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March 21, 2026ESMO Open4 citationsOpen Access

Phase III trial of infigratinib versus gemcitabine/cisplatin in adults with advanced cholangiocarcinoma with FGFR2 gene fusion or rearrangement: results and reflections on early termination of PROOF 301

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GAG.K. Abou-AlfaIBI. BorbathSRS. Roychowdhury

Key Points

  • The study aims to evaluate the efficacy of infigratinib in treating advanced cholangiocarcinoma with FGFR2 gene fusion or rearrangement.
  • Conducted a Phase III trial comparing infigratinib to gemcitabine/cisplatin.
  • Focused on adults with advanced cholangiocarcinoma and FGFR2 alterations.
  • Addressed challenges in studies involving rare tumor subpopulations.
  • Early termination of the trial reduced the ability to draw firm conclusions about infigratinib's efficacy.
  • Highlights the challenges in powering studies for rare tumors and the importance of collaboration in regulatory frameworks.

Abstract

Early termination limited the ability to draw definitive conclusions on the efficacy of infigratinib as first-line treatment of FGFR2-rearranged CCA. This study illustrates the challenges of powering confirmatory studies in biomarker-selected subpopulations of rare tumors and highlights the need for regulatory collaboration to develop pragmatic frameworks for assessing novel therapies in ultra-rare malignancies.

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Cite This Study

Abou-Alfa et al. (2026) studied this question.

synapsesocial.com/papers/69be34886e48c4981c672c2chttps://doi.org/10.1016/j.esmoop.2026.106306
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