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The Effect of Intramyocardial Injection of Mesenchymal Precursor Cells on Myocardial Function in Patients Undergoing LVAD Implantation

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ClinicalTrials.gov Identifier: NCT01442129
Recruitment Status : Completed
First Posted : September 28, 2011
Results First Posted : October 13, 2014
Last Update Posted : May 1, 2015
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Deborah Ascheim, Icahn School of Medicine at Mount Sinai

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Heart Failure
Cardiomyopathy
Ventricular Dysfunction
Interventions Biological: MPC Intramyocardial injection
Biological: Control Solution
Enrollment 30

Recruitment Details The trial was conducted in 11 U.S. centers with a Data and Clinical Coordinating Center (DCC); International Center for Health Outcomes and Innovation Research [InCHOIR], Icahn School of Medicine at Mount Sinai under an investigational new drug application. Enrollment began in May 2012, and the last patient was enrolled in August 2012.
Pre-assignment Details  
Arm/Group Title MPC Intramyocardial Injection Control Solution
Hide Arm/Group Description

Intramyocardial injections of 25 million MPCs

Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation

Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO

50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO: Injection of control solution during the LVAD implantation.

Period Title: Overall Study
Started 20 10
Completed 20 [1] 10
Not Completed 0 0
[1]
Treatment withheld from 1 patient randomized before results of screening PRA known
Arm/Group Title MPC Intramyocardial Injection Control Solution Total
Hide Arm/Group Description

Intramyocardial injections of 25 million MPCs

Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation

Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO

50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO: Injection of control solution during the LVAD implantation.

Total of all reporting groups
Overall Number of Baseline Participants 20 10 30
Hide Baseline Analysis Population Description
Patients with end-stage heart failure, either ischemic or non-ischemic etiology, who are being evaluated for LVAD implantation as a bridge-to-transplant (BTT) or destination therapy (DT)
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants 10 participants 30 participants
55.1  (15.4) 62.2  (7.8) 57.4  (13.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 10 participants 30 participants
Female
3
  15.0%
2
  20.0%
5
  16.7%
Male
17
  85.0%
8
  80.0%
25
  83.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 10 participants 30 participants
Hispanic or Latino
1
   5.0%
0
   0.0%
1
   3.3%
Not Hispanic or Latino
19
  95.0%
10
 100.0%
29
  96.7%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 10 participants 30 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
6
  30.0%
2
  20.0%
8
  26.7%
White
14
  70.0%
8
  80.0%
22
  73.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 20 participants 10 participants 30 participants
20 10 30
Cardiomyopathy  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 20 participants 10 participants 30 participants
Ischemic 7 4 11
Non-Ischemic 13 6 19
Indication for LVAD   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 20 participants 10 participants 30 participants
Bridge to Transplantation 7 3 10
Destination Therapy 13 7 20
[1]
Measure Description:

Bridge to Transplantation indicates that the LVAD is used to help patients in end stage heart failure who are awaiting a heart transplant.

Destination Therapy indicates that the LVAD is planned for long-term use for patients in end stage heart failure who are not eligible for transplant.

1.Primary Outcome
Title Intervention Related Adverse Events
Hide Description The primary safety endpoint of this study is the incidence of the following potential study-intervention related adverse events within 90 days post intervention (LVAD implantation + intramyocardial injection of study product): infectious myocarditis, myocardial rupture, neoplasm, hypersensitivity reaction, and immune sensitization.
Time Frame 90 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title MPC Intramyocardial Injection Control Solution
Hide Arm/Group Description:

Intramyocardial injections of 25 million MPCs

Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation

Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO

50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO: Injection of control solution during the LVAD implantation.

Overall Number of Participants Analyzed 20 10
Measure Type: Number
Unit of Measure: events
0 0
2.Secondary Outcome
Title Functional Status and Ventricular Function
Hide Description

The key efficacy endpoint of this study is functional status and ventricular function, while weaned from LVAD support, at 90 days post intervention (LVAD implantation + intramyocardial injection of study product). Functional status is defined by the ability to tolerate wean from LVAD support for 30 minutes without signs or symptoms of hypoperfusion, including, but not limited to symptoms of low output or signs of vascular congestion. Ventricular function will be assessed by transthoracic echocardiogram (TTE) in those patients able to be weaned for 30 minutes from LVAD support.

The number of participants who successfully tolerated the 30 minute wean from LVAD support at 90 days is reported.

Time Frame 90 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title MPC Intramyocardial Injection Control Solution
Hide Arm/Group Description:

Intramyocardial injections of 25 million MPCs

Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation

Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO

50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO: Injection of control solution during the LVAD implantation.

Overall Number of Participants Analyzed 20 10
Measure Type: Number
Unit of Measure: participants
10 2
Time Frame Adverse event data were collected for 12 months following randomization
Adverse Event Reporting Description An independent Clinical Events Committee adjudicated adverse events and causes of death. Bleeding events were defined by transfusion of ≥ 4 units of packed cells within any 24 hour period during the first 7 days post LVAD implantation, and any transfusion of packed cells within any 24 hour period thereafter.
 
Arm/Group Title MPC Intramyocardial Injection Control Solution
Hide Arm/Group Description

Intramyocardial injections of 25 million MPCs

Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation

Intramyocardial injections of 50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO

50% Alpha-MEM/42.5% ProFreeze NAO Freezing Medium/7.5% DMSO: Injection of control solution during the LVAD implantation.

All-Cause Mortality
MPC Intramyocardial Injection Control Solution
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
MPC Intramyocardial Injection Control Solution
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   19/20 (95.00%)      9/10 (90.00%)    
Blood and lymphatic system disorders     
Hemolysis  1  1/20 (5.00%)  1 2/10 (20.00%)  2
Venous Thromboembolism  1  1/20 (5.00%)  1 1/10 (10.00%)  1
Cardiac disorders     
Cardiac Arrrest  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Pericardial Fluid Collection  1  3/20 (15.00%)  3 0/10 (0.00%)  0
Right Heart Failure  1  3/20 (15.00%)  3 3/10 (30.00%)  3
Sustained Ventricular Arrhythmia  1  2/20 (10.00%)  2 2/10 (20.00%)  2
Sustained Supraventricular Arrhythmia  1  2/20 (10.00%)  2 2/10 (20.00%)  2
General disorders     
Major Bleeding  1  8/20 (40.00%)  71 5/10 (50.00%)  30
Baker's Cyst  1  0/20 (0.00%)  0 1/10 (10.00%)  1
Elevated WBC, LDH  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Shortness of Breath  1  0/20 (0.00%)  0 1/10 (10.00%)  1
Rib Pain  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Sub-therapeutic Anticoagulation  1  3/20 (15.00%)  4 0/10 (0.00%)  0
Hepatobiliary disorders     
Hepatic Dysfunction  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Infections and infestations     
Major Infection - Localized Non-Device Infection  1  4/20 (20.00%)  4 2/10 (20.00%)  2
Major Infection - Internal Pump Component Inflow or Outflow Tract Infection  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Sepsis  1  3/20 (15.00%)  3 1/10 (10.00%)  1
Major Infection  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Injury, poisoning and procedural complications     
Device Malfunction - Pump Failure  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Intraoperative Bleeding  1  2/20 (10.00%)  2 1/10 (10.00%)  1
Device Malfunction - Non Pump Failure  1  3/20 (15.00%)  3 0/10 (0.00%)  0
Device Malfunction - Pump Thrombus Suspected  1  1/20 (5.00%)  1 1/10 (10.00%)  1
Device Malfunction - Pump Thrombus Confirmed  1  3/20 (15.00%)  4 1/10 (10.00%)  1
Nervous system disorders     
Neurological Dysfunction - Other  1  1/20 (5.00%)  1 0/10 (0.00%)  0
TIA  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Ischemic Stroke  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Hemorrhagic Stroke  1  0/20 (0.00%)  0 1/10 (10.00%)  1
Toxic Metabolic Encephalopathy  1  2/20 (10.00%)  2 0/10 (0.00%)  0
Renal and urinary disorders     
Acute Renal Dysfunction  1  4/20 (20.00%)  4 1/10 (10.00%)  1
Chronic Renal Dysfunction  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Respiratory Failure  1  5/20 (25.00%)  5 2/10 (20.00%)  2
Pleural Effusion  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, INTERMACS
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
MPC Intramyocardial Injection Control Solution
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/20 (35.00%)      4/10 (40.00%)    
Blood and lymphatic system disorders     
Hemolysis  1  2/20 (10.00%)  2 0/10 (0.00%)  0
Venous Thromboembolism  1  1/20 (5.00%)  2 0/10 (0.00%)  0
Cardiac disorders     
Sustained Supraventricular Arrhythmia  1  3/20 (15.00%)  3 0/10 (0.00%)  0
Gastrointestinal disorders     
Ileus  1  1/20 (5.00%)  1 0/10 (0.00%)  0
Hepatobiliary disorders     
Hepatic Dysfunction  1  1/20 (5.00%)  1 1/10 (10.00%)  1
Infections and infestations     
Localized Non-Device Infection  1  3/20 (15.00%)  5 2/10 (20.00%)  2
Renal and urinary disorders     
Acute Renal Dysfunction  1  0/20 (0.00%)  0 1/10 (10.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, INTERMACS
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Deborah D. Ascheim, MD, Associate Professor, Clinical Director of Research, InCHOIR
Organization: Mount Sinai
Phone: 212-659-9567
Responsible Party: Deborah Ascheim, Icahn School of Medicine at Mount Sinai
ClinicalTrials.gov Identifier: NCT01442129     History of Changes
Other Study ID Numbers: GCO 08-1078-00006
U01HL088942 ( U.S. NIH Grant/Contract )
U01HL088942-04 ( U.S. NIH Grant/Contract )
711 ( Other Identifier: Ct Surgery Network Research Group )
First Submitted: September 26, 2011
First Posted: September 28, 2011
Results First Submitted: October 7, 2014
Results First Posted: October 13, 2014
Last Update Posted: May 1, 2015