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Safety and Efficacy Study of Stem Cell Transplantation to Treat Dilated Cardiomyopathy

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ClinicalTrials.gov Identifier: NCT00629018
Recruitment Status : Completed
First Posted : March 5, 2008
Results First Posted : May 12, 2015
Last Update Posted : May 12, 2015
Information provided by (Responsible Party):

Study Description
Brief Summary:

Several studies have documented that transplantation of bone marrow-derived cells (BMC) following acute myocardial infarction is associated with a reduction in infarct scar size and improvements in left ventricular function and perfusion. The available evidence in humans suggests that BMC transplantation is associated with improvements in physiologic and anatomic parameters in both acute myocardial infarction and chronic ischemic heart disease, above and beyond the conventional therapy. In particular, intracoronary application of BMC is proved to be safe and was associated with significant improvement in the left ventricular ejection fraction (LVEF) in patients with chronic heart failure.

In contrast to ischemic heart failure, the data on effects of BMC transplantation in patients with dilated cardiomyopathy are limited to pre-clinical studies. In a rat model of dilated cardiomyopathy, intramyocardial delivery of pluripotent mesenchymal cells improved LVEF, possibly through induction of myogenesis and angiogenesis, as well as by inhibition of myocardial fibrosis, suggesting that the beneficial effects of stem cell transplantation in dilated cardiomyopathy may primarily be related to their ability to supply large amounts of angiogenic, antiapoptotic, and mitogenic factors. Similarly, transplantation of cocultured mesenchymal stem cells and skeletal myoblasts was shown to improve LVEF in a murine model of Chagas disease.

Study Aim:

To define the clinical effects of BMC transplantation in dilated cardiomyopathy in a pilot clinical study investigating the effects of intracoronary CD34+ cell transplantation on functional, structural, neurohormonal, and electrophysiologic parameters in patients with end-stage dilated cardiomyopathy.

Condition or disease Intervention/treatment Phase
Dilated Cardiomyopathy Biological: CD34+ autologous stem cell transplantation Drug: Bone Marrow Stimulation Biological: SC therapy Phase 2

Detailed Description:
Patients were randomly allocated in a 1:1 ratio to receive intracoronary transplantation of autologous CD34+ stem cells (SC group) or no intracoronary infusion (control group). At the time of enrollment, and at yearly intervals thereafter, we performed detailed clinical evaluation, echocardiography, 6-minute walk test, and measured plasma levels of NT-proBNP. To better-define the potential role of inflammatory response, we also measured plasma inflammatory markers (tumor necrosis factor [TNF]-α and interleukin [IL]-6) at the time of CD34+ stem cell injection.

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 110 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: The Effects of Autologous Intracoronary Stem Cell Transplantation In Patients With End-Stage Dilated Cardiomyopathy
Study Start Date : May 2006
Primary Completion Date : April 2013
Study Completion Date : April 2013

Arms and Interventions

Arm Intervention/treatment
Experimental: SC Group

SC therapy,'Bone Marrow Stimulation','CD34+ autologous stem cell transplantation':

In the SC group, CD34+ cells were mobilized by granulocyte colony-stimulating factor and collected via apheresis. Patients underwent myocardial scintigraphy and cells were injected in the artery supplying segments with the greatest perfusion defect

Biological: CD34+ autologous stem cell transplantation
Peripheral blood stem cells will be mobilized by daily subcutaneous injections of filgrastim; CD34+ cells will be collected via apheresis and labeled with technetium. Patients will undergo myocardial perfusion scintigraphy for myocardial viability assessment and the collected CD34+ cells will be injected intracoronary in the artery supplying the segments of reduced tracer accumulation
Drug: Bone Marrow Stimulation
Patients will undergo filgrastim stimulation and viability assessment using the same protocol as in Arm 1. However, in this group, no intracoronary stem cell delivery will be performed; the patients will receive placebo (saline).
Other Name: G-CSF stimulation
Biological: SC therapy
In the SC group, CD34+ cells were mobilized by granulocyte colony-stimulating factor and collected via apheresis. Patients underwent myocardial scintigraphy and cells were injected in the artery supplying segments with the greatest perfusion defect
No Intervention: Controls
Patients receiving no cell therapy.

Outcome Measures

Primary Outcome Measures :
  1. Heart Failure Mortality [ Time Frame: 5 years ]
  2. Changes in Left Ventricular Ejection Fraction [ Time Frame: 5 years ]
    Left ventricular ejection fraction measured by echocardiography

Secondary Outcome Measures :
  1. Changes in Exercise Capacity [ Time Frame: 5 years ]
  2. Changes in Electrophysiologic Properties of Ventricular Myocardium [ Time Frame: 6 months ]
  3. Changes in Plasma Inflammatory Markers [ Time Frame: 6 months ]
  4. Changes in Left Ventricular Function [ Time Frame: 5 years ]

Eligibility Criteria

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Normal coronary angiogram
  • Left ventricular ejection fraction < 40%
  • NYHA III or IV heart failure symptoms
  • Bone marrow reactivity (G-CSF test)
  • Presence of viable myocardium

Exclusion Criteria:

  • Hematologic malignancy
  • Multiorgan failure
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00629018

Ljubljana University Medical Center
Ljubljana, Slovenia, 1000
Sponsors and Collaborators
University Medical Centre Ljubljana
Blood Transfusion Centre of Slovenia
Stanford University
Study Director: Guillermo Torre Amione, MD, PhD Methodist DeBakey Heart Center, Houston TX, USA
Study Director: Francois Haddad, MD Stanford University
More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Bojan Vrtovec, prof. dr. Bojan vrtovec, University Medical Centre Ljubljana
ClinicalTrials.gov Identifier: NCT00629018     History of Changes
Other Study ID Numbers: DCM-SCT1
First Posted: March 5, 2008    Key Record Dates
Results First Posted: May 12, 2015
Last Update Posted: May 12, 2015
Last Verified: April 2015

Keywords provided by Bojan Vrtovec, University Medical Centre Ljubljana:
Stem Cells
Heart Failure
Dilated Cardiomyopathy

Additional relevant MeSH terms:
Cardiomyopathy, Dilated
Heart Diseases
Cardiovascular Diseases
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs