Long-term follow-up of a phase Ⅱ clinical trial on pUDK-hepatocyte growth factor treatment for rest pain and ulcers caused by critical limb ischemia
10.3760/cma.j.cn114015-20230918-00686
- VernacularTitle:重组质粒-肝细胞生长因子基因治疗严重肢体缺血导致静息痛和溃疡Ⅱ期临床试验的长期随访研究
- Author:
Shijun CUI
1
;
Jianming GUO
1
;
Zhu TONG
1
;
Lianrui GUO
1
;
Yongquan GU
1
Author Information
1. 首都医科大学宣武医院血管外科，北京 100053
- Publication Type:Journal Article
- Keywords:
Peripheral arterial disease;
Critical limb ischemia;
Gene therapy;
pUDK-hepatocyte growth factor;
Clinical trial, phase Ⅱ;
Long-term follow-up
- From:
Adverse Drug Reactions Journal
2024;26(4):193-197
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To explore the long-term efficacy and safety of gene therapy with pUDK-hepatocyte growth factor (pUDK-HGF) for rest pain and ulcers caused by critical limb ischemia.Methods:Long-term follow-up were conducted through outpatient and telephone on patients who completed the pUDK-HGF Phase Ⅱ randomized double-blind placebo-controlled trial. The occurrence of tumors was observed, and tumor markers detection, fundus examination, visual analogue scale (VAS), and lower limb CT angiography (CTA) were performed according to voluntary principle. The results were analyzed descri-ptively and statistically.Results:A total of 53 patients were included in the analysis, of which 15 (28.3%) were in the placebo group and 38 (71.7%) were in the pUDK-HGF treatment group in the Phase Ⅱ clinical trial. The median follow-up time was 2.8 years, ranging from 1.7 to 3.5 years. During the follow-up period, no tumor was found in the 53 patients. Among the 38 patients in the pUDK-HGF treatment group, 18 underwent comprehensive examination and evaluation, including tumor markers, fundus and CTA examination, and patients with resting pain underwent VAS evaluation. Among them, 1 patient had transient mild elevation of carcinoembryonic antigen, and no abnormal tumor markers were found in the other 17 patients; no proliferative retinal vasculopathy was found in the fundus examination. At the end of the phase Ⅱ clinical trial (out-group), 3 were effective and 2 were ineffective of the 5 patients with rest pain; at the end of this follow-up period, 4 were evaluated as effectiveness and 1 as ineffectiveness according to CTA, and 5 were all evaluated as effectiveness according to VAS. Of the 13 patients with ulcer, 9 were evaluated as effectiveness and 4 were as ineffectiveness according to CTA at out-group; 10 were evaluated as effectiveness and 3 were as ineffectiveness at the end of this follow-up.Conclusions:pUDK-HGF had relatively good safety in the treatment of rest pain and ulcers caused by critical limb ischemia. No risk of carcinogenesis and proliferative retinal vasculopathy has been found, and the long-term efficacy of pUDK-HGF is good.
