Table 1.
Key positive clinical trials enrolling patients with BCG-unresponsive NMIBC.
| Agent/ Target |
NCT/ Acronym |
Phase | Primary Endpoint | Patients Enrolled | Median Follow Up | Results |
|---|---|---|---|---|---|---|
|
Pembrolizumab * ICI Anti-PD1 IgG4/kappa |
NCT02625961 KEYNOTE-057 [19] |
II | CRR of high-risk NMIBC | Cohort A (CIS): 101 pts Cohort B (Non-CIS): 47 pts |
36.4 mos. | Cohort A: 41% (39 out of 96 pts, 95% CI 30.7–51.1%) |
|
Atezolizumab ICI Anti-PD-L1 IgG1 |
NCT02844816 SWOG S1605 [20] |
II | CRR at 25 weeks in CIS-cohort | CIS cohort: 70 pts pre-planned Non-CIS cohort: 65 pts pre-planned | NR | CIS cohort: 27% (20 out of 74 pts, 95% CI NR) |
|
Nadofaragene firadenovec rAd-IFNa2b/Syn3 |
NCT02773849 [21] | III | CRR at 12 mos. in CIS-cohort | CIS-cohort: 107 pts Non-CIS cohort: 50 pts |
19.7 mos. | CIS-cohort: 53.4% (55 out of 103 patients, 95% CI 43.3–63.3%) |
|
Oportuzumab Monatox EpCAM scFv linked to ETA |
NCT02449239 [22] | III | CRR in CIS-cohort | 126 pts CIS-cohort: 89 pts |
NR | CIS-cohort: 40% (95% CI NR) |
* FDA approved. NCT, Number Clinical Trial; ICI, Immune Checkpoint Inhibitor; PD1, Programmed cell Death protein-1; IgG, Immunoglobulin G; CRR, Complete Response Rate; NMIBC, Non-Muscle-Invasive Bladder Cancer; CIS, Carcinoma In Situ; mos., months; pts, patients; PD-L1, Programmed death-Ligand 1; NR, Not reported; rAd-IFNa2b, non-replicating recombinant adenovirus type 5 (Ad5)-vector encoding the interferon alpha-2b; EpCAM, Epithelial Cell Adhesion Molecule; scFv, single-chain Fragment variable; ETA, Pseudomonas exotoxin A; BCG, Bacillus Calmette–Guérin.