Natural History Study of Children and Adults With Neuroendocrine Neoplasms (NEN)s

Natural History Study of Children and Adults With Neuroendocrine Neoplasms (NENs)

Background:

Neuroendocrine neoplasms (NENs) are rare tumors that originate in neuroendocrine cells. NENs can affect almost any part of the body. People with low-grade tumors can live many years. But high-grade tumors can be very aggressive. Researchers want to learn more about this type of cancer. This may help them design better treatments and supportive care studies.

Objective:

To gain a better understanding of neuroendocrine neoplasms.

Eligibility:

People starting at age 3 and older who have or are suspected of having NENs and are enrolled in protocol 19C0016, Natural History and Biospecimen Acquisition Study for Children and Adults with Rare Solid Tumors

Design:

Participants will be screened with questions about their medical history. This may be done over the telephone or in person.

Participants medical records, test results, and imaging results will be reviewed. They may have scans and blood tests. They may sign a separate consent form for some of the tests.

Participants will complete paper or electronic surveys. The surveys will ask about the effects of cancer on their wellbeing.

Participants may give samples of their tumors from previous surgeries or biopsies. These samples will be used to study their tumor genes.

Participants will get advice on how to manage their NENs. They will also get recommendations about potential treatment options.

Participants home doctors will be contacted every 6 to 12 months. They will give medical data such as imaging and test results.

Participants may have follow-up visits at NIH every 6 to 12 months.

Participants will contact researchers if there are any changes in their tumor.

Participants will be followed on this study for life.

Study Overview

• Status: Terminated
• Conditions: Neuroendocrine Tumors; Carcinoma, Neuroendocrine

Detailed Description

Background:

• Neuroendocrine neoplasm (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 are heterogeneous group of neoplasms with unique tumor biology, natural history, and clinical management issues.
• Their incidence has increased over the last decade, with an incidence of 6 per 100,000 persons a year and they represent 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. Whereas poorly differentiated neuroendocrine carcinomas (NECs) are all high-grade carcinomas that resemble small cell carcinoma or large cell carcinoma 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 should be used is unclear and controversial.

Objective:

-Characterize the natural history of neuroendocrine neoplasms (NENs). Data will include clinical presentation, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival.

Eligibility:

• Subjects with confirmed or suspicion of NENs.
• Age greater than or equal to 3 years old

Design:

• This protocol is a subprotocol of protocol 19C0016 Natural History and Biospecimen Accrual Study for Children and Adults with Rare Solid Tumors . After enrollment on the master protocol and undergoing evaluations detailed in the master protocol, patients will be enrolled on this subprotocol specific for NENs.
• Medical histories will be documented, and patients followed throughout the course of their illnesses, with particular attention to patterns of disease recurrence and progression, response to therapies, duration of responses and hormone production in patients with hormone production as a manifestation of their disease. Tumor growth rates will also be calculated throughout the course of the disease.

• Study Type: Observational
• Enrollment (Actual): 30

Contacts and Locations

• Study Contact: Name: Sarah Kelley; Phone Number: (240) 753-1971; Email: sarah.kelley@nih.gov
• Study Contact Backup: Name: Jaydira Del Rivero, M.D.; Phone Number: (240) 858-3851; Email: delriveroj@mail.nih.gov

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Institutes of Health Clinical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

primary clinical

Description

• INCLUSION CRITERIA:
• Enrollment into NCI protocol 19C0016 'Natural History and Biospecimen Acquisition Study for Children and Adults with Rare Solid Tumors'.
• Age greater than or equal to 3 years old
• Ability of subject or parent/guardian to understand and the willingness to sign a written consent document.

• Subjects with:

  --histologically or cytologically documented NENs, 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-HIAA), gastrin, somatostatin, catecholamines, metanephrines, calcitonin, fasting insulin, C-peptide (proinsulin), glucagon, anterior pituitary hormones, OR
• Suspicion of NEN (from any site/origin) on axial imaging (CT/MRI/FDG, PET/68Ga-DOTATATE scan, FDOPA PET scan).

EXCLUSION CRITERIA:

None

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Cohort 1; Subjects with confirmed/suspected NENs.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
natural history of neuroendocrine neoplasms	clinical presentation, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival	ongoing

Collaborators and Investigators

• Sponsor: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Jaydira Del Rivero, M.D., National Cancer Institute (NCI)

Publications and helpful links

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): November 24, 2020
• Primary Completion (Actual): September 6, 2022
• Study Completion (Actual): January 11, 2023

Study Registration Dates

• First Submitted: July 24, 2020
• First Submitted That Met QC Criteria: July 24, 2020
• First Posted (Actual): July 27, 2020

Study Record Updates

• Last Update Posted (Estimate): January 16, 2023
• Last Update Submitted That Met QC Criteria: January 13, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Keywords: Natural History; Clinical Outcome; Advice in the Management of Cancer; Patterns of Disease Progression; Response or Lack of Response to Therapeutic Interventions
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neoplasms; Carcinoma, Neuroendocrine; Neuroendocrine Tumors
• Other Study ID Numbers: 200139; 20-C-0139