Table 8.1.
Major trials of gene therapy and cell therapy
| End points |
||||||
|---|---|---|---|---|---|---|
| Trial | Treatment | No. of participants | AFS at 12 months (treated vs placebo) | Other end points | Treatment vs placebo | Reference |
|
| ||||||
| Gene therapy | ||||||
| TALISMAN | FGF | 125 | 73% vs 48% (P = .009) | Nikol,522 2008 | ||
| TAMARIS | FGF | 525 | 63% vs 67% (P = .48) | Belch,524 2011 | ||
| HGF-STAT | HGF | 104 | No difference | Change in TcPo2at 6 months | 25.2 mm Hg in high-dose group vs 9.4 mm Hg in placebo group (P = .0015) | Powell,526 2008 |
| HGF-0205 | HGF | 27 | No difference | Change in TBI at 6 months | +0.05 vs −0.17 (P = 0.047) | Powell,525 2010 |
| Shigematsu et al | HGF | 40 | No difference | Improvement in rest pain or reduction in ulcer size | 70.4% vs 30.8% (P = .014) | Shigematsu,527 2010 |
| Cell therapy | ||||||
| Iafrati et al | Autologous bone marrow | 97 | No difference | Improvement in pain at 6 months | 58% vs 26% (P = .025) | Iafrati,528,530 2011, 2016 |
| Improvement in TBI at 6 months | 0.48 vs 0.012 (P = .02) | |||||
| RESTORE-CLI | Expanded autologous stem cells | 72 | No difference | Combined outcomes (1-year freedom from major amputation, mortality, increased wound size, new gangrene) | 40% vs 67% (P = .045) | Powell,532 2012 |
| MOBILE | Autologous bone marrow cells | 152 | 80% vs 69% (P = not significant) | Murphy,529,5312011, 2016 | ||
| JUVENTAS | BMMNCs | 160 | 77% vs 84% (at 6 months) No difference |
Major amputation at 6 months | 19% vs 13% (P = not significant) | Teraa,535 2015 |
AFS, Amputation-free survival; BMMNCs, bone marrow mononuclear cells; FGF, fibroblast growth factor; HGF, hepatocyte growth factor; JUVENTAS, Rejuvenating Endothelial Progenitor Cells via Transcutaneous Intra-arterial Supplementation; MOBILE, MarrowStim treatment of limb ischemia in subjects with severe peripheral arterial disease; TBI, toe-brachial index; TcPo2, transcutaneous oximetry.