Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outline
Trial Contact Information
Registry Information

Alternate Title

Diagnostic and Genetic Study to Identify Pancreatic Lesions in Patients With Von Hippel-Lindau Syndrome

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
No phase specified	Biomarker/Laboratory analysis, Diagnostic, Natural history/Epidemiology	Closed	12 and over	NCI	NCI-03-C-0145 NCT00062166

Objectives

1. Identify pancreatic lesions (e.g., simple cysts, microcystic adenomas, neuroendocrine tumors, or other solid lesions of the pancreas) in patients with von Hippel-Lindau syndrome (VHL).
2. Follow patients with VHL and pancreatic manifestations by serial examination with non-invasive imaging studies (e.g., CT scan, MRI, and/or abdominal ultrasound).
3. Correlate rate of lesion growth with clinical measures of disease progression (e.g., symptoms) in patients with solid lesions of the pancreas.
4. Determine VHL mutation status and subtype the mutations for potential correlation with disease severity in these patients.
5. Obtain tissue from the pancreatic lesions of these patients for genetic analysis, including comparative genomic hybridization, tissue proteomics, and cDNA microarray analysis.
6. Determine the time from initial presentation with pancreatic tumors to the time that surgery is recommended in these patients.

Entry Criteria

Disease Characteristics:

• Diagnosis of von Hippel-Lindau syndrome (VHL) by the Urologic Oncology Branch using germ line analysis OR clinical criteria and family history



• Must have at least 1 of the following pancreatic manifestations of VHL documented by a non-invasive imaging study:
  • Pancreatic cyst(s)
  • Solid lesions suspicious for microcystic adenoma(s)
  • Solid enhancing lesions suspicious for pancreatic neuroendocrine tumor(s)
  • Any other solid lesion(s) of the pancreas

Prior/Concurrent Therapy:

Biologic therapy

• Not specified

Chemotherapy

• Not specified

Endocrine therapy

• Not specified

Radiotherapy

• Not specified

Surgery

• Not specified

Patient Characteristics:

Age

• 12 and over

Performance status

• Not specified

Life expectancy

• Not specified

Hematopoietic

• Not specified

Hepatic

• Not specified

Renal

• Not specified

Other

• Willing to return to the National Institutes of Health for follow-up
• Willing to undergo serial non-invasive imaging

Expected Enrollment

A total of 300 patients will be accrued for this study within 5 years.

Outline

Patients undergo non-invasive imaging studies (e.g., CT scan with contrast, MRI, and/or abdominal ultrasound). Blood and urine samples are collected for laboratory analysis. Blood samples are also drawn for genetic germ line mutational analysis.

All patients are offered genetic counseling.

Patients with cystic disease of the pancreas only (no solid lesions) are followed with non-invasive imaging studies at least every 2 years.

Patients with solid lesions of the pancreas that are suspicious for pancreatic neuroendocrine tumors (PNETs) and have not reached size criteria for surgery are followed annually with CT scans and MRI.

Patients with solid lesions of the pancreas that are suspicious for PNETs and have reached size criteria for surgery or are symptomatic for PNETs may undergo surgical management. Patients with cystic disease who develop symptoms attributable to the cysts may also undergo surgical management.

In patients who undergo surgical resection, tissue is removed for further genetic analysis, including comparative genomic hybridization, cDNA microarray analysis, and tissue proteomics.

Trial Contact Information

Trial Lead Organizations

NCI - Center for Cancer Research

Steven Rosenberg, MD, PhD, Principal investigator		Ph: 301-496-4164 Email: sar@nih.gov

Registry Information
Official Title		Evaluation of the Natural History and Management of Pancreatic Lesions Associated with Von Hippel-Lindau
Trial Start Date		2003-04-11
Registered in ClinicalTrials.gov		NCT00062166
Date Submitted to PDQ		2003-04-28
Information Last Verified		2008-11-30

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