Phase II trial dedicated to non-selected, pretreated cutaneous angiosarcoma: Efficacy of nivolumab (AngioCheck Study).
Level 4 - case-series / case-control
Single-arm, open-label phase II clinical trial without a concurrent control group.
PubMed 40472568 · doi:10.1016/j.ejca.2025.115537
What was done
This multicenter, open-label, single-arm phase II study (AngioCheck) evaluated the efficacy and safety of nivolumab (480 mg every 4 weeks) in 23 patients with histologically confirmed cutaneous angiosarcoma previously treated with docetaxel or paclitaxel across 11 institutions in Japan. The primary endpoint was centrally reviewed overall response rate (ORR). Secondary endpoints included investigator-assessed ORR, overall survival (OS), progression-free survival (PFS), and an exploratory tumor mutational burden (TMB) biomarker analysis.
What was found
- Centrally reviewed ORR was 13.0% (3 partial responses [PR]; 90% CI: 3.7–30.4%), which did not meet the predefined success threshold. - Investigator-assessed ORR was 21.7% (5 PR). - Median PFS was 59 days (90% CI: 57–112). - Median OS was 259 days (90% CI: 188.0–387.0). - In TMB analysis (n=16), TMB-high patients (n=7) had 0 PR and 3 stable disease (SD), while non-TMB-high patients (n=9) had 2 PR and 1 SD; no significant association between TMB status and response was identified despite a numerically higher disease control rate in the TMB-high group (42.9% vs. 33.3%).
Why it matters
This study provides prospective evidence that nivolumab monotherapy has limited efficacy in unselected, taxane-pretreated cutaneous angiosarcoma and that high tumor mutational burden does not clearly predict response.
Limits
The trial lacked a randomized control group and had a small sample size (n=23, with TMB analysis restricted to 16 patients). The population was recruited entirely from Japanese institutions, and detailed toxicity/safety data were not reported in the abstract.