Research Papers: Gerotarget (Focus on Aging):
Rationale and design of the allogeneiC human mesenchymal stem cells (hMSC) in patients with aging fRAilTy via intravenoUS delivery (CRATUS) study: A phase I/II, randomized, blinded and placebo controlled trial to evaluate the safety and potential efficacy of allogeneic human mesenchymal stem cell infusion in patients with aging frailty
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Samuel Golpanian1,2, Darcy L. DiFede1, Marietsy V. Pujol1, Maureen H. Lowery3, Silvina Levis-Dusseau3,5, Bradley J. Goldstein1,7, Ivonne H. Schulman1,3,6, Bangon Longsomboon1, Ariel Wolf1, Aisha Khan1, Alan W. Heldman1,3,4, Pascal J. Goldschmidt-Clermont3,4 and Joshua M. Hare1,3,4
1 Interdisciplinary Stem Cell Institute, University of Miami Miller School of Medicine, Miami, FL, USA
2 Department of Surgery, University of Miami Miller School of Medicine, Miami, FL, USA
3 Department of Medicine, University of Miami Miller School of Medicine, Miami, FL, USA
4 Division of Cardiology, University of Miami Miller School of Medicine, Miami, FL, USA
5 Division of Endocrinology, University of Miami Miller School of Medicine, Miami, FL, USA
6 Division of Nephrology, University of Miami Miller School of Medicine, Miami, FL, USA
7 Department of Otolaringology, University of Miami Miller School of Medicine, Miami, FL, USA
Joshua M. Hare, email:
Keywords: aging, frailty, mesenchymal stem cells, allogeneic, Gerotarget
Received: December 20, 2015 Accepted: January 30, 2016 Published: February 25, 2016
Frailty is a syndrome associated with reduced physiological reserves that increases an individual’s vulnerability for developing increased morbidity and/or mortality. While most clinical trials have focused on exercise, nutrition, pharmacologic agents, or a multifactorial approach for the prevention and attenuation of frailty, none have studied the use of cell-based therapies. We hypothesize that the application of allogeneic human mesenchymal stem cells (allo-hMSCs) as a therapeutic agent for individuals with frailty is safe and efficacious. The CRATUS trial comprises an initial non-blinded phase I study, followed by a blinded, randomized phase I/II study (with an optional follow-up phase) that will address the safety and pre-specified beneficial effects in patients with the aging frailty syndrome. In the initial phase I protocol, allo-hMSCs will be administered in escalating doses via peripheral intravenous infusion (n=15) to patients allocated to three treatment groups: Group 1 (n=5, 20 million allo-hMSCs), Group 2 (n=5, 100 million allo-hMSCs), and Group 3 (n=5, 200 million allo-hMSCs). Subsequently, in the randomized phase, allo-hMSCs or matched placebo will be administered to patients (n=30) randomly allocated in a 1:1:1 ratio to one of two doses of MSCs versus placebo: Group A (n=10, 100 million allo-hMSCs), Group B (n=10, 200 million allo-hMSCs), and Group C (n=10, placebo). Primary and secondary objectives are, respectively, to demonstrate the safety and efficacy of allo-hMSCs administered in frail older individuals. This study will determine the safety of intravenous infusion of stem cells and compare phenotypic outcomes in patients with aging frailty.
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