This phase II clinical trial investigated the efficacy and safety of intramuscular injection of plasmid pUDK-HGF, which encodes the human hepatocyte growth factor gene in patients with critical limb ischemia. Resting pain patients (n = 119) and patients with leg ulcers (n = 121) were enrolled as two cohorts and randomized to receive pUDK-HGF treatment on days 0, 14, and 28. In the resting pain cohort, the proportion of patients with complete pain relief on day 180 after receiving pUDK-HGF injection, as the primary outcome, was significantly higher than that of the placebo group on the same day (p = 0.0148). More responders with >50% pain reduction were also observed in the pUDK-HGF groups than in the placebo groups (p = 0.0168). In the ulcer cohort of patients, pUDK-HGF treatment tended to be superior to the placebo in the percentage of patients with both complete ulcer healing and >50% ulcer healing. No significant differences in the incidence of adverse events (AEs) or serious AEs were observed among the groups. The mid-dose pUDK-HGF (6 mg) was the most efficacious, and is therefore an appropriate dose for use in a phase III clinical trial.

中文翻译：

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pUDK-HGF 基因治疗缓解 CLI 休息疼痛和溃疡：II 期、双盲、随机安慰剂对照试验

这项 II 期临床试验调查了肌内注射质粒 pUDK-HGF 的疗效和安全性，该质粒编码人类肝细胞生长因子基因，用于严重肢体缺血患者。静息痛患者 ( n  = 119) 和腿部溃疡患者 ( n  = 121) 被纳入两个队列，并在第 0、14 和 28 天随机接受 pUDK-HGF 治疗。在静息痛队列中，患者的比例作为主要结果，接受 pUDK-HGF 注射后第 180 天疼痛完全缓解的患者在同一天明显高于安慰剂组（p  = 0.0148）。与安慰剂组相比，在 pU​​DK-HGF 组中也观察到更多的疼痛减轻 > 50% 的反应者（p = 0.0168）。在溃疡患者队列中，pUDK-HGF 治疗在溃疡完全愈合和 > 50% 溃疡愈合的患者百分比方面往往优于安慰剂。在各组之间未观察到不良事件 (AE) 或严重 AE 的发生率存在显着差异。中等剂量的 pUDK-HGF (6 mg) 是最有效的，因此是用于 III 期临床试验的合适剂量。