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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

159 participants

Primary outcome timeframe

up to 6 months

Results posted on

2019-11-26

Participant Flow

Screening started in 2014. First patient randomized in July 2015. Last patient randomized in August 2017.

Participant milestones

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

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

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 months

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.

Outcome measures

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 months

Safety as assessed by number of study intervention-related adverse events

Outcome measures

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 months

Echocardiographic 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 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months and 12 months

Quality 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 months

Cognitive 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 months

Cognitive 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 months

Cognitive 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 months

Cognitive 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 months

Cognitive 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 months

Cognitive 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 months

Length of stay of index hospitalization

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

Frequency and cause of readmissions

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

Hospital resource use

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

functional status, defined by the number of temporary weans from LVAD support tolerated

Outcome measures

Outcome data not reported

Adverse Events

MPC Intramyocardial Injection

Serious events: 88 serious events
Other events: 33 other events
Deaths: 15 deaths

Control Solution

Serious events: 41 serious events
Other events: 12 other events
Deaths: 8 deaths

Serious adverse events

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

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

Phone: 212-659-9567

Results disclosure agreements

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