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

Completed

• Conditions: Gastroenteropancreatic Neuroendocrine Tumor; Lung Neuroendocrine Neoplasm
• Interventions: Procedure: NETest

• Registration Number: NCT02948946
• Lead Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-10-27
• Study First Submitted QC: 2016-10-27
• Study First Posted: 2016-10-31
• Study Start: 2017-01-09
• Primary Completion: 2018-08-24
• Study Completion: 2020-09-15
• Status Verified: 2021-07
• Last Update Submitted: 2021-08-03
• Last Update Posted: 2021-08-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 105

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Neuroendocrine Tumor (NET) Cohort	NETest	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.
Non-NET Cohort	NETest	Participants with histologically or cytologically proven diagnosis of any grade, any stage gastrointestinal (GI) malignancies.

Primary Outcome Measures

Name	Time	Method
Rate of Successful Test Results Per Cohort	12 months	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.

Trial Locations

Locations (1)

H. Lee Moffitt Cancer Center and Research Institute; 🇺🇸 Tampa, Florida, United States