Trial results for neoadjuvant pembrolizumab in combination with gemcitabine for urothelial carcinoma were posted on ClinicalTrials.gov on 2025-03-18. The study found a 61% pathologic muscle invasive response rate in cisplatin-eligible participants.
Background
Pembrolizumab (Keytruda) is an immunotherapy drug. This study investigated its use in subjects with muscle invasive bladder cancer or urothelial cancer who are candidates for neoadjuvant therapy.
Trial design
This completed Phase 1/Phase 2 trial (NCT02365766) enrolled 83 participants with Urothelial Carcinoma or Bladder Cancer. The study was designed as a two-part trial, with a one-arm Phase 1b portion followed by a two-arm Phase 2 portion. Participants were stratified into two cohorts based on their eligibility for cisplatin therapy.
Key results
Key results from the study include:
- For the Rate of Pathologic Muscle Invasive Response (PaIR):
- In Cohort I (Cisplatin Eligible), the rate was 61%.
- In Cohort II (Cisplatin Ineligible), the rate was 51.5%.
- For Relapse-Free Survival (RFS) at 18 Months:
- In Cohort I (Cisplatin Eligible), the rate was 83%.
- In Cohort II (Cisplatin Ineligible), the rate was 57.4%.
- For Overall Survival (OS) at 5 Years:
- In Cohort I (Cisplatin Eligible), the rate was 71.9%.
- In Cohort II (Cisplatin Ineligible), the rate was 56.6%.
- For the Radical Cystectomy (RC) Rate:
- In Cohort I (Cisplatin Eligible), the rate was 86%.
- In Cohort II (Cisplatin Ineligible), the rate was 89%.
What this means
The observed rates for pathologic muscle invasive response, relapse-free survival, and overall survival suggest that neoadjuvant pembrolizumab in combination with gemcitabine may offer clinical benefit for patients with muscle invasive bladder cancer or urothelial cancer. The differences in outcomes between cisplatin-eligible and cisplatin-ineligible cohorts highlight the importance of patient stratification in treatment planning.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT02365766, titled 'Neoadjuvant Pembrolizumab in Combination With Gemcitabine Therapy in Cis-eligible/Ineligible UC Subjects,' were posted on 2025-03-18 on clinicaltrials.gov.