• Kathleen D Liu, Jennifer G Wilson, Hanjing Zhuo, Lizette Caballero, Melanie L McMillan, Xiaohui Fang, Katherine Cosgrove, Carolyn S Calfee, Jae-Woo Lee, Kirsten N Kangelaris, Jeffrey E Gotts, Angela J Rogers, Joseph E Levitt, Jeanine P Wiener-Kronish, Kevin L Delucchi, Andrew D Leavitt, David H McKenna, B Taylor Thompson, and Michael A Matthay.
• Departments of Nephrology and Anesthesia, University of California, San Francisco, CA, USA.
• Ann Intensive Care. 2014 Jan 1;4:22.

BackgroundDespite 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.MethodsThis 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).ResultsThis 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.ConclusionsExperience 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.Trial RegistrationClinical Trials RegistrationNCT01775774 and NCT02097641.

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