- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04074135
Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine Tumors
Evaluation of the Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine Tumors
Background:
People with von Hippel-Lindau (VHL) can have problems with a variety of organs, such as the pancreas. The disease can cause tumors of the pancreas. This can result in life-threatening complications. Researchers want to learn more about these pancreatic tumors and how to better detect them. This may help them design better future treatment and care for people with VHL disease.
Objective:
To better understand VHL disease that affects the pancreas and to test whether adding a certain type of scan (68-Gallium DOTATATE PET/CT) can further detect tumors.
Eligibility:
People ages 12 and older with VHL that causes tumors and cysts to grow in the pancreas
Design:
Participants will be screened with their medical records and imaging studies.
Participants will have an initial evaluation:
Participants will have their body examined by different doctors. This will depend on what types of symptoms they have.
Participants will have blood and urine tests
Participants will have images made of their body using one or more machines: They made have a CT or PET/CT scan in which they lie on a table that moves through a big ring. They may have an MRI in which they lie on a table that moves into a big tube. They may have an ultrasound that uses a small stick that produces sound waves to look at the body.
After the first visit, participants will be asked to return to the NIH. Some of the tests performed at the first visit will be repeated. Depending on their disease status, visits will be once a year or every 2 years for life.
Study Overview
Status
Intervention / Treatment
Detailed Description
Background:
- Patients with the von Hippel-Lindau (VHL) familial cancer syndrome demonstrate manifestations in a variety of organs, including the pancreas. Pancreatic manifestations can range from benign cysts and micro cystic adenomas to neuroendocrine tumors of the pancreas which are capable of regional and distant metastases. These neuroendocrine tumors can result in life-threatening complications.
- This protocol is designed to identify VHL patients with pancreatic manifestations and to follow these patients with serial imaging studies, germ line genetic analysis, discovery of serum biomarkers, and novel imaging modalities such as 68-Gallium DOTATATE PET/CT scan.
Objective:
- To comprehensively and longitudinally evaluate the natural history of patients with VHL pancreatic neuroendocrine tumors and cystic lesions, estimating and defining their clinical spectrum.
Eligibility:
- Patients greater than or equal to 12 years of age diagnosed with VHL.
Design:
- Demographic data will be collected from the medical record and patient interview for each patient participant. Data will be securely stored in a computerized database.
- Patients will be evaluated by the urology, neurosurgery, brain oncology and/or ophthalmology personnel as indicated to rule out or manage other manifestations of VHL such as hemangioblastoma, renal cell cancer, and pheochromocytoma.
- Anatomical and functional clinical imaging studies, research blood and urine sample collection will be performed at each scheduled visit. Research 68-Gallium DOTATATE PET/CT imaging studies in adult subjects will be performed, no more than once per year.
- Surgical resection of solid lesions of the pancreas will be recommended based on previously published criteria.
- Based on our analysis of likelihood of tumor growth or risk of metastasis, data will be analyzed every two years and appropriate revisions will be made to the surgical management guidelines, if indicated by data analysis.
- Projected accrual will be 40 patients per year for a total of 15 years. Considering some patients may have a screen failure, we anticipate accruing of 740 patients on this protocol.
Study Type
Enrollment (Estimated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Naris Nilubol, M.D.
- Phone Number: (240) 760-6154
- Email: niluboln@mail.nih.gov
Study Contact Backup
- Name: Kristine J Villaruel
- Phone Number: (240) 858-7033
- Email: villaruelkf@mail.nih.gov
Study Locations
-
-
Maryland
-
Bethesda, Maryland, United States, 20892
- Recruiting
- National Institutes of Health Clinical Center
-
Contact:
- For more information at the NIH Clinical Center contact National Cancer Institute Referral Office
- Phone Number: 888-624-1937
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
INCLUSION CRITIERIA:
Subjects who have been diagnosed with VHL using the following criteria:
-- Identification of a heterozygous germline pathogenic variant in VHL by molecular genetic testing.
or
-- Clinical criteria
Subjects with at least 1 pancreatic manifestation of VHL as documented on any non-invasive imaging study. These manifestations may include:
- Pancreatic cyst(s)
- Solid lesions suspicious for microcystic adenoma(s)
- Solid enhancing lesions suspicious for PNET(s)
- Any other solid lesion(s) of the pancreas
- Age greater than or equal to 12 years.
- Ability of subject to understand and the willingness to sign a written informed consent document.
EXCLUSION CRITERIA:
1. Inability of subject to undergo serial non-invasive imaging.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Diagnostic
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 1/ Arm 1
Study natural history of VHL pancreatic neuroendocrine tumors with yearly 68-Gallium DOTATATE PET/CT research scans.
|
68-Gallium DOTATATE, to be used in yearly PET/CT research scans
|
No Intervention: 2/ Arm 2
Study natural history of VHL pancreatic neuroendocrine tumors without research scans.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Characterization of the natural and clinical histories of VHL pancreatic neuroendocrine tumors and cystic lesions
Time Frame: 15 years
|
Characterization of the natural and clinical histories of VHL pancreatic neuroendocrine tumors and cystic lesions
|
15 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
predictive value of 68-Gallium DOTATATE PET/CT imaging in patient population
Time Frame: at each scheduled visit
|
mean uptake between these two groups of adult patients in which per patient uptake is defined as the average uptake of multiple lesions detected in each patient
|
at each scheduled visit
|
growth rate in solid pancreatic tumors
Time Frame: at each scheduled visit
|
graded tumor biopsy
|
at each scheduled visit
|
distribution of time from initial presentation with pancreatic tumors to the time that surgery is recommended
Time Frame: at each scheduled visit, until surgery is recommended
|
median amount of time from initial presentation with pancreatic tumors to the time that surgery is recommended
|
at each scheduled visit, until surgery is recommended
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Naris Nilubol, M.D., National Cancer Institute (NCI)
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Cardiovascular Diseases
- Vascular Diseases
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms by Site
- Neoplasms, Glandular and Epithelial
- Endocrine System Diseases
- Congenital Abnormalities
- Digestive System Neoplasms
- Endocrine Gland Neoplasms
- Genetic Diseases, Inborn
- Neuroectodermal Tumors
- Neoplasms, Germ Cell and Embryonal
- Neoplasms, Nerve Tissue
- Pancreatic Diseases
- Abnormalities, Multiple
- Adenoma
- Neurocutaneous Syndromes
- Ciliopathies
- Angiomatosis
- Pancreatic Neoplasms
- Neoplasms
- Neuroendocrine Tumors
- Von Hippel-Lindau Disease
- Adenoma, Islet Cell
Other Study ID Numbers
- 190135
- 19-C-0135
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Time Frame
IPD Sharing Access Criteria
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
- ICF
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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