RAGNAR

NCT04083976 📎

Regimen

Experimental
Erdafitinib 8 mg PO QD (dose titration to 9 mg based on phosphate levels), continuous 28-day cycle — pan-FGFR inhibitor (covers FGFR1-4)
Control
single-arm / no comparator

Population

Advanced solid tumors with FGFR1-4 alterations (fusions, mutations, or amplifications) after ≥1 prior therapy; tumor-agnostic design spanning ≥15 tumor types. BTC cohort: n=27 of 217 total (CCA/BTC subgroup); FGFR2 fusions the dominant alteration in BTC patients consistent with ICC biology. FGFR alterations detected by central or local NGS.

Key finding

RAGNAR supported FDA's tumor-agnostic accelerated approval of erdafitinib (September 2024) for FGFR-altered solid tumors after chemotherapy. The BTC cohort (n=27) showed responses consistent with the overall basket, validating FGFR2 targeting in CCA within a broader tumor-agnostic framework. Erdafitinib differs from pemigatinib/futibatinib in its pan-FGFR activity (covers FGFR1-4 and also addresses mutations in addition to fusions). Hyperphosphatemia was the key class-effect toxicity; ocular toxicity (central serous retinopathy) required monitoring.

Source: PMID 37541273

Timeline

    Guideline citations

    • NCCN BTC (p.40)