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Updated: Aug 5, 2026
Quantitation of Intra-peritoneal Ovarian Cancer Metastasis
Published on: July 18, 2016
Phase II randomized study of PM01183 versus topotecan in patients with platinum-resistant/refractory advanced ovarian
A Poveda1, J M Del Campo2, I Ray-Coquard3
1Department of Gynecologic Oncology, Instituto Valenciano de Oncología, Valencia.
Background:
PM01183 is a new compound that blocks active transcription, produces DNA breaks and apoptosis, and affects the inflammatory microenvironment. PM01183 showed strong antitumor activity in preclinical models of cisplatin-resistant epithelial ovarian cancer.
Patients And Methods:
Patients with platinum-resistant/refractory ovarian cancer were included in a two-stage, controlled, randomized (in a second stage), multicenter, phase II study. Primary endpoint was overall response rate (ORR) by RECIST and/or GCIG criteria. The exploratory first stage (n = 22) confirmed the activity of PM01183 as a single agent at 7.0 mg flat dose every 3 weeks (q3wk). The second stage (n = 59) was randomized and controlled with topotecan on days 1-5 q3wk or weekly (every 4 weeks, q4wk).
Results:
ORR was 23% (95% CI, 13%-37%) for 52 PM01183-treated patients. Median duration of response was 4.6 months (95% CI, 2.5-6.9 months), and 23% (95% CI, 0%-51%) of responses lasted 6 months or more. Ten of the 12 confirmed responses were reported for 33 patients with platinum-resistant disease [ORR = 30% (95% CI, 16%-49%)]; for the 29 patients treated with topotecan in the second stage, no responses were found. Median PFS for all PM01183-treated patients was 4.0 months (95% CI, 2.7-5.6 months), and 5.0 months (95% CI, 2.7-6.9 months) for patients with platinum-resistant disease. Grade 3/4 neutropenia in 85% of patients; febrile neutropenia in 21% and fatigue (grade 3 in 35%) were the principal safety findings for PM01183.
Conclusion:
PM01183 is an active drug in platinum-resistant/refractory ovarian cancer and warrants further development. The highest activity was observed in platinum-resistant disease. Its safety profile indicates the dose should be adjusted to body surface area (mg/m2).
Trial Code:
EudraCT 2011-002172-16.
Insights
PM01183 demonstrates significant antitumor activity in platinum-resistant ovarian cancer, showing a 23% overall response rate. Further development is warranted, with dose adjustments based on body surface area recommended.
Area of Science:
- Oncology
- Pharmacology
- Clinical Trials
Background:
- PM01183 is a novel compound with demonstrated preclinical efficacy in cisplatin-resistant epithelial ovarian cancer.
- It functions by inhibiting transcription, inducing DNA damage and apoptosis, and modulating the tumor microenvironment.
Purpose of the Study:
- To evaluate the efficacy and safety of PM01183 in patients with platinum-resistant or refractory ovarian cancer.
- To compare PM01183 with topotecan in a randomized phase II setting.
Main Methods:
- A two-stage, randomized, controlled, multicenter phase II study was conducted.
- The first stage assessed PM01183 as a single agent (7.0 mg flat dose q3wk).
- The second stage randomized patients to PM01183 or topotecan (days 1-5 q3wk or weekly).
Main Results:
- The overall response rate (ORR) for PM01183 was 23% (95% CI, 13%-37%).
- In platinum-resistant disease, the ORR was 30% (95% CI, 16%-49%), with a median progression-free survival (PFS) of 5.0 months.
- Topotecan showed no responses in the second stage. Grade 3/4 neutropenia (85%), febrile neutropenia (21%), and fatigue (35%) were key safety concerns.
Conclusions:
- PM01183 exhibits significant activity in platinum-resistant/refractory ovarian cancer, particularly in platinum-resistant disease.
- Further clinical development of PM01183 is recommended.
- The safety profile suggests dose optimization to mg/m2 is necessary.
