Background

Early phase oncology trials offer access to novel therapies for patients with advanced malignancies; however, real-world Australian data on patient outcomes is limited. Here, we review the experiences of the early phase oncology trials unit at Cabrini Health.

Methods

A retrospective cohort analysis was conducted of all patients referred for early phase oncology clinical trials at Cabrini Health between January 2019 and December 2024. Patient demographics, prior therapy, trial characteristics, screening outcomes, best response, toxicity and overall survival (OS) were analysed.

Results

There were 271 referrals pertaining to 218 patients. Median age was 64 years [range, 30-92]. Females comprised 66% of the cohort, reflecting a high number of gynaecological cancers (36% of all referrals) seen at Cabrini. Most patients (79%) were from Metropolitan Melbourne; 6% were from regional Victoria or interstate. ECOG was 0-1 in 90% of patients and 128 (45.6%) had received ≥3 prior lines of palliative intent therapy.

Trial enrolment was high: 198 referrals (73%) passed screening and resulted in commencement on an early phase trial and 45 (21%) patients were referred to 2 or more trials. Among these, 3 (1.5%) patients achieved a complete response, 22 (11.1%) had a partial response and 55 (27.8%) had stable disease as best response per RECIST/iRECIST; providing a disease control rate (DCR) of 40%. Clinically significant grade 2 or grade 3–5 TRAEs occurred in 55 (27.8%) of patients. Median duration on study was 62 days [2-629] and median OS was 7.9 months [IQR: 2.5 – 11.7].


Conclusion

This study of an Australian early phase oncology trials program demonstrated a high DCR, with a median OS of >7 months in heavily pretreated patients. These results highlight the importance of early referral of patients to a Phase I/II clinical trial, as clinically meaningful responses may be obtained.