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Review
. 2025 Sep:139:102982.
doi: 10.1016/j.ctrv.2025.102982. Epub 2025 Jun 26.

A systematic review of phase I trials in patients with ovarian cancer

Giuliana Pavone  1 , Federica Martorana  1 , Vincenza Ricco  2 , Esteban Andres Ciliberti  3 , Marta Nerone  3 , Cristiana Sessa  3 , Ilaria Colombo  4
Affiliations
Review

A systematic review of phase I trials in patients with ovarian cancer

Giuliana Pavone et al. Cancer Treat Rev. 2025 Sep.

Abstract

Background: Ovarian cancer (OC) is the leading cause of death among gynecological malignancies, with limited treatment options for advanced and platinum-resistant disease. This systematic review analyzes phase I trials to assess recent therapeutic advancements.

Methods: We performed a systematic review of phase I trials in OC, published between 2012 and 2023, retrieving data on trial characteristics and outcomes. Studies were classified according to the tested treatment strategies into chemotherapy-only (CO), chemotherapy + non-chemotherapy agents (CNC) and chemotherapy-free (CF).

Results: 78 trials were included, with more than 50 % of them published in the last four years. Overall, chemotherapy and immunotherapy were the most investigated agents. Fourteen trials (17.9 %) evaluated a CO strategy, 42 (53.8 %) a CNC combination and 22 (28.2 %) a CF therapy. Dose-limiting toxicities and toxic deaths were observed in 71 % and 100 % of CO studies, in 45.2 % and 21 % of CNC trials and in 37.4 % and 13.6 % of CF trials, respectively. CNC regimens outperformed the other treatment types in terms of efficacy outcomes, including overall response rate (11.5 % CO; 32.2 % CNC; 25.5 % CF), clinical benefit rate (40 % CO; 62 % CNC; 52 % CF) and median progression free survival (mPFS 5.9 months CO; 6.45 months CNC; 4.85 months CF). Trials enrolling platinum resistant or agnostic patients displayed worse clinical outcomes.

Conclusions: In the last years, there has been an increasing number of phase 1 trials assessing new agents and new combinations in patients with OC. Chemotherapy-free strategies display a more favorable safety profile, while regimens combining CNC agents seem to be more effective compared to CO approaches.

Keywords: Drug development; Ovarian cancer; phase I trials.

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Conflict of interest statement

Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: : CS: SO Consultant for gynaecological cancer ESMO Extended member women for oncology, ESMO Member of the compliance committee, Merck DMC member of the MK-3475-C93 study, DMC member of the MK-2870-005 study. FM: Invited speaker: Daichii-Sankyo, Eli-lilly, Gilead, Novartis, Pfizer; Research grant: Gilead; Travel grants: Eli-Lilly, Gilead, Pfizer. IC: declares institutional funding for clinical trials as PI fromAstraZeneca, Merck Sharp & Dhome, Vivesto, Tolremo, Orion, Bayer, lncyte, consultancy/advisor role from AstraZeneca, GlaxoSmithKline, Merck Sharp & Dhome, AbbVie, Biontech, lncyte, Beigene outside the submitted work. EAS, GP, MN, and VR: None declared.

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