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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT05064150
Other study ID # 202104599
Secondary ID
Status Enrolling by invitation
Phase
First received
Last updated
Start date May 10, 2022
Est. completion date June 2026

Study information

Verified date June 2024
Source University of Iowa
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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 12/31/2023 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 (~3,000 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.


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.


Recruitment information / eligibility

Status Enrolling by invitation
Enrollment 3010
Est. completion date June 2026
Est. primary completion date June 2026
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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 12/31/2023, 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

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
United States Regents of the University of Michigan Ann Arbor Michigan
United States The University of North Carolina at Chapel Hill Chapel Hill North Carolina
United States The Medical University of South Carolina Charleston South Carolina
United States The Ohio State University Columbus Ohio
United States UT Southwestern Medical Center Dallas Texas
United States 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 Medical College of Wisconsin, Inc Milwaukee Wisconsin
United States Allina Health System Minneapolis Minnesota
United States Vanderbilt University Medical Center Nashville Tennessee
United States University of Pittsburgh Pittsburgh Pennsylvania
United States Mayo Clinic Rochester Rochester Minnesota
United States University of Utah Salt Lake City Utah

Sponsors (19)

Lead Sponsor Collaborator
University of Iowa Allina Health System, Healing NET Foundation, Mayo Clinic, Medical College of Wisconsin, Medical University of South Carolina, Neuroendocrine Cancer Awareness Network, Neuroendocrine Tumor Research Foundation, Northern California CarciNET Community, Ohio State University, Patient-Centered Outcomes Research Institute, University of Florida, University of Kansas Medical Center, University of Michigan, University of North Carolina, Chapel Hill, University of Pittsburgh Medical Center, University of Texas Southwestern Medical Center, University of Utah, Vanderbilt University Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary European Organisation for Research and Treatment of Cancer - Quality of Life Questionnaire (EORTC QLQ-C30) Incorporates five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status / QoL scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease. Responses to the QLQ-C30 will be linearly transformed to a 0-100 scale using EORTC guidelines, a higher score represents a higher ("better") level of functioning, or a higher ("worse") level of symptoms. Change in score across baseline, 6, 12, and 18 month time points.
Primary European Organisation for Research and Treatment of Cancer - Quality of Life Questionnaire - Neuroendocrine Carcinoid Module (EORTC QLQ-GI.NET21) The QLQ-GINET21 contains a total of 21 items: four single-item assessments relating to muscle and/or bone pain (MBP), body image (BI), information (INF) and sexual functioning (SX), together with 17 items organized into five proposed scales: endocrine symptoms (ED; three items), GI symptoms (GI; five items), treatment-related symptoms (TR; three items), social functioning (SF) of the new module (SF21; three items) and disease-related worries (DRW; three items). Responses to the QLQ-GINET21 will be linearly transformed to a 0-100 scale using EORTC guidelines, with higher scores reflecting more severe symptoms. Change in score across baseline, 6, 12, and 18 month time points.
Primary Sequencing of treatment regimens from electronic medical records (% of patients using modality) Ordering of treatment receipt i.e.: first-line, 2nd line 3rd line therapies Up to 5 years
Primary Renal function Creatinine clearance loss (per/Yr) Change in score across baseline, 6, 12, and 18 month time points.
Secondary Norfolk Carcinoid Symptom Score Patient-reported symptoms Change in score across baseline, 6, 12, and 18 month time points.
Secondary Experiences with cancer care (from CANCORS) 13 items to assess cancer care as described at https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2953972/ Change in score across baseline, 6, 12, and 18 month time points.
Secondary Progression-free survival Time to event 1-, 3-, and 5-year
Secondary Overall survival Time to event Up to 5 years
Secondary Adverse toxicities Incident acute renal failure, dialysis and liver failure during follow-up Up to 5 years
Secondary Presence of Acute Renal Failure Diagnosis Common Data Model (CDM) diagnosis codes for acute renal failure and dialysis Up to 5 years
Secondary Health related Quality of Life (HRQoL) by PRRT regimen Changes in HRQOL Change in score across baseline, 6, 12, and 18 month time points.
Secondary Symptom scores by PRRT regimen Changes in symptom scores Change in score across baseline, 6, 12, and 18 month time points.
Secondary Renal toxicity (creatinine clearance) by PRRT isotope Creatinine clearance loss (per/Yr) 177Lu vs 90Y Up to 5 years
Secondary Renal toxicity of PRRT by primary tumor location & grade 3 disease Creatinine clearance loss (per/Yr) GEP-NETs vs lung NETs and G3 Up to 5 years
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