Aim: This study examined the clinical characteristics and outcomes of patients enrolled in phase 0 and 1 clinical trials at Liverpool Cancer Therapy Centre.
Methods: We collected data retrospectively from the medical records of patients enrolled in phase 0 and 1 clinical trials from 2012 to July 2018 at Liverpool Cancer therapy Centre. Previously reported data of 40 patients have been included in this analysis. We collected patient demographics, cancer type, sites of metastatic disease, previous lines of treatment, duration on trial, overall survival, toxicity, reason for ceasing treatment and deaths within 30 days.
Results: There were 100 patients treated in 102 episodes over the period studied. The median age was 64 years (range 31-88). Performance status was either ECOG 0 (33%) or 1(73%). Most patients had gastrointestinal cancers (40%) followed by genitourinary cancers (33%). The median number of previous lines of systemic treatment was 2 (range 0-13). The adverse events were grade1 in 61 %, grade 2 in 22% and grade 3 in 8 % of patients. At the time of data collection, 59% were dead, 23% were alive and 18% unknown. The median duration on trial was 54 days (range 5-479). The median overall survival from trial commencement was 137.5 days (range 5-1743). Sixteen patients (15%) died within 30 days of last drug administration. The most common reason for ceasing treatment was progressive disease (60%). Five patients (5%) stopped treatment due to toxicity.
Conclusion: Our data suggest that phase 1 trials are a therapeutic option for oncology patients with limited options. Toxicity was manageable and predominately Grade 1. Patient selection is important and a minority of patients progressed rapidly during, or soon after, participating in a trial.