Table 2.
Summary of cardiovascular gene therapy and genetic decoy-based clinical trials
| DiseaseTarget | Trans gene | Study | Vector | Delivery system | Placebo | No | Primary end points | Result |
|---|---|---|---|---|---|---|---|---|
| CLI | HGF | Morishita et al. (2004) | Plasmid | IM | No | 6 | Pain, ABI, Ulcer | Improved |
| VEGF | Baumgartner et al. (1998) | Plasmid | Catheter | No | 9 | ABI, limb salvage | Improved | |
| VEGF | Isner et al. (1996) | Plasmid | Catheter | No | 1 | Angiography, Doppler | Improved | |
| PVD | VEGF | Kim et al. (2004) | Plasmid | IM | No | 9 | Angiography, ABI, pain | Improved |
| VEGF | Rajagopalan et al. (2001) | Adenovirus | IM | No | 5 | Endothelial activity | Improved | |
| Intermittent claudification | VEGF | Rajagopalan et al. (2003) (RAVE) | Adenovirus | IM | Yes | 105 | Peak walking time | NSD |
| VEGF | ||||||||
| Kusumanto et al. (2006) | Plasmid | IM | Yes | 27 | Amputation rate at 100 days | NSD | ||
| Angina | VEGF | Losordo et al. (2002) | Plasmid | Catheter | Yes | 19 | CCS angina class | Improved |
| Fortuin et al. (2003) | Plasmid | Thoracotomy | No | 5 | CCS angina class | Improved | ||
| FGF | Grines et al. (2002) (AGENT I) | Adenovirus | Catheter | Yes | 79 | ETT | Trend | |
| Grines et al. (2003) (AGENT II) | Adenovirus | IC injection | Yes | 52 | Reversible perfusion defect size | Trend | ||
| VBG | E2F decoy | Mann et al. (1999) (PREVENT I) | DNA | Instilled | Yes | 41 | Safety and occlusions | Trend |
| Grube et al. (2001) (PREVENT II) | DNA | Instilled | Yes | 200 | Occlusion, vessel wall thickness | Improved (30%) | ||
| Conte et al. (2005) (PREVENT III) | DNA | Instilled | Yes | 1404 | Reintervention | NSD | ||
| Alexander et al. (2005) (PREVENT IV) | DNA | Instilled | Yes | 3014 | Angiographic failure | NSD | ||
| ISR | VEGF | Laitinen et al. (2000) | PL/Lipo | I/P catheter | No | 15 | Safety, angiography | NSD |
| Hedman et al. (2003) | PL/Lipo/Ad | I/P catheter | No | 103 | Angiography, restenosis | NSD | ||
| (KAT I) | Myocardial perfusion |
Abbreviations: ABI, ankle-brachial index; Ad, adenovirus; CCS, Canadian Cardiovascular Society; CLI, chronic limb ischaemia; ETT, excercise treadmill test; FGF, fibroblast growth factor; HGF, hepatocyte growth factor; I/P, infusion-perfusion; IC, intracoronary; IM, intramuscular; ISR, in-stent restenosis; Lipo, liposome; NSD, no significant difference; PL, plasmid; PVD, peripheral vascular disease; VBG, vascular bypass graft; VEGF, vascular endothelial growth factor.