Clinical Utility Assay as a Biomarker for Gastroenteropancreatic and Lung Neuroendocrine Tumors

Study Overview
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Brief Summary
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The purpose of this study is to evaluate how well an investigational blood test performs. The study will look at the sensitivity and specificity of a blood-based multitranscriptome assay (NETest).

Official Title
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The Clinical Utility of a Blood-Based Multitranscriptome Assay as a Biomarker for Gastroenteropancreatic and Lung Neuroendocrine Tumors

Conditions
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Gastroenteropancreatic Neuroendocrine Tumor, Lung Neuroendocrine Neoplasm

Intervention / Treatment
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* Procedure: NETest


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: NET Cohort- Patients with histologically or cytologically proven diagnosis of any grade, any stage NET of GEP or lung origin; In the first stage of the study (initial 50 patients) only patients with stage IV, well-differentiated tumors (G1/G2) will be enrolled. Patients with stable or progressive disease, as documented on a scan (CT, MRI); Progression status will be documented on case report form (CRF). Allowed prior therapies include: a.) Surgery (tumor surgery at least four weeks prior to study entry); b.) Locoregional therapy such as: chemoembolization, radio-embolization, radiofrequency ablation, radiotherapy at least six weeks prior to study entry; c.) Any number of previous lines of systemic therapy, providing that cytotoxic therapies (chemotherapy, PRRT) have been discontinued at least 4 weeks prior to study entry. Non-NET Cohort - Healthy participants Patients with histologically or cytologically proven diagnosis of any grade, any stage GI malignancies. Exclusion Criteria: NET Cohort - Patients on treatment with cytotoxic agents (chemotherapy, PRRT). Patients with renal insufficiency or congestive heart failure. No other active malignancy within 3 years of enrolment except adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated stage I or II cancer from which the patient is currently in complete remission. Non-NET Cohort Patients with GI malignancies with neuroendocrine differentiation.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Neuroendocrine Tumor (NET) Cohort<br>Participants with histologically or cytologically proven diagnosis of any grade, any stage NET of gastroenteropancreatic (GEP) or lung origin; In the first stage of the study (initial 50 patients) only potential participants with stage IV, well-differentiated tumors (G1/G2) will be enrolled. | Procedure: NETest<br>* 5 mL of blood will be drawn from participants for testing.<br>|
| Non-NET Cohort<br>Participants with histologically or cytologically proven diagnosis of any grade, any stage gastrointestinal (GI) malignancies. | Procedure: NETest<br>* 5 mL of blood will be drawn from participants for testing.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of Successful Test Results Per Cohort | Investigators have designed the study to test the null hypothesis that the NETest has a sensitivity and specificity of 70% or less in NET patients. The sample size calculation has been based on the assumption that a sensitivity and specificity of greater than 90% would generate further interest in the test for unselected NET patients. Power and type 1 error will be 99% and 5% respectively. Under this model, 80 or more positive tests in the cohort of 100 patients with NETs would lead to the rejection of the null hypothesis, suggesting that the NETest is sensitive. Likewise, 80 or more negative tests in 100 patients without NETs will suggest that NETest is specific. An interim analysis will be performed to rule out the futility of the NETest. Futility will be defined as a rate of false positive or false negative >25%. Hence, if 12 or more false positives or negatives are observed among the first 50 participants, the study will be suspended, pending review by the investigators. | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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lung cancer, pancreatic cancer