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

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, Michael A. Matthay

Research output: Contribution to journalArticlepeer-review

37 Scopus citations

Abstract

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. Trial registration: Clinical Trials Registration: NCT01775774 and NCT02097641.

Original languageEnglish (US)
Article number22
Pages (from-to)1-9
Number of pages9
JournalAnnals of Intensive Care
Volume4
Issue number1
DOIs
StatePublished - Dec 1 2014

Bibliographical note

Publisher Copyright:
© 2014, Liu et al.; licensee Springer.

Keywords

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

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