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RecruitingNCT05237934Updated Sep 24, 2026

Tissue Procurement and Natural History Study of Neuroendocrine Neoplasms (NENs) Including Adrenocortical Carcinoma (ACC)

An observational study in Neuroendocrine Tumors and Carcinoma, Neuroendocrine, sponsored by National Cancer Institute (NCI). Recruiting at 1 site in United States. Open to participants aged 18 Years to 120 Years. Per ClinicalTrials.gov, last updated 2026-09-24.

Sponsored by National Cancer Institute (NCI) · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
300
Ages
18 Years to 120 Years
Sex
All
01

Study summary

Background:

Neuroendocrine neoplasm (NENs)are rare cancers arising from the neuroendocrine cells and can affect almost any part of the body. They vary from low grade neuroendocrine tumors (NETs) to high grade neuroendocrine carcinomas (NECs). These tumors often occur in the gastrointestinal tract, pancreas, lungs, adrenal medulla (pheochromocytomas) or adrenal cortex (adrenocortical cancer) and other areas of the body mentioned below:

  • Gastroenteropancreatic neuroendocrine tumors (GEP-NET): stomach, duodenum, pancreas, colon, appendix, etc.
  • Liver and gallbladder
  • Adrenal tumors
  • Pituitary gland
  • Thyroid gland: medullary thyroid carcinoma
  • Parathyroid tumors
  • Pulmonary neuroendocrine tumors: typical and atypical carcinoid, small cell lung cancer (SCLC), large cell neuroendocrine carcinoma (LCNEC)
  • Extrapulmonary small cell cancer
  • Peripheral nervous system tumors: paraganglioma, neuroblastoma)
  • Breast and genitourinary tract

Their rates are rising in the United States and worldwide. Researchers want to learn more about NENs through this natural history study.

Objective:

To study the natural history of people with NENs and obtain samples from them to learn more about the disease. The clinical management of all NETs is not standardized, with only a few FDA-approved therapies and we would like to learn which combination therapeutic approach should be used, how long treatment should be continued, and in what subgroup of NENs a particular treatment option should be used.

Eligibility:

People aged 18 and older who have or are suspected to have NENs or ACC.

Design:

Participants will be screened with a medical history.

Participants will have a physical exam. Their symptoms and their ability to perform their normal activities will be reviewed. They will have blood and urine tests.

Participants will receive recommendations for managing their disease and potential treatment options. They will be able to ask as many questions as they would like.

Participants may provide saliva and blood, a for research. They will give tumor samples from a previous surgery or biopsy.

Participants may have optional biopsies. During biopsies, cancer tissue will be obtained using a needle and syringe. Tissue will be taken from the liver, lung, or a lymph node. Participants may have an imaging scan or ultrasound to help locate the tumor or area to be biopsied. They will receive local anesthesia and may be sedated.

Participants will complete a questionnaire about their family medical history.

Participants will have follow-up visits every 12 months. They will have physical exams and give samples. If their health changes, they may have extra visits. If they cannot visit NIH, they (or their doctor) will be contacted by phone or email.

Participants will take part in the study for all their life.

Read the detailed description

Background:

  • Neuroendocrine neoplasms (NENs) are divided into neuroendocrine tumors (NETs) and neuroendocrine carcinomas (NECs). These are rare malignancies occurring for example in the gastrointestinal tract, islets of the pancreas, lung, adrenal medulla, thyroid C-cells, etc., and is a heterogeneous group of neoplasms with unique tumor biology, natural history, and clinical management issues.
  • The annual occurrence of NENs has been increasing in the United States (US) and worldwide. The current incidence in the US is about 6 per 100,000 persons a year and represents 0.46% of all malignancies.
  • Most NETs are sporadic, but they can be part of familial cancer syndromes such as multiple endocrine neoplasia type 1 (MEN1), neurofibromatosis type 1 (NF1), or Von Hippel-Lindau (VHL) syndrome. Poorly differentiated NECs are all high-grade carcinomas that resemble small cell carcinoma or large cell NEC of the lung.
  • Treatment for localized NETs is surgical resection, however, a variety of therapeutic options are available for patients with advanced NETs. When to apply a given option, what combination therapeutic approach should be used, how long treatment should be continued, and in what subgroup of patients a particular treatment option should be used is unclear and controversial.

Objective:

-To comprehensively and longitudinally evaluate the natural history of participants with NENs and allow sample acquisition for use in the study of NENs

Eligibility:

  • Participants with confirmed or suspected NENs including ACC
  • Age >=18 years old

Design:

  • This protocol is a bio-specimen collection and natural history protocol in which samples will be collected from participants with NENs (from well-differentiated to poorly differentiated neuroendocrine neoplasm).
  • An accrual ceiling of 300 participants is planned over an accrual period of 10 years.
02

Conditions studied

  • Neuroendocrine Tumors
  • Carcinoma, Neuroendocrine

Keywords

  • Clinical Outcome
  • Advice in the Management of Cancer
  • Patterns of Disease Progression
  • Response or Lack of Response to Therapeutic Interventions
  • Natural History
03

Who can participate

Ages eligible
18 Years to 120 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Probability sample

Study population

primary clinical

Inclusion criteria

  • Age >= 18 years old
  • The ability of the participant to understand and the willingness to sign a written consent document.
  • Participants with the documentation of:

    • histological or cytological confirmation of NENs or adrenocortical cancer

OR

--biochemical evidence of neuroendocrine tumor (serum/urinary) based on elevated levels of chromogranin A, pancreatic polypeptide, neuron-specific enolase, vasoactive intestinal polypeptide, serotonin (urinary 5-hydroxyindoleacetic acid (5-HIAA)), gastrin, somatostatin, catecholamines, metanephrines, calcitonin, fasting insulin, Cpeptide (proinsulin), glucagon, anterior pituitary hormones

OR

--Suspicion of NEN (from any site/origin) on axial imaging (computed tomography (CT)/ magnetic resonance imaging (MRI) / fluorodeoxyglucose (FDG) positron emission tomography (PET) / 68Ga-Dotatate scan

OR

--a germline genetic variant that predisposes to NETs including ACC.

Exclusion criteria

EXCLUSION CRITERIA:

None

04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
300 participants (estimated)

Groups and cohorts

  • Cohort 1

    Participants with confirmed/suspected NENs including ACC

05

What researchers measure

Primary outcomes

  1. natural history of neuroendocrine neoplasms including ACC

    To comprehensively and longitudinally evaluate the natural history of participants with NENs and allow sample acquisition for use in the study of NENs

    Time frame: ongoing

06

Study locations

1 of 1 sites recruiting
  • National Institutes of Health Clinical Center
    Bethesda, Maryland 20892, United States
    • For more information at the NIH Clinical Center contact National Cancer Institute Referral Office · Contact · 888-624-1937
    • Anna Rivero · Contact · anna.rivero@nih.gov · (301) 451-7007
    Recruiting
07

References and documents

Individual participant data

Plan to share: Yes — All IPD recorded in the medical record will be shared with intramural investigators upon request.@@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.

Supporting information: Study protocol, Sap, Icf

08

Registry details

Key details

Study ID
NCT05237934
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Feb 14, 2022
Start date
Apr 25, 2022
Primary completion
Dec 31, 2032 (estimated)
Completion
Dec 31, 2032 (estimated)
Last update
Sep 24, 2026

Study contacts

Anna Liza F Rivero
Contact
anna.rivero@nih.gov
(240) 858-7946
Jaydira Del Rivero, M.D.
Contact
delriveroj@mail.nih.gov
(240) 858-3851
Jaydira Del Rivero, M.D.
principal investigator · National Cancer Institute (NCI)

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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