Clinical trial NCT03048266: Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in MEN1 Patients
Official title: Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in Multiple Endocrine Neoplasia Type 1 Patients
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Multiple Endocrine Neoplasia
Keywords: Multiple endocrine neoplasia
MEN1
Aggressive pancreatic neuroendocrine tumors
PNET
Non-aggressive pancreatic neuroendocrine tumors
Metabolomics
Genetics
Biomarkers
Lead sponsor: M.D. Anderson Cancer Center
Sponsor class: OTHER
First posted: 2017-02-09
Enrollment: 629
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: RETROSPECTIVE
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.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Relationship of Serum Global Metabolic Profiles with Subsequent Development of Aggressive PNET
timeFrame: 10 years
Secondary outcomes:
measure: Prediction of Occurrence of Aggressive PNET among MEN1 Patients by Examining Patterns of Serum Metabolic Biomarkers
timeFrame: 10 years
measure: 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)
timeFrame: 10 years
Other outcomes:
Locations:
city: Houston
country: United States
facility: University of Texas MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Nancy D. Perrier, MD
role: PRINCIPAL_INVESTIGATOR
References: