Trial Outcomes & Findings for Stem Cell Therapy in Patients With Severe Heart Failure & Undergoing Left Ventricular Assist Device Placement (NCT NCT00869024)
NCT ID: NCT00869024
Last Updated: 2020-03-13
Results Overview
Safety as measured by the total number of adverse events per group.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
25 participants
Primary outcome timeframe
24 months
Results posted on
2020-03-13
Participant Flow
Participant milestones
| Measure |
Stem Cell Therapy
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
Overall Study
STARTED
|
16
|
9
|
|
Overall Study
COMPLETED
|
14
|
4
|
|
Overall Study
NOT COMPLETED
|
2
|
5
|
Reasons for withdrawal
| Measure |
Stem Cell Therapy
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
Overall Study
Death
|
2
|
3
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Protocol Violation
|
0
|
1
|
Baseline Characteristics
Stem Cell Therapy in Patients With Severe Heart Failure & Undergoing Left Ventricular Assist Device Placement
Baseline characteristics by cohort
| Measure |
Stem Cell Therapy
n=16 Participants
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
n=9 Participants
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Age, Continuous
|
62.3 years
STANDARD_DEVIATION 8.68 • n=5 Participants
|
65.1 years
STANDARD_DEVIATION 8.6 • n=7 Participants
|
63.3 years
STANDARD_DEVIATION 8.58 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=5 Participants
|
9 participants
n=7 Participants
|
25 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 monthsSafety as measured by the total number of adverse events per group.
Outcome measures
| Measure |
Stem Cell Therapy
n=16 Participants
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
n=4 Participants
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
Safety of Cell Delivery
|
1 adverse events
|
0 adverse events
|
PRIMARY outcome
Timeframe: baseline, 10 weeksChange in LAD segments from baseline to 10 weeks. PET scan viability is reported by segment on a scale of 0-4. A score of 0, 1, or 2 are categorized as viable/healthy heart tissue and a score of 3 or 4 are categorized as not viable/scar tissue. No change or better in viability will be reported to determine safety of cell injection. Measurement is reported as number of segment that remained the same or improved were considered "safe" for stem cell injection.
Outcome measures
| Measure |
Stem Cell Therapy
n=15 Participants
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
n=4 Participants
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
Improvement in Myocardial Viability by PET/CT Scan
Anterior wall base segment
|
14 safe segments
|
4 safe segments
|
|
Improvement in Myocardial Viability by PET/CT Scan
Anterior wall mid segment
|
14 safe segments
|
4 safe segments
|
|
Improvement in Myocardial Viability by PET/CT Scan
Anterior lateral wall base segment
|
15 safe segments
|
4 safe segments
|
|
Improvement in Myocardial Viability by PET/CT Scan
Anterior lateral wall mid segment
|
15 safe segments
|
4 safe segments
|
PRIMARY outcome
Timeframe: 24 monthsNumber of participants who expired during the study.
Outcome measures
| Measure |
Stem Cell Therapy
n=16 Participants
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
n=4 Participants
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
Combined End Points of Death
|
2 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 10 weeksLVAD turn-down was completed at 10 weeks. Hemodynamic measurements were taken and reported with nominal LVAD support and then again at peak exercise. LVAD turn-down protocol was followed to ensure safety of patient while turning down their LVAD support. After each turn-down we waited 10 minutes and repeated ECHO, RHC, LVAD parameters, vital signs. Stopping parameters for turn down: 1. Significant symptoms (clinician judgement, although low threshold to stop test) 2. CVP\>20 or increase by more than 10 (e.g., 5 to 16) 3. PCWP\>25 or increase by more than 10 (e.g., 11 to 22) 4. Hypotension 5. Increase in LVIDd by \>1.5 cm 6. Aortic valve opening minimally (less than 1 in 5 beats, e.g.) The number of patients that could be turn-down without meeting stopping rules and were able to exercise were reported per group.
Outcome measures
| Measure |
Stem Cell Therapy
n=12 Participants
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
n=5 Participants
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
Number of Participants Turned Down Without Meeting LVAD Stopping Rules
|
12 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 10 weeksChange in left ventricular dimensions assessed by ECHO at baseline compared to 10 weeks with LVAD turn-down to 6000 RPMs.
Outcome measures
| Measure |
Stem Cell Therapy
n=14 Participants
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
n=6 Participants
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
Change in Left Ventricular Dimensions
Change from baseline to 10 weeks in LVEDD
|
-0.49 cm
Standard Deviation 1.004
|
-0.57 cm
Standard Deviation 0.76
|
|
Change in Left Ventricular Dimensions
Change from baseline to 10 weeks in LVESD
|
-0.73 cm
Standard Deviation 1.07
|
-0.62 cm
Standard Deviation 0.89
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Data collection was insufficient for data analysis. Samples were collected but no histological analysis performed.
Data collection was insufficient for data analysis. Samples were collected but no histological analysis performed.
Outcome measures
Outcome data not reported
Adverse Events
Stem Cell Therapy
Serious events: 13 serious events
Other events: 0 other events
Deaths: 2 deaths
Placebo
Serious events: 7 serious events
Other events: 0 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Stem Cell Therapy
n=16 participants at risk
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells in Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
Placebo
n=9 participants at risk
Intramyocardial Delivery Placebo solution into Patients with Severe LV Dysfunction and LVAD Support
Intramyocardial Delivery of Bone Marrow Derived Mononuclear Cells: Ten separate injections will be delivered into the LV free wall (20 X 106 cells / 400 micro lit).
|
|---|---|---|
|
General disorders
Fever
|
6.2%
1/16 • Number of events 2 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
11.1%
1/9 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Cardiac disorders
Transient ischemic attack
|
0.00%
0/16 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
11.1%
1/9 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Cardiac disorders
Cerebrovascular accident
|
12.5%
2/16 • Number of events 2 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
0.00%
0/9 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Blood and lymphatic system disorders
Increased bleeding
|
43.8%
7/16 • Number of events 11 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
11.1%
1/9 • Number of events 2 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Cardiac disorders
Syncope
|
6.2%
1/16 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
0.00%
0/9 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Cardiac disorders
Palpitations
|
0.00%
0/16 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
11.1%
1/9 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Cardiac disorders
Increased frequency of Atrial arrhythmias
|
12.5%
2/16 • Number of events 2 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
11.1%
1/9 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Cardiac disorders
Increased frequency of Ventricular arrhythmias
|
18.8%
3/16 • Number of events 5 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
33.3%
3/9 • Number of events 3 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
General disorders
Sepsis
|
6.2%
1/16 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
11.1%
1/9 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Blood and lymphatic system disorders
Endocarditis
|
6.2%
1/16 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
0.00%
0/9 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Infections and infestations
Any infection
|
37.5%
6/16 • Number of events 8 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
33.3%
3/9 • Number of events 3 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Hepatobiliary disorders
New Liver Failure
|
0.00%
0/16 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
22.2%
2/9 • Number of events 2 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Renal and urinary disorders
New Renal Failure
|
0.00%
0/16 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
22.2%
2/9 • Number of events 2 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Any Malignancies
|
6.2%
1/16 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
0.00%
0/9 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
|
General disorders
Shock
|
6.2%
1/16 • Number of events 1 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
22.2%
2/9 • Number of events 2 • 24 months
Adverse events are classified as serious or non-serious. A serious adverse event is any AE that is: fatal life-threatening requires or prolongs hospital stay results in persistent or significant disability or incapacity An important medical event
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place