Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in MEN1 Patients

Study Overview
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Brief Summary
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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.

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

Conditions
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Multiple Endocrine Neoplasia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The study will include all patients with a confirmed MEN1 diagnosis (clinical, genetic or familial criteria). No prior history of PNET. Exclusion Criteria: N/A
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 |
| --- | --- |
| MEN1 Patients Who Have Developed Aggressive PNETs-Cases<br> | |
| MEN1 Patients Who Have Developed Non-Aggressive PNETs-Controls<br> | |

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Multiple endocrine neoplasia, MEN1, Aggressive pancreatic neuroendocrine tumors, PNET, Non-aggressive pancreatic neuroendocrine tumors, Metabolomics, Genetics, Biomarkers