Trial Outcomes & Findings for Effectiveness of Stem Cell Treatment for Adults With Ischemic Cardiomyopathy (The FOCUS Study) (NCT NCT00824005)

NCT ID: NCT00824005

Last Updated: 2015-07-01

Results Overview

The VO2(max) is assessed using the Naughton treadmill protocol.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 92 participants
• Primary outcome timeframe: Measured at Baseline and Month 6
• Results posted on: 2015-07-01

Participant Flow

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.

Participant milestones

Measure	Placebo Injections Participants will receive placebo injections.	Active Stem Cell Injections Participants will receive active stem cell injections.
Overall Study STARTED	31	61
Overall Study COMPLETED	31	59
Overall Study NOT COMPLETED	0	2

Reasons for withdrawal

Measure	Placebo Injections Participants will receive placebo injections.	Active Stem Cell Injections Participants will receive active stem cell injections.
Overall Study Death	0	1
Overall Study Lost to Follow-up	0	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Placebo Injections n=31 Participants Participants will receive placebo injections.	Active Stem Cell Injections n=61 Participants Participants will receive active stem cell injections.	Total n=92 Participants Total of all reporting groups
Age, Continuous	62.32 years STANDARD_DEVIATION 8.25 • n=5 Participants	63.95 years STANDARD_DEVIATION 10.90 • n=7 Participants	63.4 years STANDARD_DEVIATION 10.1 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	8 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants	53 Participants n=7 Participants	82 Participants n=5 Participants
Region of Enrollment United States	31 participants n=5 Participants	61 participants n=7 Participants	92 participants n=5 Participants

PRIMARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and 6 month VO2max data available are included.

The VO2(max) is assessed using the Naughton treadmill protocol.

Outcome measures

Measure	Placebo Injections n=27 Participants Participants received placebo injections.	Active Stem Cell Injections n=52 Participants Participants received active stem cell injections.
Change in Maximal Oxygen Consumption (VO2max)	-0.6 mL/kg/min Standard Deviation 3.2	0.4 mL/kg/min Standard Deviation 2.8

PRIMARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month LVESV data available are included.

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.

Outcome measures

Measure	Placebo Injections n=28 Participants Participants received placebo injections.	Active Stem Cell Injections n=54 Participants Participants received active stem cell injections.
Change in Left Ventricular End Systolic Volume (LVESV)as Assessed Via Echo	0 mL/m2 Standard Deviation 10.8	-0.9 mL/m2 Standard Deviation 11.6

PRIMARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month reversible defect data available are included.

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.

Outcome measures

Measure	Placebo Injections n=26 Participants Participants received placebo injections.	Active Stem Cell Injections n=50 Participants Participants received active stem cell injections.
Change in Reversible Defect Size	-2.7 percentage of reversible defect Standard Deviation 18.2	-3.9 percentage of reversible defect Standard Deviation 25.4

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: The small number of patients without contraindications for MRI (n=17) precluded performing informative analysis on the MRI data.

Regional wall motion as measured by cardiac MRI (in patients who are not contraindicated)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: The small number of patients without contraindications for MRI (n=17) precluded performing informative analysis on the MRI data.

Regional blood flow improvement as measured by cardiac MRI (in patients who are not contraindicated)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month wall motion data available are included.

Movement of the left ventricular wall measured in mm from baseline to six months.

Outcome measures

Measure	Placebo Injections n=26 Participants Participants received placebo injections.	Active Stem Cell Injections n=55 Participants Participants received active stem cell injections.
Regional Wall Motion by Echocardiography	0 mm Standard Deviation 0.4	0 mm Standard Deviation 0.5

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month CCS data available are included.

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.

Outcome measures

Measure	Placebo Injections n=22 Participants Participants received placebo injections.	Active Stem Cell Injections n=44 Participants Participants received active stem cell injections.
Clinical Improvement in CCS Classification (Angina Pectoris)	-0.3 units on a scale Standard Deviation 0.7	-0.5 units on a scale Standard Deviation 0.8

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month NYHA class data available are included.

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.

Outcome measures

Measure	Placebo Injections n=30 Participants Participants received placebo injections.	Active Stem Cell Injections n=55 Participants Participants received active stem cell injections.
Clinical Improvement in NYHA Classification	-0.1 units on a scale Standard Deviation 0.7	-0.3 units on a scale Standard Deviation 0.9

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month anti-anginal medication data available are included.

Number of participants with a decrease in anti-anginal medication (nitrates needed weekly)

Outcome measures

Measure	Placebo Injections n=28 Participants Participants received placebo injections.	Active Stem Cell Injections n=50 Participants Participants received active stem cell injections.
Number of Participants With a Decrease in Anti-anginal Medication	0 participants	2 participants

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month 6 minute walk data available are included.

Exercise time and level as assessed via six minute walk test. (change in number of feet walked)

Outcome measures

Measure	Placebo Injections n=29 Participants Participants received placebo injections.	Active Stem Cell Injections n=51 Participants Participants received active stem cell injections.
Exercise Time and Level	80 feet Standard Deviation 415	184 feet Standard Deviation 407

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month BNP data available are included.

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.

Outcome measures

Measure	Placebo Injections n=20 Participants Participants received placebo injections.	Active Stem Cell Injections n=30 Participants Participants received active stem cell injections.
Serum BNP Levels in Patients With CHF BNP	63 IUs Standard Deviation 249	28 IUs Standard Deviation 117
Serum BNP Levels in Patients With CHF pro-BNP	234 IUs Standard Deviation 1636	497 IUs Standard Deviation 1637

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month LV diastolic data available are included.

Left ventricular (LV) diastolic dimension as assessed by contrast echocardiography

Outcome measures

Measure	Placebo Injections n=28 Participants Participants received placebo injections.	Active Stem Cell Injections n=54 Participants Participants received active stem cell injections.
LV Diastolic Dimension	-8.5 mL Standard Deviation 34	0.9 mL Standard Deviation 30

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Incidence of major adverse cardiac events between baseline and 6 months. (Incidence rate)

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)

Outcome measures

Measure	Placebo Injections n=31 Participants Participants received placebo injections.	Active Stem Cell Injections n=61 Participants Participants received active stem cell injections.
Incidence of a Major Adverse Cardiac Event	4 events	5 events

SECONDARY outcome

Timeframe: Measured at Baseline and Month 6

Population: Only participants with both baseline and six month fixed defect data available are included.

Fixed total defect is the stress total defect minus the reversible component.

Outcome measures

Measure	Placebo Injections n=25 Participants Participants received placebo injections.	Active Stem Cell Injections n=52 Participants Participants received active stem cell injections.
Reduction in Fixed Perfusion Defect(s)Via SPECT	1.9 percentage of defect that is fixed Standard Deviation 7.7	1.2 percentage of defect that is fixed Standard Deviation 8.7

Adverse Events

Placebo Injections

Serious events: 6 serious events

Other events: 11 other events

Deaths: 0 deaths

Active Stem Cell Injections

Serious events: 9 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo Injections n=31 participants at risk Participants received placebo injections.	Active Stem Cell Injections n=61 participants at risk Participants received active stem cell injections.
Cardiac disorders Chest pain	6.5% 2/31 • Number of events 3 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	6.6% 4/61 • Number of events 4 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)
Cardiac disorders Tachycardia	3.2% 1/31 • Number of events 1 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	4.9% 3/61 • Number of events 7 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)
Cardiac disorders Heart Failure	9.7% 3/31 • Number of events 5 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	3.3% 2/61 • Number of events 5 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)

Other adverse events

Measure	Placebo Injections n=31 participants at risk Participants received placebo injections.	Active Stem Cell Injections n=61 participants at risk Participants received active stem cell injections.
Cardiac disorders Tachycardia	9.7% 3/31 • Number of events 3 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	4.9% 3/61 • Number of events 5 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)
Gastrointestinal disorders Nausea	6.5% 2/31 • Number of events 4 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	3.3% 2/61 • Number of events 3 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)
Blood and lymphatic system disorders Anemia	0.00% 0/31 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	4.9% 3/61 • Number of events 4 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)
Cardiac disorders Change in BNP levels	9.7% 3/31 • Number of events 3 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	13.1% 8/61 • Number of events 8 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)
Respiratory, thoracic and mediastinal disorders Shortness of breath	9.7% 3/31 • Number of events 3 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)	4.9% 3/61 • Number of events 3 • Events reported are from Randomization Date to the 6 month endpoint data collection window (i.e. 210 days post intervention)

Additional Information

Lemuel Moye, MD, PhD

UT-Houston School of Public Health

Phone: 713-500-9518

Email: Lemmoye@msn.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place