68-Gallium DOTATATE Scan for Von Hippel-Lindau Syndrome

Overseen By Naris Nilubol, M.D.

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: National Cancer Institute (NCI)

Disqualifiers: Inability to undergo imaging

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

740 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

What data supports the effectiveness of the treatment 68-Gallium DOTATATE for Von Hippel-Lindau Syndrome?

68-Gallium DOTATATE PET/CT scans are highly accurate for detecting tumors in Von Hippel-Lindau disease because these tumors often have cells that express somatostatin receptors, which the scan can identify effectively. ^{1 2 3 4 5}

Is 68-Gallium DOTATATE safe for use in humans?

68-Gallium DOTATATE has been used safely in humans for imaging neuroendocrine tumors, as it helps identify tumors with specific receptors. There is no specific mention of safety concerns in the studies reviewed, suggesting it is generally considered safe for use in humans. ^{1 4 5 6 7}

How does the 68-Gallium DOTATATE scan treatment differ for Von Hippel-Lindau Syndrome?

The 68-Gallium DOTATATE scan is unique because it uses a special imaging technique to detect tumors in Von Hippel-Lindau Syndrome by targeting somatostatin receptors, which are often present in these tumors. This method is particularly useful for identifying various types of tumors associated with the syndrome, making it a valuable tool for diagnosis and management compared to traditional imaging methods. ^{1 4 5 8 9}

Research Team

Naris Nilubol, M.D.

Principal Investigator

National Cancer Institute (NCI)

Eligibility Criteria

This trial is for people aged 12 or older with von Hippel-Lindau (VHL) disease, which causes tumors and cysts in the pancreas. Participants must have a confirmed VHL diagnosis through genetic testing or clinical criteria and be able to give informed consent.

Inclusion Criteria

I have pancreatic issues related to VHL, confirmed by imaging.

I have been diagnosed with VHL based on genetic testing or clinical criteria.

I am 12 years old or older.

Exclusion Criteria

I cannot have repeated imaging tests.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Initial Evaluation

Participants undergo a comprehensive evaluation including physical examination, blood and urine tests, and imaging studies such as CT, PET/CT, MRI, or ultrasound.

1-2 weeks

1 visit (in-person)

Longitudinal Monitoring

Participants are monitored with annual or biennial visits for imaging studies, blood and urine sample collection, and potential surgical recommendations based on tumor growth or metastasis risk.

Ongoing (annually or biennially)

1 visit per year or every 2 years (in-person)

Follow-up

Participants are monitored for safety and effectiveness after any surgical intervention or significant treatment changes.

4 weeks

Treatment Details

Interventions

• 68-Gallium DOTATATE

Trial Overview The study aims to understand how VHL affects the pancreas and assess if a new type of scan called 68-Gallium DOTATATE PET/CT can better detect pancreatic tumors in these patients.

Participant Groups

Group I: 1/ Arm 1 Experimental Treatment 1 Intervention

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

Group II: 2/ Arm 2 Active Control 1 Intervention

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

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Findings from Research

In a study involving 36 patients with von Hippel-Lindau (VHL) disease, 68Ga-DOTATATE PET/CT demonstrated a higher detection rate of lesions compared to traditional imaging methods like CT and MRI, making it a promising tool for monitoring tumors associated with VHL.

68Ga-DOTATATE PET/CT not only showed superior detection capabilities but also offered lower radiation exposure, suggesting it could be a safer and more effective option for the surveillance of VHL-related tumors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The utility of 68Gallium-DOTATATE PET/CT in the detection of von Hippel-Lindau disease associated tumors. Shell, J., Tirosh, A., Millo, C., et al.[2021]

In a study of 20 patients with von Hippel-Lindau disease, (68)Ga-DOTATOC PET/CT detected pancreatic neuroendocrine tumors (pNETs) in 55% of patients, outperforming traditional imaging methods like CT and MRI, which identified only 45%.

The study suggests that (68)Ga-DOTATOC PET/CT is a more effective screening tool for pNETs in vHLD patients compared to the previously recommended (111)In-octreoscan, as it identified a higher proportion of tumors without any patients showing progressive disease during follow-up.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Potential role of (68)Ga-DOTATOC PET/CT in screening for pancreatic neuroendocrine tumour in patients with von Hippel-Lindau disease. Prasad, V., Tiling, N., Denecke, T., et al.[2019]

In a study of 81 patients with neuroendocrine tumors (NETs), Ga-68 DOTATATE PET/CT was more effective than F-18 FDG PET/CT in detecting positive scans in patients with low and intermediate-grade tumors, with 82.1% of DOTATATE scans positive compared to 54.5% for FDG (P = 0.04).

For high-grade, poorly-differentiated NETs, both DOTATATE and FDG PET/CT scans were equally effective, with all patients showing positive results, indicating that while DOTATATE is superior for lower-grade tumors, it does not provide additional benefits for high-grade tumors compared to FDG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ga-68 DOTATATE PET/CT and F-18 FDG PET/CT in the evaluation of low and intermediate versus high-grade neuroendocrine tumors. You, H., Kandathil, A., Beg, M., et al.[2021]

In a study of 14 patients with Von Hippel-Lindau (VHL) disease, the 68Ga-DOTANOC PET/CT scan successfully detected a total of 67 lesions across various sites, demonstrating its efficacy in identifying both benign and malignant tumors associated with this hereditary condition.

The 68Ga-DOTANOC PET/CT scan may serve as a convenient one-stop imaging solution for VHL patients, potentially replacing multiple separate imaging tests, thus saving time and reducing costs for patients undergoing screening and follow-up evaluations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

68Ga-DOTANOC PET/CT for Screening and Surveillance of Von Hippel-Lindau (VHL) disease. Shamim, SA., Arora, G., Kumar, N., et al.[2023]

68Ga-DOTANOC PET/CT imaging can reveal somatostatin receptor expression in various conditions, including in cases of Von Hippel-Lindau syndrome, which is linked to multiple tumor types.

In a specific case, the imaging technique showed tracer uptake in swollen eyelids of a patient suspected of having Von Hippel-Lindau syndrome, but clinical examination later confirmed the condition as viral conjunctivitis, highlighting the importance of accurate diagnosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Incidental Detection of Viral Conjunctivitis on 68Ga-DOTANOC PET/CT in a Patient Suspected With Von Hippel-Lindau Syndrome. Mathur, Y., Krishnaraju, VS., Singh, H., et al.[2023]

68 Ga-DOTATATE PET/CT imaging proved effective in identifying previously unknown primary tumors and metastatic disease in sinonasal neuroendocrine tumors, highlighting its potential for better diagnosis compared to conventional imaging methods.

In two cases of esthesioneuroblastoma, 68 Ga-DOTATATE detected abnormal uptake that was not visible on 18F-FDG PET/CT, suggesting it may be crucial for treatment planning, including identifying candidates for targeted therapies like 177 Lu-DOTATATE.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The value of 68 Gallium-DOTATATE PET/CT in sinonasal neuroendocrine tumor management: A case series. Liu, KY., Goldrich, DY., Ninan, SJ., et al.[2021]

Gallium-68 DOTATATE imaging effectively identified the primary tumor site in 45% of patients with neuroendocrine tumors (NETs) of unknown primary origin, highlighting its utility in disease localization.

The study found that systemic exposure to long-acting somatostatin analogs did not significantly affect the mean Standardized Uptake Value (SUV) in gallium-68 DOTATATE scans, suggesting that these treatments do not compromise imaging quality.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Post FDA approval analysis of 200 gallium-68 DOTATATE imaging: A retrospective analysis in neuroendocrine tumor patients. Chauhan, A., El-Khouli, R., Waits, T., et al.[2023]

Ga-68 somatostatin receptor PET/CT imaging is effective for diagnosing and managing various malignancies associated with Von Hippel-Lindau (VHL) disease, which includes a range of tumors such as hemangioblastomas and pheochromocytomas.

This imaging technique serves as a comprehensive tool for evaluating and monitoring the multiple tumor types that can arise in patients with VHL disease, highlighting its potential as a 'one-stop-shop' for clinical management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ga-68 Somatostatin Receptor PET/CT in von Hippel-Lindau Disease. Oh, JR., Kulkarni, H., Carreras, C., et al.[2021]

A rare case of metastatic renal cell carcinoma was identified in a 66-year-old man with von Hippel-Lindau syndrome, showing 68Ga-DOTATATE uptake in multiple locations, including the pancreas and spine.

The presence of 68Ga-DOTATATE avidity in VHL-associated cancers can influence clinical decision-making, highlighting the importance of this imaging technique in detecting and managing such rare metastatic presentations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

68Ga-DOTATATE Avid Metastatic Vertebral Renal Cell Carcinoma in the Setting of von Hippel-Lindau Syndrome. Patel, M., Jha, A., Antic, T., et al.[2023]

References

1. Ireland pubmed.ncbi.nlm.nih.gov

The utility of 68Gallium-DOTATATE PET/CT in the detection of von Hippel-Lindau disease associated tumors. [2021]

2. Germany pubmed.ncbi.nlm.nih.gov

Potential role of (68)Ga-DOTATOC PET/CT in screening for pancreatic neuroendocrine tumour in patients with von Hippel-Lindau disease. [2019]

3. England pubmed.ncbi.nlm.nih.gov

Ga-68 DOTATATE PET/CT and F-18 FDG PET/CT in the evaluation of low and intermediate versus high-grade neuroendocrine tumors. [2021]

4. Germany pubmed.ncbi.nlm.nih.gov

68Ga-DOTANOC PET/CT for Screening and Surveillance of Von Hippel-Lindau (VHL) disease. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Incidental Detection of Viral Conjunctivitis on 68Ga-DOTANOC PET/CT in a Patient Suspected With Von Hippel-Lindau Syndrome. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

The value of 68 Gallium-DOTATATE PET/CT in sinonasal neuroendocrine tumor management: A case series. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Post FDA approval analysis of 200 gallium-68 DOTATATE imaging: A retrospective analysis in neuroendocrine tumor patients. [2023]

8. Germany pubmed.ncbi.nlm.nih.gov

Ga-68 Somatostatin Receptor PET/CT in von Hippel-Lindau Disease. [2021]

9. Germany pubmed.ncbi.nlm.nih.gov

68Ga-DOTATATE Avid Metastatic Vertebral Renal Cell Carcinoma in the Setting of von Hippel-Lindau Syndrome. [2023]