Study of PEP02 as a Second Line Therapy for Metastatic Pancreatic Cancer

Gemcitabine monotherapy or a gemcitabine-based combination regimen is the standard first line therapy for advanced pancreatic cancer. After disease progression, there is no standard treatment available. In animal studies and a previous phase I trial, PEP02 has shown anti-tumor activity and preliminary efficacy in pancreatic cancer. In addition, a phase II study of free-form irinotecan single agent has already shown encouraging activity as second-line treatment for patients with advanced pancreatic cancer refractory to gemcitabine. The liposome formulation of PEP02 theoretically has therapeutic advantages over free-form irinotecan, such as site-specific delivery and extended release of drug. Hence PEP02 may be able to provide better efficacy than free-form irinotecan.

The primary purpose of this phase II study is to evaluate the activity of PEP02 as a second-line therapy in patients with metastatic pancreatic cancer failed to gemcitabine treatment. The primary goal is to measure the 3-month survival rate. An optimal Simon's 2-stage design will be used for this exploratory phase II study.

Inclusion criteria

• Histologically or cytologically confirmed adenocarcinoma of exocrine pancreas
• Metastatic disease
• Documented disease progression after treatment with 1 line of prior gemcitabine-based regimen
• Karnofsky performance status equal or more than 70

Exclusion criteria

• With active CNS metastases
• With clinically significant gastrointestinal disorder (e.g., bleeding, inflammation, occlusion, or diarrhea > grade 1)
• Major surgery or radiotherapy within 4 weeks
• Prior participation in any investigational drug study within 4 weeks
• With prior irinotecan treatment