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. 2019 Mar 1;10(3):189. doi: 10.3390/genes10030189

Table 3.

Examples of Clinical Trials Conducted with RNA Viral Vectors.

Viral Vector Disease Phase Response Ref
Toca 511 RV HGG Phase I Prolonged survival [122]
Toca 511 RV HGG Phase II/III Study in progress [123]
GRV CGD Phase I/II Resolved bacterial & fungal infections [124]
LV hCEF-CT CT Phase 0 Established parameters for Phase I CT trial [125]
LentiGlobin BB305 β-thalassemia Phase I/II Reversion of need of red-blood cell transfusions [126]
LV pELPs 19-BB-z CLL Case report CLL remission for 10 months [127]
LV CTL019 ALL Phase I Sustained ALL remission, prolonged survival [128]
LV CTL019 ALL Approved FDA approval for refractory/relapsed ALL [129]
LV-FVIII Hemophilia A Phase 0 Potential cure, life-long production of FVIII [130]
LV-FIX Hemophilia B Phase 0 High level expression of [131]
LV ProSavin® PD Phase I/II Improvements in motor behavior [132]
LVsh5/C46 HIV Phase I Safe, protection of the immune system [133]
VEE-gB/pp65 CMV Phase I CMV-specific antibodies [136]
VEE-gag HIV Phase I Low-level antibody responses [137]
VEE-CEA Breast, colorectal, pancreatic CA Phase I Prolonged survival [138]
VEE-PSMA CRPC Phase I Neutralizing antibodies [139]
LipoVIL12 Melanoma, kidney CA Phase I Safe delivery, tumor targeting [24]
VSV-ZEBOV EBOV Phase I Safe administration, antibody responses [140]
VSVΔG-ZEBOV EBOV Phase I Safe administration, sustainable IgG responses [141]
VSV-ZEBOV EBOV Phase I/II Better tolerability, reduced antibody responses [142]
VSVΔG-ZEBOV EBOV Phase III Better immune response than for Ad vector [143]
VSV-ZEBOV EBOV Phase III Safe administration, protection against EBOV [144]
VSV-ZEBOV EBOV Phase III Safe administration, protection against EBOV [145]
VSV-ZEBOV EBOV Phase II/III Safe administration, no EBOV cases [146]
MV-EZ CTCL Phase I Regression of CTCL lesions [147]
MV-CEA Ovarian CA Phase I Stable disease [148]
MV-CEA Glioblastoma Phase I No dose-related toxicity in initial patients [149]
MV-NIS Myeloma Phase I Complete response in one patient [150]
NDV-TAA Prostate CA Phase II Prolonged survival [151]
NDV PV701 Solid tumors Phase II Progression-free survival [152]
NDV Melanoma Phase II/III Failure to provide superiority to controls [153]
NDV Colorectal CA Phase II Prolonged survival [154]
CVA21 Melanoma Phase I/II Good tolerance, anti-tumor activity [155]
CVA2 1 + ICB Melanoma Phase II Enhanced anti-tumor activity [156]
CVA21 + PLMAb Melanoma Phase 1b Stable disease [157]

Ad, adenovirus; ALL, Acute lymphocytic leukemia; CA, carcinoma; CAV21, Coxsackievirus A21; CEA, carcinoembryonic antigen; CGD, chronic granulomatous disease; CMV, cytomegalovirus; CRPC, castration resistant metastatic prostate cancer; CT, cystic fibrosis; CLL, chronic lymphocytic leukemia; CTCL, cutaneous T cell lymphoma; EBOV, Ebola virus; GRV, gammaretrovirus; HGG, high-grade glioma; HIV, human immunodeficiency virus; ICL, immune checkpoint blockade; IL, interleukin; LipoVIL-12; liposome-encapsulated SFV-IL-12; LV, lentivirus; MV, measles virus; NDV, Newcastle disease virus; NIS, sodium iodide symporter; PD, Parkinson’s disease; PLMab, pembrolizumab; PSMA, prostate-specific membrane antigen; RV, retroviral vector; TAA, tumor-associated antigen; VEE, Venezuelan equine encephalitis virus; VSV, vesicular stomatitis virus; ZEBOV, Ebola virus Zaire strain.