Table 2.
Cationic Liposomal Delivery System for Cancer Gene Therapy in Clinical Trials
| Name | Drug | Carrier Components | Administration Route | Condition | ClinicalTrials.gov Identifier (Phase) | Sponsor and/or Affiliations | First Posted |
|---|---|---|---|---|---|---|---|
| Atu027 | PKN3 siRNA | AtuFECT01-DPhyPE/DSPE-PEG-2000 | i.v. | pancreatic cancer | NCT01808638 (I/II) | Silence Therapeutics | 2013 |
| SGT-94 | RB94 pDNA | DOTAP/DOPE | i.v. | solid tumors | NCT01517464 (I) | Synergene Therapeutics | 2012 |
| SGT-53 | HWTp53 pDNA, PD1 antibody | DOTAP/DOPE | i.v. | glioblastoma, solid tumors, pancreatic cancer | NCT02340156 (II) | Synergene Therapeutics | 2015 |
| NCT02340117 (II) | |||||||
| Lipo-MERIT | (NY-ESO-1, MAGE-A3, tyrosinase and TPTE) RNAs | DOTMA/DOPE | i.v. | stage IIIB–IV melanoma | NCT02410733 (I) | Biopharmaceutical New Technologies | 2015 |
| Tusc2-nanoparticles | Tusc2 pDNA | DOTAP/Chol | i.v. | lung cancer | NCT01455389 (I/II) | MD Anderson Cancer Center | 2011 |
| Liposomal-DNA complexes | interleukin-2 pDNA | DOTMA/Chol | i.t. | head and neck cancer | NCT00006033 (II) | H. Lee Moffitt Cancer Center and Research Institute | 2004 |
| DC-Chol liposomes | EGFR antisense DNA | DC-Chol | i.t. | head and neck cancer | NCT00009841 (I) | University of Pittsburgh | 2004 |
| pbi-shRNA STMN1 lipoplexes | bi-shRNA-stathmin 1 pDNA | DOTAP/Chol | i.t. | solid tumors | NCT01505153 (I) | Gradalis | 2012 |
| IGTM-101 | (HSTK, cIFNβ, hIL-2, hGM-CSF) pDNA | DMRIE/DOPE | intratumoral/peritumoral injection | melanoma | NCT03338777 (I) terminateda | Hospital Italiano de Buenos Aires | 2017 |
Source: https://clinicaltrials.gov. PKN3, protein kinase N3; DPhyPE, 1,2-diphytanoyl-sn-glycero-3-phosphoethanolamine; RB94, an N-terminal truncated retinoblastoma protein; HWTp53, human wild-type tumor protein 53; PD1, programmed cell death-1; NY-ESO-1, New York esophageal squamous cell carcinoma 1; MAGE-A3 melanoma antigen family A3; TPTE, transmembrane phosphatase with tensin homology; Tusc2, tumor suppressor candidate 2; STMN1, stathmin 1; DOTMA, N-[1-(2,3-dioleyloxy)propyl]-N,N,N-trimethylammonium chloride; HSTK, herpes simplex thymidine kinase; cIFNβ, canine interferon β; hIL-2, human interleukin-2; hGM-CSF, human granulocyte-macrophage colony-stimulating factor; DMRIE, 1,2-dimyristyl oxypropyl-3-dimethyl-hydroxyethylammonium bromide; i.v., intravenous; i.t., intratumoral.
Failure to achieve primary objective, terminated at 2020.