Videos
Infigratinib in Patients With FGFR2 Fusion-Positive Cholangiocarcinoma
05/19/2026
Clinical Summary:
- Design/Context: The phase 3 PROOF 301 trial assessed infigratinib in patients with FGFR2 fusion-positive advanced cholangiocarcinoma.
- Key Outcomes: The study faced major accrual limitations despite extensive international screening efforts, limiting interpretation of efficacy outcomes.
- Clinical Relevance: These findings underscore the need for innovative trial designs and alternative end points to accelerate development of targeted therapies in rare molecularly defined cancers.
Ghassan Abou-Alfa, MD, Memorial Sloan Kettering Cancer Center, New York, New York, discusses results from the phase 3 PROOF 301 trial evaluating infigratinib in patients with FGFR2 fusion-positive cholangiocarcinoma.
The study highlights the challenges of conducting large randomized trials in rare molecular subsets and raises important questions about trial design and end points in precision oncology.
Source:
Abou-Alfa GK, Borbath I, Roychowdhury S, et al. 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. ESMO Open. Published online: March 17, 2026. doi: 10.1016/j.esmoop.2026.106306
