Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03831958|
Recruitment Status : Recruiting
First Posted : February 6, 2019
Last Update Posted : March 8, 2019
The pituitary gland produces hormones. A tumor in this gland can cause it to produce too much of the hormone cortisol. Too much cortisol in the body causes Cushing disease. This disease causes many problems. Some of these problems might persist after the disease is cured.
To find out the long-term effects of exposure to high levels of cortisol during childhood and adolescence.
People ages 10-42years who were diagnosed with Cushing disease before age 21 and are now cured and have normal or low cortisol levels
People related to someone with Cushing disease
Participants will be screened with a medical history.
Participants will complete an online survey. This will include questions about their or their child s physical and mental health.
All participants will be seen at 5 -year intervals after cure of Cushing disease (5yr, 10yr, 15yr, 20yr (last visit))
Participants who have a relative with Cushing disease will have a medical history and blood tests or cheek swabs.
Participants who have the disease will have:
DXA scan: A machine will x-ray the participant s body to measure bone mineral content.
For participants who are still growing, a hand x-ray
Participants with the disease may also have:
Hormone stimulation test: Participants will get a hormone or another substance that will be measured.
Serial hormone sampling: Participants blood will be measured several times through a thin plastic tube in an arm vein.
Urine tests: Participants urine may be collected over 24 hours.
MRI: Participants may have a dye injected into a vein. They will lie on a table that slides into a machine. The machine will take pictures of the body.
|Condition or disease|
Cushing Disease (CD) describes the state of hypercortisolemia secondary to cortisol producing pituitary adenomas. The rarity of the disease (2-5 new cases per million, 1 out of 10 in children) and the subtle initial findings result in prolonged undiagnosed hypercortisolemia, that increases the risk for significant complications, including obesity, height deceleration, hyperlipidemia, hyperglycemia, hypertension, osteoporosis, immunodeficiency, and others. Although hypercortisolemia usually resolves after successful resection of the pituitary adenoma, the reversal of the abovementioned complications and the long-term effects of the previous prolonged exposure of the body to supraphysiologic levels of cortisol have not been clarified, especially when hypercortisolemia occurs during childhood.
Previous studies have addressed the possible complications after resolution of hypercortisolemia, but most of them refer to adult patients. Amongst the described complications, suppression of the pituitary hormones, such as the growth and thyroid hormone axes, and persistent increase of the body mass index (BMI) and abnormal fat distribution, have been described in limited number of pediatric patients followed for a few years after cure. Other complications, such as components of the metabolic syndrome or cardiovascular dysfunction, have not been extensively studied in children. Furthermore, equally important are the long-term effects of glucocorticoids on other aspects of health. For example, it has been previously described that the neurocognitive function of children with CD declines the first year after treatment. This can potentially result in impaired quality of life, lower education level, and lower job and life satisfaction in the future; however, the long-term neurocognitive sequelae of Cushing syndrome (CS) diagnosed in childhood have not been studied.
This study aims to provide novel insight on the long-term effects of hypercortisolemia on the developing child, their underlying pathogenetic mechanisms, their evolution over time, and the risk factors for developing them. This will assist in designing methods to closely monitor or prevent them in the future. Certain results of this study could potentially apply to children with iatrogenic CS, which is much more common due to the widespread use of pharmacologic doses of glucocorticoids in malignancies, autoimmune and atopic disorders.
|Study Type :||Observational|
|Estimated Enrollment :||600 participants|
|Official Title:||Long-Term Follow UP of Survivors of Pediatric Cushing Disease|
|Actual Study Start Date :||March 4, 2019|
|Estimated Primary Completion Date :||January 3, 2040|
|Estimated Study Completion Date :||January 3, 2040|
survivor of pediatric Cushing disease
Family member of survivor of pediatric Cushing disease
- Difference in body mass index z-score of subjects previously treated for Cushing disease compared to general population [ Time Frame: baseline, 5, 10, 15, 20 years ]The primary outcome measure of the study is the difference in the Body Mass Index (BMI) z-score of the patients previously treated for pediatric CD compared to the general population, as calculated by data derived from the NHANES study.
- Prevalence of endocrine and non-endocrine abnormalities [ Time Frame: baseline, 5, 10, 15, 20 years ]The secondary outcomes will be to describe the prevalence of other endocrine and non- endocrine abnormalities after successful treatment of CD in childhood, including: growth hormone, thyroid, gonadal, adrenal function; bone mineral density; glucose metabolism; lipid profile; cardiovascular abnormalities; immunologic changes; coagulation function; psychiatric diseases, behavioral symptomology; neurocognitive function; and quality of life.
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): NCT03831958
|Contact: Margaret F Keil, C.R.N.P.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||Margaret F Keil, C.R.N.P.||Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)|