Trial results for a Phase 2 study (NCT02314169) investigating nivolumab with or without ipilimumab in patients with refractory metastatic anal canal cancer were posted on ClinicalTrials.gov on 2026-04-22. The study's Part A, evaluating nivolumab monotherapy, demonstrated an overall response rate of 24%. In Part B, the combination of nivolumab and ipilimumab achieved a median progression-free survival of 3.7 months.

Background

The study investigated nivolumab, an immunotherapy agent, alone or in combination with ipilimumab, for treating patients with anal canal cancer that had not responded to previous treatment and had spread to other parts of the body. Both nivolumab and ipilimumab are monoclonal antibodies that aim to help the body's immune system attack cancer cells.

Trial design

The study, NCT02314169, was a Phase 2 trial designed to evaluate nivolumab with or without ipilimumab. It enrolled 143 participants with Anal Canal Squamous Cell Carcinoma, Metastatic Anal Canal Carcinoma, or Stage IV Anal Cancer AJCC v8. The trial was structured into two parts: Part A investigated nivolumab monotherapy, while Part B compared nivolumab alone (Arm I) against nivolumab combined with ipilimumab (Arm II).

Key results

For Part A, evaluating nivolumab monotherapy, the Overall Response Rate was 24% (percentage of patients with a response).

In Part B, Arm I, participants treated with nivolumab had a median Progression-free Survival (PFS) of 2.9 months. For Part B, Arm II, the combination of nivolumab and ipilimumab resulted in a median PFS of 3.7 months.

Statistical analysis comparing the median PFS between the treatment arms in Part B using a one-sided log rank test yielded a p-value of 0.25. A Cox Proportional Hazard analysis showed a value of 0.98 (90.0% CI: 0.64 to 1.51). A Chi-squared analysis resulted in a p-value of 0.89.

What this means

The trial results indicate that nivolumab monotherapy demonstrated an objective response rate in patients with refractory metastatic anal canal cancer. While the combination of nivolumab and ipilimumab showed a numerically longer median progression-free survival compared to nivolumab alone in Part B, the statistical analysis (p-value of 0.25) suggests this difference was not statistically significant. These findings provide insights into the activity of immunotherapy options in this challenging patient population.

Source

The information for these trial results was sourced from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT02314169, titled "Nivolumab With or Without Ipilimumab in Treating Patients With Refractory Metastatic Anal Canal Cancer," were posted on 2026-04-22 on clinicaltrials.gov.