"pUDK-HGF Gene Therapy to Relieve CLI Rest Pain and Ulcer: A Phase II, Double-Blind, Randomized Placebo-Controlled Trial"

作者全名："Gu, Yongquan; Cui, Shijun; Liu, Changjian; Zhao, Jichun; Li, Ming; Li, Yiqing; Yang, Xinglong; Lv, Bonan; Li, Mingzhang; Zhao, Wenguang; Guo, Wei; Huang, Jianhua; Huang, Wen; Qiu, Zhenming; Zhao, Jun; Yin, Ping; Qin, Tingting; Zhu, Dan; Sun, Wenjie; Ren, Keyun; Lu, Yuxin; Cheng, Xiaochen; Du, Li; Xiao, Fengjun; Zhang, Qinglin; Wu, Zuze"

作者地址："[Gu, Yongquan; Cui, Shijun] Capital Med Univ, Xuan Wu Hosp, Vasc Surg Dept, 45 Changchun St, Beijing, Peoples R China; [Liu, Changjian] Nanjin Drum Tower Hosp, Vasc Surg Dept, Nanjing, Peoples R China; [Zhao, Jichun] Sichuan Univ, West China Hosp, Vasc Surg Dept, Chengdu, Peoples R China; [Li, Ming] Zhejiang Univ, Vasc Surg Dept, Affiliated Hosp 1, Med Sch, Hangzhou, Peoples R China; [Li, Yiqing] Wuhan Union Hosp, Vasc Surg Dept, Wuhan, Peoples R China; [Yang, Xinglong] Nanchang Univ, Gen Surg Dept, Affiliated Hosp 1, Nanchang, Jiangxi, Peoples R China; [Lv, Bonan] Hebei Gen Hosp, Gen Surg Dept, Shijiazhuang, Hebei, Peoples R China; [Li, Mingzhang] Baotou Cent Hosp, Gen Surg Dept, Baotou, Peoples R China; [Zhao, Wenguang] Jilin Univ, Vasc Surg Dept, Bethune Hosp 1, Changchun, Peoples R China; [Guo, Wei] Chinese Peoples Liberat Army Gen Hosp, Vasc Surg Dept, Beijing, Peoples R China; [Huang, Jianhua] Cent South Univ, Vasc Surg Dept, Xiangya Hosp, Changsha, Peoples R China; [Huang, Wen] Chongqin Med Univ, Vasc Surg Dept, Affiliated Hosp 1, Chongqing, Peoples R China; [Qiu, Zhenming] Taizhou Hosp Zhejiang Prov, Gen Surg Dept, Linhai, Peoples R China; [Zhao, Jun] Shanghai Jiao Tong Univ, Gen Surg Dept, Shanghai Peoples Hosp 6, Shanghai, Peoples R China; [Yin, Ping; Qin, Tingting] Huazhong Univ Sci & Technol, Tongji Med Coll, Sch Publ Hlth, Wuhan, Peoples R China; [Zhu, Dan; Sun, Wenjie; Ren, Keyun] Humanwell Healthcare Grp Co Ltd, Wuhan, Peoples R China; [Lu, Yuxin; Cheng, Xiaochen; Du, Li; Xiao, Fengjun; Zhang, Qinglin; Wu, Zuze] Beijing Inst Radiat Med, Dept Expt Hematol & Biochem, 27 Taiping Rd, Beijing, Peoples R China"

通信作者："Gu, YQ (corresponding author), Capital Med Univ, Xuan Wu Hosp, Vasc Surg Dept, 45 Changchun St, Beijing, Peoples R China.; Zhang, QL (corresponding author), Beijing Inst Radiat Med, Dept Expt Hematol & Biochem, 27 Taiping Rd, Beijing, Peoples R China."

来源：HUMAN GENE THERAPY

ESI学科分类：MOLECULAR BIOLOGY & GENETICS

WOS号：WOS:000653280400001

JCR分区：Q1

影响因子：4.2

年份：2021

卷号：32

期号：15-16

开始页：839

结束页：849

文献类型：Article

关键词：peripheral arterial disease; critical limb ischemia; gene therapy; hepatocyte growth factor; pUDK-HGF; phase II clinical trial

摘要："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. This study was approved by the China Food and Drug Administration (2013L00637), China Clinical Trial Registry URL: www.chinadrugtrials.org.cn. Unique Identifier: 20130378."

基金机构："National Science and Technology Major Project, China [2014ZX09101-045-002]"

基金资助正文："This work was supported by the National Science and Technology Major Project, China, No. 2014ZX09101-045-002."