Clinical Trial: Regorafenib in Treating Patients With Advanced or Metastatic Neuroendocrine Tumors

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase II Study of Single Agent Regorafenib in Patients With Advanced/Metastatic Neuroendocrine Tumors

Brief Summary: This phase II trial studies regorafenib in treating patients with neuroendocrine tumors that have spread from the primary site (place where it started) to other places in the body. Regorafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To assess progression-free survival (PFS) in advanced/metastatic in patients with carcinoid or pancreatic islet cell tumors.

SECONDARY OBJECTIVES:

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.

OUTLINE:

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.

Current Primary Outcome: PFS [ Time Frame: Time from start of treatment to time of progression or death on study whichever comes first, assessed at 6 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• 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.

Original Secondary Outcome:

• Tumor response rate, evaluated using the new international criteria proposed by the Response Evaluation Criteria in Solid Tumors 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 NCI 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.

Dates:
Date Received: October 3, 2014
Date Started: August 16, 2016
Date Completion: August 16, 2020
Last Updated: April 18, 2017
Last Verified: April 2017