Safety and efficacy of ixmyelocel-T: an expanded, autologous multi-cellular therapy, in dilated cardiomyopathy

Circ Res. 2014 Sep 26;115(8):730-7. doi: 10.1161/CIRCRESAHA.115.304554. Epub 2014 Aug 20.


Rationale: Ixmyelocel-T is associated with a wide range of biological activities relevant to tissue repair and regeneration.

Objective: To evaluate the safety and efficacy of ixmyelocel-T in 2 prospective randomized phase 2A Trials administered via minithoracotomy or intramyocardial catheter injections in patients with dilated cardiomyopathy (DCM) stratified by ischemic or nonischemic status.

Methods and results: In IMPACT-DCM, patients were randomized to either ixmyelocel-T or standard-of-care control in a 3:1 ratio (n=39); ixmyelocel-T was administered intramyocardially via minithoracotomy. In Catheter-DCM, patients were randomized to either ixmyelocel-T or standard of care control in a 2:1 ratio (n=22); ixmyelocel-T was administered intramyocardially using the NOGA Myostar catheter. Only patients randomized to ixmyelocel-T underwent bone marrow aspiration and injections. In the 2 studies, a total of 61 patients were randomized, and 59 were treated or received standard of care. Fewer ischemic patients treated with ixmyelocel-T experienced a major adverse cardiovascular event during follow-up when compared with control patients. A similar benefit was not seen in the nonischemic patients. Heart failure exacerbation was the most common major adverse cardiovascular event. Ixmyelocel-T treatment was associated with improved New York Heart Association class, 6-minute walk distance, and Minnesota Living with Heart Failure Questionnaire scores in the ischemic population relative to control; a similar trend was not observed in the nonischemic population.

Conclusions: Intramyocardial injection with ixmyelocel-T reduces major adverse cardiovascular event and improves symptoms in patients with ischemic DCM but not in patients with nonischemic DCM.

Trial registration: NCT00765518 NCT01020968.

Keywords: cardiomyopathy, dilated; clinical trial; heart failure; stem cell.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Cardiomyopathy, Dilated / pathology
  • Cardiomyopathy, Dilated / physiopathology
  • Cardiomyopathy, Dilated / therapy*
  • Catheters
  • Cell Transplantation / adverse effects
  • Cell Transplantation / methods*
  • Cell- and Tissue-Based Therapy / adverse effects
  • Cell- and Tissue-Based Therapy / methods*
  • Female
  • Follow-Up Studies
  • Heart Function Tests
  • Humans
  • Ischemia / therapy*
  • Male
  • Middle Aged
  • Myocardium / pathology
  • Prospective Studies
  • Tomography, Emission-Computed, Single-Photon
  • Transplantation, Autologous
  • Treatment Outcome

Associated data