Trial results for the combination of nivolumab, cabozantinib s-malate, and ipilimumab in poorly differentiated neuroendocrine tumors were posted on ClinicalTrials.gov on 2025-12-12. The study, NCT04079712, reported a median duration of response of 664 days for participants in the treatment arm.
Background
The trial investigated a triple combination therapy for patients with poorly differentiated neuroendocrine tumors, including metastatic large cell neuroendocrine carcinoma, metastatic neuroendocrine carcinoma, metastatic neuroendocrine neoplasm, and metastatic small cell neuroendocrine carcinoma. The combination included cabozantinib, which may block enzymes needed for tumor cell growth, and immunotherapies nivolumab and ipilimumab, which aim to enhance the immune system's attack on cancer.
Trial design
The Phase 2 trial (NCT04079712) enrolled 17 participants with poorly differentiated neuroendocrine tumors, specifically metastatic large cell neuroendocrine carcinoma, metastatic neuroendocrine carcinoma, metastatic neuroendocrine neoplasm, and metastatic small cell neuroendocrine carcinoma. The study evaluated the combination of cabozantinib s-malate, nivolumab, and ipilimumab.
Key results
Key measurements from the trial included:
- For the outcome "Number of Participants With a Response," values of 2 participants and 14 participants were reported.
- Median Progression Free Survival (PFS) was 85 days (Full Range).
- For the outcome "Number of Participants Reporting Adverse Events," values of 0, 3, 7, and 6 participants were reported.
- For the outcome "Disease Control Rate (DCR)," values of 9 participants and 7 participants were reported.
- Median Duration of Response (DOR) was 664 days (Full Range).
What this means
The results indicate that the combination of nivolumab, cabozantinib, and ipilimumab demonstrated a median duration of response of 664 days in this small cohort of patients with poorly differentiated neuroendocrine tumors. While the overall number of participants with a response was limited, the observed duration of response for those who responded suggests potential clinical activity for this triple therapy in a difficult-to-treat patient population.
Source
The information for these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT04079712, titled 'Testing the Combination of XL184 (Cabozantinib), Nivolumab, and Ipilimumab for Poorly Differentiated Neuroendocrine Tumors,' were posted on 2025-12-12 on clinicaltrials.gov.