Clinical trial · Interventional
A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations
A Phase 2 Study of Erdafitinib in Subjects With Advanced Solid Tumors and FGFR Gene Alterations
NCT04083976CI-TRIAL-00110241RAGNARcompletedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 18, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260918-000001
Summary
Brief summary (as posted)
The purpose of this study is to evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) in adult and pediatric participants with advanced solid tumors with fibroblast growth factor receptor (FGFR) alterations (mutations or gene fusions). It will also evaluate ORR in pediatric participants with advanced solid tumors and FGFR alterations.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Advanced Solid Tumor | Solid Neoplasm | CURATED_BROADER | 0.80 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Erdafitinib | Drug | Erdafitinib | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Erdafitinib
- description
- Participants with fibroblast growth factor receptor (FGFR) mutations and FGFR gene fusions will receive a dose of erdafitinib oral tablets until disease progression, intolerable toxicity, withdrawal of consent, decision by the investigator to discontinue treatment, or end of data collection timepoint if there is clinical benefit in the opinion of the investigator, has been achieved.
- interventionNames
- Drug: Erdafitinib
Primary outcomes (2)
- measure
- Broad Panel and Pediatric Cohorts: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC)
- timeFrame
- Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 4 years
- description
- ORR is defined as the percentage of participants who achieved a complete response (CR), or partial response (PR) based on Response Assessment in Neuro-Oncology (RANO) criteria. According to RANO criteria whereby overall RANO response is based on both radiographic response (CR: disappearance of all target lesions; PR: sum of products of diameters \[SPD\] decreased by \>=50 percent \[%\] from baseline value) and clinical performance status with steroid dose information.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 6 Years
Show eligibility criteria text
Inclusion Criteria: * Histologic demonstration of an unresectable, locally advanced, or metastatic solid tumor malignancy with an fibroblast growth factor receptor (FGFR) mutation or FGFR gene fusion * Measurable disease * Participant must have received at least one prior line of systemic therapy in the advanced, unresectable, or metastatic setting; or is a child or adolescent participant with a newly-diagnosed solid tumor and no acceptable standard therapies * Documented progression of disease, defined as any progression that requires a change in treatment, prior to full study screening Exclusion Criteria: * Has had prior chemotherapy, targeted therapy, or treatment with an investigational anticancer agent within 15 days or less than or equal to (\<=) 5 half-lives of the agent (whichever is longer) and up to a maximum of 30 days before the first dose of erdafitinib * The presence of FGFR gatekeeper and resistance mutations * Histologic demonstration of urothelial carcinoma * Hematologic malignancy (i.e., myeloid and lymphoid neoplasms * For non-small cell lung cancer participants only: pathogenic somatic mutations or gene fusions in the following genes: EGFR, ALK, ROS1, NTRK, BRAF V600E and KRAS * Active malignancies other than for disease requiring therapy
References
Publications (2)
- DERIVEDCarranza O, Pant S, Schuler M, Witt O, Geoerger B, Loriot Y, Farouk Sait S, Arnold D, Dal Molin GZ, Lugowska I, Winter H, Baldini C, Corassa M, Thomas S, Crow L, Xia Y, Triantos S, Sweiti H, Reardon DA. Erdafitinib in Patients With Advanced Solid Tumors With FGFR Alterations (RAGNAR): Results of Tumor-Specific Analyses and Secondary Cohorts. JCO Precis Oncol. 2026 Jul;10(7):e2501094. doi: 10.1200/PO-25-01094. Epub 2026 Jul 15. PMID 42456086
- DERIVEDPant S, Schuler M, Iyer G, Witt O, Doi T, Qin S, Tabernero J, Reardon DA, Massard C, Minchom A, Lugowska I, Carranza O, Arnold D, Gutierrez M, Winter H, Stuyckens K, Crow L, Najmi S, Hammond C, Thomas S, Santiago-Walker A, Triantos S, Sweiti H, Loriot Y; RAGNAR Investigators. Erdafitinib in patients with advanced solid tumours with FGFR alterations (RAGNAR): an international, single-arm, phase 2 study. Lancet Oncol. 2023 Aug;24(8):925-935. doi: 10.1016/S1470-2045(23)00275-9. PMID 37541273