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Prenatal Dex Study (Prenatal Dex)

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ClinicalTrials.gov Identifier: NCT02795871
Recruitment Status : Recruiting
First Posted : June 10, 2016
Last Update Posted : February 23, 2017
Sponsor:
Information provided by (Responsible Party):
Hospices Civils de Lyon

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.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Behavioral: Neuropsychological and cognitive assessment Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 500 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Multicentric Evaluation of in Utero Dexamethasone (DEX) on the Cognitive Development of Children at Risk of Congenital Adrenal Hyperplasia
Actual Study Start Date : October 2016
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: Group D+ 1
Girls and boys at risk of CAH treated in utero by Dexamethasone but unaffected.
Behavioral: Neuropsychological and cognitive assessment
Experimental: Group D+ 2
Girls and boys affected by CAH and treated in utero by Dexamethasone.
Behavioral: Neuropsychological and cognitive assessment
Active Comparator: : Group D - 1
Girls and boys not affected by CAH and not treated in utero by Dexamethasone.
Behavioral: Neuropsychological and cognitive assessment
Active Comparator: Group D - 2
Girls and boys affected by CAH and not treated in utero by Dexamethasone.
Behavioral: Neuropsychological and cognitive assessment
Group D - 3
Girls and boys enrolled in school closed to Lyon
Behavioral: Neuropsychological and cognitive assessment



Primary Outcome Measures :
  1. 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.



Secondary Outcome Measures :
  1. Children Memory Scale (CMS) [ Time Frame: 3 months ]

    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

  2. Rey figure test [ Time Frame: 3 months ]
    Neurocognitive profile of children assessed by visuoperceptual test (Rey Figure test) at Visit 2

  3. Revised - Child Measure of Anxiety Scale (R- CMAS) score [ Time Frame: 15 months ]
    Emotional profile assessed by Revised - Child Measure of Anxiety Scale (R- CMAS) score

  4. Mood Depressive Inventory for Children (MDI- C) score [ Time Frame: 15 months ]
    Emotional profile assessed by Mood Depressive Inventory for Children (MDI- C) score

  5. Child Behavior Checklist (CBCL) score [ Time Frame: 15 months ]
    Emotional profile assessed by Child Behavior Checklist (CBCL) score

  6. Evaluation of the benefits of in utero DEX in preventing or reducing the virilization of external genitalia in CAH girls [ Time Frame: Day 1 ]
    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.



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Ages Eligible for Study:   6 Years to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02795871


Contacts
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Contact: Véronique TARDY-GUIDOLLET, MD PHD (0)4.72.12.96.35 ext +33 veronique.tardy@chu-lyon.fr
Contact: Annie IUNG, PHD (0)4 72 40 68 24 annie.iung@chu-lyon.fr

Locations
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France
Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est. 59 boulevard Pinel Recruiting
Bron, France, 69677
Contact: Véronique TARDY-GUIDOLLET, MD PHD    (0)4.72.12.96.35 ext +33    veronique.tardy@chu-lyon.fr   
Sponsors and Collaborators
Hospices Civils de Lyon
Investigators
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Principal Investigator: Véronique TARDY-GUIDOLLET, MD PHD Groupement Hospitalier Est - Laboratoire d'endocrinologie moléculaire et maladies rares - Centre de biologie et de pathologie Est.

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Responsible Party: Hospices Civils de Lyon
ClinicalTrials.gov Identifier: NCT02795871     History of Changes
Other Study ID Numbers: 69HCL14_0447
First Posted: June 10, 2016    Key Record Dates
Last Update Posted: February 23, 2017
Last Verified: February 2017

Keywords provided by Hospices Civils de Lyon:
Congenital Adrenal Hyperplasia
Prenatal treatment
Dexamethasone
Neuropsychological assessment
Cognitive development

Additional relevant MeSH terms:
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Hyperplasia
Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders
Dexamethasone
Dexamethasone acetate
BB 1101
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors