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Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy.

Publication ,  Journal Article
Greene, SJ; Epstein, SE; Kim, RJ; Quyyumi, AA; Cole, RT; Anderson, AS; Wilcox, JE; Skopicki, HA; Sikora, S; Verkh, L; Tankovich, NI; Butler, J ...
Published in: J Cardiovasc Med (Hagerstown)
April 2017

AIMS: This article describes an ongoing study investigating the safety and efficacy of ischemia-tolerant mesenchymal stem cell (MSC) therapy in patients with nonischemic heart failure and dysfunctional viable myocardium without scarring. This study will follow principles of the previously described mechanistic translational-phase concept whereby the effect of the study agent on laboratory and imaging markers of cardiac structure and function will be tested in a small homogenous cohort with the goal to enhance the understanding of the effect of interventions on cardiac remodeling and performance. STUDY DESIGN: This single-blind, placebo-controlled, crossover, multicenter, randomized study will assess the safety, tolerability, and preliminary efficacy of a single intravenous (i.v.) dose of allogeneic ischemia-tolerant MSCs in individuals with heart failure of nonischemic cause, ejection fraction 40% or less, and dysfunctional viable myocardium who have been receiving guideline-directed medical therapy. Eligible patients will have no evidence of baseline replacement scarring on delayed-enhancement cardiac magnetic resonance (CMR). Approximately 20 patients will be randomized in a 1 : 1 ratio to receive an i.v. infusion of ischemia-tolerant MSCs or placebo. At 90 days, the two groups will undergo crossover and received the alternative treatment. The primary endpoint is safety, as evaluated through at least 1-year post-MSC infusion. Additional efficacy endpoints will include measures of cardiac structure and function, as evaluated by serial cine-CMR and transthoracic echocardiography at 90 and 180 days post-initial infusion. CONCLUSION: This pilot study will explore the safety and effects on cardiac structure and function of i.v. injection of ischemia-tolerant MSCs in a small homogenous cohort of nonischemic heart failure patients with reduced ejection fraction and absent replacement scarring on CMR. This study also represents a prospective mechanistic translational-phase study using baseline and serial CMR imaging in heart failure patients and serves as a potential model for design of future heart failure trials (ClinicalTrials.gov identifier: NCT02467387).

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Published In

J Cardiovasc Med (Hagerstown)

DOI

EISSN

1558-2035

Publication Date

April 2017

Volume

18

Issue

4

Start / End Page

283 / 290

Location

United States

Related Subject Headings

  • Ventricular Remodeling
  • Ventricular Function, Left
  • United States
  • Treatment Outcome
  • Time Factors
  • Stroke Volume
  • Single-Blind Method
  • Research Design
  • Recovery of Function
  • Pilot Projects
 

Citation

APA
Chicago
ICMJE
MLA
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Greene, S. J., Epstein, S. E., Kim, R. J., Quyyumi, A. A., Cole, R. T., Anderson, A. S., … Butler, J. (2017). Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy. J Cardiovasc Med (Hagerstown), 18(4), 283–290. https://doi.org/10.2459/JCM.0000000000000303
Greene, Stephen J., Stephen E. Epstein, Raymond J. Kim, Arshed A. Quyyumi, Robert T. Cole, Allen S. Anderson, Jane E. Wilcox, et al. “Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy.J Cardiovasc Med (Hagerstown) 18, no. 4 (April 2017): 283–90. https://doi.org/10.2459/JCM.0000000000000303.
Greene SJ, Epstein SE, Kim RJ, Quyyumi AA, Cole RT, Anderson AS, et al. Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy. J Cardiovasc Med (Hagerstown). 2017 Apr;18(4):283–90.
Greene, Stephen J., et al. “Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy.J Cardiovasc Med (Hagerstown), vol. 18, no. 4, Apr. 2017, pp. 283–90. Pubmed, doi:10.2459/JCM.0000000000000303.
Greene SJ, Epstein SE, Kim RJ, Quyyumi AA, Cole RT, Anderson AS, Wilcox JE, Skopicki HA, Sikora S, Verkh L, Tankovich NI, Gheorghiade M, Butler J. Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy. J Cardiovasc Med (Hagerstown). 2017 Apr;18(4):283–290.

Published In

J Cardiovasc Med (Hagerstown)

DOI

EISSN

1558-2035

Publication Date

April 2017

Volume

18

Issue

4

Start / End Page

283 / 290

Location

United States

Related Subject Headings

  • Ventricular Remodeling
  • Ventricular Function, Left
  • United States
  • Treatment Outcome
  • Time Factors
  • Stroke Volume
  • Single-Blind Method
  • Research Design
  • Recovery of Function
  • Pilot Projects