Regorafenib in Treating Patients With Advanced or Metastatic Neuroendocrine Tumors
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02259725|
Recruitment Status : Active, not recruiting
First Posted : October 8, 2014
Last Update Posted : January 25, 2019
|Condition or disease||Intervention/treatment||Phase|
|Gastrinoma Glucagonoma Insulinoma Metastatic Gastrointestinal Carcinoid Tumor Pancreatic Polypeptide Tumor Pulmonary Carcinoid Tumor Recurrent Gastrointestinal Carcinoid Tumor Recurrent Islet Cell Carcinoma Somatostatinoma||Drug: regorafenib Other: laboratory biomarker analysis||Phase 2|
I. To assess progression-free survival (PFS) in advanced/metastatic in patients with carcinoid or pancreatic islet cell tumors.
I. To assess overall survival and response rate in advanced/metastatic poor prognosis in patients with carcinoid or pancreatic islet cell tumors.
II. To assess the toxicity of patients treated with regorafenib. III. To explore markers of angiogenesis as they relate to outcome in carcinoid and pancreatic islet cell tumors.
Patients receive regorafenib orally (PO) once daily (QD) on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3 months.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Study of Single Agent Regorafenib in Patients With Advanced/Metastatic Neuroendocrine Tumors|
|Actual Study Start Date :||August 16, 2016|
|Estimated Primary Completion Date :||August 16, 2020|
|Estimated Study Completion Date :||August 16, 2021|
Experimental: Treatment (regorafenib)
Patients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Other: laboratory biomarker analysis
- PFS [ Time Frame: Time from start of treatment to time of progression or death on study whichever comes first, assessed at 6 months ]Two parallel Simon's 2-stage phase II trials will be conducted to evaluate the efficacy of regorafenib in patients with advanced carcinoid (cohort A) or pancreatic islet cell tumors (cohort B). Will be summarized as a proportion of patients who are alive and progression-free among all patients in the primary data analysis set. The 95% confidence intervals (CIs) will be calculated using the Wilson method. Will be analyzed using Kaplan-Meier (KM) curves. The median PFS and 95% CIs will be calculated. The probability of 6-month PFS will be estimated from the KM curve too.
- Tumor response rate, evaluated using the new international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) Committee [ Time Frame: Up to 4 years ]Will be calculated as a proportion of patients who have either a complete or partial response among all patients in the primary data analysis set. The 95% CIs will be given.
- Overall survival [ Time Frame: From start of treatment until death due to any cause, assessed up to 4 years ]The 95% CIs will be calculated using the Wilson method. Will be analyzed using KM curves.
- Incidence of adverse events, assessed according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v4.0 [ Time Frame: Up to 4 years ]Toxicity profile will be summarized by attribution: regorafenib-related and all reported, course: course 1 and all courses, type, and grade: grade 1-2, 3-4, and 5.
- Biomarkers such as messenger ribonucleic acid (mRNA) levels or germline variations of genes related to angiogenesis [ Time Frame: Up to 4 years ]The associations between biomarkers and clinical outcomes (6-month PFS, response, PFS, and overall survival) will be analyzed in univariate analysis first using appropriate methods. Multivariable analyses will be conducted to evaluate the independent effect of a marker on clinical outcome. All tests will be two-sided at a significance level of 0.05. P values will be adjusted for multiple comparisons.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02259725
|United States, Arizona|
|Mayo Clinic in Arizona|
|Scottsdale, Arizona, United States, 85259|
|United States, California|
|USC Norris Comprehensive Cancer Center|
|Los Angeles, California, United States, 90033|
|Hoag Memorial Hospital Presbyterian|
|Newport Beach, California, United States, 92663|
|USC Norris Oncology Hematology‐Newport Beach|
|Newport Beach, California, United States, 92663|
|United States, Washington|
|Seattle Cancer Care Alliance|
|Seattle, Washington, United States, 98109|
|Principal Investigator:||Syma Iqbal, MD||University of Southern California|