Understanding and managing pancreatic tumors in patients with Von Hippel-Lindau disease

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

Phase 2 Interventional National Institutes of Health Clinical Center (CC) · NCT04074135

This study is testing how pancreatic tumors behave in people with Von Hippel-Lindau disease to improve detection and management of these tumors.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment740 (estimated)
Ages12 Years and up
SexAll
SponsorNational Institutes of Health Clinical Center (CC) NIH
Locations1 site (Bethesda, Maryland)
Trial IDNCT04074135 on ClinicalTrials.gov

What this trial studies

This study aims to investigate the natural history of pancreatic neuroendocrine tumors associated with Von Hippel-Lindau (VHL) disease. Participants aged 12 and older with VHL will undergo comprehensive evaluations, including medical record reviews, imaging studies, and blood tests. The study will utilize advanced imaging techniques, specifically the 68-Gallium DOTATATE PET/CT scan, to enhance tumor detection and monitoring. By following these patients longitudinally, researchers hope to better understand the clinical spectrum of pancreatic manifestations in VHL.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 12 and older diagnosed with Von Hippel-Lindau disease who have pancreatic manifestations.

Not a fit: Patients without a diagnosis of VHL or those unable to undergo serial non-invasive imaging may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved detection and management strategies for pancreatic tumors in VHL patients, potentially reducing life-threatening complications.

How similar studies have performed: While there have been studies on VHL and pancreatic tumors, the specific use of 68-Gallium DOTATATE PET/CT for this purpose is a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria
* INCLUSION CRITIERIA:

  1. 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
  2. Participants 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 participant to understand and the willingness to sign a written informed consent document.

EXCLUSION CRITERIA:

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

Where this trial is running

Bethesda, Maryland

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions VHL Pancreatic Neuroendocrine TumorsVon Hippel-Lindau DiseaseNeuroendocrine TumorsPancreatic tumors68-Gallium DOTATATEMEN1 syndromeNETestPNET
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.