Long-term effects of childhood Cushing disease

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

This study looks at the long-term health effects in people who were treated for Cushing disease as children to see how it has impacted their physical and mental well-being.

Quick facts

• Study type: Observational
• Enrollment: 600 (estimated)
• Ages: 2 Years to 90 Years
• Sex: All
• Sponsor: National Institutes of Health Clinical Center (CC) (nih)
• Drugs / interventions: radiation
• Locations: 1 site (Bethesda, Maryland)
• Trial ID: NCT03831958 on ClinicalTrials.gov

What this trial studies

This observational study aims to investigate the long-term consequences of Cushing disease in individuals who were diagnosed before the age of 21 and have since been cured. Participants aged 10-42 will undergo a medical history screening and complete an online survey regarding their physical and mental health. Follow-up visits will occur at five-year intervals to assess various health outcomes related to their previous exposure to high cortisol levels. The study seeks to clarify the persistent complications associated with childhood hypercortisolemia, including metabolic and endocrine issues.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 10-42 who were diagnosed with Cushing disease before age 21 and are currently cured with normal or low cortisol levels.

Not a fit: Patients who are currently pregnant or have significant medical, physical, or psychiatric conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term health challenges faced by survivors of pediatric Cushing disease, leading to improved management strategies.

How similar studies have performed: While previous studies have explored complications in adult patients, this study's focus on pediatric survivors is relatively novel and has not been extensively tested.

Eligibility criteria

* 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.

Where this trial is running

Bethesda, Maryland

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (RECRUITING)

Study contacts

• Principal investigator: Deborah P Merke, M.D. — Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
• Study coordinator: Deborah P Merke, M.D.

Conditions: Cushing Disease, Body Mass Index Z Score, Adult Height, Endocrine Function, Neuropsychological Outcome, Metabolic Syndrome, Natural History