CTNI-61. A PHASE 2 STUDY OF ERDAFITINIB IN PATIENTS WITH RECURRENT OR PROGRESSIVE IDH-WILD TYPE GLIOMA WITH FGFR-TACC GENE FUSIONS: SAFETY RUN-IN COHORT RESULTS
Bibliographic record
Abstract
Abstract FGFR-TACC gene fusions (F-T) are the most prevalent fusions in adult glioma, present in 3-6% of IDH-wild-type glioma. F-T emerge as truncal alterations during gliomagenesis, are independent predictors of favorable outcome in gliomas, and are retained in recurrent glioblastoma. In vitro and in vivo, F-T exhibit strong oncogenic activity and confer sensitivity to FGFR inhibitors. Erdafitinib is a potent, oral pan-FGFR tyrosine kinase inhibitor, FDA-approved for salvage therapy of metastatic urothelial carcinoma with FGFR alterations. Although responses to erdafitinib in F-T glioma have been reported as part of basket trials, no clinical study has yet been focused only on F-T gliomas. Here, we present the safety run-in cohort results of a multicenter, single-arm phase 2 study to assess erdafitinib in patients with recurrent/progressive F-T glioma. This cohort was designed to evaluate safety, tolerability and determine the recommended phase 2 dose (RP2D). Six adult patients with recurrent F-T glioma with measurable disease were treated with erdafitinib 8 mg daily continuous dose (median age: 63 years [range 52-72]; female 50%, GBM: 100%). With reporting adverse events (AEs) occurred within dose-limiting toxicity (DLT) period of Cycle 1 (4 weeks), all patients had treatment-emergent AE, mainly grade (G) 1/2. One out of 6 patients experienced a DLT with G3 AE - central serous retinopathy - leading to treatment discontinuation. There was one treatment-unrelated G3 AE - cerebral edema. Five patients had hyperphosphatemia (4 G1 and 1 G2). Preliminary efficacy data was available for 5 patients with best ORR of 1 CR, 1 PR, 2 SD, and 1 PD. The 2 patients with SD have both been on treatment for over 6 months with tumor reduction not yet meeting the 50% required for PR. Based on the safety review committee evaluation, erdafitinib 8mg continuous dose was confirmed as the RP2D. The expansion cohort is on-going.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".