This randomized pilot phase I trial studies the side effects and best method of delivery of bone marrow derived mesenchymal stem cells (MSCs) in improving heart function in patients with heart failure caused by anthracyclines (a type of chemotherapy drug used in cancer treatment). MSCs are a type of stem cell that can be removed from bone marrow and grown into many different cell types that can be used to treat cancer and other diseases, such as heart failure. Bone marrow derived MSCs may promote heart muscle cells repair and lead to reverse remodeling and ultimately improve heart function and decrease morbidity and mortality from progression to advanced heart failure.
PRIMARY OBJECTIVE: I. To demonstrate the safety of allogeneic human mesenchymal stem cells (hMSCs) administered by intravenous infusion in patients with left ventricular (LV) dysfunction and heart failure secondary to chemotherapy with anthracyclines. SECONDARY OBJECTIVE: I. To demonstrate the efficacy of allogeneic hMSCs administered by intravenous infusion in patients with left ventricular dysfunction (left ventricular ejection fraction \[LVEF\] \< 40%) and heart failure secondary to treatment with anthracyclines. OUTLINE: Patients are randomized to 1 of 2 treatment arms. ARM I: Patients receive allogeneic hMSCs intravenously (IV) over 10-20 minutes once weekly for 4 weeks and standard of care drugs for heart failure. ARM II: Patients receive only standard of care drugs for heart failure. After completion of study treatment, patients are followed up monthly for 6 months and then at 12 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
7
Correlative studies
Undergo mesenchymal stem cell infusion
Undergo mesenchymal stem cell infusion
M D Anderson Cancer Center
Houston, Texas, United States
Incidence of adverse events of intravenous injection of human mesenchymal stem cells (hMSCs) in patients with recent onset left ventricular systolic dysfunction from anthracyclines
Statistical analyses of safety will be descriptive. In particular, will provide a table of adverse events.
Time frame: Up to 6 months
Change in improvement in left ventricular systolic function by 7 points
Change from baseline left ventricular ejection fraction (LVEF) at 6 months after randomization, expressed as a percentage. The comparison will be between the two groups of patients.
Time frame: Baseline up to 6 months
Development of acute heart failure decompensation
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 6 months
Emergency center visits for heart failure
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 6 months
Need for new pacemaker/automatic implantable cardioverter defibrillator
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 6 months
New onset arrhythmias
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 6 months
Sudden cardiac death
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 6 months
Acute pulmonary edema
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 6 months
Asymptomatic decrease in left ventricular ejection fraction (LVEF) of > 10%
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Baseline to up to 6 months
Heart failure admission
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 6 months
Progression free survival
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Time frame: Up to 12 months
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