Ignite Creation Date:
2025-12-24 @ 3:55 PM
Ignite Modification Date:
2026-02-22 @ 1:00 PM
Study NCT ID:
NCT00617292
Status:
UNKNOWN
Last Update Posted:
2008-12-09
First Post:
2008-01-31
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000312', 'term': 'Adrenal Hyperplasia, Congenital'}, {'id': 'C535979', 'term': 'Congenital adrenal hyperplasia due to 21 hydroxylase deficiency'}], 'ancestors': [{'id': 'D047808', 'term': 'Adrenogenital Syndrome'}, {'id': 'D012734', 'term': 'Disorders of Sex Development'}, {'id': 'D014564', 'term': 'Urogenital Abnormalities'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D043202', 'term': 'Steroid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D006058', 'term': 'Gonadal Disorders'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_CONTROL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 233}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2008-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2008-12', 'completionDateStruct': {'date': '2009-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2008-12-08', 'studyFirstSubmitDate': '2008-01-31', 'studyFirstSubmitQcDate': '2008-02-05', 'lastUpdatePostDateStruct': {'date': '2008-12-09', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2008-02-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2009-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Prevalence of hypertension and obesity', 'timeFrame': 'Throughout the study'}, {'measure': '"Normal" masculinization of unaffected females treated prenatally with dexamethasone', 'timeFrame': 'Throughout the study'}, {'measure': 'Normal masculinization of male fetuses partially treated prenatally with dexamethasone', 'timeFrame': 'Throughout the study'}, {'measure': 'Memory-related cognitive function', 'timeFrame': 'Throughout the study'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['21-hydroxylase deficiency', '21OHD', 'CAH'], 'conditions': ['Adrenal Hyperplasia, Congenital']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': "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.\n\nThis 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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Participants in this study will include children who received prenatal dexamethasone treatment as fetuses and their mothers.', 'healthyVolunteers': True, 'eligibilityCriteria': "Inclusion Criteria:\n\nFor all participants:\n\n* English-speaking\n* Has undergone DNA testing for mutations in the CYP21A2 gene\n\nFor children who received prenatal dexamethasone treatment:\n\n* Genetic confirmation of 21OHD diagnosis\n* Received full or partial prenatal dexamethasone treatment\n\nFor children in the control group:\n\n* Did not receive prenatal dexamethasone treatment\n\nFor mothers:\n\n* History of at-risk pregnancy for a fetus affected with 21OHD\n* Genetic confirmation of child's diagnosis\n\nExclusion Criteria:\n\n* Any mental disorder that could prevent understanding of study materials\n* Current or past steroid use for reasons other than CAH (i.e., asthma, lupus, rheumatoid arthritis)"}, 'identificationModule': {'nctId': 'NCT00617292', 'briefTitle': 'Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers', 'organization': {'class': 'NIH', 'fullName': 'Office of Rare Diseases (ORD)'}, 'officialTitle': 'Long-Term Outcome in Offspring and Mothers of Dexamethasone-Treated Pregnancies at Risk for Classical Congenital Adrenal Hyperplasia Owing to 21-Hydroxylase Deficiency', 'orgStudyIdInfo': {'id': 'RDCRN 5610'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Category 1, Group 1', 'description': 'Children who have 21OHD and received prenatal dexamethasone treatment'}, {'label': 'Category 1, Group 2', 'description': 'Children who have 21OHD and did not receive prenatal dexamethasone treatment (control)'}, {'label': 'Category 2', 'description': 'Mothers of children who received prenatal dexamethasone treatment'}]}, 'contactsLocationsModule': {'locations': [{'zip': '10029', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Claire Gilbert, MS', 'role': 'CONTACT', 'email': 'claire.gilbert@mssm.edu', 'phone': '212-241-7099'}, {'name': 'Maria I. New, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Madeline Harbison, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Karen Lin-Su, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Robert Wilson, PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Saroj Nimkarn, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Susan Baker, PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Heino Meyer-Bahlburg, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Mount Sinai School of Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jean Wilson, MD', 'role': 'CONTACT', 'email': 'jean.wilson@utsouthwestern.edu', 'phone': '214-648-3494'}, {'name': 'Jean Wilson, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Richard Auchus, MD, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'University of Texas Southwestern Medical Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'city': 'São Paulo', 'state': 'São Paulo', 'status': 'NOT_YET_RECRUITING', 'country': 'Brazil', 'contacts': [{'name': 'Ivo Arnhold, MD', 'role': 'CONTACT', 'email': 'iarnhold@usp.br', 'phone': '55-11-3069-7512'}, {'name': 'Ivo Arnhold, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Berenice Mendonca, MD, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'University of Sao Paolo', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'city': 'Lyon', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Pierre Chatelain, MD', 'role': 'CONTACT', 'email': 'pierre.chatelain@chu-lyon.fr', 'phone': '04-72-38-58-73'}, {'name': 'Pierre Chatelain, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Maguelone Forest, MD, PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Michael David, MD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'University of Lyon', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}], 'centralContacts': [{'name': 'Claire Gilbert', 'role': 'CONTACT', 'email': 'claire.gilbert@mssm.edu'}], 'overallOfficials': [{'name': 'Maria I. New, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Icahn School of Medicine at Mount Sinai'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Office of Rare Diseases (ORD)', 'class': 'NIH'}, 'responsibleParty': {'oldNameTitle': 'Maria I. New, MD', 'oldOrganization': 'Mount Sinai School of Medicine'}}}}