Trial Outcomes & Findings for Effect of Intramyocardial Injection of Mesenchymal Precursor Cells on Heart Function in People Receiving an LVAD (NCT NCT00927784)

NCT ID: NCT00927784

Last Updated: 2019-03-05

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: Measured within 90 days of study entry
• Results posted on: 2019-03-05

Participant Flow

80 patients with end-stage CHF and clinical indication for implantation with an FDA-approved LVAD as a bridge-to-transplant were to be enrolled. 40 patients in the low dose cohort(25M)/40 patients in the high dose cohort(75M). Trial enrollment was permanently suspended upon 10 patients enrolled in the low dose cohort due to a change in NIH funding.

Participant milestones

Measure	Low Dose Cohort: Treatment Group Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Overall Study STARTED	8	2
Overall Study COMPLETED	6	2
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	Low Dose Cohort: Treatment Group Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Overall Study Death	1	0
Overall Study Withdrawal by Subject	1	0

Baseline Characteristics

Effect of Intramyocardial Injection of Mesenchymal Precursor Cells on Heart Function in People Receiving an LVAD

Baseline characteristics by cohort

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.	Total n=10 Participants Total of all reporting groups
Age, Continuous	56 years STANDARD_DEVIATION 14 • n=5 Participants	51 years STANDARD_DEVIATION 5 • n=7 Participants	55 years STANDARD_DEVIATION 12 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	2 Participants n=7 Participants	8 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	7 Participants n=5 Participants	2 Participants n=7 Participants	9 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	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants	2 Participants n=7 Participants	6 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	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants	2 participants n=7 Participants	10 participants n=5 Participants

PRIMARY outcome

Timeframe: Measured within 90 days of study entry

Outcome measures

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Incidence of Infectious Myocarditis	0 participants	0 participants

PRIMARY outcome

Timeframe: Measured within 90 days of study entry

Outcome measures

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Incidence of Myocardial Rupture	0 participants	0 participants

PRIMARY outcome

Timeframe: Measured within 90 days of study entry

Outcome measures

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Incidence of Neoplasm	0 participants	0 participants

PRIMARY outcome

Timeframe: Measured within 90 days of study entry

Outcome measures

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Incidence of Hypersensitivity Reaction	0 participants	0 participants

PRIMARY outcome

Timeframe: Measured within 90 days of study entry

Outcome measures

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Incidence of Immune Sensitization	0 participants	0 participants

SECONDARY outcome

Timeframe: up to 12 months

Population: data not collected

The secondary endpoints assessed during the LVAD wean include echocardiographic assessments, 6 minute walk, ability to tolerate wean from LVAD support, duration of ability to tolerate wean from LVAD support, and neuronal function.

Measured at 60 days, 90 days, and every 60 days thereafter following LVAD implantation until heart transplantation or 12 months, whichever comes first

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

This includes Device Malfunction-Pump Thrombus-Suspected and Internal Pump Component, Inflow, or Outflow Tract Infection

Outcome measures

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Incidence of Study Intervention-related Adverse Events	2 participants	0 participants

SECONDARY outcome

Timeframe: up to 12 months

Outcome measures

Measure	Low Dose Cohort: Treatment Group n=8 Participants Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 Participants Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Incidence of All Serious Adverse Events	27 events	1 events

SECONDARY outcome

Timeframe: up to 12 months

Population: Study terminated early. Data not collected or analyzed for this Outcome Measure.

Number of patients who experienced donor-specific HLA sensitization post-randomization in each treatment arm.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

Population: Study terminated early. Data not collected or analyzed for this Outcome Measure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

Population: Study terminated early. Data not collected or analyzed for this Outcome Measure.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

Population: Study terminated early. Data not collected or analyzed for this Outcome Measure.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 months

Population: Study terminated early. Data not collected or analyzed for this Outcome Measure.

Outcome measures

Outcome data not reported

Adverse Events

Low Dose Cohort: Treatment Group

Serious events: 8 serious events

Other events: 0 other events

Deaths: 0 deaths

Low Dose Cohort: Control Group

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Low Dose Cohort: Treatment Group n=8 participants at risk Intramyocardial injection of study drug (25 million MPC) (Revasacor TM)at the time of LVAD implantation	Low Dose Cohort: Control Group n=2 participants at risk Intramyocardial injection of vehicle (50% alpha-MEM/42.5% proFreeze NAO Freezing Medium/7.5%DMSO) at the time of LVAD implantation.
Blood and lymphatic system disorders Intra-operative Bleeding	37.5% 3/8 • Number of events 3 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Blood and lymphatic system disorders Major Bleeding (Non-Intra-Operative)	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Blood and lymphatic system disorders Venous Thromboembolism	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Blood and lymphatic system disorders Other Bleeding	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Cardiac disorders Cardiac Arrhythmias	87.5% 7/8 • Number of events 9 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Cardiac disorders Other (Volume Overload)	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Cardiac disorders Other (decompensated heart failure)	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Cardiac disorders Right Heart Failure	25.0% 2/8 • Number of events 2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Vascular disorders Other (vasodilatory shock periop)	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Blood and lymphatic system disorders Device Malfunction-Pump Thrombus-Suspected	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Infections and infestations Major Infection - Percutaneous Site Infection	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Infections and infestations Major Infection - Internal Pump Component, Inflow, or Outflow Tract Infection	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Nervous system disorders Neurological Dysfunction - Ischemic Stroke	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Nervous system disorders Neurological Dysfunction - TIA	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Nervous system disorders Neurological Dysfunction - Other	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Renal and urinary disorders Renal Dysfunction - Acute	12.5% 1/8 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	0.00% 0/2 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).
Endocrine disorders Other (New diagnosis of Type II diabetes)	0.00% 0/8 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).	50.0% 1/2 • Number of events 1 • Continuous from randomization until induction of anesthesia for cardiac transplant or 12 months, whichever comes first. Adverse events were documented and reported by staff at the study sites as they occurred per the protocol definitions. All adverse events were adjudicated by an Independent Clinical Events Committee (CEC).

Other adverse events

Adverse event data not reported

Additional Information

Deborah Ascheim, MD, Principal Investigator, Data Coordinating Center

Mount Sinai School of Medicine

Phone: 212-659-9567

Email: deborah.ascheim@mssm.edu

Results disclosure agreements

• Principal investigator is a sponsor employee The site investigators have the right to independently publish results of their portion of the study conducted at their site 1 year following study conclusion; the site investigator must submit to the DCC for review and comment,a copy of any proposed manuscript resulting from research at least thirty (30) days prior to estimated publication date. If no response is received within thirty (30) days of the date the manuscript was submitted to the DCC, publication may proceed without delay.
• Publication restrictions are in place

Restriction type: OTHER