Natural History and Genetic Study of Patients With Von Hippel-Lindau Syndrome and Pancreatic Lesions
Last Modified: 3/31/2009 First Published: 5/23/2003
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 | Protocol IDs |
|---|
| No phase specified | Biomarker/Laboratory analysis, Diagnostic, Natural history/Epidemiology | Closed | 12 and over | NCI-03-C-0145
NCT00062166 |
Objectives - 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).
- Follow patients with VHL and pancreatic manifestations by serial examination with non-invasive imaging studies (e.g., CT scan, MRI, and/or abdominal ultrasound).
- Correlate rate of lesion growth with clinical measures of disease progression (e.g., symptoms) in patients with solid lesions of the pancreas.
- Determine VHL mutation status and subtype the mutations for potential correlation with disease severity in these patients.
- Obtain tissue from the pancreatic lesions of these patients for genetic analysis, including comparative genomic hybridization, tissue proteomics, and cDNA microarray analysis.
- 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 Chemotherapy Endocrine therapy Radiotherapy Surgery Patient Characteristics:
Age Performance status Life expectancy Hematopoietic Hepatic Renal Other - Willing to return to the National Institutes of Health for follow-up
- Willing to undergo serial non-invasive imaging
Expected Enrollment 300A 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 | | | |
| 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 1 | | | Date Submitted to PDQ | | 2003-04-28 | | | Information Last Verified | | 2008-11-30 |
Note: The purpose of most clinical trials listed in this database is to test new cancer treatments, or new methods of diagnosing, screening, or preventing cancer. Because all potentially harmful side effects are not known before a trial is conducted, dose and schedule modifications may be required for participants if they develop side effects from the treatment or test. The therapy or test described in this clinical trial is intended for use by clinical oncologists in carefully structured settings, and may not prove to be more effective than standard treatment. A responsible investigator associated with this clinical trial should be consulted before using this protocol.
Table of Links
| 1 | http://clinicaltrials.gov/ct/show/NCT00062166 |
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