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. Author manuscript; available in PMC: 2019 Oct 4.
Published in final edited form as: Curr Opin Oncol. 2014 May;26(3):305–320. doi: 10.1097/CCO.0000000000000064

Table 2.

Reported and ongoing trials of targeted agents in nonmuscle-invasive bladder cancer

Table 2a. Reported trials of targeted agents in NMIBC

Author Year Agent Target Intravesical therapy Study type n RR %
Kowalski [15] 2010 Oportuzumab monatox EpCAM None Phase I 64 NR
Hendricksen [12] 2012 Apaziquone Mitomycin-C analogue, apoptosis Apaziquone Phase II 53 NR
Falke [14■■] 2012 TMX-101 TLR-7 None Phase I 16 NR
Kowalski [13] 2012 Oportuzumab monatox EpCAM None Two-arm, phase II 46 44
Table 2b. Ongoing trials of targeted agents in nonmuscle-invasive bladder cancer

PI Start year Agent Target Intravesicaltherapy Study type Target enrollment Clinicaltrials.gov ID

Lamm [17] 2008 DTA-H19/PEI DNA plasmid, DT-A gene, H19 transcription factors None Phase IIb intermediate risk with prior intravesical induction 47 NCT00595088
Weizer [18] 2009 Sunitinib VEGFR-1/2, C-KIT, PDGFR a/b, FLT3, RET BCG Phase II high grade 36 NCT00794950
Dalbagni [19] 2010 Everolimus PI3K/Akt/mTOR Gemcitabine Phase I/II BCG failure 45 NCT01259063
Fishman [20] 2011 Lenalidomide Apoptosis, antiangiogenesis, and immune modulation BCG Phase II with prior BCG induction 70 NCT01373294
Fishman [16] 2011 Rituximab CD20 BCG Phase II high grade 70 NCT01373294
Sun [21] 2011 Apaziquone Mitomycin-C analogue, apoptosis None Randomized phase III low risk 658 NCT01469221
Dinney [22] 2012 rAD-IFN/Syn3 Antiangiogenesis and immune modulation None Randomized phase II high-grade BCG failure 40 NCT01687244
Rosser [23] 2012 ALT-801 p53 Gemcitabine Phase Ib/II BCG failure 52 NCT01625260
Jichlinski [24] 2012 recMAGE-A3+AS-15 MAGE-A3 vaccine BCG Phase I high grade 24 NCT01498172
Shapiro [25] 2012 Valproic acid Thrombospondin-1 BCG/MMC Phase 0 50 NCT01738815
Lamm [26] 2013 TMX-101 TLR-7 None Phase II with CIS 12 NCT01731652
Hahn [27] 2013 Dovitinib FGFR3 None Phase II BCG failure with FGFR3 mutation overexpression 50 NCT01732107
Steinberg [28] 2013 HS-410 gp96-chaperoned peptides BCG Phase I/II high grade with prior BCG induction phase I/II high grade with prior BCG induction 93 NCT02010203
Agarwal [29] 2013 PANVAC MUC-1, CEA BCG Randomized phase II BCG failure 54 NCT02015104

BCG, Bacillus Calmette-Guerin; CIS, carcinoma in situ; EpCAM, epithelial cell adhesion molecule; FGFR, fibroblast growth factor receptor; MMC, mitomycin C; mTOR, mammalian target of rapamycin; n, number of patients enrolled; NR, not reported; PDGFR, platelet-derived growth factor receptor; PI, principal investigator; RR, response rate; TLR, toll-like receptor; VEGFR, vascular endothelial growth factor receptor.