Recruiting

Observational Study

Sponsor:

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Code:

NCT03831958

Conditions

Cushing Disease

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Accepted

Study Details

Brief summary:

Background:

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.

Objective:

To find out the long-term effects of exposure to high levels of cortisol during childhood and adolescence.

Eligibility:

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

Design:

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:

Physical exam

Blood tests

Cheek swab

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.

...

Conditions

Cushing Disease

Study ID

NCT03831958

Start date

Mar 4, 2019

Status verified date

Jul 16, 2026

Completion date

Jan 3, 2040

Anticipated

Primary completion date

Jan 3, 2040

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Accepted

  • INCLUSION CRITERIA:

1. Males and females 10-42 years old (subjects) who were previously diagnosed and had successful treatment of CD before the age of 21 years old. Patients who have undergone therapies other than surgical resection (such as radiation or medical treatment) will be eligible to participate.
2. Normocortisolemia or hypocortisolemia at the time of the study (as documented within past 6 months of recruitment) documented as urine free cortisol or midnight/afternoon serum or salivary cortisol levels within or below the normal range or documented panhypopituitarism (on glucocorticoid replacement).
3. Patients or a legal guardian (in case of cognitively impaired adults or children) must provide assent/consent at the time of the recruitment.
4. Family members (2- 90 yrs.) of patients with a family history of pituitary tumors and who agree to participate in the DNA/linkage analysis study.

EXCLUSION CRITERIA:

1. Pregnancy
2. Patients with any medical, physical, psychiatric, or social conditions, which, in the opinion of the investigators, would make participation in this protocol not in their best interest, will be excluded from the on-site visit of the study. Patients who are critically ill, unstable, or with severe organ failure that may affect/limit the endocrine evaluation and place unsustainable demands on Clinical Center or NICHD resources will be excluded. They will still be offered the opportunity to participate in the online questionnaire part of the study.

Study Design

Enrollment

600 participants

Anticipated

Interventions and Outcome Measures

Arms

Family member

Family member of survivor of pediatric Cushing disease

Subjects

survivor of pediatric Cushing disease

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

Deborah P Merke, M.D.

(301) 496-0718dmerke@nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

800-411-1222prpl@cc.nih.gov

More Information

Sponsor

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Last update posted

Sep 1, 2026

Last verified

Jul 16, 2026

Keywords

  • Body Mass Index Z Score
  • Adult Height
  • Endocrine Function
  • Neuropsychological Outcome
  • Metabolic Syndrome
  • Natural History

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) on 2026-09-01.