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Effectiveness of Stem Cell Treatment for Adults With Ischemic Cardiomyopathy (The FOCUS Study)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00824005
Recruitment Status : Completed
First Posted : January 16, 2009
Results First Posted : April 4, 2013
Last Update Posted : July 1, 2015
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Cardiovascular Cell Therapy Research Network (CCTRN)
Information provided by (Responsible Party):
Dr Lemuel A Moye III, The University of Texas Health Science Center, Houston

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Chronic Ischemic Heart Disease
Left Ventricular Dysfunction
Angina
Ischemic Cardiomyopathy
Interventions Biological: Adult stem cells
Biological: Placebo
Enrollment 92
Recruitment Details Enrollment took place at five Network centers and their associated satellite facilities between April 29, 2009 and April 18, 2011. The main centers are located in Ohio, Texas, Florida, Minnesota, and Tennessee. Study brochures, patient informational DVDs, and clinical trials.gov were among the tools used for recruitment.
Pre-assignment Details  
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description Participants will receive placebo injections. Participants will receive active stem cell injections.
Period Title: Overall Study
Started 31 61
Completed 31 59
Not Completed 0 2
Reason Not Completed
Death             0             1
Lost to Follow-up             0             1
Arm/Group Title Placebo Injections Active Stem Cell Injections Total
Hide Arm/Group Description Participants will receive placebo injections. Participants will receive active stem cell injections. Total of all reporting groups
Overall Number of Baseline Participants 31 61 92
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 31 participants 61 participants 92 participants
62.32  (8.25) 63.95  (10.90) 63.4  (10.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants 61 participants 92 participants
Female
2
   6.5%
8
  13.1%
10
  10.9%
Male
29
  93.5%
53
  86.9%
82
  89.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 31 participants 61 participants 92 participants
31 61 92
1.Primary Outcome
Title Change in Maximal Oxygen Consumption (VO2max)
Hide Description The VO2(max) is assessed using the Naughton treadmill protocol.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and 6 month VO2max data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 27 52
Mean (Standard Deviation)
Unit of Measure: mL/kg/min
-0.6  (3.2) 0.4  (2.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Compare the change in the cell group to the change in the placebo group.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.169
Comments No adjustment for multiple comparisons
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.0
Confidence Interval (2-Sided) 95%
-0.42 to 2.34
Parameter Dispersion
Type: Standard Error of the mean
Value: 2.9
Estimation Comments [Not Specified]
2.Primary Outcome
Title Change in Left Ventricular End Systolic Volume (LVESV)as Assessed Via Echo
Hide Description Echocardiographic measurements were performed by an echocardiographic core laboratory. LVESVs were calculated by the modified biplane Simpson method, using myocardial contrast to enhance endocardial definition. To account for patient body surface area, LVESV indices are reported.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month LVESV data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 28 54
Mean (Standard Deviation)
Unit of Measure: mL/m2
0  (10.8) -0.9  (11.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Change in the difference of end systolic volume over time.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.856
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.0
Confidence Interval (2-Sided) 95%
-10.05 to 12.07
Parameter Dispersion
Type: Standard Error of the mean
Value: 23.9
Estimation Comments [Not Specified]
3.Primary Outcome
Title Change in Reversible Defect Size
Hide Description Adenosine myocardial perfusion (SPECT) tests were collected at baseline and 6 months to identify change in ischemic (reversible) defects. SPECT imaging was performed at rest and after adenosine infusion over 4 minutes. To enhance the detection of viability on resting images, sublingual nitroglycerin was administered 15 minutes before injecting technetium Tc 99m sestamibi for the resting image.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month reversible defect data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 26 50
Mean (Standard Deviation)
Unit of Measure: percentage of reversible defect
-2.7  (18.2) -3.9  (25.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Change in percent of the defect that is reversible
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.835
Comments Is the change in percent reversible defect the same between the two groups
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.2
Confidence Interval (2-Sided) 95%
-12.5 to 10.1
Parameter Dispersion
Type: Standard Error of the mean
Value: 22.3
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Regional Wall Motion by MRI (in Eligible Patients)
Hide Description Regional wall motion as measured by cardiac MRI (in patients who are not contraindicated)
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The small number of patients without contraindications for MRI (n=17) precluded performing informative analysis on the MRI data.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Regional Blood Flow Improvement by MRI (in Eligible Patients)
Hide Description Regional blood flow improvement as measured by cardiac MRI (in patients who are not contraindicated)
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The small number of patients without contraindications for MRI (n=17) precluded performing informative analysis on the MRI data.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title Regional Wall Motion by Echocardiography
Hide Description Movement of the left ventricular wall measured in mm from baseline to six months.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month wall motion data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 26 55
Mean (Standard Deviation)
Unit of Measure: mm
0  (0.4) 0  (0.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Difference in the change between the two groups
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.471
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.1
Confidence Interval (2-Sided) 95%
-0.30 to 0.14
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.5
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Clinical Improvement in CCS Classification (Angina Pectoris)
Hide Description Clinical improvement in Canadian Cardiovascular Society (CCS) functional classification of angina pectoris. The CCS scale ranges from Class I (best)"able to conduct ordinary daily activity without causing angina" to Class IV (worst) "Inability to perform any physical activity without discomfort; anginal symptoms may be present at rest." Patients receive a rating of 1-4 for their anginal symptoms. Results reflect the mean change in the total score over time.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month CCS data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 22 44
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.3  (0.7) -0.5  (0.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Change in average improvement in Canadian Class Score over time between the two groups.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.227
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.25
Confidence Interval (2-Sided) 95%
-0.66 to 0.16
Parameter Dispersion
Type: Standard Deviation
Value: 0.784
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Clinical Improvement in NYHA Classification
Hide Description Clinical improvement in New York Heart Association (NYHA) classification. The NYHA scale ranges from 1 (best)"Mild- no limitation of physical activity due to heart failure" to 4 (worst) "Severe-Unable to carry out any physical activity without discomfort due to heart failure". Patients receive a rating of 1-4 for their heart failure symptoms. Results reflect the mean change in the total score over time.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month NYHA class data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 30 55
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.1  (0.7) -0.3  (0.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Difference in the change in NYHA score between the two groups
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.361
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.176
Confidence Interval (2-Sided) 95%
-0.56 to 0.21
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.845
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Number of Participants With a Decrease in Anti-anginal Medication
Hide Description Number of participants with a decrease in anti-anginal medication (nitrates needed weekly)
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month anti-anginal medication data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 28 50
Measure Type: Number
Unit of Measure: participants
0 2
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Difference in the change in anti-anginal meds across groups
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.28
Comments [Not Specified]
Method Chi-squared
Comments [Not Specified]
Method of Estimation Estimation Parameter Difference in the proportion of particip
Estimated Value 0.04
Confidence Interval (2-Sided) 95%
-0.01 to 0.09
Parameter Dispersion
Type: Standard Deviation
Value: 0.045
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Exercise Time and Level
Hide Description Exercise time and level as assessed via six minute walk test. (change in number of feet walked)
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month 6 minute walk data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 29 51
Mean (Standard Deviation)
Unit of Measure: feet
80  (415) 184  (407)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Change in six minute walk distance
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.302
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 104
Confidence Interval (2-Sided) 95%
-95 to 303
Parameter Dispersion
Type: Standard Deviation
Value: 409
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Serum BNP Levels in Patients With CHF
Hide Description Serum b-type natriuretic peptide (BNP) levels in patients with congestive heart failure (CHF). A minority number of patients had pro-BNP collected versus regular BNP; these numbers are reported in the analysis population description.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month BNP data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 20 30
Mean (Standard Deviation)
Unit of Measure: IUs
BNP 63  (249) 28  (117)
pro-BNP 234  (1636) 497  (1637)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Difference in the change in BNP(reg) between cell and placebo group
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.55
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -34.7
Confidence Interval (2-Sided) 95%
-150 to 80
Parameter Dispersion
Type: Standard Deviation
Value: 176
Estimation Comments [Not Specified]
12.Secondary Outcome
Title LV Diastolic Dimension
Hide Description Left ventricular (LV) diastolic dimension as assessed by contrast echocardiography
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month LV diastolic data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 28 54
Mean (Standard Deviation)
Unit of Measure: mL
-8.5  (34) 0.9  (30)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Change in the difference of end diastolic volume between the two groups over time.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.198
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 9.5
Confidence Interval (2-Sided) 95%
-5.03 to 23.93
Parameter Dispersion
Type: Standard Deviation
Value: 31.2
Estimation Comments [Not Specified]
13.Secondary Outcome
Title Incidence of a Major Adverse Cardiac Event
Hide Description

Incidence of major adverse cardiac events (new MI, rehospitalization for PCI in coronary artery territories that were treated, death, or rehospitalization for acute coronary syndrome and for congestive heart failure).

(Incidence rate)

Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Incidence of major adverse cardiac events between baseline and 6 months. (Incidence rate)
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 31 61
Measure Type: Number
Unit of Measure: events
4 5
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments The difference in the incidence of major adverse cardiac events between the two groups over time. (Incidence rate)
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.47
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Difference in the incidence rates
Estimated Value -0.047
Confidence Interval (2-Sided) 95%
-0.133 to 0.039
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.044
Estimation Comments [Not Specified]
14.Secondary Outcome
Title Reduction in Fixed Perfusion Defect(s)Via SPECT
Hide Description Fixed total defect is the stress total defect minus the reversible component.
Time Frame Measured at Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only participants with both baseline and six month fixed defect data available are included.
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description:
Participants received placebo injections.
Participants received active stem cell injections.
Overall Number of Participants Analyzed 25 52
Mean (Standard Deviation)
Unit of Measure: percentage of defect that is fixed
1.9  (7.7) 1.2  (8.7)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Injections, Active Stem Cell Injections
Comments Difference in the change between the two groups
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.7
Confidence Interval (2-Sided) 95%
-4.78 to 3.36
Parameter Dispersion
Type: Standard Error of the mean
Value: 8.4
Estimation Comments [Not Specified]
Time Frame Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Injections Active Stem Cell Injections
Hide Arm/Group Description Participants received placebo injections. Participants received active stem cell injections.
All-Cause Mortality
Placebo Injections Active Stem Cell Injections
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Injections Active Stem Cell Injections
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/31 (19.35%)      9/61 (14.75%)    
Cardiac disorders     
Chest pain   2/31 (6.45%)  3 4/61 (6.56%)  4
Tachycardia   1/31 (3.23%)  1 3/61 (4.92%)  7
Heart Failure   3/31 (9.68%)  5 2/61 (3.28%)  5
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Injections Active Stem Cell Injections
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   11/31 (35.48%)      19/61 (31.15%)    
Blood and lymphatic system disorders     
Anemia   0/31 (0.00%)  0 3/61 (4.92%)  4
Cardiac disorders     
Tachycardia  [1]  3/31 (9.68%)  3 3/61 (4.92%)  5
Change in BNP levels   3/31 (9.68%)  3 8/61 (13.11%)  8
Gastrointestinal disorders     
Nausea   2/31 (6.45%)  4 2/61 (3.28%)  3
Respiratory, thoracic and mediastinal disorders     
Shortness of breath   3/31 (9.68%)  3 3/61 (4.92%)  3
Indicates events were collected by systematic assessment
[1]
Tachycardia not requiring hospitalization or a change to current therapy
Sample size was small and required large improvements in order to show significant treatment effects. SPECT often underestimates reversibility and viability in those with multivessel disease. Presence of cardiac devices limited MRI evaluation.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Lemuel Moye, MD, PhD
Organization: UT-Houston School of Public Health
Phone: 713-500-9518
Responsible Party: Dr Lemuel A Moye III, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT00824005     History of Changes
Other Study ID Numbers: 580
U01HL087318 ( U.S. NIH Grant/Contract )
1 U01-HL-087318-01 (Project 3)
First Submitted: January 15, 2009
First Posted: January 16, 2009
Results First Submitted: August 30, 2012
Results First Posted: April 4, 2013
Last Update Posted: July 1, 2015