Trial Outcomes & Findings for Safety & Efficacy of Intramyocardial Injection of Mesenchymal Precursor Cells on Myocardial Function in LVAD Recipients (NCT NCT02362646)
NCT ID: NCT02362646
Last Updated: 2019-11-26
Results Overview
functional status, defined by the number of temporary weans from LVAD support tolerated over the 6 months post-randomization. A successful wean is the ability to tolerate temporary weaning from LVAD support for 30 minutes without sustained symptoms of worsening heart failure. Wean failures are defined as inability to tolerate the temporary wean for 30 minutes; death; or patient too unstable, in the judgment of the primary heart failure cardiologist, to tolerate the wean attempt.
COMPLETED
PHASE2
159 participants
up to 6 months
2019-11-26
Participant Flow
Screening started in 2014. First patient randomized in July 2015. Last patient randomized in August 2017.
Participant milestones
| Measure |
MPC Intramyocardial Injection
MPC Intramyocardial Injection: Intramyocardial injection of 150 million mesenchymal precursor cells (MPCs) at the time of LVAD implantation
|
Control Solution
Control Solution: Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO
|
|---|---|---|
|
Overall Study
STARTED
|
106
|
53
|
|
Overall Study
COMPLETED
|
68
|
29
|
|
Overall Study
NOT COMPLETED
|
38
|
24
|
Reasons for withdrawal
| Measure |
MPC Intramyocardial Injection
MPC Intramyocardial Injection: Intramyocardial injection of 150 million mesenchymal precursor cells (MPCs) at the time of LVAD implantation
|
Control Solution
Control Solution: Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO
|
|---|---|---|
|
Overall Study
Death
|
15
|
8
|
|
Overall Study
Withdrawal by Subject
|
2
|
2
|
|
Overall Study
received transplant
|
21
|
14
|
Baseline Characteristics
Safety & Efficacy of Intramyocardial Injection of Mesenchymal Precursor Cells on Myocardial Function in LVAD Recipients
Baseline characteristics by cohort
| Measure |
MPC Intramyocardial Injection
n=106 Participants
MPC Intramyocardial Injection: Intramyocardial injection of 150 million mesenchymal precursor cells (MPCs) at the time of LVAD implantation
|
Control Solution
n=53 Participants
Control Solution: Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO
|
Total
n=159 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
55.5 years
STANDARD_DEVIATION 12.3 • n=5 Participants
|
56.9 years
STANDARD_DEVIATION 11.7 • n=7 Participants
|
56 years
STANDARD_DEVIATION 12.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
94 Participants
n=5 Participants
|
47 Participants
n=7 Participants
|
141 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
100 Participants
n=5 Participants
|
52 Participants
n=7 Participants
|
152 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
16 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
82 Participants
n=5 Participants
|
40 Participants
n=7 Participants
|
122 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to 6 monthsfunctional status, defined by the number of temporary weans from LVAD support tolerated over the 6 months post-randomization. A successful wean is the ability to tolerate temporary weaning from LVAD support for 30 minutes without sustained symptoms of worsening heart failure. Wean failures are defined as inability to tolerate the temporary wean for 30 minutes; death; or patient too unstable, in the judgment of the primary heart failure cardiologist, to tolerate the wean attempt.
Outcome measures
| Measure |
Control Solution
n=53 Participants
Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO
Control Solution
|
MPC Intramyocardial Injection
n=106 Participants
MPC Intramyocardial Injection: Intramyocardial injection of 150 million mesenchymal precursor cells (MPCs) at the time of LVAD implantation
|
|---|---|---|
|
Number of Temporary Weans From LVAD Support Tolerated
|
0.58 number of weans
Standard Deviation 0.45
|
0.61 number of weans
Standard Deviation 0.41
|
PRIMARY outcome
Timeframe: up to 6 monthsSafety as assessed by number of study intervention-related adverse events
Outcome measures
| Measure |
Control Solution
n=53 Participants
Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO
Control Solution
|
MPC Intramyocardial Injection
n=106 Participants
MPC Intramyocardial Injection: Intramyocardial injection of 150 million mesenchymal precursor cells (MPCs) at the time of LVAD implantation
|
|---|---|---|
|
Number of Participants With Adverse Events
Other than Serious AE
|
12 Participants
|
33 Participants
|
|
Number of Participants With Adverse Events
Serious AE
|
44 Participants
|
88 Participants
|
SECONDARY outcome
Timeframe: up to 12 monthsEchocardiographic assessments of the myocardial size and function by transthoracic echocardiography with LVAD at full support, and as tolerated following 6-Minute Walk Test (MWT) while weaned from LVAD support (for patients who tolerate wean from LVAD support for 30 minutes)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 12 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 12 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 months and 12 monthsQuality of life will be assessed with the Kansas City Cardiomyopathy Questionnaire (KCCQ), a widely used tool in heart failure populations, and the Short Form 12 (SF12), a widely used overall health status measure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months and 12 monthsCognitive performance will be assessed Hopkins Verbal Learning Test. Neurocognitive testing will be administered by clinical site personnel who have been trained and certified for test administration by the Neurocognitive Core lab personnel.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months and 12 monthsCognitive performance will be assessed using Trailmaking Tests A and B. Neurocognitive testing will be administered by clinical site personnel who have been trained and certified for test administration by the Neurocognitive Core lab personnel.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months and 12 monthsCognitive performance will be assessed using the MCG Complex Figures. Neurocognitive testing will be administered by clinical site personnel who have been trained and certified for test administration by the Neurocognitive Core lab personnel.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months and 12 monthsCognitive performance will be assessed using the MCG Complex Figures. Neurocognitive testing will be administered by clinical site personnel who have been trained and certified for test administration by the Neurocognitive Core lab personnel.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months and 12 monthsCognitive performance will be assessed using the Digit Symbol Substitution Test. Neurocognitive testing will be administered by clinical site personnel who have been trained and certified for test administration by the Neurocognitive Core lab personnel.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months and 12 monthsCognitive performance will be assessed using the Controlled Oral Word Association. Neurocognitive testing will be administered by clinical site personnel who have been trained and certified for test administration by the Neurocognitive Core lab personnel.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 12 monthsLength of stay of index hospitalization
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 12 monthsFrequency and cause of readmissions
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 12 monthsHospital resource use
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 12 monthsfunctional status, defined by the number of temporary weans from LVAD support tolerated
Outcome measures
Outcome data not reported
Adverse Events
MPC Intramyocardial Injection
Control Solution
Serious adverse events
| Measure |
MPC Intramyocardial Injection
n=106 participants at risk
Intramyocardial injections of 150 million MPCs
MPC Intramyocardial Injection: Intramyocardial injection of 150 million mesenchymal precursor cells at the time of LVAD implantation
|
Control Solution
n=53 participants at risk
Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO
Control Solution
|
|---|---|---|
|
General disorders
Bleeding
|
48.1%
51/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
52.8%
28/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Cardiac disorders
Sustained ventricular dysrhythmia requiring defibrillation or cardioversion
|
24.5%
26/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
15.1%
8/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Cardiac disorders
Sustained supraventricular dysrhythmia requiring drug treatment or cardioversion
|
11.3%
12/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
17.0%
9/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Injury, poisoning and procedural complications
Pump thrombus confirmed
|
9.4%
10/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Injury, poisoning and procedural complications
Pump thrombus suspected
|
8.5%
9/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Injury, poisoning and procedural complications
Nonpump thrombus related
|
0.94%
1/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Injury, poisoning and procedural complications
Minor device malfunction
|
1.9%
2/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Blood and lymphatic system disorders
Hemolysis
|
3.8%
4/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
3.8%
2/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Infections and infestations
Localized nondevice infection
|
15.1%
16/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
15.1%
8/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Infections and infestations
Sepsis
|
13.2%
14/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
11.3%
6/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Infections and infestations
Percutaneous site and/or pocket infection
|
5.7%
6/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
9.4%
5/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Nervous system disorders
Toxic metabolic encephalopathy
|
4.7%
5/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
7.5%
4/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Nervous system disorders
Ischemic stroke
|
5.7%
6/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
1.9%
1/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Nervous system disorders
Intracranial hemorrhage
|
5.7%
6/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Nervous system disorders
Transient ischemic attack
|
2.8%
3/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
1.9%
1/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Nervous system disorders
Other neurological dysfunction
|
4.7%
5/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Renal and urinary disorders
Renal dysfunction
|
9.4%
10/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
9.4%
5/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Cardiac disorders
Right heart failure
|
17.9%
19/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
18.9%
10/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
General disorders
Potential Inflammatory Responses
|
0.94%
1/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Cardiac disorders
Pericardial Fluid Collection
|
7.5%
8/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Cardiac disorders
Vasodilatory State
|
2.8%
3/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Hepatobiliary disorders
Hepatic Dysfunction
|
0.94%
1/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
1.9%
1/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Vascular disorders
Hypertension
|
3.8%
4/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
1.9%
1/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Cardiac disorders
MI (Non-perioperative)
|
0.94%
1/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Psychiatric disorders
Psychiatric Episode
|
0.94%
1/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
15.1%
16/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
18.9%
10/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Vascular disorders
Arterial Non-CNS Thromboembolism
|
0.94%
1/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
0.00%
0/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Blood and lymphatic system disorders
Venous Thromboembolism Event
|
1.9%
2/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
1.9%
1/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Injury, poisoning and procedural complications
Wound Dehiscence
|
0.94%
1/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
1.9%
1/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
5.7%
6/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Cardiac disorders
Sustained SVT dysrhytmia
|
13.2%
14/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
Other adverse events
| Measure |
MPC Intramyocardial Injection
n=106 participants at risk
Intramyocardial injections of 150 million MPCs
MPC Intramyocardial Injection: Intramyocardial injection of 150 million mesenchymal precursor cells at the time of LVAD implantation
|
Control Solution
n=53 participants at risk
Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO
Control Solution
|
|---|---|---|
|
Cardiac disorders
Sustained supraventricular dysrhythmia
|
13.2%
14/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Infections and infestations
Localized nondevice infection
|
15.1%
16/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
15.1%
8/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
5.7%
6/106 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
5.7%
3/53 • 6 months for adverse events 12 months for mortality
Trial specific protocol-defined adverse event
|
Additional Information
Annetine Gelijns, PhD
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place