Clinical outcomes in phase I trials across solid tumors and hematological malignancies.

Abstract

e19014 Background: Low expectations of clinical benefit from Phase I cancer trials may hinder patient participation and delay study findings. Although progress in cancer drug development, shifting from cytotoxic agents to targeted therapies and immunotherapies, may have improved treatment responses, comprehensive data remain limited. This study aims to evaluate clinical outcomes for solid tumors and hematological malignancies from Phase I trials. Methods: Between 2018 and 2020, a total of 231 patients with advanced solid tumors and hematological malignancies were enrolled in a Phase I clinical trial at the Phase I Unit, Department of Oncology, Rigshospitalet, Denmark. Response rates for solid tumor patients were assessed using RECIST 1.1, while specific methods were used for hematological patients, of whom a large majority were lymphoma patients whose response was assessed according to the Lugano Classification. The response rate was defined as the percentage of patients achieving complete or partial response, while the clinical benefit rate was defined as complete response, partial response, or stable disease with no progression 90 days after C1D1. Results: A total of 231 patients were analyzed; comprising 56% (n=129) male and 44% (n=102) female, with a median age of 64 years. Solid tumors were present in 63.6% (n=147) patients, while 36.4% (n=84) had hematological malignancies. The response rate was 6.1% for solid tumors and 60.6% for hematological malignancies. The clinical benefit rate was 36.4% for solid tumors and 62.8% for hematological malignancies. The median progression free survival was 2.76 months (95% CI: 1.90–3.62) for solid tumors and 5.52 months (95% CI: 2.89–8.15) for hematological malignancies. The median survival was 8.21 months (95% CI: 6.75–9.68) for solid tumors and 21.42 months (95% CI: 9.15–33.69) for hematological malignancies. Conclusions: This study presents promising outcomes of Phase I cancer trials, with particularly encouraging response rates in hematological malignancies. While response rates in early-stage clinical trials vary by patient population and trial availability, this study reflects outcomes from a major academic center across diagnoses and clinical trials.