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| Sponsored by: |
Office of Rare Diseases (ORD) |
|---|---|
| Information provided by: | Office of Rare Diseases (ORD) |
| ClinicalTrials.gov Identifier: | NCT00617292 |
Purpose
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.
| Condition |
|---|
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Adrenal Hyperplasia, Congenital |
| Study Type: | Observational |
| Study Design: | Case Control, Prospective |
| Official Title: | Long-Term Outcome in Offspring and Mothers of Dexamethasone-Treated Pregnancies at Risk for Classical Congenital Adrenal Hyperplasia Owing to 21-Hydroxylase Deficiency |
| Estimated Enrollment: | 233 |
| Study Start Date: | January 2008 |
| Estimated Study Completion Date: | July 2009 |
| Estimated Primary Completion Date: | July 2009 (Final data collection date for primary outcome measure) |
| Groups/Cohorts |
|---|
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Category 1, Group 1
Children who have 21OHD and received prenatal dexamethasone treatment
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Category 1, Group 2
Children who have 21OHD and did not receive prenatal dexamethasone treatment (control)
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Category 2
Mothers of children who received prenatal dexamethasone treatment
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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.
Eligibility| Ages Eligible for Study: | 12 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | Yes |
| Sampling Method: | Non-Probability Sample |
Participants in this study will include children who received prenatal dexamethasone treatment as fetuses and their mothers.
Inclusion Criteria:
For all participants:
For children who received prenatal dexamethasone treatment:
For children in the control group:
For mothers:
Exclusion Criteria:
Contacts and Locations| Contact: Claire Gilbert | claire.gilbert@mssm.edu |
| United States, New York | |
| Mount Sinai School of Medicine | Recruiting |
| New York, New York, United States, 10029 | |
| Contact: Claire Gilbert, MS 212-241-7099 claire.gilbert@mssm.edu | |
| Principal Investigator: Maria I. New, MD | |
| Sub-Investigator: Madeline Harbison, MD | |
| Sub-Investigator: Karen Lin-Su, MD | |
| Sub-Investigator: Robert Wilson, PhD | |
| Sub-Investigator: Saroj Nimkarn, MD | |
| Sub-Investigator: Susan Baker, PhD | |
| Sub-Investigator: Heino Meyer-Bahlburg, PhD | |
| United States, Texas | |
| University of Texas Southwestern Medical Center | Not yet recruiting |
| Dallas, Texas, United States, 75390 | |
| Contact: Jean Wilson, MD 214-648-3494 jean.wilson@utsouthwestern.edu | |
| Principal Investigator: Jean Wilson, MD | |
| Sub-Investigator: Richard Auchus, MD, PhD | |
| Brazil, SP | |
| University of Sao Paolo | Not yet recruiting |
| Sao Paolo, SP, Brazil | |
| Contact: Ivo Arnhold, MD 55-11-3069-7512 iarnhold@usp.br | |
| Principal Investigator: Ivo Arnhold, MD | |
| Sub-Investigator: Berenice Mendonca, MD, PhD | |
| France | |
| University of Lyon | Recruiting |
| Lyon, France | |
| Contact: Pierre Chatelain, MD 04-72-38-58-73 pierre.chatelain@chu-lyon.fr | |
| Principal Investigator: Pierre Chatelain, MD | |
| Sub-Investigator: Maguelone Forest, MD, PhD | |
| Sub-Investigator: Michael David, MD | |
| Study Chair: | Maria I. New, MD | Mount Sinai School of Medicine |
More Information
| Responsible Party: | Mount Sinai School of Medicine ( Maria I. New, MD ) |
| Study ID Numbers: | RDCRN 5610 |
| Study First Received: | January 31, 2008 |
| Last Updated: | December 8, 2008 |
| ClinicalTrials.gov Identifier: | NCT00617292 History of Changes |
| Health Authority: | United States: Federal Government |
|
21-hydroxylase deficiency 21OHD CAH |
|
Anti-Inflammatory Agents Dexamethasone Gonadal Disorders Hormone Antagonists Hormones, Hormone Substitutes, and Hormone Antagonists Adrenal Gland Diseases Adrenogenital Syndrome Antiemetics Sex Differentiation Disorders Adrenocortical Hyperfunction Hormones 21-hydroxylase-deficient Congenital Adrenal Hyperplasia Metabolism, Inborn Errors |
Adrenal Hyperplasia Adrenal Hyperplasia, Congenital Epinephrine Metabolic Disorder Dexamethasone acetate Metabolic Diseases Antineoplastic Agents, Hormonal Endocrine System Diseases Glucocorticoids Hyperplasia Genetic Diseases, Inborn Endocrinopathy Peripheral Nervous System Agents |
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Anti-Inflammatory Agents Dexamethasone Antineoplastic Agents Gonadal Disorders Physiological Effects of Drugs Hormones, Hormone Substitutes, and Hormone Antagonists Adrenal Gland Diseases Adrenogenital Syndrome Antiemetics Sex Differentiation Disorders Adrenocortical Hyperfunction Hormones Metabolism, Inborn Errors Pathologic Processes |
Therapeutic Uses Adrenal Hyperplasia, Congenital Steroid Metabolism, Inborn Errors Metabolic Diseases Antineoplastic Agents, Hormonal Gastrointestinal Agents Endocrine System Diseases Glucocorticoids Pharmacologic Actions Hyperplasia Genetic Diseases, Inborn Autonomic Agents Peripheral Nervous System Agents Central Nervous System Agents |
