Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in MEN1 Patients

Sponsor

M.D. Anderson Cancer Center (Other)

Overall Status

Recruiting

CT.gov ID

NCT03048266

Collaborator

(none)

629

Enrollment

Location

131.9

Anticipated Duration (Months)

4.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Objectives:

The aim of the present study is to assess the significance of metabolomics and genetics in diagnosing and survival evaluation for pNET in the periodic follow-up of MEN1 patients.

Aim 1: To evaluate the relationship of serum global metabolic profiles with subsequent development of aggressive PNET and evaluate patients survival in a nested case-control study of MEN1 patients who have developed aggressive PNETs (cases) and MEN1 patients who have developed non-aggressive PNETs (controls).

Aim 2: Validate the top serum metabolites identified from Aim 1 in MEN1 patients who have developed aggressive PNETs and MEN1 patients who have developed non-aggressive PNETs, using a targeted metabolomics approach.

Aim 3: Prospectively identify the potential miRNA biomarkers of serum with miRNA sequencing in MEN1 patients who have developed aggressive PNETs (cases) and MEN1 patients who have developed non-aggressive PNETs (controls).

Aim 4: Validate the potential miRNA biomarkers identified from Aim 1 in MEN1 patients who have developed aggressive PNETs and in MEN1 patients who have developed non-aggressive PNETs, using a targeted qRT-PCR approach (in serums), as well as to see the relationship of potential miRNA biomarkers with patients survival.

Condition or Disease	Intervention/Treatment	Phase
Multiple Endocrine Neoplasia

Study Design

Study Type:

Observational

Anticipated Enrollment :

629 participants

Observational Model:

Case-Control

Time Perspective:

Retrospective

Official Title:

Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in Multiple Endocrine Neoplasia Type 1 Patients

Actual Study Start Date :

Nov 5, 2015

Anticipated Primary Completion Date :

Nov 1, 2025

Anticipated Study Completion Date :

Nov 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
MEN1 Patients Who Have Developed Aggressive PNETs-Cases
MEN1 Patients Who Have Developed Non-Aggressive PNETs-Controls

Outcome Measures

Primary Outcome Measures

Relationship of Serum Global Metabolic Profiles with Subsequent Development of Aggressive PNET [10 years]
Review of samples from 50 cases (aggressive PNET) and 50 controls (non-aggressive PNET).

Secondary Outcome Measures

Prediction of Occurrence of Aggressive PNET among MEN1 Patients by Examining Patterns of Serum Metabolic Biomarkers [10 years]
Advanced metabolomics technology used to demonstrate that various patterns of serum metabolic biomarkers can predict the occurrence of aggressive PNET among MEN1 patients.
Prospectively Identify Potential miRNA Biomarkers of Serum with miRNA Sequencing in MEN1 Patients Who Have Developed Aggressive PNETs (Cases) and MEN1 Patients Who Have Developed Non-Aggressive PNETs (Controls) [10 years]
The levels of miRNA in each group defined as mean ±SD. One-way ANOVA used to identify possible associations between miRNA concentrations and clinicopathological features of aggressive patients.