Phase 2
Completed N=316
A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations
Advanced Solid Tumor
Source: ClinicalTrials.gov NCT04083976 ↗
Enrolled (actual)
316
Serious AEs
42.7%
Results posted
Feb 2025
Primary outcomePrimary: Broad Panel and Pediatric Cohorts: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC) — 29.5; 66.7 Percentage of participants
Summary
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Broad Panel and Pediatric Cohorts: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC) |
29.5; 66.7 | — |
| PRIMARY Core Panel Cohort: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC) |
26.6 | — |
| SECONDARY Objective Response Rate (ORR) as Assessed by Investigators Assessment |
— | — |
| SECONDARY Duration of Responses (DOR) |
— | — |
| SECONDARY Disease Control Rate (DCR) |
— | — |
| SECONDARY Clinical Benefit Rate (CBR) |
— | — |
| SECONDARY Progression-free Survival (PFS) |
— | — |
| SECONDARY Overall Survival (OS) |
— | — |
| SECONDARY Number of Participants With Adverse Events (AEs) |
— | — |
| SECONDARY Number of Participants With Adverse Events (AEs) by Severity |
— | — |
| SECONDARY Pediatric Cohort: Plasma Concentration of Erdafitinib |
— | — |
| SECONDARY Change From Baseline in European Organization for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30) for Participants >=18 Years |
— | — |
| SECONDARY Change From Baseline in Pediatric Functional Assessment Of Cancer Therapy-Brain (Peds FACT-Br) |
— | — |
| SECONDARY Change From Baseline in Patient Global Impression of Symptom Severity (PGIS) |
— | — |
| SECONDARY Change From Baseline in Patient Global Impression of Change (PGIC) |
— | — |
| SECONDARY Change From Baseline in European Quality of Life -5 Dimensions-5 Levels (EQ-5D-5L) |
— | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT04083976). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.