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NCT Number: NCT00629018

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

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.

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Key information

Conditions

Dilated Cardiomyopathy Cardiomegaly Cardiomyopathies Cardiomyopathy, Dilated Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heart Diseases Heart Failure Laminopathies

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Ljubljana University Medical Center

Ljubljana, 1000, Slovenia

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

CD34+ autologous stem cell transplantation

Biological

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

Bone Marrow Stimulation

Drug

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 names: G-CSF stimulation

SC therapy

Biological

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

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

University Medical Centre Ljubljana

Other

Collaborators

  • Blood Transfusion Centre of Slovenia
  • Stanford University

Registry information

Official study title

The Effects of Autologous Intracoronary Stem Cell Transplantation In Patients With End-Stage Dilated Cardiomyopathy

Important dates

Study start
2006
Primary completion
2013
Study completion
2013
First posted
Mar 5, 2008
Registry last updated
May 12, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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