Trial Outcomes & Findings for The Effect of Intramyocardial Injection of Mesenchymal Precursor Cells on Myocardial Function in Patients Undergoing LVAD Implantation (NCT NCT01442129)

Last Updated: 2015-05-01

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

90 days

Results posted on

2015-05-01

Participant Flow

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.

Participant milestones

Participant milestones
Measure	MPC Intramyocardial Injection Intramyocardial injections of 25 million MPCs Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation	Control Solution 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 Study STARTED	20	10
Overall Study COMPLETED	20	10
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	MPC Intramyocardial Injection n=20 Participants Intramyocardial injections of 25 million MPCs Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation	Control Solution n=10 Participants 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 n=30 Participants Total of all reporting groups
Age, Continuous	55.1 years STANDARD_DEVIATION 15.4 • n=5 Participants	62.2 years STANDARD_DEVIATION 7.8 • n=7 Participants	57.4 years STANDARD_DEVIATION 13.6 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	17 Participants n=5 Participants	8 Participants n=7 Participants	25 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=5 Participants	10 Participants n=7 Participants	29 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	6 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) White	14 Participants n=5 Participants	8 Participants n=7 Participants	22 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	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants	10 participants n=7 Participants	30 participants n=5 Participants
Cardiomyopathy Ischemic	7 participants n=5 Participants	4 participants n=7 Participants	11 participants n=5 Participants
Cardiomyopathy Non-Ischemic	13 participants n=5 Participants	6 participants n=7 Participants	19 participants n=5 Participants
Indication for LVAD Bridge to Transplantation	7 participants n=5 Participants	3 participants n=7 Participants	10 participants n=5 Participants
Indication for LVAD Destination Therapy	13 participants n=5 Participants	7 participants n=7 Participants	20 participants n=5 Participants

PRIMARY outcome

Timeframe: 90 days

Outcome measures

Outcome measures
Measure	MPC Intramyocardial Injection n=20 Participants Intramyocardial injections of 25 million MPCs Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation	Control Solution n=10 Participants 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.
Intervention Related Adverse Events	0 events	0 events

SECONDARY outcome

Timeframe: 90 days

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.

Outcome measures

Outcome measures
Measure	MPC Intramyocardial Injection n=20 Participants Intramyocardial injections of 25 million MPCs Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation	Control Solution n=10 Participants 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.
Functional Status and Ventricular Function	10 participants	2 participants

Adverse Events

MPC Intramyocardial Injection

Serious events: 19 serious events

Other events: 7 other events

Deaths: 0 deaths

Control Solution

Serious events: 9 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	MPC Intramyocardial Injection n=20 participants at risk Intramyocardial injections of 25 million MPCs Mesenchymal Precursor Cell Injection: Intramyocardial injection of 25 million mesenchymal precursor cells at the time of LVAD implantation	Control Solution n=10 participants at risk 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.
Gastrointestinal disorders Ileus	5.0% 1/20 • Number of events 1 • Adverse event data were collected for 12 months following randomization 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.	0.00% 0/10 • Adverse event data were collected for 12 months following randomization 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.
Cardiac disorders Sustained Supraventricular Arrhythmia	15.0% 3/20 • Number of events 3 • Adverse event data were collected for 12 months following randomization 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.	0.00% 0/10 • Adverse event data were collected for 12 months following randomization 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.
Blood and lymphatic system disorders Hemolysis	10.0% 2/20 • Number of events 2 • Adverse event data were collected for 12 months following randomization 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.	0.00% 0/10 • Adverse event data were collected for 12 months following randomization 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.
Hepatobiliary disorders Hepatic Dysfunction	5.0% 1/20 • Number of events 1 • Adverse event data were collected for 12 months following randomization 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.	10.0% 1/10 • Number of events 1 • Adverse event data were collected for 12 months following randomization 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.
Infections and infestations Localized Non-Device Infection	15.0% 3/20 • Number of events 5 • Adverse event data were collected for 12 months following randomization 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.	20.0% 2/10 • Number of events 2 • Adverse event data were collected for 12 months following randomization 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.
Renal and urinary disorders Acute Renal Dysfunction	0.00% 0/20 • Adverse event data were collected for 12 months following randomization 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.	10.0% 1/10 • Number of events 1 • Adverse event data were collected for 12 months following randomization 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.
Blood and lymphatic system disorders Venous Thromboembolism	5.0% 1/20 • Number of events 2 • Adverse event data were collected for 12 months following randomization 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.	0.00% 0/10 • Adverse event data were collected for 12 months following randomization 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.

Additional Information

Deborah D. Ascheim, MD, Associate Professor, Clinical Director of Research, InCHOIR

Mount Sinai

Phone: 212-659-9567

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place