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Open Access 01-12-2014 | Research

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

Authors: 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

Published in: Annals of Intensive Care | Issue 1/2014

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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Title: 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
Authors: 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
Publication date: 01-12-2014
Publisher: Springer Paris
Published in: Annals of Intensive Care / Issue 1/2014
Electronic ISSN: 2110-5820
DOI: https://doi.org/10.1186/s13613-014-0022-z

Keynote webinar | Spotlight on medication adherence

Springer Medicine

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

Abstract

Background

Methods

Results

Conclusions

Trial registration

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background

Methods

Results

Conclusions

Trial registration

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