Study Shows Potential of Cabozantinib to Treat Patients With Advanced Neuroendocrine Tumors

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An interim analysis of the CABINET trial has shown that the phase III pivotal trial has met its primary endpoint, according to a press release from the Alliance for Clinical Trials in Oncology. Cabozantinib demonstrated statistically significant and clinically meaningful improvements in progression-free survival for patients with advanced neuroendocrine tumors, the Alliance reported.


“Patients with progressive neuroendocrine tumors have limited treatment options. At present, after progression on previous therapies, the treatment path is unclear—underscoring the need for additional options for this disease that is rising in incidence,” stressed Jennifer Chan, MD, MPH, Clinical Director of the Gastrointestinal Cancer Center and Director of the Program in Carcinoid and Neuroendocrine Tumors at Dana-Farber Cancer Institute as well as Study Chair of the CABINET trial.

About 12,000 patients are diagnosed with neuroendocrine tumors per year. The tumors develop from cells in the diffuse neuroendocrine system and are most common in the gastrointestinal tract, lungs, and pancreas. Most neuroendocrine tumors grow slowly, but some are capable of rapidly advancing and metastasizing to other parts of the body.

Researchers have identified several treatment options for patients with neuroendocrine tumors—including surgery, liver-directed therapy, somatostatin analogs, chemotherapy, targeted therapy, and peptide receptor radionuclide therapy.

Study Methods and Results

In the new interim analysis of the phase III CABINET pivotal trial ( identifier NCT03375320), researchers randomly assigned 290 patients with advanced neuroendocrine tumors—93 of whom had pancreatic neuroendocrine tumors and 197 of whom had extra-pancreatic neuroendocrine tumors—who experienced disease progression after prior systemic therapy to receive cabozantinib or placebo.

The researchers noted that the patients must have had measurable disease per RECIST 1.1 criteria and must have experienced disease progression after at least one U.S. Food and Drug Administration (FDA)-approved line of prior therapy other than somatostatin analogs. The primary endpoint was progression-free survival in each cohort. Upon confirmation of disease progression, the patients were unblinded, and those receiving placebo were permitted to cross over to open-label therapy with cabozantinib. Secondary endpoints included overall survival, radiographic response rate, and safety.

The researchers found that cabozantinib showed efficacy benefit for patients with pancreatic and extra-pancreatic neuroendocrine tumors. The safety profile of cabozantinib was consistent with its known safety profile, and the researchers discovered no new safety signals.

The Data and Safety Monitoring Board recommended that the researchers end the trial early as a result of efficacy findings that will be discussed with the FDA. Detailed results from the trial will be presented at an upcoming scientific meeting.


“These promising findings from the CABINET trial … are welcome news and show the potential for cabozantinib to address important unmet needs for this community,” Dr. Chan emphasized.

"The Alliance and NCTN have a long and established history of successful practice changing cancer clinical trials. The results of [the] CABINET [trial] add to this important work to further improve the outcomes of patients with these rare tumors," suggested Suzanne George, MD, Associate Professor of Medicine at Harvard Medical School, Clinical Director at the Center for Sarcoma and Bone Oncology at Dana-Farber Cancer Institute, Interim Group Chair of the Alliance for Clinical Trials in Oncology.

Disclosure: The CABINET trial was sponsored by the National Cancer Institute.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.