Phase II study of trabectedin in pretreated patients with advanced colorectal cancer.

Abstract Purpose This open-label, nonrandomized, phase II study was aimed at evaluating the efficacy and toxicity of trabectedin over a 3-hour intravenous infusion every 3 weeks in patients with pretreated advanced colorectal cancer. Patients and Methods Twenty-one patients were enrolled: 5 patients (23.8%) were treated with 1650 μg/m 2 , 10 patients (47.6%) with 1300 μg/m 2 , and 6 patients (28.6%) with 1100 μg/m 2 . Response to treatment was assessed according to World Health Organization criteria, and toxicities were graded according to National Cancer Institute Common Toxicity Criteria, version 2.0. Results The median number of treatment cycles per patient was 2 (range, 1–6 cycles). No objective responses were reported. Four patients (19%; 95% confidence interval [CI], 5.5%–41.9%) exhibited stable disease lasting for a median of 3.6 months (range, 2.4–4.9 months). The median time to progression was 1.5 months (95% CI, 1.3–1.6 months), and the median overall survival was 4.4 months (95% CI, 3–7.5 months; n = 2 censored). The main grade 3/4 toxicities were transient asymptomatic transaminase increase (alanine aminotransferase, 66.7% of patients; aspartate aminotransferase, 57.1%) and neutropenia (42.8%). No toxic deaths were reported. Conclusion Trabectedin 1300 μg/m 2 given as a 3-hour intravenous infusion every 3 weeks was well tolerated but lacked activity in pretreated advanced-stage colorectal cancer. Therefore, further clinical trials with this trabectedin schedule as a single agent are not warranted.