Molecular therapy : the journal of the American Society of Gene Therapy · 2025
Seltoplasmid promotes ulcer healing versus placebo for treating patients with chronic limb-threatening ischemia: HOPE CLTI-2 trial.
СтатьяМногоцентровое исследованиеРандомизированное исследованиеКлиническое исследование III фазыНазвание и аннотация: Перевод пока не подготовлен
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Di Xiao, Liu Changwei, Sun Siqiao, Qin Jinbao, Guo Xueli, Chen Yikuan, Li Xin, Zhang Hongkun, Liu Ming, Yang Liu, Zhao Hui, Lu Shaoying, Huang Jingyong, Zhang Yunfeng, Li Jun, Lin Xiaolei, Yao Kai, Tang Jingdong, Wang Jian, Gao Zhanfeng, Wang Jinjun, Huang Xiaojin, Xu Songshan, Liu Yue, Han Wei, Ni Leng, Ye Wei, Zheng Yuehong, Chen Yuexin, Liu Bao
Аннотация
Intramuscular injection of donaperminogene seltoplasmid (recombinant human hepatocyte growth factor plasmids) represents a gene therapy that treats patients with chronic limb-threatening ischemia (CLTI). The HOPE CLTI-2 trial was a phase 3, multicenter, double-blind, placebo-controlled study aimed to evaluate the efficacy and safety of seltoplasmid in patients with Rutherford class 5 CLTI. This study did not require participants to be ineligible for revascularization, allowing enrollment of patients with CLTI caused by either atherosclerosis (ASO) or Buerger disease (or thromboangiitis obliterans [TAO]). The primary endpoint was the complete ulcer healing rate at 6 months. A total of 242 participants (53.3% ASO versus 46.7% TAO) were enrolled, with 161 receiving seltoplasmid and 81 receiving placebo. Complete ulcer healing was achieved in 70 patients in the seltoplasmid group compared to 15 patients in the placebo group, resulting in an adjusted healing rate difference of 26.1% (95% confidence interval [CI]: 15.1%-37.0%; p < 0.001). The hazard ratio for healing was 2.31 (95% CI: 1.32-4.05; p = 0.004). The benefits of seltoplasmid on ulcer healing persisted in both TAO and ASO subgroups. Serious adverse events were rare. Our study demonstrated that seltoplasmid significantly improved ulcer healing rates in patients with Rutherford class 5 CLTI compared to placebo.