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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.

Publication ,  Journal Article
Golpanian, S; DiFede, DL; Pujol, MV; Lowery, MH; Levis-Dusseau, S; Goldstein, BJ; Schulman, IH; Longsomboon, B; Wolf, A; Khan, A; Heldman, AW ...
Published in: Oncotarget
March 15, 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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Published In

Oncotarget

DOI

EISSN

1949-2553

Publication Date

March 15, 2016

Volume

7

Issue

11

Start / End Page

11899 / 11912

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Survival Rate
  • Research Design
  • Regenerative Medicine
  • Prognosis
  • Middle Aged
  • Mesenchymal Stem Cells
  • Mesenchymal Stem Cell Transplantation
  • Male
  • Infusions, Intravenous
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Golpanian, Samuel, Darcy L. DiFede, Marietsy V. Pujol, Maureen H. Lowery, Silvina Levis-Dusseau, Bradley J. Goldstein, Ivonne H. Schulman, et al. “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.Oncotarget 7, no. 11 (March 15, 2016): 11899–912. https://doi.org/10.18632/oncotarget.7727.

Published In

Oncotarget

DOI

EISSN

1949-2553

Publication Date

March 15, 2016

Volume

7

Issue

11

Start / End Page

11899 / 11912

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Survival Rate
  • Research Design
  • Regenerative Medicine
  • Prognosis
  • Middle Aged
  • Mesenchymal Stem Cells
  • Mesenchymal Stem Cell Transplantation
  • Male
  • Infusions, Intravenous