD
Organizational Affiliation
Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est.
Official's Role
Principal Investigator
Facility Information:
Facility Name
Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est. 59 boulevard Pinel
City
Bron
ZIP/Postal Code
69677
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Véronique TARDY-GUIDOLLET, MD PHD
Phone
(0)4.72.12.96.35
Ext
+33
Email
veronique.tardy@chu-lyon.fr

12. IPD Sharing Statement

Learn more about this trial

Prenatal Dex Study

We'll reach out to this number within 24 hrs