Neuroendocrine Tumors - Patient Reported Outcomes

NCT ID: NCT05064150

Last Updated: 2025-12-18

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 2539 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-05-10
• Study Completion Date: 2026-06-30

Brief Summary

With so many therapeutic options available (i.e.: biologic therapy, liver directed therapy, radiotherapy and chemotherapy), the purpose of this project is to partner with patients on comparative effectiveness research (CER) to achieve the goal of alleviating undue toxicity, and optimizing effectiveness and sequencing of therapy for neuroendocrine tumors (NET) patients. We will conduct a study of all newly occurring GEP-NET and lung NET cases aged 18 years and older diagnosed between 01/01/2018 through 09/30/2024 across 14 sites participating in the National Patient-Centered Clinical Research Network (PCORnet), enrolling an average of 215 patients per site over the 3 year study period (2515 patients total), allowing up to 60 months of follow-up for medical record outcomes. Participants will complete four online or paper surveys over 18 months; these surveys will focus on patient-reported outcomes, including questions on quality of life, treatment decisions, and experiences with cancer care. Survey data will be linked to participant medical record data to achieve study aims.

Detailed Description

NETs are a group of neoplasms that occur most frequently in the gastrointestinal tract, pancreas and the lungs, collectively referred to as gastroenteropancreatic (GEP-NETs) and lung-NETs. There are currently fewer than 180,000 patients living with this condition in the United States, meeting the criteria for rare disease status. NETs are typically slow growing, with vague signs and a myriad of symptoms (carcinoid syndrome) leading to diagnostic delays. Thus, NET patients typically experience a prolonged clinical course with active disease, and many have significant symptom burdens. However, assessment of quality of life outside of therapy trials remains scarce and of poor quality. What's more, over half of GEP-NETs are diagnosed with spread of their disease at diagnosis and are not candidates for curative surgery. Fortunately, many of these tumors are amenable to long-term medical treatment with somatostatin analogues (SSAs)- which slow down the production of hormones, especially serotonin, which helps to control the symptoms of carcinoid syndrome. However, living with distant spread of the disease increases the probability for the disease to progress. Following failure of first-line SSA therapy there are no clear consensus guidelines as to the optimum sequencing of other therapeutic options. NET patients are left wondering not only 'what therapy would be best to try next?', but 'if I were to take this option now, what treatment options will be closed off to me in the future?' and clinicians are unsure as to how best to tailor treatment selection on the characteristics of the patient and their tumor.

There is currently no large nationally recruiting prospective (forward in time) observational study of NET patients. Our large study will robustly generate real-world evidence on the frequency and sequence of commonly used treatments for GEP and lung NET patients in relation to Patient Reported Outcomes (PROs) and survival/progression, endpoints that matter most to NET patients, their caregivers, and clinicians involved in their care. Given the lack of consensus guidelines as to the optimum sequencing of treatments, evidence generated in this study will aid patient (and clinician) navigation and selection of the next most appropriate therapy, accounting for the preferences and needs of the individual patient, whilst respecting the underlying profile of their tumor. Moreover, the infrastructure this study will generate (i.e.: electronic identification of NET patients, entry and completion of tumor table data in PCORnet, and a unique NET patient health record portal), will foster future CER studies in NETs and other rare diseases.

The four specific aims of this project are:

1. To describe the frequency of treatment regimens received by line of therapy, and examine their association with symptom burden and changes in 6, 12 and 18 month health-related quality of life (HRQoL) outcomes. The influence of patient preferences, beliefs, attitudes, and experience of care on choice of these treatment regimens will also be examined.

2. To examine the association of patient, clinical, and tumor characteristics on the selection of first-line and beyond treatment regimens and compare the effects of common treatment sequences on frequency of subsequent treatments received and outcomes of overall survival and disease progression.

3. To compare the effectiveness of peptide receptor radionuclide therapy (PRRT) regimens on outcomes of renal toxicity, disease progression, and patient-reported symptoms and HRQoL.

4. To disseminate lessons learned and expand enrollment of the prospective cohort to patient advocate organizations, and to use the infrastructure developed to aid in the study of other rare diseases.

Conditions

Neuroendocrine Tumors; Gastroenteropancreatic Neuroendocrine Tumor; Lung Neuroendocrine Neoplasm; Neuroendocrine Carcinoma

Keywords

Patient-reported outcomes; Neuroendocrine; Quality of Life; Survival; Treatment sequencing; Symptom burden

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

NET patient observational cohort

Patients diagnosed with lung or gastrointestinal neuroendocrine tumors

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• (1) Adults age 18 years or older at time of NET diagnosis
• (2) Diagnosis of GEP-NET or lung NET between 1/1/2018 and 09/30/2024, as evidenced by
• (a) medical record information on diagnoses and/or medications and/or treatments and/or test results and/or clinical notes and/or procedures and/or encounters and/or tumor characteristics, and
• (b) patient self-attestation of their diagnosis.

Exclusion Criteria

Any GEP-NET/Lung NET prior to 1/1/18, as evidenced by medical record information on diagnoses and/or medications and/or treatments and/or test results and/or clinical notes and/or procedures and/or encounters and/or tumor characteristics

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Allina Health System

OTHER

Sponsor Role: collaborator

Medical College of Wisconsin

OTHER

Sponsor Role: collaborator

University of Kansas Medical Center

OTHER

Sponsor Role: collaborator

University of Texas Southwestern Medical Center

OTHER

Sponsor Role: collaborator

University of Utah

OTHER

Sponsor Role: collaborator

University of Florida

OTHER

Sponsor Role: collaborator

Ohio State University

OTHER

Sponsor Role: collaborator

University of Michigan

OTHER

Sponsor Role: collaborator

University of Pittsburgh Medical Center

OTHER

Sponsor Role: collaborator

Medical University of South Carolina

OTHER

Sponsor Role: collaborator

University of North Carolina, Chapel Hill

OTHER

Sponsor Role: collaborator

Vanderbilt University Medical Center

OTHER

Sponsor Role: collaborator

Mayo Clinic

OTHER

Sponsor Role: collaborator

Northern California CarciNET Community

UNKNOWN

Sponsor Role: collaborator

Neuroendocrine Cancer Awareness Network

UNKNOWN

Sponsor Role: collaborator

Neuroendocrine Tumor Research Foundation

UNKNOWN

Sponsor Role: collaborator

Healing NET Foundation

UNKNOWN

Sponsor Role: collaborator

Patient-Centered Outcomes Research Institute

OTHER

Sponsor Role: collaborator

University of Iowa

OTHER

Sponsor Role: lead

Responsible Party

Michael A O'Rorke

Assistant Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Michael O'Rorke, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Iowa

Locations

University of Florida

Gainesville, Florida, United States

University of Iowa

Iowa City, Iowa, United States

University of Kansas Medical Center Research Institute, Inc

Kansas City, Kansas, United States

Regents of the University of Michigan

Ann Arbor, Michigan, United States

Allina Health System

Minneapolis, Minnesota, United States

Mayo Clinic Rochester

Rochester, Minnesota, United States

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

The Ohio State University

Columbus, Ohio, United States

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

The Medical University of South Carolina

Charleston, South Carolina, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

UT Southwestern Medical Center

Dallas, Texas, United States

University of Utah

Salt Lake City, Utah, United States

Medical College of Wisconsin, Inc

Milwaukee, Wisconsin, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Other Identifiers

202104599

Identifier Type: -

Identifier Source: org_study_id