Long-Term Follow-Up of Survivors of Pediatric Cushing Disease

A vizsgálat adatai

Ki végzi

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

Azonosító

NCT03831958

A regiszteradat frissítve

2026. november 12.

Eredeti leírás

Background:A teljes eredeti leírásKevesebb

Long-Term Follow-UP of Survivors of Pediatric Cushing Disease

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 >=5 years 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.

Kizáró feltételek: 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; Pregnancy: Pregnancy will be evaluated only in participants of reproductive age (from 10 years old until 60 years of age unless menopause has already occurred per clinical report of the participant); For participants enrolled as external participants or under the Linkage study (where research activities include no more than blood draws), any female who could possibly become pregnant will be screened using clinical criteria (history, with pregnancy testing only if indicated) for exclusion and this information will be documented in the consent process note in EMR. If a participant has initially been registered as external location and then presents on-site, then pregnancy test will be performed if within the reproductive age group.

INCLUSION CRITERIA:; Males and females >=5 years of age or older (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; 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); Patients or a legal guardian (in case of cognitively impaired adults or children) must provide assent/consent at the time of the recruitment; Or,; 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.

ClinicalTrials.gov NCT03831958