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Ann Intensive Care. 2014 Jul 3;4:22. doi: 10.1186/s13613-014-0022-z. eCollection 2014.

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 information

1
Departments of Nephrology and Anesthesia, University of California, San Francisco, CA, USA.
2
Departments of Emergency Medicine and Critical Care Medicine, University of California, San Francisco, CA, USA.
3
Cardiovascular Research Institute, University of California, San Francisco, CA, USA.
4
Clinical Laboratories, Bone Marrow Center, University of California, San Francisco, CA, USA.
5
Division of Pulmonary and Critical Care Medicine, Department of Medicine, Massachusetts General Hospital, Boston, MA, USA.
6
Division of Pulmonary and Critical Care Medicine, Department of Medicine, University of California, San Francisco, CA, USA.
7
Department of Anesthesia, University of California, San Francisco, CA, USA.
8
Division of Hospital Medicine, Department of Medicine, University of California, San Francisco, CA, USA.
9
Division of Pulmonary and Critical Care Medicine, Department of Medicine, Stanford University, Stanford, CA, USA.
10
Department of Anesthesia, Critical Care and Pain Medicine, Massachusetts General Hospital, Harvard University, Cambridge, MA, USA.
11
Department of Psychiatry, University of California, San Francisco, CA, USA.
12
Department of Laboratory Medicine, University of California, San Francisco, CA, USA.
13
Division of Transfusion Medicine, Department of Laboratory Medicine and Pathology, University of Minnesota, Minneapolis, MN, USA.
14
Departments of Medicine and Anesthesia and the Cardiovascular Research Institute, University of California, San Francisco, CA, USA.

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.

KEYWORDS:

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

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