Phase 1 trial of HR070803 (an Irinotecan liposome) in combination with 5-fluorouracil, leucovorin, and oxaliplatin for untreated advanced or metastatic pancreatic ductal adenocarcinoma

Abstract Background This study assessed the safety, preliminary antitumor activity, and pharmacokinetics of HR070803 (a novel liposomal irinotecan) in combination with 5-FU/LV and oxaliplatin for treatment-naive patients with unresectable locally advanced or metastatic pancreatic ductal adenocarcino...

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Main Authors: Qiang Xu, Xue Zhao, Xianze Wang, Ruizhe Zhu, Yuejuan Cheng, Tao Xia, Heshui Wu, He Tian, Yuping Sun, Mingjun Zhang, Chuntao Gao, Deliang Fu, Xiaojie Wu, Tongsen Zheng, Xiaoyu Yin, Yili Chen, Xiaobing Chen, Zhihua Li, Rufu Chen, Xue Yang, Huan Wang, Quanren Wang, Xiaohong Han, Wenming Wu
Format: Article
Language:English
Published: BMC 2025-07-01
Series:BMC Medicine
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Online Access:https://doi.org/10.1186/s12916-025-04234-4
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Summary:Abstract Background This study assessed the safety, preliminary antitumor activity, and pharmacokinetics of HR070803 (a novel liposomal irinotecan) in combination with 5-FU/LV and oxaliplatin for treatment-naive patients with unresectable locally advanced or metastatic pancreatic ductal adenocarcinoma (PDAC). Methods This multicenter, open-label, single-arm, dose-escalation phase 1 study recruited treatment-naive patients aged 18–70 years with unresectable locally advanced or metastatic PDAC. Treatment doses were escalated from 40/60 (HR070803 40 mg/m2 plus 5-FU/LV and oxaliplatin 60 mg/m2) to 60/60 and 60/85. The primary endpoints were maximum tolerated dose (MTD) and the recommended phase 2 dose (RP2D). Secondary endpoints included safety, preliminary antitumor activity, and pharmacokinetics. Results A total of 41 patients were enrolled, including 6, 17, and 18 patients in the 40/60, 60/60, and 60/85 group, respectively. Only one patient in the 60/60 group experienced dose-limiting toxicities of grade 3 increased alanine aminotransferase and grade 3 increased aspartate aminotransferase, and the MTD was not reached. Adverse events of grade ≥ 3 were reported in 31 (75.6%) patients, with the most common being decreased neutrophil count and increased gamma-glutamyltransferase. No treatment discontinuation occurred owing to adverse events, and there were no treatment-related deaths. The overall response (complete or partial response) rate was 29.3% in the total population. Pharmacokinetic results demonstrated prolonged circulation and slow release of free irinotecan. Conclusions HR070803 plus 5-FU/LV and oxaliplatin demonstrated an acceptable toxicity, good antitumor activity, and favorable pharmacokinetic profile as a first-line treatment for patients with unresectable locally advanced or metastatic PDAC. Based on the comprehensive data obtained during the dose escalation and dose expansion stages, HR070803 60 mg/m2 plus 5-FU/LV and oxaliplatin 85 mg/m2 was chosen as the RP2D. Trial registration Clinical trials.gov NCT04796948; registered March 15, 2021.
ISSN:1741-7015