Neoplasms

All Conditions

Symptoms and General Pathology

Rare Diseases

Carcinoma

Neuroendocrine Tumors

Carcinoma, Neuroendocrine

Disease Progression

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Nerve Tissue

Adenocarcinoma

Neoplasms, Glandular and Epithelial

Neuroendocrine Tumor

Neuroepithelioma

Jaydira Del Rivero, M.D.

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.

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.

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

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

clinical presentation, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival

National Cancer Institute (NCI)

Gland and Hormone Related Diseases

Adrenocortical Carcinoma

Adrenal Cortex Neoplasms

Adrenal Gland Neoplasms

Endocrine Gland Neoplasms

Adrenal Cortex Diseases

Adrenal Gland Diseases

Endocrine System Diseases

Participants with confirmed/suspected NENs including ACC

Sarah Kelley

For more information at the NIH Clinical Center contact National Cancer Institute Referral Office

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, blood, and stool samples 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 6 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.

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.

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

.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.

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

Digestive System Diseases

Carcinoma, Hepatocellular

Liver Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Liver Diseases

Fibrolamellar Carcinoma

Tim F Greten, M.D.

Background:

Fibrolamellar Hepatocellular Carcinoma (FL-HCC) is a rare liver cancer. It usually occurs in young people who have no history of liver disease. Currently the only effective treatment option is surgery that removes the tumor and part of the liver. Researchers want to study the course of the disease to learn more about it.

Objective:

To collect samples from people with FL-HCC to learn more about the disease and help develop new treatments.

Eligibility:

People any age with FL-HCC

Design:

Participants must be enrolled on another NIH protocol.

Participants will have at least 1 study visit. They will have:

* A medical and cancer history
* A physical exam
* A review of their symptoms and their ability to do normal activities
* Tests to produce images of the body. They may have a scan (CT) that uses a small amount of radiation. Or they may have a scan (MRI) that uses a magnetic field. These will examine the chest, abdomen, and pelvis.
* Blood tests

Researchers will study previous tumor samples if they are available.

If participants come to NIH for visits on other studies, data will be collected about their disease, tests, treatments, and responses. Tumor tissue will be collected if participants are having it taken for a procedure.

All other participants will be contacted to collect this data. They will be contacted once a month for 1 year and 2 times a year after that.

Participants will be asked to contact researchers when their health changes. They may come in for more tests.

Background:

* Fibrolamellar hepatocellular carcinoma (FL-HCC) is a rare hepatocellular carcinoma accounting for 0.5-9% of primary liver cancer in various case series, which is usually not associated with elevated serum alpha fetoprotein (AFP) levels, is enriched in younger age groups, and is not associated with underlying liver disease.
* Reports on the characteristics of patients with FL-HCC as well as predictors of recurrence and survival are scarce, largely due to the rarity of this tumor. The only potentially curative treatment option for FL-HCC patients who have resectable disease is surgery: either liver resection (LR) or liver transplantation (LT). However, disease recurrence after complete surgical resection is high, ranging from 33-100%. The clinical outcome of patients with unresectable disease is suboptimal with median survival of less than 12 months and no patient surviving beyond 5 years.
* The role of neoadjuvant and adjuvant therapies, including systemic chemotherapy, remains poorly defined and has been reported to have only a modest or no therapeutic effect. Platinum-based chemotherapy in pediatric patients with FL-HCC resulted a partial response in 31% of patients on imaging but a 3-year survival of only 22%. To date no targeted therapy has been shown to be of any value in this disease.
* The natural history of FL-HCC varies greatly with most patients surviving only months while others can live with the disease for years. While one cannot exclude an immune or other host component as responsible for the diverse clinical courses, it is also possible that there may be a genetic basis for this phenomenon. A novel somatic recurrent 400 kb deletion on the short arm of chromosome 19, giving rise to an in-frame DNAJB1 PRKACA gene fusion was found in FL-HCC. mTOR signaling is significantly activated in FL-HCC compared to other liver malignancies. A bio-specimen repository will be a major step towards more comprehensive studies of this very rare and unusual tumor and allow us to begin to characterize subgroups within the disease.
* Patients with rare tumors seek expert advice in the management of their care. A natural history study would establish a more formal mechanism for such referrals, while allowing the systematic collection of epidemiologic data as well as much needed tumor samples.

Objective:

- Characterize the natural history of Fibrolamellar hepatocellular carcinoma (FL-HCC), including clinical presentation, family history, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival.

Eligibility:

- Subjects of all ages with histologically or cytologically proven FL-HCC.

Design:

* Participants will undergo a comprehensive study entry evaluation including clinical phenotyping and imaging of tumor sites. Computed tomography scans of the thorax, abdomen and pelvis may be performed if clinically indicated; occasionally, magnetic resonance imaging may be performed for the visualization of lesions in the liver, spine, or other anatomic sites if clinically indicated.
* Medical histories will be documented, and participants will be followed throughout the course of their illnesses, with attention to patterns of disease recurrence and progression, response to therapies and duration of responses. As part of this natural history study, growth rates of target tumor lesions will also be calculated throughout the course of the disease whenever available.
* Blood and tumor samples will be obtained at study entry and while on study.

Natural History Study of Children and Adults With Fibrolamellar Hepatocellular Carcinoma

A Natural History Study of Children and Adults With Fibrolamellar Hepatocellular Carcinoma

Characterize the natural history of Fibrolamellar hepatocellular carcinoma (FL- HCC), including clinical presentation, family history, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival

Study title	NCT ID	Conditions	Eligibility	Organization
Natural History Study of Children and Adults With Neuroendocrine Neoplasms (NEN)s	NCT04488263	Neuroendocrine ... Carcinoma, Neur...	3 Years -	National Institutes of Health Clinical Center (CC)
Tissue Procurement and Natural History Study of Neuroendocrine Neoplasms (NENs) Including Adrenocortical Carcinoma (ACC)	NCT05237934	Neuroendocrine ... Carcinoma, Neur...	18 Years -	National Institutes of Health Clinical Center (CC)
Tissue Procurement and Natural History Study of Neuroendocrine Neoplasms (NENs) Including Adrenocortical Carcinoma (ACC)	NCT05237934	Neuroendocrine ... Carcinoma, Neur...	18 Years -	National Institutes of Health Clinical Center (CC)
Natural History Study of Children and Adults With Fibrolamellar Hepatocellular Carcinoma	NCT03748927	Hepatocellular ... Hepatocellular ...	1 Year - 99 Years	National Institutes of Health Clinical Center (CC)
Natural History Study of Children and Adults With Neuroendocrine Neoplasms (NEN)s	NCT04488263	Neuroendocrine ... Carcinoma, Neur...	3 Years -	National Institutes of Health Clinical Center (CC)
Natural History Study of Children and Adults With Fibrolamellar Hepatocellular Carcinoma	NCT03748927	Hepatocellular ... Hepatocellular ...	1 Year - 99 Years	National Institutes of Health Clinical Center (CC)

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