Trial Outcomes & Findings for A Study to Assess the Effect of Intravenous Dose of (aMBMC) to Subjects With Non-ischemic Heart Failure (NCT NCT02467387)
NCT ID: NCT02467387
Last Updated: 2020-01-07
Results Overview
As identified in the SAP, the safety analysis was the primary objective and was evaluated by the number of AEs
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
23 participants
Primary outcome timeframe
Total AEs and SAEs within 450 days post-infusion
Results posted on
2020-01-07
Participant Flow
34 patients have been screened for eligibility between June, 2014 and April, 2016 at Emory University Medical Center, Atlanta, GA, Northwestern University Medical Center, Chicago, IL, University of Pennsylvania Medical Center, Philadelphia, PA, and Stony Brook University Medical Center, Stony Brook, NY.
23 out of 34 patients have been randomized and 22 received the study intervention. Of those not randomized, 10 did not meet inclusion criteria and 1 could not tolerate CMR.
Participant milestones
| Measure |
itMSC, Then Placebo
Participants first received itMSC 1.5 million per kilogram of weight, infused in the corresponding hospital facility at speed of 1 mL/minute (cell concentration 1 million/mL). After 90 days patients completed treatment at 90 days, and thus constituted the control treatment. After 90 day follow-up testing patients received Placebo and followed up for another 90 days. Total efficacy follow-up - 180 days. Safety measurements 0, 60, 90, days 270 and 450.
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Placebo, Then itMSC
Participants first received Placebo, infused in the corresponding hospital facility at speed of 1 mL/minute. After 90 days patients completed Placebo Control and related testing, and received itMSC treatment 1.5 million per kilogram of weight, and thus constituted the itMSC treated group for another 90 days.Total efficacy follow-up - 180 days. Safety measurements 0, 60, 90, days 270 and 450.
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|---|---|---|
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First Intervention
STARTED
|
11
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12
|
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First Intervention
Received Treatment as Randomized
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10
|
12
|
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First Intervention
COMPLETED
|
10
|
12
|
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First Intervention
NOT COMPLETED
|
1
|
0
|
|
Second Intervention
STARTED
|
10
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12
|
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Second Intervention
COMPLETED
|
7
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12
|
|
Second Intervention
NOT COMPLETED
|
3
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Assess the Effect of Intravenous Dose of (aMBMC) to Subjects With Non-ischemic Heart Failure
Baseline characteristics by cohort
| Measure |
Study Population
n=22 Participants
itMSC, then Placebo: Participants first received itMSC 1.5 million per kilogram of weight, infused in the corresponding hospital facility at speed of 1 mL/minute (cell concentration 1 million/mL). After 90 days patients completed treatment at 90 days, and thus constituted the control treatment. After 90 day follow-up testing patients received Placebo and followed up for another 90 days. Total efficacy follow-up - 180 days. Safety follow-up - 240 days.
Placebo, then itMSC: Participants first received Placebo, infused in the corresponding hospital facility at speed of 1 mL/minute. After 90 days patients completed Placebo Control and related testing, and received itMSC treatment 1.5 million per kilogram of weight, and thus constituted the itMSC treated group for another 90 days.Total efficacy follow-up - 180 days. Safety follow-up - 240 days. and thus constituted the control treatment.
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|---|---|
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Age, Continuous
|
47.3 years
STANDARD_DEVIATION 12.8 • n=5 Participants
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Sex: Female, Male
Female
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9 Participants
n=5 Participants
|
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Sex: Female, Male
Male
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13 Participants
n=5 Participants
|
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Black or African American
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7 Participants
n=5 Participants
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Race (NIH/OMB)
White
|
15 Participants
n=5 Participants
|
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
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Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
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Weight
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92.5 kg
STANDARD_DEVIATION 19.96 • n=5 Participants
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BMI
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32.24 kg/m^2
STANDARD_DEVIATION 7.56 • n=5 Participants
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New York Heart Association (NYHA) Class
Class II
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21 Participants
n=5 Participants
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New York Heart Association (NYHA) Class
Class III
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1 Participants
n=5 Participants
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Systolic Blood Pressure
|
120 mmHg
STANDARD_DEVIATION 17.1 • n=5 Participants
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Diastolic Blood Pressure
|
74 mmHg
STANDARD_DEVIATION 9.6 • n=5 Participants
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Heart Rate
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78 bpm
STANDARD_DEVIATION 14.2 • n=5 Participants
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N-terminlal-proB-type Natriuretic Peptide (NT-proBNP)
|
212 pg/mL
STANDARD_DEVIATION 841 • n=5 Participants
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Troponin I
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0.0009 ug/mL
STANDARD_DEVIATION 0.0003 • n=5 Participants
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Serum Sodium
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138 mmol/L
STANDARD_DEVIATION 2 • n=5 Participants
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Serum Creatinine
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0.96 mg/dL
STANDARD_DEVIATION 0.23 • n=5 Participants
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Aspartate Aminotransferase (AST)
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21.86 International Units/milliliter (IU/mL)
STANDARD_DEVIATION 8.64 • n=5 Participants
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Alanine Aminotransferase (ALT)
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21.82 IU/L
STANDARD_DEVIATION 9.57 • n=5 Participants
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Total bilirubin
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0.8 mg/dL
STANDARD_DEVIATION 0.4 • n=5 Participants
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Albumin
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4.2 g/dL
STANDARD_DEVIATION 0.36 • n=5 Participants
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History of Hypertension
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5 Participants
n=5 Participants
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History of Diabetes
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3 Participants
n=5 Participants
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History of Atrial Fibrillation
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2 Participants
n=5 Participants
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History of Kidney Disease
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1 Participants
n=5 Participants
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Left Ventricular Ejection Fraction (LVEF)
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31.6 %
STANDARD_DEVIATION 9.8 • n=5 Participants
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Left Ventricular End Systolic Volume (LVESV)
|
189.6 mL
STANDARD_DEVIATION 114.2 • n=5 Participants
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Left Ventricular End-Diastolic Volume (LVEDV)
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264.9 mL
STANDARD_DEVIATION 120.4 • n=5 Participants
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Loop diuretic medication
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16 Participants
n=5 Participants
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angiotensin converting enzyme inhibitors (ACEI) or Angiotensin II Receptor Blockers (ARB) medication
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22 Participants
n=5 Participants
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Mineralocorticoid Receptor Antagonist medication
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18 Participants
n=5 Participants
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Beta-blocker
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22 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Total AEs and SAEs within 450 days post-infusionPopulation: All 22 participants that received itMSC treatment were included. All adverse events are divided into serious and non-serious.
As identified in the SAP, the safety analysis was the primary objective and was evaluated by the number of AEs
Outcome measures
| Measure |
Experimental
n=22 Participants
Participants received itMSC
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Placebo
n=20 Participants
Participants received Placebo
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|---|---|---|
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Safety Will be Evaluated by Number of AE
Adverse Events
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38 number of events
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35 number of events
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Safety Will be Evaluated by Number of AE
Serious Adverse Events
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1 number of events
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1 number of events
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Safety Will be Evaluated by Number of AE
Cell related adverse events
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1 number of events
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0 number of events
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Safety Will be Evaluated by Number of AE
All-cause hospitalization
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0 number of events
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0 number of events
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Safety Will be Evaluated by Number of AE
All-cause death
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0 number of events
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0 number of events
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SECONDARY outcome
Timeframe: Baseline to Day 90Population: The secondary efficacy endpoint according to the protocol was the change in LVEF from baseline to Day 90 post-initial infusion.
The secondary efficacy endpoint was the change in LVEF from baseline to Day 90 post-initial infusion. Participants with data available at each time point.
Outcome measures
| Measure |
Experimental
n=12 Participants
Participants received itMSC
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Placebo
n=12 Participants
Participants received Placebo
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|---|---|---|
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Change in LVEF From Baseline to Day 90 Post-initial Infusion.
Baseline (%)
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34.3 percentage of ejection volume
Standard Deviation 7.91
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34.5 percentage of ejection volume
Standard Deviation 7.49
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Change in LVEF From Baseline to Day 90 Post-initial Infusion.
90 days (%)
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34.1 percentage of ejection volume
Standard Deviation 9.7
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36.7 percentage of ejection volume
Standard Deviation 5.42
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Adverse Events
Experimental
Serious events: 1 serious events
Other events: 18 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Experimental
n=22 participants at risk
Subjects that received experimental drug, 22 subjects
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Placebo
n=20 participants at risk
Subjects that received placebo, 20 subjects
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|---|---|---|
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Cardiac disorders
atrial fibrillation
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4.5%
1/22 • Number of events 1 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
5.0%
1/20 • Number of events 1 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
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Other adverse events
| Measure |
Experimental
n=22 participants at risk
Subjects that received experimental drug, 22 subjects
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Placebo
n=20 participants at risk
Subjects that received placebo, 20 subjects
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|---|---|---|
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General disorders
General Disorders and administration site condition: chest pain, fatigue, pyrexia
|
13.6%
3/22 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
55.0%
11/20 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
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Investigations
Venous Pressure Jugular Increased
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27.3%
6/22 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
20.0%
4/20 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
|
Infections and infestations
Upper Respiratory Tract, Nasopharyngitis
|
13.6%
3/22 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
45.0%
9/20 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
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Metabolism and nutrition disorders
Fluid Overload, Dehydration
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36.4%
8/22 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
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20.0%
4/20 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
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Nervous system disorders
Dizziness, headache
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45.5%
10/22 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
0.00%
0/20 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
9.1%
2/22 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
30.0%
6/20 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
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Musculoskeletal and connective tissue disorders
Muscle Spasms
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27.3%
6/22 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
|
5.0%
1/20 • 450 days
All AE summaries were restricted to treatment-emergent adverse events (TEAEs), and TEAEs were defined as those AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study. Treatment-emergent AEs by severity (severe, other), relationship to study treatment ("related" or "not related"). Reported treshold 5%.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place