Phase 2 trials of investigational agents for pancreatic cancer treatment do not usually progress to phase 3, according to a study being presented at the 2017 Gastrointestinal Cancers Symposium.1

Treatment outcomes for patients with locally-advanced or metastatic pancreatic cancer are minimally improved since the 1970s. This study was intended to elucidate reasons for this lack of improvement by reviewing results from phase 2 and 3 trials conducted between 1978 and 2015.

One hundred and forty-nine phase 2 trials of 7499 patients with pancreatic cancer were identified, of which the pooled objective response rate was 13.8%. Authors of 56% of the phase 2 trials recommended the investigational agents progress to phase 3, but only 11% did so.

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Less than 10% of novel investigational agents proceeded to phase 3 study. Of all reviewed phase 2 studies investigating a novel agent, “only 1 trial was enriched for the biomarker.”

Three agents in phase 3 trials improved clinical outcomes.

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The authors concluded that phase 2 results do not predict progression to phase 3 study or successful results thereof. These conclusions may be explained by factors including “overly optimistic interpretations” of study results, ineffective investigational agents, and heterogeneity among patients.


  1. Tang M, Chen J, Goldstein D, et al. Correlation of phase 2 trials (Ph2t) results with outcomes of Phase 3 trials (Ph3t) of investigational agents (IA) in locally advanced and metastatic pancreas cancer (LAMPC). J Clin Oncol. 2017;35(suppl):4S. Abstract 227.