PDF(Pubmed)
Abstract：
BACKGROUND: Alport syndrome (AS) is one of the most common fatal hereditary renal diseases in human, with a high risk of progressing to end-stage renal disease without effective treatments. Mesenchymal stem cells (MSCs) have recently emerged as a promising therapeutic strategy for chronic kidney disease. However, the safety and therapeutic potential of MSC transfusion for patients with AS are still need to be confirmed. Therefore, we have designed a clinical trial to evaluate the hypothesis that intravenous infusion of human umbilical cord-derived MSC (hUC-MSC) is safe, feasible, and well-tolerated in children with AS.
METHODS: We report the protocol of the first prospective, open-label, single-arm clinical trial to evaluate the safety and preliminary efficacy of hUC-MSC transfusion in children with early-stage AS. Paediatric patients diagnosed with AS who have persistent albuminuria will be candidates for screening. Twelve eligible patients are planned to recruit and will receive hUC-MSC infusions under close safety monitoring, and complete the efficacy assessments at scheduled follow-up visits. The primary endpoints include the occurrence of adverse events to assess safety and the albuminuria level for efficacy evaluation. Secondary endpoint assessments are based on haematuria and glomerular filtration measurements. Each patient\'s efficacy endpoints will be evaluated against their baseline levels. Additionally, the underlying mechanism of hUC-MSC therapy will be explored through transcriptomic and proteomic analysis of blood and urine samples.
BACKGROUND: The protocol (V.1.0, date 17 January 2015) was approved by the institutional review board of the Affiliated Taihe Hospital of Hubei University of Medicine (ethical approval 03 March 2015). Written informed consent will be obtained from the patient and/or guardians before study specific process. In addition to publication in a peer-reviewed scientific journal, a lay summary of study will be available for participants and the public on the Chinese Organization for Rare Disorders website (http://www.cord.org.cn/).
BACKGROUND: ISRCTN62094626.
METHODS: We report the protocol of the first prospective, open-label, single-arm clinical trial to evaluate the safety and preliminary efficacy of hUC-MSC transfusion in children with early-stage AS. Paediatric patients diagnosed with AS who have persistent albuminuria will be candidates for screening. Twelve eligible patients are planned to recruit and will receive hUC-MSC infusions under close safety monitoring, and complete the efficacy assessments at scheduled follow-up visits. The primary endpoints include the occurrence of adverse events to assess safety and the albuminuria level for efficacy evaluation. Secondary endpoint assessments are based on haematuria and glomerular filtration measurements. Each patient\'s efficacy endpoints will be evaluated against their baseline levels. Additionally, the underlying mechanism of hUC-MSC therapy will be explored through transcriptomic and proteomic analysis of blood and urine samples.
BACKGROUND: The protocol (V.1.0, date 17 January 2015) was approved by the institutional review board of the Affiliated Taihe Hospital of Hubei University of Medicine (ethical approval 03 March 2015). Written informed consent will be obtained from the patient and/or guardians before study specific process. In addition to publication in a peer-reviewed scientific journal, a lay summary of study will be available for participants and the public on the Chinese Organization for Rare Disorders website (http://www.cord.org.cn/).
BACKGROUND: ISRCTN62094626.
摘要：
背景：Alport综合征（AS）是人类最常见的致命遗传性肾脏疾病之一，如果没有有效的治疗，进展为终末期肾病的风险很高。间充质干细胞（MSC）最近已成为慢性肾脏疾病的有希望的治疗策略。然而,MSC输血治疗AS患者的安全性和治疗潜力仍有待证实.因此,我们设计了一项临床试验来评估静脉输注人脐带源性MSC（hUC-MSC）是安全的假设，可行,并且在患有AS的儿童中耐受性良好。
方法：我们报告了第一个前瞻性，开放标签，单臂临床试验评估早期AS患儿输注hUC-MSC的安全性和初步疗效。诊断为AS且有持续性白蛋白尿的儿科患者将是筛查的候选人。计划招募12名符合条件的患者，并将在密切的安全监测下接受hUC-MSC输注，并在预定的随访中完成疗效评估。主要终点包括用于评估安全性的不良事件的发生和用于疗效评估的白蛋白尿水平。次要终点评估基于血尿和肾小球滤过测量。每个患者的疗效终点将根据他们的基线水平进行评估。此外,hUC-MSC治疗的潜在机制将通过血液和尿液样本的转录组学和蛋白质组学分析来探索。
背景：该方案（V.1.0，日期2015年1月17日）由湖北医药大学附属太和医院机构审查委员会批准（伦理批准2015年3月3日）。在研究特定过程之前，将从患者和/或监护人获得书面知情同意书。除了在同行评审的科学杂志上发表外，研究摘要将在中国罕见疾病组织网站（http：//www。cord.org.cn/)。
背景：ISRCTN62094626。
方法：我们报告了第一个前瞻性，开放标签，单臂临床试验评估早期AS患儿输注hUC-MSC的安全性和初步疗效。诊断为AS且有持续性白蛋白尿的儿科患者将是筛查的候选人。计划招募12名符合条件的患者，并将在密切的安全监测下接受hUC-MSC输注，并在预定的随访中完成疗效评估。主要终点包括用于评估安全性的不良事件的发生和用于疗效评估的白蛋白尿水平。次要终点评估基于血尿和肾小球滤过测量。每个患者的疗效终点将根据他们的基线水平进行评估。此外,hUC-MSC治疗的潜在机制将通过血液和尿液样本的转录组学和蛋白质组学分析来探索。
背景：该方案（V.1.0，日期2015年1月17日）由湖北医药大学附属太和医院机构审查委员会批准（伦理批准2015年3月3日）。在研究特定过程之前，将从患者和/或监护人获得书面知情同意书。除了在同行评审的科学杂志上发表外，研究摘要将在中国罕见疾病组织网站（http：//www。cord.org.cn/)。
背景：ISRCTN62094626。
