VHL Pancreatic Neuroendocrine TumorsPhase 2NCT04074135
Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine Tumors
A Phase 2 study testing 68-Gallium DOTATATE in people with VHL Pancreatic Neuroendocrine Tumors and Von Hippel-Lindau Disease, run by National Cancer Institute (NCI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- VHL Pancreatic Neuroendocrine Tumors, Von Hippel-Lindau Disease, Neuroendocrine Tumors
- What is being tested?
- 68-Gallium DOTATATE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 740 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jun 2020to 1 Jul 2036
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants 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
- Participants with at least 1 pancreatic manifestation of VHL as documented on any non-invasive imaging study. These manifestations may include:
- Pancreatic cyst(s)
Show all 11Show fewer
- 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 participant to understand and the willingness to sign a written informed consent document.
Who cannot join
- 1. Inability of participant to undergo serial non-invasive imaging.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact National Cancer Institute Referral Office, 888-624-1937
About this study
Official title: 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.
Read more
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.
Read the 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 participants with VHL pancreatic neuroendocrine tumors and cystic lesions, estimating and defining their clinical spectrum.
Eligibility:
- Participants 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 participant. Data will be securely stored in a computerized database.
- Participants 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 participants 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.
- Up to 700 participants may receive the research 68-Gallium DOTATATE PET/CT imaging on this protocol.
- Being tested
- 68-Gallium DOTATATE
- Keywords
- Pancreatic tumors, 68-Gallium DOTATATE, MEN1 syndrome, NETest, PNET
- Medical subject headings
- von Hippel-Lindau Disease, Neuroendocrine Tumors, Pancreatic Neoplasms, Multiple Endocrine Neoplasia Type 1, Neuroectodermal Tumors, Primitive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
68-Gallium DOTATATEDrug
68-Gallium DOTATATE, to be used in yearly PET/CT research scans
| Study group | Role | What they receive |
|---|---|---|
| 1/ Arm 1 | Experimental | Study natural history of VHL pancreatic neuroendocrine tumors with yearly 68-Gallium DOTATATE PET/CT research scans. Receives: Drug: 68-Gallium DOTATATE |
| 2/ Arm 2 | No intervention | Study natural history of VHL pancreatic neuroendocrine tumors without research scans. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterization of the natural and clinical histories of VHL pancreatic neuroendocrine tumors and cystic lesions
Characterization of the natural and clinical histories of VHL pancreatic neuroendocrine tumors and cystic lesions
Measured: 15 years
Secondary outcomes (3)
predictive value of 68-Gallium DOTATATE PET/CT imaging in patient population
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
Measured: at each scheduled visit
growth rate in solid pancreatic tumors
graded tumor biopsy
Measured: at each scheduled visit
distribution of time from initial presentation with pancreatic tumors to the time that surgery is recommended
median amount of time from initial presentation with pancreatic tumors to the time that surgery is recommended
Measured: at each scheduled visit, until surgery is recommended
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2019
- Study start2 Jun 2020
- Primary completion30 Jan 2036 (estimated)
- Study completion1 Jul 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VHL Pancreatic Neuroendocrine TumorsStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 740 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Naris Nilubol, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04074135
- Sponsor's protocol ID
- 190135
- Other IDs
- 19-C-0135
- Status last verified
- 13 Oct 2026
- First submitted
- 28 Aug 2019
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.