GEMPAX was an open-label (both researchers and patients know which treatment is being administered), randomised (randomly assigns participants into an experimental group or a control group) phase III clinical trial (compares the safety and effectiveness of the new treatment against the current standard treatment) designed to assess the efficacy and tolerability of gemcitabine plus paclitaxel (GEMPAX) versus gemcitabine alone as second-line treatment (when first-line treatment has failed) for patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) who previously received FOLFIRINOX: 5-fluorouracil, oxaliplatin, and irinotecan (as first line-treatment).

This phase III clinical trial included 211 patients with mPDAC (median age of 64 [30-86] and 62% men). From these, 140 patients received GEMPAX and 71 patients received only gemcitabine. Median follow-up for surviving patients was 13.4 months in the gemcitabine/paclitaxel group and 13.8 months in the gemcitabine group. Median overall survival was 6.4 months (95% confidence interval [CI] = 5.2–7.4 months) in the gemcitabine/paclitaxel group vs 5.9 months (95% CI = 4.6–6.9 months) in the gemcitabine group (hazard ratio [HR] = 0.87, 95% CI = 0.63–1.20, P = 0.4095).

Adverse effects from treatment-related grade ≥ 3 occurred in 58.0% of patients in the GEMPAX group vs 21.7% of the gemcitabine group, i.e., thrombocytopenia – when platelet count is very low (19.6% vs 4.3%), neutropenia – low number of white blood cells called neutrophils (15.9% vs 15.7%), anemia (15.2% vs 4.3%), neuropathy – damage of the nerves that can cause pain, weakness, numbness or tingling (12.3% vs 0%), and asthenia – generalised weakness and/or lack of energy (10.1% vs 2.9%). Adverse events of any cause resulted in treatment discontinuation in 16.7% vs 2.9% of patients. One treatment-related death occurred in the combination group due to acute respiratory distress.

Results showed that GEMPAX improved the objective response rate (ORR – the proportion of patients with a complete or partial response to treatment) and progression-free survival (PFS – the time during and after the treatment that a patient lives with the disease, but it does not get worse) but did not improve overall survival (OS – the time from the diagnosis to the death) when compared to gemcitabine alone. 

References

ClinicalTrials.gov ID NCT03943667

De La Fouchardière C, et al. Gemcitabine and Paclitaxel Versus Gemcitabine Alone After 5-Fluorouracil, Oxaliplatin, and Irinotecan in Metastatic Pancreatic Adenocarcinoma: A Randomized Phase III PRODIGE 65-UCGI 36-GEMPAX UNICANCER Study. J Clin Oncol. 2024 Jan 17:JCO2300795. doi: 10.1200/JCO.23.00795.