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

作者：Liu Kathleen D^*; Wilson Jennifer G; Zhuo Hanjing; Caballero Lizette; McMillan Melanie L; Fang Xiaohui; Cosgrove Katherine; Calfee Carolyn S; Lee Jae Woo; Kangelaris Kirsten N; Gotts Jeffrey E; Rogers Angela J; Levitt Joseph E; Wiener Kronish Jeanine P; Delucchi Kevin L; Leavitt Andrew D; McKenna David H; Thompson B Taylor; Matthay Michael A

来源：Annals of Intensive Care, 2014, 4(1): 22.

DOI：10.1186/s13613-014-0022-z

• Acute lung injury
• Clinical trial
• Mesenchymal stem/stromal cell
• Pulmonary edema

Background: 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.
Methods: 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).
Results: 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.
Conclusions: 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.

• 出版日期2014-7-3