Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers

The recruitment status of this study is unknown because the information has not been verified recently.
Verified December 2008 by Office of Rare Diseases (ORD).
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00617292
First received: January 31, 2008
Last updated: December 8, 2008
Last verified: December 2008

January 31, 2008
December 8, 2008
January 2008
July 2009   (final data collection date for primary outcome measure)
  • Prevalence of hypertension and obesity [ Time Frame: Throughout the study ] [ Designated as safety issue: No ]
  • "Normal" masculinization of unaffected females treated prenatally with dexamethasone [ Time Frame: Throughout the study ] [ Designated as safety issue: No ]
  • Normal masculinization of male fetuses partially treated prenatally with dexamethasone [ Time Frame: Throughout the study ] [ Designated as safety issue: No ]
  • Memory-related cognitive function [ Time Frame: Throughout the study ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT00617292 on ClinicalTrials.gov Archive Site
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Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers
Long-Term Outcome in Offspring and Mothers of Dexamethasone-Treated Pregnancies at Risk for Classical Congenital Adrenal Hyperplasia Owing to 21-Hydroxylase Deficiency

Congenital adrenal hyperplasia (CAH) is a genetic disorder that affects the amount of steroids that the body forms. The most common form of CAH is 21-hydroxylase deficiency (21OHD), which leads to cortisol deficiency and causes the development of mature masculine characteristics in newborn, prepubescent, and grown females, and prepubescent males. Prenatal treatment with dexamethasone, a corticosteroid, has been shown to reduce the masculinization of genitalia. However, the long-term effects of dexamethasone on the children who received it as fetuses and on mothers who were exposed to it while they were pregnant have not been determined. This study will investigate potential long-term adverse side effects of prenatal dexamethasone treatment in children and young adults who received dexamethasone as fetuses and their mothers who were exposed to it during pregnancy.

CAH is a genetic steroidogenesis disorder. The most common form, 21OHD, leads to cortisol deficiency and, in turn, an excess of androgen, a hormone that promotes the development and maintenance of male sex characteristics. As a result of this androgen excess, prepubescent males and newborn, prepubescent, and grown females exhibit mature masculine characteristics. Prenatal treatment with dexamethasone, a corticosteroid that decreases androgen levels, has been shown to prevent the development of abnormal genitalia in female infants. The long-term effects of this treatment, however, have not been evaluated. This study will determine whether prenatal dexamethasone treatment causes any long-term side effects by examining children and young adults who received dexamethasone as fetuses and their mothers, who were exposed to dexamethasone while pregnant.

This study has three parts. In Part 1 of the study, participants will provide written consent for release of their medical records from their physicians. Participants' physicians will then complete a medical form and/or provide copies of selected medical records for each participant. Parts 2 and 3 can be completed in 1 day. In Part 2 of the study, participants will complete questionnaires in their homes. Participants will answer questions about the following experiences: medical procedures, such as hormone treatment and genital surgery; education; work; hobbies; play activities and chores during childhood; identification with the male or female gender; relationships with parents; interest in being a parent; and overall adjustment. Part 3 of the study will consist of neuropsychological testing at the study site. This testing will focus on memory, attention, and overall cognitive abilities.

Observational
Observational Model: Case Control
Time Perspective: Prospective
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Non-Probability Sample

Participants in this study will include children who received prenatal dexamethasone treatment as fetuses and their mothers.

Adrenal Hyperplasia, Congenital
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  • Category 1, Group 1
    Children who have 21OHD and received prenatal dexamethasone treatment
  • Category 1, Group 2
    Children who have 21OHD and did not receive prenatal dexamethasone treatment (control)
  • Category 2
    Mothers of children who received prenatal dexamethasone treatment
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
233
July 2009
July 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

For all participants:

  • English-speaking
  • Has undergone DNA testing for mutations in the CYP21A2 gene

For children who received prenatal dexamethasone treatment:

  • Genetic confirmation of 21OHD diagnosis
  • Received full or partial prenatal dexamethasone treatment

For children in the control group:

  • Did not receive prenatal dexamethasone treatment

For mothers:

  • History of at-risk pregnancy for a fetus affected with 21OHD
  • Genetic confirmation of child's diagnosis

Exclusion Criteria:

  • Any mental disorder that could prevent understanding of study materials
  • Current or past steroid use for reasons other than CAH (i.e., asthma, lupus, rheumatoid arthritis)
Both
12 Years and older
Yes
Contact: Claire Gilbert claire.gilbert@mssm.edu
United States,   Brazil,   France
 
NCT00617292
RDCRN 5610
Yes
Maria I. New, MD, Mount Sinai School of Medicine
Office of Rare Diseases (ORD)
Not Provided
Study Chair: Maria I. New, MD Mount Sinai School of Medicine
Office of Rare Diseases (ORD)
December 2008

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP