Table A1.
Trial Summaries of the key clinical trials investigating the role of neoadjuvant, adjuvant, and systemic therapies in the management of UTUC. DFS = Disease Free Survival, ORR = Objective Response Rate, PRR = Pathological Response Rate, PCR = Pathologic Complete Response, OS = Overall Survival.
| Name of Trial or Study Drug | NCT Identifier | Trial Setting | Clinical Setting | Number of Study Subjects/UTUC Patients | Primary Measure | Treatment Efficacy | Treatment |
|---|---|---|---|---|---|---|---|
| POUT | NCT01993979 | Phase 3 | Adjuvant | 261 total/261 UTUC patients | Disease-free Survival | 3-year DFS: 77% vs. 46% (Treatment vs. Surveillance) | Chemotherapy (Cisplatin or Carboplatin) vs. Surveillance |
| PURE-02 | NCT02736266 | Phase 2 | Neoadjuvant | 10 total/10 UTUC | Pathological Response | Not Reported | Pembrolizumab |
| Checkmate 274 | NCT02632409 | Phase 3 | Adjuvant | 709 total/149 UTUC patients | Disease-free Survival | DFS: 21.2 months vs. 20.8 months (Nivolumab vs. Placebo) | Nivolumab vs. Placebo |
| Imvigor-210 | NCT02108652 | Phase 2 | Advanced | 119 total/33 UTUC patients | Objective Response Rate | Not Reported | Atezolizumab |
| AMBASSADOR | NCT03244384 | Phase 3 | Adjuvant | 739 total | Overall Survival, Disease-free Survival | DFS: 16.9 months vs. 8.3 months (Pembrolizumab vs. Surveillance) |
Pembrolizumab vs. Surveillance |
| Infigratanib (Lyou et al. [47]) |
NCT01004224 | Phase 1b | Advanced | 67 total/8 UTUC patients | Objective Response Rate | ORR: 31% | Infigratanib |
| Erdafitinib (Loriot et al. [46]) |
NCT02365597 | Phase 2 | Advanced | 99 total/99 UTUC patients | Objective Response Rate | ORR: 40% | Erdafitinib |
| Gemcitabine and Cisplatin (Coleman et al. [33]) | NCT01261728 | Phase 2 | Neoadjuvant | 57 total/57 UTUC patients | Pathological Response Rate | PRR: 63% | Gemcitabine and Cisplatin |
| aMVAC vs. GCa (Margulis et al. [29]) | NCT02412670 | Phase 2 | Neoadjuvant | 30 total/30 UTUC patients | Pathologic Complete Response | PCR: 14% (aMVAC arm) |
Accelerated Methotrexate, Vinblastine, Doxorubicin, Cisplatin vs. Gemcitabine and Carboplatin |
| Nivolumab plus Ipilimumab (Teo M. et al. [37]) |
NCT03520491 | Phase 2 | Neoadjuvant | 45 total | Pathologic Complete Response | Not Reported | Nivolumab vs. Nivolumab + Ipilimumab |
| ABACUS | NCT02662309 | Phase 2 | Neoadjuvant | 96 total | Pathologic Complete Response | PCR: 31% | Atezolizumab |
| DANUBE | NCT02516241 | Phase 3 | Advanced | 1032 total | Overall Survival | OS: 15.1 months vs. 12.1 months (Treatment vs. Standard of Care) |
Durvalumab + Tremelimumab vs. SOC |
| iNDUCT | NCT04617756 | Phase 2 | Neoadjuvant | 50 total | Pathologic Complete Response | Not Reported | Durvalumab + Gemcitabine/(Cisplatin or Carboplatin) |