Phase 2
N=107
A Study of Erdafitinib Versus Investigator Choice of Intravesical Chemotherapy in Participants Who Received Bacillus Calmette-Guérin (BCG) and Recurred With High Risk Non-Muscle-Invasive Bladder Cancer (NMIBC)
Urinary Bladder Neoplasms
Bottom Line
View on ClinicalTrials.gov: NCT04172675 ↗Enrolled (actual)
107
Serious AEs
20.0%
Results posted
Aug 2025
Primary outcome: Primary: Cohort 1: Recurrence-Free Survival (RFS) — NA; 11.60 Months — p==0.0007
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Erdafitinib (Drug); Investigator Choice (Gemcitabine) (Drug); Investigator Choice (Mitomycin C) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Janssen Research & Development, LLC
- Primary completion
- Jul 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Cohort 1: Recurrence-Free Survival (RFS) |
NA; 11.60 | =0.0007 sig |
| SECONDARY Cohort 1: Recurrence-Free Survival Rate at 6 Months and 12 Months |
0.96; 0.73; 0.79; 0.44 | — |
| SECONDARY Cohort 1: Time to Progression |
NA; NA | — |
| SECONDARY Cohort 1: Overall Survival |
NA; NA | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
49; 19; 16; 18 | — |
| SECONDARY Plasma Concentrations of Erdafitinib |
592; 525; 605; 635; 603; 685 | — |
Summary
The purpose of this study is to evaluate recurrence-free survival (RFS) in participants treated with erdafitinib vs Investigator's Choice, for participants with high-risk non-muscle-invasive bladder cancer (NMIBC) who harbor fibroblast growth factor receptor (FGFR) mutations or fusions, and who recurred after bacillus calmette-guerin (BCG) therapy.
Eligibility Criteria
Inclusion Criteria
- Histologically confirmed, recurrent, non-muscle-invasive urothelial carcinoma of the bladder. Variant pathology are allowed
- Tumor with specified fibroblast growth factor receptor (FGFR) mutations or fusions
- Bacillus Calmette- Guerin (BCG)-unresponsive after adequate BCG therapy or BCG experienced participants
- Refuses or is not eligible for cystectomy (Cohort 1 and Cohort 2 only)
- Eastern Cooperative Oncology Group (ECOG) performance status Grade 0-1
- Must sign an informed consent form (ICF) (or their legally acceptable representative must sign) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study
- A woman of childbearing potential must have a negative pregnancy test (beta-human chorionic gonadotropin [beta-hCG]) (urine or serum) within 7 days before randomization (Cohort 1) or the first dose of study drug (Cohort 2 and Cohort 3)
- Adequate bone marrow, liver, and renal function as specified in the protocol
Exclusion Criteria
- Histologically confirmed, muscle-invasive (T2 or higher stage) urothelial carcinoma of the bladder
- Histopathology demonstrating any small cell component, pure adenocarcinoma, pure squamous cell carcinoma, or pure squamous CIS of the bladder
- Prior treatment with an FGFR inhibitor
- Active malignancies other than the disease being treated under study. The only allowed exceptions are: (a) skin cancer treated within the last 24 months that is considered completely cured (b) adequately treated lobular carcinoma in situ (LCIS) and ductal CIS (c) history of localized breast cancer and receiving antihormonal agents, or history of localized prostate cancer (N0M0) and receiving androgen deprivation therapy
- Current central serous retinopathy or retinal pigment epithelial detachment of any grade
Data sourced from ClinicalTrials.gov (NCT04172675). 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.