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.
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.
- 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.