Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in MEN1 Patients

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

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.

Study Overview

• Status: Recruiting
• Conditions: Multiple Endocrine Neoplasia

• Study Type: Observational
• Enrollment (Anticipated): 629

Contacts and Locations

• Study Contact: Name: Nancy D. Perrier, MD; Phone Number: 713-792-6940; Email: NPerrier@mdanderson.org

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • University of Texas MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Study subjects identified from a cohort of MEN1 patients. These patients had previously consented to collection of clinical data, blood, and parathyroid tissue for research purposes at MD Anderson Cancer Center.

Description

Inclusion Criteria:

1. The study will include all patients with a confirmed MEN1 diagnosis (clinical, genetic or familial criteria).
2. No prior history of PNET.

Exclusion Criteria:

N/A

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
MEN1 Patients Who Have Developed Aggressive PNETs-Cases
MEN1 Patients Who Have Developed Non-Aggressive PNETs-Controls

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Relationship of Serum Global Metabolic Profiles with Subsequent Development of Aggressive PNET	Review of samples from 50 cases (aggressive PNET) and 50 controls (non-aggressive PNET).	10 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Prediction of Occurrence of Aggressive PNET among MEN1 Patients by Examining Patterns of Serum Metabolic Biomarkers	Advanced metabolomics technology used to demonstrate that various patterns of serum metabolic biomarkers can predict the occurrence of aggressive PNET among MEN1 patients.	10 years
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)	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.	10 years

Collaborators and Investigators

• Sponsor: M.D. Anderson Cancer Center
• Investigators: Principal Investigator: Nancy D. Perrier, MD, M.D. Anderson Cancer Center

Publications and helpful links

Helpful Links

• University of Texas MD Anderson Cancer Center Website

Study record dates

Study Major Dates

• Study Start (Actual): November 5, 2015
• Primary Completion (Anticipated): November 1, 2025
• Study Completion (Anticipated): November 1, 2026

Study Registration Dates

• First Submitted: February 7, 2017
• First Submitted That Met QC Criteria: February 7, 2017
• First Posted (Estimate): February 9, 2017

Study Record Updates

• Last Update Posted (Actual): May 23, 2019
• Last Update Submitted That Met QC Criteria: May 21, 2019
• Last Verified: May 1, 2019

More Information

Terms related to this study

• Keywords: Genetics; Metabolomics; Biomarkers; MEN1; PNET; Multiple endocrine neoplasia; Aggressive pancreatic neuroendocrine tumors; Non-aggressive pancreatic neuroendocrine tumors
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Endocrine System Diseases; Digestive System Neoplasms; Genetic Diseases, Inborn; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neoplastic Syndromes, Hereditary; Pancreatic Diseases; Adenoma; Neoplasms, Multiple Primary; Pancreatic Neoplasms; Neoplasms; Neuroendocrine Tumors; Endocrine Gland Neoplasms; Adenoma, Islet Cell; Multiple Endocrine Neoplasia
• Other Study ID Numbers: PA15-0822

Drug and device information, study documents

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