Clinical trial NCT02270567: RegisterNET - A Registry for Neuroendocrine Tumors in the USA
Official title: A Registry for Neuroendocrine Tumors in the USA
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Neuroendocrine Tumor
Carcinoid
Neuroendocrine Carcinoma
Lead sponsor: Wren Laboratories LLC
Sponsor class: INDUSTRY
First posted: 2014-10-21
Enrollment: 400
Sex: ALL
Minimum age (years): 21
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with a histologically confirmed diagnosis of a neuroendocrine tumour

Brief summary: Neuroendocrine tumors are derived from the neuroendocrine system of the gastroenteropancreatic and bronchopulmonary tract systems. Treatment options include surgery, medical and ablative therapies as well as peptide-receptor radionuclide therapy. Survival is linked to early and accurate diagnoses or to the effective detection of disease recurrence and/or treatment failure. One challenge is to develop accurate non-invasive blood tests that can detect neuroendocrine tumor activity. A second challenge is to evaluate the effectiveness of molecular biomarkers in the natural history of this disease. RegisterNET registry aims at collecting data and blood samples from patients presenting with a NET in the USA. Data will be entered prospectively and anonymized after informed consent. All physicians who treat neuroendocrine tumor patients are invited to participate to the registry. Data will be evaluated within regular time frames, focusing on diagnostic accuracy for biomarkers in the different types and grades of tumors, treatment modalities and patient outcomes (e.g. disease recurrence and survival), thereby contributing to an understanding of the role of biomarkers in tumor management.

Detailed description: Background: Survival for neuroendocrine tumors is linked to early and accurate diagnoses or to the effective detection of disease recurrence and/or treatment failure. Non-invasive biomarkers have been identified that can improve diagnosis and prognosis of patients. Little, however, is known about the utility of these markers in clinical practice.

Objective: To systematically and prospectively collect clinical information and blood samples from neuroendocrine tumors in the USA based a histologically confirmed diagnosis.

Methods: All neuroendocrine tumors (gastroenteropancreatic and pulmonary) are following informed consent. Data will be entered prospectively and anonymized. Patient history including a quality of life survey are completed by contributing physicians and blood sample is collected for analysis. All information will be transferred to the database. Evaluation of treatment modalities and patient outcomes (e.g. disease recurrence) will be assessed at follow-up times.

The primary objectives of the project are to:

* monitor patients with neuroendocrine tumors
* provide descriptive statistical analyses
* assessment of diagnostic accuracy of molecular-based blood tests

The secondary objectives of the project include:

* assessment of disease recurrence
* analysis of patient survival

Analyses will include:

1. Descriptive statistical analyses including demographics, site, treatment, QoL assessment.
2. Clinical follow-up and blood chemistry results.
3. Correlation analyses between blood results and clinical data. This will include assessment of the time at which the blood chemistry results significantly (and consistently) increases and the time of tumor recurrence and an evaluation whether the change in blood results is predictive of disease recurrence.

Eligibility criteria: Inclusion Criteria:

* Gastroenteropancreatic neuroendocrine tumor
* Bronchopulmonary neuroendocrine tumor
* Gastroenteropancreatic neuroendocrine carcinoma
* Patient provides informed consent

Exclusion Criteria:

* N/A

Primary outcomes:
measure: Tumor-related recurrence
timeFrame: 5 years
measure: Tumor-related diagnosis
timeFrame: 1 year
measure: Tumor-related mortality
timeFrame: 10 years
Secondary outcomes:
measure: Quality of Life Evaluation
timeFrame: 5 years
measure: Biomarker prediction of treatment response and disease relapse
timeFrame: 10 years
Other outcomes:
Locations:
city: Branford
country: United States
facility: Wren Laboratories
state: Connecticut
Study officials:
References:
citation: Modlin IM, Drozdov I, Kidd M. The identification of gut neuroendocrine tumor disease by multiple synchronous transcript analysis in blood. PLoS One. 2013 May 15;8(5):e63364. doi: 10.1371/journal.pone.0063364. Print 2013.
citation: Modlin IM, Drozdov I, Kidd M. Gut neuroendocrine tumor blood qPCR fingerprint assay: characteristics and reproducibility. Clin Chem Lab Med. 2014 Mar;52(3):419-29. doi: 10.1515/cclm-2013-0496.
citation: Modlin IM, Drozdov I, Alaimo D, Callahan S, Teixiera N, Bodei L, Kidd M. A multianalyte PCR blood test outperforms single analyte ELISAs (chromogranin A, pancreastatin, neurokinin A) for neuroendocrine tumor detection. Endocr Relat Cancer. 2014 Aug;21(4):615-28. doi: 10.1530/ERC-14-0190.
citation: Modlin IM, Drozdov I, Bodei L, Kidd M. Blood transcript analysis and metastatic recurrent small bowel carcinoid management. BMC Cancer. 2014 Aug 5;14:564. doi: 10.1186/1471-2407-14-564.
citation: Liu E, Paulson S, Gulati A, Freudman J, Grosh W, Kafer S, Wickremesinghe PC, Salem RR, Bodei L. Assessment of NETest Clinical Utility in a U.S. Registry-Based Study. Oncologist. 2019 Jun;24(6):783-790. doi: 10.1634/theoncologist.2017-0623. Epub 2018 Aug 29.
citation: Kidd M, Kitz A, Drozdov I, Modlin I. Neuroendocrine Tumor Omic Gene Cluster Analysis Amplifies the Prognostic Accuracy of the NETest. Neuroendocrinology. 2021;111(5):490-504. doi: 10.1159/000508573. Epub 2020 May 11.