Multiple Endocrine NeoplasiaNCT03048266
Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in MEN1 Patients
An observational study of Multiple Endocrine Neoplasia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Multiple Endocrine Neoplasia
- How many people
- 629 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Nov 2015to 1 Nov 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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.
Who may be able to join
- The study will include all patients with a confirmed MEN1 diagnosis (clinical, genetic or familial criteria).
- No prior history of PNET.
Who cannot join
- N/A
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: 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.
Read more
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.
- Conditions
- Multiple Endocrine Neoplasia
- Keywords
- Multiple endocrine neoplasia, MEN1, Aggressive pancreatic neuroendocrine tumors, PNET, Non-aggressive pancreatic neuroendocrine tumors, Metabolomics, Genetics, Biomarkers
- Medical subject headings
- Multiple Endocrine Neoplasia, Neuroectodermal Tumors, Primitive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| MEN1 Patients Who Have Developed Aggressive PNETs-Cases | Not described | |
| MEN1 Patients Who Have Developed Non-Aggressive PNETs-Controls | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 10 years
Secondary outcomes (2)
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.
Measured: 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.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2017 (estimated)
- Study start5 Nov 2015
- Primary completion1 Nov 2025 (estimated)
- Study completion1 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple Endocrine NeoplasiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Retrospective
- Enrolment
- 629 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Nancy D. Perrier, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03048266
- Sponsor's protocol ID
- PA15-0822
- Status last verified
- May 2019
- First submitted
- 7 Feb 2017
- Last updated
- 23 May 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in Multiple Endocrine Neoplasia Type 1 Patients
- ClinicalTrials.gov ID
- NCT03048266
- Acronym / sponsor ID
- PA15-0822
- Conditions
- Multiple Endocrine Neoplasia
- Keywords
- Multiple endocrine neoplasia, MEN1, Aggressive pancreatic neuroendocrine tumors, PNET, Non-aggressive pancreatic neuroendocrine tumors, Metabolomics, Genetics, Biomarkers
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 629 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 5 Nov 2015 (actual)
- Primary completion
- 1 Nov 2025 (estimated)
- Study completion
- 1 Nov 2026 (estimated)
- First posted
- 9 Feb 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case-control
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MEN1 Patients Who Have Developed Aggressive PNETs-Cases | – | – |
| MEN1 Patients Who Have Developed Non-Aggressive PNETs-Controls | – | – |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- The study will include all patients with a confirmed MEN1 diagnosis (clinical, genetic or familial criteria).
- No prior history of PNET.
Key exclusion criteria (1 listed)
- N/A
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Relationship of Serum Global Metabolic Profiles with Subsequent Development of Aggressive PNET
Time frame: 10 years
Review of samples from 50 cases (aggressive PNET) and 50 controls (non-aggressive PNET).
2 secondary outcomes
Prediction of Occurrence of Aggressive PNET among MEN1 Patients by Examining Patterns of Serum Metabolic Biomarkers
Time frame: 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)
Time frame: 10 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2019.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nancy D. Perrier, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Multiple Endocrine Neoplasia, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Nancy D. Perrier, MD (713-792-6940, NPerrier@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relationship of Serum Global Metabolic Profiles with Subsequent Development of Aggressive PNET (time frame: 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2019.