Trial results for a Phase 3 study (NCT03390504) involving pembrolizumab as a comparator in advanced urothelial cancer were posted on ClinicalTrials.gov on 2025-05-14. In one cohort, pembrolizumab demonstrated a median overall survival (OS) of 11.07 months when compared to erdafitinib. The study also compared erdafitinib to chemotherapy in another cohort.
Background
Pembrolizumab (Keytruda) is an established treatment option in oncology. This trial investigated its use as a comparator in participants with advanced urothelial cancer.
Trial design
This Phase 3 study (NCT03390504) enrolled 629 participants with advanced urothelial cancer harboring selected fibroblast growth factor receptor (FGFR) aberrations. The trial was designed to evaluate the efficacy of erdafitinib against chemotherapy (vinflunine or docetaxel) in Cohort 1, and against pembrolizumab in Cohort 2. Cohort 1 included participants who had progressed after 1 or 2 prior treatments, with at least one anti-programmed death ligand 1 (PD-[L]1) agent. Cohort 2 included participants who had progressed after 1 prior treatment not containing an anti-PD-(L)1 agent.
Key results
Key results for overall survival (OS), progression-free survival (PFS), and objective response rate (ORR) were reported:
- Overall Survival (OS)
- In Cohort 1 (Erdafitinib vs. Chemotherapy):
- Erdafitinib (8 mg/9 mg): Median OS was 12.06 months.
- Chemotherapy (Vinflunine or Docetaxel): Median OS was 8.74 months.
- In Cohort 2 (Erdafitinib vs. Pembrolizumab):
- Erdafitinib (8 mg/9 mg): Median OS was 10.91 months.
- Pembrolizumab (200 mg): Median OS was 11.07 months.
- Progression Free Survival (PFS) as per RECIST v1.1
- In Cohort 1 (Erdafitinib vs. Chemotherapy):
- Erdafitinib (8 mg/9 mg): Median PFS was 5.39 months.
- Chemotherapy (Vinflunine or Docetaxel): Median PFS was 2.73 months.
- In Cohort 2 (Erdafitinib vs. Pembrolizumab):
- Erdafitinib (8 mg/9 mg): Median PFS was 4.44 months.
- Pembrolizumab (200 mg): Median PFS was 2.69 months.
- Objective Response Rate (ORR) Per RECIST v1.1
- In Cohort 1 (Erdafitinib vs. Chemotherapy):
- Erdafitinib (8 mg/9 mg): ORR was 46.9 Percentage of participants.
- Chemotherapy (Vinflunine or Docetaxel): ORR was 12.6 Percentage of participants.
- In Cohort 2 (Erdafitinib vs. Pembrolizumab):
- Erdafitinib (8 mg/9 mg): ORR was 40.0 Percentage of participants.
- Pembrolizumab (200 mg): ORR was 21.6 Percentage of participants.
- For Erdafitinib vs. Chemotherapy (Cohort 1): A Hazard Ratio (HR) of 0.65 (95.0% Confidence Interval: 0.48, 0.86) was reported, with a p-value of 0.0031 (Log Rank method).
- For Erdafitinib vs. Pembrolizumab (Cohort 2): A Hazard Ratio (HR) of 1.16 (95.0% Confidence Interval: 0.92, 1.48) was reported, with a p-value of 0.2121 (Stratified log-rank test method).
Analyses of overall survival included:
What this means
In Cohort 1, erdafitinib demonstrated a statistically significant improvement in overall survival, progression-free survival, and objective response rate compared to chemotherapy in participants with advanced urothelial cancer who had received prior anti-PD-(L)1 treatment. In Cohort 2, where erdafitinib was compared to pembrolizumab, the median overall survival was similar between the two treatments, with erdafitinib showing a median OS of 10.91 months and pembrolizumab showing 11.07 months. This difference was not statistically significant (HR 1.16, p=0.2121). However, erdafitinib did show numerically higher median progression-free survival and objective response rates compared to pembrolizumab in this cohort.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT03390504, titled "A Study of Erdafitinib Compared With Vinflunine or Docetaxel or Pembrolizumab in Participants With Advanced Urothelial Cancer and Selected Fibroblast Growth Factor Receptor (FGFR) Gene Aberrations," were posted on 2025-05-14 on clinicaltrials.gov.