人脐带间充质干细胞治疗膝关节骨性关节炎的临床试验方案  

Human umbilical cord mesenchymal stem cells in the treatment of knee osteoarthritis: study protocol for a clinical trial

作　　者：杨子逸[1] 林剑浩[1] 邢丹[1] 王斌[1] 侯云飞[1] Yang Zi-yi;Lin Jian-hao;Xing Dan;Wang Bin;Hou Yun-fei(Arthritis Clinic&Research Center,Peking University People’s Hospital,Beijing 100044,China)

机构地区：[1]北京大学人民医院关节病诊疗研究中心,北京市100044

出　　处：《中国组织工程研究》2018年第9期1407-1412,共6页Chinese Journal of Tissue Engineering Research

基　　金：北京大学人民医院研究与发展基金资助课题(RDH2017-05)~~

摘　　要：背景:骨关节炎是一种严重危害人类健康的慢性骨关节疾病。细胞治疗骨关节炎近年来发展较快,并得到了广泛关注。脐带来源的间充质干细胞治疗骨关节炎既具有其他来源间充质干细胞扩增分化容易,具有抗炎和招募作用等优势,又具有细胞年轻化、获得量大、无伦理问题、取材过程无痛苦、高增殖特性、多能分化特性、低免疫源性、无致瘤性等特点,是目前临床细胞治疗最常用的种子细胞。目的:拟利用同种异体脐带来源间充质干细胞治疗人膝关节骨关节炎,获得脐带来源间充质干细胞治疗骨关节炎的有效性和安全性数据,以期为干细胞治疗骨关节炎提供必要的理论基础和临床依据。方法:试验在中国北京市,北京大学人民医院关节病诊疗研究中心完成。在获得受试者的知情同意以及伦理委员会审批后(该试验前两部分均已注册(https://register.clinicaltrials.gov/),编号分别为NCT03357770及NCT03358654,第三部分需要根据前两部分安全性评价后再行进行),按照既定的纳入/排除标准招募受试者。临床试验共分为3个部分:第一部分纳入3组受试者,每组3人,分别接受高、中、低3种等级的细胞剂量治疗,3组受试者在注射后进行随访,评价剂量限制性毒性,确立最大耐受剂量。第二部分即单臂临床试验,继续招募9名受试者进行干预试验,注射剂量为第一步试验确定的最高剂量,受试者在注射后进行随访,评价治疗的安全性及有效性。第三部分采用随机对照研究的试验设计,按照随机化的原则将受试者随机分为2组,每组7人,分别给予干细胞治疗和传统的透明质酸治疗,在试验期间,保证评价者盲、受试者盲以及干预者盲。在术后既定时间点进行随访,评价干预措施的安全性和疗效数据。主要终点是参考NCI-CTCAE标准,出现不明原因的局部及全身症状或死亡终止试验。预期结果:参考既往�BACKGROUND:Osteoarthritis(OA)is a kind of chronic bone and joint disease which seriously endangers human health.Cell therapy for OA has aroused widespread concern and gotten rapid development in recent years.Umbilical cord-derived mesenchymal stem cells(UC-MSCs)have the advantages of easy amplification and differentiation,anti-inflammation and recruiting function such as MSCs from other sources.Furthermore,UC-MSCs are young cells that have large quantity,no ethical problems,high proliferative potential and pluripotent differentiation.UC-MSCs have been the most commonly used seed cells in clinical cell therapy.OBJECTIVE:To evaluate the efficacy and safety of UC-MSCs in the treatment of human knee OA to provide theoretical and clinical basis for stem cell therapy of OA.METHODS:The trail will be completed in Arthritis Clinic&Research Center,Beijing,China.Participants will be recruited according to established inclusion/exclusion criteria after obtaining the informed consent and the approval of the Ethics Committee(the first and second parts of the trial have been registered(https://register.clinicaltrials.gov/),with the identifier No.NCT03357770 and NCT03358654,and the third part will be carried out according to the conclusion of the first and second parts).The clinical trial will be divided into three parts:in the first part three groups will be recruited.Each group will contain three participants.The three groups of participants will be treated with high,medium and low dose of MSCs,respectively.Participants will be followed up to evaluate dose-limiting toxicity so as to determine the maximum tolerated dose.The second part will be a single-arm clinical trial.Nine participants will be recruited.The injection dose will be the maximum tolerated dose determined in the first part.Participants will be followed up to evaluate the safety and efficacy of the treatment.The third part will be a randomized controlled trial.Participants will be randomly divided into two groups(n=7 per group)and treated with MSCs and hyaluronic

关 键 词：干细胞 骨关节炎 组织工程 

分 类 号：R394.2[医药卫生—医学遗传学]