Design and implementation of the START (STem cells for ARDS Treatment) trial, a phase 1/2 trial of human mesenchymal stem/stromal cells for the treatment of moderate-severe acute respiratory distress syndrome.
Design and implementation of the START (STem cells for ARDS Treatment) trial, a phase 1/2 trial of human mesenchymal stem/stromal cells for the treatment of moderate-severe acute respiratory distress syndrome.
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DOI：
10.1186/s13613-014-0022-z
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发表时间：
2014
影响因子：
8.1
通讯作者：
Matthay MA
中科院分区：
文献类型：
--
作者：
Liu KD;Wilson JG;Zhuo H;Caballero L;McMillan ML;Fang X;Cosgrove K;Calfee CS;Lee JW;Kangelaris KN;Gotts JE;Rogers AJ;Levitt JE;Wiener-Kronish JP;Delucchi KL;Leavitt AD;McKenna DH;Thompson BT;Matthay MA
尽管在支持性护理方面取得了进展，但中-重度急性呼吸窘迫综合征(ARDS)与高死亡率有关，因此需要新的治疗方法来治疗这种疾病。来自小鼠、大鼠、绵羊和体外灌流的人肺模型的令人信服的临床前数据支持使用人间充质干细胞(MSCs)作为一种新的静脉疗法用于ARDS的早期治疗。本文描述了START(用于ARDS治疗的干细胞)试验的研究设计和实施过程中遇到的挑战，START(用于ARDS的干细胞治疗)试验是一项1/2期的人类骨髓间充质干细胞治疗中-重度ARDS的试验。计划招募69名受试者(9名受试者在第一阶段，60名受试者在第二阶段接受骨髓间充质干细胞或安慰剂治疗，比例为2：1)。这份报告描述了重症受试者1期试验独有的研究设计特征，以及在ICU实施基于细胞的治疗试验的具体挑战。在设计和实施START研究期间获得的经验将有助于研究人员规划未来以ICU为基础的第一阶段临床试验，以及其他急性疾病的基于细胞的治疗试验。临床试验注册：NCT01775774和NCT02097641。
Despite advances in supportive care, moderate-severe acute respiratory distress syndrome (ARDS) is associated with high mortality rates, and novel therapies to treat this condition are needed. Compelling pre-clinical data from mouse, rat, sheep and ex vivo perfused human lung models support the use of human mesenchymal stem (stromal) cells (MSCs) as a novel intravenous therapy for the early treatment of ARDS. This article describes the study design and challenges encountered during the implementation and phase 1 component of the START (STem cells for ARDS Treatment) trial, a phase 1/2 trial of bone marrow-derived human MSCs for moderate-severe ARDS. A trial enrolling 69 subjects is planned (9 subjects in phase 1, 60 subjects in phase 2 treated with MSCs or placebo in a 2:1 ratio). This report describes study design features that are unique to a phase 1 trial in critically ill subjects and the specific challenges of implementation of a cell-based therapy trial in the ICU. Experience gained during the design and implementation of the START study will be useful to investigators planning future phase 1 clinical trials based in the ICU, as well as trials of cell-based therapy for other acute illnesses. Clinical Trials Registration: NCT01775774 and NCT02097641.
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DOI：
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发表时间：
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作者：
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通讯作者：
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