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
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.
Authors
- M. Javle
- L. Goyal
- C. Tamaş
- D.F. van Veenhuyzen
- S. Roychowdhury
- R.T. Shroff
- D.M. Savastano
- C. Xu
- D.-Y. Oh
- G.K. Abou-Alfa
- T. Macarulla
- J. Solanas
- E. Freas
- I. Borbath
- A. Lamarca
Institutions
- Hebron University (PS)
- Cliniques Universitaires Saint-Luc (BE)
- Memorial Sloan Kettering Cancer Center (US)
- The University of Texas MD Anderson Cancer Center (US)
- University of Arizona (US)
- Cornell University (US)
- The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (US)
- Seoul National University Hospital (KR)
- Helsinn (Switzerland) (CH)
- Vall d'Hebron Hospital Universitari (ES)
- Hospital Universitario Fundación Jiménez Díaz (ES)
- Center for Cancer Research (US)
- Io Therapeutics (United States) (US)
- Helsinn Therapeutics (United States) (US)
- University of Arizona Cancer Center
- UCLouvain (BE)
Publication Details
- Journal
- ESMO Open
- Published
- 2026-03-17
- DOI
- https://doi.org/10.1016/j.esmoop.2026.106306
- Primary Topic
- Fibroblast Growth Factor Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00