عنوان

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.

Number

LA2jf572q0

Title Proper

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.

General Material Designation

[Article]

First Statement of Responsibility

Liu, Kathleen DWilson, Jennifer GZhuo, HanjingCaballero, LizetteMcMillan, Melanie LFang, XiaohuiCosgrove, KatherineCalfee, Carolyn SLee, Jae-WooKangelaris, Kirsten NGotts, Jeffrey ERogers, Angela JLevitt, Joseph EWiener-Kronish, Jeanine PDelucchi, Kevin LLeavitt, Andrew Det al.

Text of Note

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.

Date of Publication

2014

Title

UCSF

Electronic name

[Article]

277831

a

Y