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Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine Tumors

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ClinicalTrials.gov Identifier: NCT04074135
Recruitment Status : Not yet recruiting
First Posted : August 29, 2019
Last Update Posted : October 18, 2019
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Brief Summary:

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.


Condition or disease
VHL Pancreatic Neuroendocrine Tumors Von Hippel-Lindau Disease Neuroendocrine Tumors

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 imaging studies, research blood and urine sample collection will be performed at each scheduled visit.
  • 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 620 patients on this protocol.

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Study Type : Observational
Estimated Enrollment : 620 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evaluation of the Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine Tumors
Estimated Study Start Date : October 23, 2019
Estimated Primary Completion Date : January 30, 2036
Estimated Study Completion Date : July 1, 2036


Group/Cohort
1/ Cohort 1
Subjects with VHL pancreatic neuroendocrine tumors



Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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

  2. growth rate in solid pancreatic tumors [ Time Frame: at each scheduled visit ]
    graded tumor biopsy

  3. predictive value of PET imaging in patient population [ Time Frame: at each scheduled visit ]
    mean uptake between these two groups of patients in which per patient uptake is defined as the average uptake of multiple lesions detected in each patient



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
primary clinical
Criteria
  • INCLUSION CRITIERIA:

    1. 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

    2. 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
    3. Age greater than or equal to 12 years.
    4. Ability of subject (or Legally Authorized Representative (LAR)) to understand and the willingness to sign a written informed consent document.

EXCLUSION CRITERIA:

  1. Inability of subject to undergo serial non-invasive imaging.
  2. Pregnant women are excluded from this study, as there is no direct benefit to study participation. Note: Pregnancy testing will be performed on all females of child bearing potential at screening, at each visit and prior to any imaging study using ionizing radiation or MRI with gadolinium. Those with positive pregnancy tests will be excluded.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04074135


Contacts
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Contact: Deborah E Allen, R.N. (240) 760-6184 allendeb@mail.nih.gov

Locations
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United States, Maryland
National Institutes of Health Clinical Center Not yet recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office    888-624-1937      
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Naris Nilubol, M.D. National Cancer Institute (NCI)

Additional Information:
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Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT04074135     History of Changes
Other Study ID Numbers: 190135
19-C-0135
First Posted: August 29, 2019    Key Record Dates
Last Update Posted: October 18, 2019
Last Verified: September 9, 2019
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Pancreatic tumors
68-Gallium DOTATATE
MEN1 syndrome
NETest
PNET
Additional relevant MeSH terms:
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Neuroendocrine Tumors
Adenoma, Islet Cell
Von Hippel-Lindau Disease
Neoplasms
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Adenoma
Neoplasms, Glandular and Epithelial
Pancreatic Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Endocrine Gland Neoplasms
Digestive System Diseases
Pancreatic Diseases
Endocrine System Diseases
Neurocutaneous Syndromes
Nervous System Diseases
Angiomatosis
Vascular Diseases
Cardiovascular Diseases
Ciliopathies
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn