Diagnosis and Clinical Genetics of Cushing Syndrome in Pediatrics.
Constantine A Stratakis
https://pubmed.ncbi.nlm.nih.gov/27241967Actively Recruiting
Age: 2Years - 90Years
All Genders
Healthy Volunteers
ID03831958
Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
Led by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · Updated on 2026-06-01
600
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Cushing Disease (CD) is caused by pituitary tumors that produce excess cortisol, leading to various health problems. This research focuses on understanding the long-term effects of high cortisol levels experienced during childhood and adolescence, especially after the disease has been treated and cortisol levels have normalized. The study aims to clarify complications like hormone imbalances, metabolic issues, and neurocognitive effects that may persist or develop over time in those who had pediatric CD. Participants include individuals diagnosed and successfully treated for pediatric CD before age 21, who now have normal or low cortisol levels. Family members of these patients may also join for genetic studies. The study involves regular evaluations at 5, 10, 15, and 20 years after cure, including online surveys and optional in-person visits with physical exams, blood tests, imaging scans like DXA and MRI, and neurocognitive assessments. Additional tests such as hormone stimulation and urine collection may be done for participants with the disease. During the study, participants complete medical history reviews, questionnaires about physical and mental health, and undergo clinical assessments to monitor body mass index and other health measures. Researchers track changes in endocrine function, bone density, metabolism, cardiovascular health, immune system, and quality of life over time. The study includes ongoing safety monitoring and may last up to 20 years to provide long-term insights into the effects of pediatric Cushing Disease and guide future care.
CONDITIONS
Brief Title
Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
Who Can Participate
Age: 2Years - 90Years
All Genders
Healthy Volunteers
Eligibility Criteria
You may qualify if you...
- Males and females aged 10 to 42 years who were diagnosed with and successfully treated for Cushing Disease before age 21
- Must have normal or low cortisol levels documented within the past 6 months
- Patients who had surgery, radiation, or medical treatment are eligible
- Patients or legal guardians must provide consent or assent
- Family members aged 2 to 90 years of patients with a family history of pituitary tumors who agree to DNA/linkage analysis
You will not qualify if you...
- Pregnancy
- Medical, physical, psychiatric, or social conditions that would make study participation unsafe or not in the participant's best interest
- Critically ill, unstable, or severe organ failure limiting endocrine evaluation
- Such patients may still participate in the online questionnaire but not the on-site visit
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person or virtual)
Long-term Monitoring
Duration - Up to 20 years after documented cure, with assessments at 5, 10, 15, and 20 years (+/-1 year)
Participants previously treated for pediatric Cushing Disease are observed at specific intervals to assess their health status over time, including medical diagnoses, endocrine function, neurocognitive function, and quality of life.
Online surveys and optional on-site short visits at NIH offered at each assessment interval
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
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Research Team
D
Deborah P Merke, M.D.
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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Published Research Related To This Trial
Final adult height and body mass index after cure of paediatric Cushing's disease.
J H Davies, H L Storr, K Davies...
https://pubmed.ncbi.nlm.nih.gov/15807878Anthropometric measures and fasting insulin levels in children before and after cure of Cushing syndrome.
Margaret F Keil, Jennifer Graf, Nirmal Gokarn...
https://pubmed.ncbi.nlm.nih.gov/22154461