TY - JOUR
T1 - A multinational phase 2 study of nanoliposomal irinotecan sucrosofate (PEP02, MM-398) for patients with gemcitabine-refractory metastatic pancreatic cancer
AU - Ko, A. H.
AU - Tempero, M. A.
AU - Shan, Y. S.
AU - Su, W. C.
AU - Lin, Y. L.
AU - Dito, E.
AU - Ong, A.
AU - Wang, Y. W.
AU - Yeh, C. G.
AU - Chen, L. T.
N1 - Funding Information:
This study was supported by PharmaEngine, Inc. (Taipei, Taiwan). We thank the patients and their families who participated in this international phase 2 study, and also thank the medical and nursing staff of the investigational sites for the care and support of the patients in this study. The study was registered with clinical trials.gov identifier NCT00813163. PharmaEngine (Sponsor) provided a research grant and supplied PEP02, designated as MM-398 by Merrimack Pharmaceuticals, Inc. (Cambridge, MA, USA), to conduct this study. LTC acknowledges funding from Excellence Cancer Research Center Programme (DOH101-TD-C-111-004), Department of Health, Executive Yuan, Taiwan.
PY - 2013/8/20
Y1 - 2013/8/20
N2 - Background: PEP02, also known as MM-398, is a novel nanoliposomal irinotecan that has improved pharmacokinetics and tumour bio-distribution of the free drug. This phase 2 study evaluated PEP02 monotherapy as second-line treatment for pancreatic cancer. Methods: Patients who had metastatic pancreatic adenocarcinoma, Karnofsky performance status ≥70, and had progressed following gemcitabine-based therapy were eligible. Intravenous injection of PEP02 120 mg m-2 was given every 3 weeks. Simon 2-stage design was used. The primary objective was 3-month survival rate (OS 3-month). Results: A total of 40 patients were enrolled. The most common severe adverse events included neutropenia, abdominal pain, asthenia, and diarrhoea. Three patients (7.5%) achieved an objective response, with an additional 17 (42.5%) demonstrating stable disease for a minimum of two cycles. Ten (31.3%) of 32 patients with an elevated baseline CA19-9 had a >50% biomarker decline. The study met its primary end point with an OS 3-month of 75%, with median progression-free survival and overall survival of 2.4 and 5.2 months, respectively. Conclusion: PEP02 demonstrates moderate antitumour activity with a manageable side effect profile for metastatic, gemcitabine-refractory pancreatic cancer patients. Given the limited treatment options available to this patient population, a phase 3 trial of PEP02 (MM-398), referred to as NAPOLI-1, is currently underway.
AB - Background: PEP02, also known as MM-398, is a novel nanoliposomal irinotecan that has improved pharmacokinetics and tumour bio-distribution of the free drug. This phase 2 study evaluated PEP02 monotherapy as second-line treatment for pancreatic cancer. Methods: Patients who had metastatic pancreatic adenocarcinoma, Karnofsky performance status ≥70, and had progressed following gemcitabine-based therapy were eligible. Intravenous injection of PEP02 120 mg m-2 was given every 3 weeks. Simon 2-stage design was used. The primary objective was 3-month survival rate (OS 3-month). Results: A total of 40 patients were enrolled. The most common severe adverse events included neutropenia, abdominal pain, asthenia, and diarrhoea. Three patients (7.5%) achieved an objective response, with an additional 17 (42.5%) demonstrating stable disease for a minimum of two cycles. Ten (31.3%) of 32 patients with an elevated baseline CA19-9 had a >50% biomarker decline. The study met its primary end point with an OS 3-month of 75%, with median progression-free survival and overall survival of 2.4 and 5.2 months, respectively. Conclusion: PEP02 demonstrates moderate antitumour activity with a manageable side effect profile for metastatic, gemcitabine-refractory pancreatic cancer patients. Given the limited treatment options available to this patient population, a phase 3 trial of PEP02 (MM-398), referred to as NAPOLI-1, is currently underway.
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U2 - 10.1038/bjc.2013.408
DO - 10.1038/bjc.2013.408
M3 - Article
C2 - 23880820
AN - SCOPUS:84883169930
SN - 0007-0920
VL - 109
SP - 920
EP - 925
JO - British Journal of Cancer
JF - British Journal of Cancer
IS - 4
ER -