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

NCT04074135 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: 19013519-C-0135
• Study Type: interventional
• Target: 740
• Locations: 1 country
• Sites: 1

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.

Conditions

VHL Pancreatic Neuroendocrine Tumors; Von Hippel-Lindau Disease; Neuroendocrine Tumors

Keywords

Pancreatic tumors; 68-Gallium DOTATATE; MEN1 syndrome; NETest; PNET

Outcome Measures

Primary Outcomes (1)

• 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

  15 years

Secondary Outcomes (3)

• predictive value of 68-Gallium DOTATATE PET/CT imaging in patient population

  at each scheduled visit

• growth rate in solid pancreatic tumors

  at each scheduled visit

• distribution of time from initial presentation with pancreatic tumors to the time that surgery is recommended

  at each scheduled visit, until surgery is recommended

Study Arms (2)

1/ Arm 1

EXPERIMENTAL

Study natural history of VHL pancreatic neuroendocrine tumors with yearly 68-Gallium DOTATATE PET/CT research scans.

Drug: 68-Gallium DOTATATE

2/ Arm 2

NO INTERVENTION

Study natural history of VHL pancreatic neuroendocrine tumors without research scans.

Interventions

68-Gallium DOTATATE DRUG

68-Gallium DOTATATE, to be used in yearly PET/CT research scans

1/ Arm 1

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may not qualify if:

• \. Inability of participant to undergo serial non-invasive imaging.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

von Hippel-Lindau Disease; Neuroendocrine Tumors; Pancreatic Neoplasms; Multiple Endocrine Neoplasia Type 1; Neuroectodermal Tumors, Primitive

Interventions

gallium Ga 68 dotatate

Condition Hierarchy (Ancestors)

Neurocutaneous Syndromes; Nervous System Diseases; Angiomatosis; Vascular Diseases; Cardiovascular Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Digestive System Neoplasms; Neoplasms by Site; Endocrine Gland Neoplasms; Digestive System Diseases; Pancreatic Diseases; Endocrine System Diseases; Multiple Endocrine Neoplasia; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Neoplasms, Neuroepithelial; Neoplasms, Glandular and Epithelial

Study Officials

• Naris Nilubol, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kristine J Villaruel

CONTACT

(240) 858-7033; kristinejoy.villaruel@nih.gov

Naris Nilubol, M.D.

CONTACT

(240) 760-6154; niluboln@mail.nih.gov

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 28, 2019
• First Posted: August 29, 2019
• Study Start: June 2, 2020
• Primary Completion (Estimated): January 30, 2036
• Study Completion (Estimated): July 1, 2036
• Last Updated: April 20, 2026

Record last verified: 2026-04-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.
• Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.@@@@@@Genomic data are made available via dbGaP through requests to the data custodians.

Locations

US (1)