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

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

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).

Study Overview

• Status: Completed
• Conditions: Lung Neuroendocrine Neoplasm; Gastroenteropancreatic Neuroendocrine Tumor
• Intervention / Treatment: Procedure: NETest

• Study Type: Observational
• Enrollment (Actual): 105

Contacts and Locations

Study Locations

• United States
  • Florida
    • Tampa, Florida, United States, 33612
      • H. Lee Moffitt Cancer Center and Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants to be enrolled in the study will be recruited within the Gastrointestinal (GI) Clinic of Moffitt Cancer Center.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Neuroendocrine Tumor (NET) Cohort; 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; 5 mL of blood will be drawn from participants for testing.
Non-NET Cohort; Participants with histologically or cytologically proven diagnosis of any grade, any stage gastrointestinal (GI) malignancies.	Procedure: NETest; 5 mL of blood will be drawn from participants for testing.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Publications and helpful links

Helpful Links

• Moffitt Cancer Center Clinical Trials website

Study record dates

Study Major Dates

• Study Start (Actual): January 9, 2017
• Primary Completion (Actual): August 24, 2018
• Study Completion (Actual): September 15, 2020

Study Registration Dates

• First Submitted: October 27, 2016
• First Submitted That Met QC Criteria: October 27, 2016
• First Posted (Estimate): October 31, 2016

Study Record Updates

• Last Update Posted (Actual): August 4, 2021
• Last Update Submitted That Met QC Criteria: August 3, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Keywords: lung cancer; pancreatic cancer
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroendocrine Tumors
• Other Study ID Numbers: MCC-18756

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No