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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.

  • Author(s): 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
  • et al.
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.

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