Trial Information

Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms

Background:

- Endocrine neoplasms are among the fastest growing tumors in incidence in the United
States. Between 1995 and 2005, the incidence of thyroid carcinoma has increased 98
percent.

- Tumors of the thyroid, parathyroid, adrenal gland and pancreatic neuroendocrine tumors
are among some of the most difficult tumors to clinically and histopathologically
distinguish as benign or malignant.

- Moreover, endocrine neoplasm provide an extremely important model for studying the
important molecular changes that lead to carcinogenesis because of their diverse
clinical behavior, even when having the same TNM stage and histologic features.

- The Endocrine Surgery Section of the Surgery Branch, NCI has a focus on studying the
molecular changes that are involved in Endocrine Cancer initiation and progression. In
addition, this section has primary responsibility for providing endocrine surgery
consultative services to the NIH. As such, our Section is uniquely positioned to
acquire and perform important studies on endocrine tissue to help identify diagnostic
and predictive markers, as well as, therapeutic targets that may have significant
clinical ramifications. Some of the patients enrolled in this study may be on other
protocols throughout the NIH, for which our Section is consulted in order to perform
their operation.

Objectives:

Primary Objective:

- To develop a genetic and epigenetic, metabolomic, and proteomic profile of endocrine
neoplasm that will allow us to distinguish benign from malignant tumor for each of the
endocrine histologies under study. This objective will drive the statistical endpoints of
the study.

Secondary Objectives:

- To utilize the tissue obtained from these endocrine neoplasms for studies of gene
expression and epigenetic (methylation) changes, and metabolite and protein expression.

- To obtain, when accessible, normal endocrine and other adjacent tissue for comparison
with the neoplastic tissue.

- To collect tissues from endocrine neoplasms arising in the thyroid, parathyroid,
adrenal, pancreas, and extraadrenal neuroendocrine rests for future analysis and
correlation with clinical outcome.

Eligibility:

- Patients with radiographic evidence of, biochemical evidence of, or
histologically/cytologically proven, endocrine neoplasms, including lesions of the
thyroid, parathyroid, adrenal, extra-adrenal endocrine rests, paragangliomas,
neuroblastomas and pancreas. or patients with a described pre or potentially malignant
condition that requires surgery or biopsy as a part of the standard of care treatment
and/or follow up.

- Patients must have an ECOG performance score of 0-2.

- Patients must have laboratory and physical examination parameters within acceptable
limits by standard of practice guidelines prior to biopsy or surgery.

Design:

- A tissue acquisition trial in which tissues will be obtained at the time of surgical
operation for the removal of neoplasms of the thyroid, parathyroid, adrenal, pancreas,
and paragangliomas and or extraadrenal rests of neuroendocrine tissue.

- At the time of surgical operation, blood samples will be obtained from the operative
field during the removal of neoplasms of the thyroid, parathyroid, adrenal, pancreas,
and paragangliomas and or extraadrenal neuroendocrine tissue.

- Tissue and blood will be processed at the time of collection, stored and then
transferred to Dr. Kebebew's laboratory for further processing.

- No investigational therapy will be given.

- It is anticipated that 1350 patients will be enrolled over a period of 16 years.