Phase II Randomized, Double-Blind Study of mFOLFIRINOX plus Ramucirumab versus mFOLFIRINOX plus placebo in Advanced Pancreatic Cancer Patients Open Access

Liu, Yusi (Spring 2020)

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Background - Pancreatic cancer is a kind of highly lethal cancer. The prognosis of pancreas adenocarcinoma, known as PCA made little progress in the last decade. However, FOLFIRINOX, a combination of PCA drugs, which works on Vascular Endothelial Growth Factor (VEGF) and VEGF receptor is an exception. This study is an interim study of a phase II randomized, multi-center and double-blinded study designed to compare the efficacy and safety of mFOLFIRINOX plus Ramucirumab (Arm A) versus mFOLFIRINOX plus placebo (Arm B) in patients with recurrent or metastatic pancreatic cancer (PCA). The primary endpoint is progression free survival (PFS) and secondary endpoints are overall survival and disease response.

Method and Result - The study summarized the interim analysis after total 65 subjects were enrolled (33 in Arm A and 32 in Arm B). Patients had been followed up for at least 9 months. Based on KM curve analysis, no significant difference for PFS was observed between Arm A and Arm B (p-value = 0.747). The median PFS time was 5.3 months for Arm A [95% CI (2.1, 7.7)] vs 3.6 months for Arm B [95% CI (2.1, 9.5)] and the 9-month PFS rate between the two arms are also comparable (20.7% [95% CI (15.8%,54.2%)] for Arm A vs 33.9% [95% CI (15.5%,53.3%)] for Arm B). For the secondary endpoints, the median OS between the two arms were comparable (10.5 months for Arm A [95% CI (4.3, 13)] vs 9.5 months for Arm B [95% CI (3.6, 25.1)]). For disease response, Arm B have obviously better disease (19.05% CR/PR) than Arm A (4.55%). However, this difference did not reach to the significant level yet.

Conclusion - The interim analysis result suggested that Arm B may have better PFS and OS as well as disease response than Arm A in a long run. However, at this time point, no evidence shows significant difference between Arm A and Arm B for both primary point and secondary points. Hence, we suggest that the trail should continue to enroll more subjects until reach the targeted sample size.

Table of Contents




2.1 Study Design and Subject Registration

2.2 Statistical Analysis

2.2.1 Descriptive Analysis

2.2.2 Survival Analysis

2.2.3 Interim analysis and O’Brien-Fleming approach


3.1 Descriptive Analysis

3.1.1 Summary of baseline demographic

3.1.2 Distribution of disease response base on study arms

3.2.... Survival Analysis

3.2.1 Progression-Free Survival Analysis

3.2.2 Overall Survival Analysis




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