NCT03048266

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
629

participants targeted

Target at P75+ for all trials

Timeline
6mo left

Started Nov 2015

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress96%
Nov 2015Nov 2026

Study Start

First participant enrolled

November 5, 2015

Completed
1.3 years until next milestone

First Submitted

Initial submission to the registry

February 7, 2017

Completed
2 days until next milestone

First Posted

Study publicly available on registry

February 9, 2017

Completed
8.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2025

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2026

Expected
Last Updated

May 23, 2019

Status Verified

May 1, 2019

Enrollment Period

10 years

First QC Date

February 7, 2017

Last Update Submit

May 21, 2019

Conditions

Multiple Endocrine Neoplasia

Keywords

Multiple endocrine neoplasiaMEN1Aggressive pancreatic neuroendocrine tumorsPNETNon-aggressive pancreatic neuroendocrine tumorsMetabolomicsGeneticsBiomarkers

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (2)

  • Prediction of Occurrence of Aggressive PNET among MEN1 Patients by Examining Patterns of Serum Metabolic Biomarkers

    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)

    10 years

Study Arms (2)

MEN1 Patients Who Have Developed Aggressive PNETs-Cases

MEN1 Patients Who Have Developed Non-Aggressive PNETs-Controls

Eligibility Criteria

Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

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

You may not qualify if:

  • N/A

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Multiple Endocrine NeoplasiaNeuroectodermal Tumors, Primitive

Condition Hierarchy (Ancestors)

Endocrine Gland NeoplasmsNeoplasms by SiteNeoplasmsNeoplasms, Multiple PrimaryNeoplastic Syndromes, HereditaryGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEndocrine System DiseasesNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Study Officials

  • Nancy D. Perrier, MD

    M.D. Anderson Cancer Center

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Nancy D. Perrier, MD

CONTACT

713-792-6940NPerrier@mdanderson.org

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 7, 2017

First Posted

February 9, 2017

Study Start

November 5, 2015

Primary Completion

November 1, 2025

Study Completion (Estimated)

November 1, 2026

Last Updated

May 23, 2019

Record last verified: 2019-05

Locations

US(1)