Participation of patients with gynecological cancer in phase 1 clinical trials: Experience from 2 NCI-Designated Comprehensive Cancer Centers.

person Anusha Adkoli Rutgers Robert Wood Johnson Medical School Department of Obstetrics and Gynecology, New Brunswick, NJ info_outline Anusha Adkoli, Krystle Vera, Patrick Glennan, Kevin Yeung, Demmie Aguilar, Mohammad Ghalib, Imran Chaudhary, Radhashree Maitra, Eugenia Girda, Sanjay Goel

Authors person Anusha Adkoli Rutgers Robert Wood Johnson Medical School Department of Obstetrics and Gynecology, New Brunswick, NJ info_outline Anusha Adkoli, Krystle Vera, Patrick Glennan, Kevin Yeung, Demmie Aguilar, Mohammad Ghalib, Imran Chaudhary, Radhashree Maitra, Eugenia Girda, Sanjay Goel Organizations Rutgers Robert Wood Johnson Medical School Department of Obstetrics and Gynecology, New Brunswick, NJ, Rutgers Robert Wood Johnson Medical School, New Brunswick, NJ, Rutgers Robert Wood Johnson Medical School, Piscataway, NJ, Rutgers Cancer Institute of New Jersey, New Brunswick, NJ, Montefiore Medical Center, Bronx, NY, Montefiore Medical Center/Albert Einstein College of Medicine, Bronx, NY Abstract Disclosures Research Funding No funding sources reported Background: Phase 1 trials are intended to define the safety of novel therapeutics, yet they offer alternate options long before they are approved. There is limited data regarding access and participation in phase 1 trials for gynecologic oncology patients. The purpose of this study is to assess the efficacy of phase 1 trials and to study factors of demographics, tumor characteristics, and toxicities impacting this population group. Methods: This IRB approved study was conducted via retrospective chart review at the Rutgers Cancer Institute of New Jersey and the Montefiore Einstein Comprehensive Cancer Center. Relevant variables were collected for all gynecologic oncology patients enrolled in phase 1 trials between 2000-2022. Data analysis was completed in a primarily descriptive manner. Results: Of the 335 patients enrolled, 72.2% were < 65 years of age; diagnoses of the 335 patients included ovarian/fallopian tube/peritoneal/mullerian (58.0%), uterine (26.3%), cervical (15.2%), vaginal (0.3%), and vulvar (0.3%) cancers. Patients were 50.5% White, 24.2% Black, 18.2% Hispanic, and 6.6% Asian. Trend over-time analysis (enrollment between 2000-2011 compared to 2012-2022) showed that enrollment of White patients stayed stable (51.0/50.4%), Hispanic (16.0/21.6%) and Asian (3.1%/11.5%) increased, while enrollment decreased for Black patients (29.9/16.5%). Most patients had an ECOG performance status of 1 (74.0%). Prior number of regimens received by patients included 1-3 (52.0%), 4-7 (38.1%), and 8 or more (9.4%). The median number of prior lines was 3. Metastatic disease sites included lymph nodes (51.3%), abdominal/pelvic cavity (39.4%), lung (35.8%) and liver (34.0%). Highest grade hematologic toxicities were 3 or 4 in 18.2% of patients. For non-hematologic toxicities, 28% of patients experienced grade 3-5 events. Among non-hematologic adverse events, the most common grade 3-5 events were fatigue (1.2%), diarrhea (1.1%), vomiting (0.9%), and pain (0.9%). Only 3.9% had a dose limiting toxicity. Efficacy included complete response in 1.2%, partial response in 7.0%, stable disease in 40.2%, progressive disease in 37.8%, and not evaluable in 13.4%. Median OS was 37.9 weeks. There was no difference in OS between the races (p=0.11). Conclusions: Patients with gynecologic cancers often enrolled into phase 1 trials later in the course of their disease. Most patients had stable disease and although low grade toxicities were common, very few patients experienced a DLT, suggesting that phase 1 trials are tolerable and can be considered earlier in a patient’s clinical course. This illustrates the ongoing necessity to implement strategies to overcome disparities among patients participating in early phase clinical trials, especially given the similar OS experience.