Natural History of Familial Carcinoid Tumor
ConditionCarcinoid
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-100
SponsorNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
About this trial
This study will evaluate members in families with a history of small bowel carcinoid cancer to study the natural history of those family members that have the disease, determine ways to improve early detection by performing surveillance on those at risk but without disease and to identify the gene(s) that may cause the tumors. Familial carcinoid tumors usually originate in hormone-producing cells that line the small intestine or other cells of the digestive tract. The tumors are slow-growing and usually take many years before they cause symptoms. It is known that these tumors occur more often in some families and are then passed from one generation to the next by inherited genes.
Members of families, including all siblings and offspring in which two or more immediate blood relatives have had small bowel carcinoid tumors are eligible for this study. In some cases unaffected spouses of family members diagnosed with carcinoid cancer are also requested to participate by donating a sample of blood only.
Participants undergo a medical evaluation every 3 years during a 3- to 5-day hospital stay at the NIH Clinical Center. All participants have a personal and family medical history obtained and undergo a physical examination, blood and urine tests.
People who already have a small bowel carcinoid tumor or are at risk of developing a carcinoid tumor have some or all of the following procedures to determine the presence of carcinoid tumor and its (omit next two words- location or) spread to other areas of the body:
* Video Capsule Endoscopy: Visualization of the gastrointestinal tract by ingesting a disposable, "vitamin-pill sized" video capsule that has its own camera and light source. * CT of the chest abdomen and pelvis with oral and IV contrast : X-ray examination of the chest, abdominal and pelvis organs. * 18 FDOPA Positron emission tomography (PET) with CT for localization: Nuclear imaging scan to look at tumor activity. * MRI Liver with contrast - to determine if disease has spread to liver * Gallium 68 PET/CT-limited to individuals that have residual tumor. * Clinical and research blood work
Should mid gut carcinoid tumors be found every participant will be assisted in determine what the best course of treatment will be for them.
...
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male and female subjects >= 18 years of age
Have a diagnosis of small intestinal carcinoid tumor
Have at least one blood relation with a diagnosis of either small intestinal, pulmonary, kidney or gastropancreatic neuroendocrine tumor or metastatic neuroendocrine tumor of unknown primary
Male and female subjects >= 18 years of age
Disqualifiers
Members of families with multiple endocrine neoplasia (MEN) I, MEN II or other familial tumor syndromes such as Von Hippel Lindau Syndrome and Neurofibromatosis type I and type II for which there is a known genetic predisposition to non-carcinoid tumors as well as
Any condition which, in the opinion of the investigator, would make it unsafe to participate or would prohibit completion of the protocol.
Inability to provide informed consent (Arm 1 only)
Pregnant or breastfeeding (Arm 1 only)
Trial population
Community sample
Trial design
Design model
Family-based
Time perspective
Prospective
Treatments tested in this trial
[18F]-DOPA
Drug18F-DOPA PET/CT Scan.
Treatment groups
1,600 Participants
are divided into 2 treatment groupsGroup A: Arm 11 intervention
Group B: Arm 21 intervention
Trial outcomes
Primary outcomes
1
Study the natural history of familial carcinoid tumors
Incidence, age of onset, symptoms, the appropriate diagnostic (biochemical and imaging) modalities, location, histology and metastatic potential of the tumors, metabolic sequelae of the tumor, and clinical and biochemical prognostic factors
Time frame
End of studySecondary outcomes
1
Compare the sensitivity and specificity of various imaging modalities: computed tomography (CT) with IV contrast and oral Volumen, 18F-DOPA PET/CT scan, [68Ga]DOTATATE PET/CT scan and endoscopic modalities for diagnosing and following carcinoid ...
Enrollment of a sufficient number of subjects to allow for the determination of sensitivity and specificity for each imaging and endoscopic modality to show statistically significant superiority for one modality relative to the others
Time frame
End of study2
Sequester DNA from peripheral blood for genotyping (including sequencing) with the intention of localizing a susceptibility gene/s responsible for the familial occurrence of the disease
Determination of the gene mutation responsible for the disease in each family
Time frame
End of study3
Collect tumor specimens for histologic evaluation, culturing of intestinal organoids, and genotyping (including DNA and RNA sequencing)
Sufficient number of tumors collected to correlate disease grade with natural history of disease and assess the variability in disease grade
Time frame
End of study4
Screen for occult disease and determine whether early detection affects the natural history of the disease
Change in survival
Time frame
End of studySponsors and contacts
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Source ClinicalTrials.gov