Myocardial Infarction
Conditions
Keywords
Myocardial Infarction, Heart Attack, Stem cell, Left ventricular dysfunction, Congestive heart failure
Brief summary
The purpose of this study is to determine whether Allogeneic Cardiosphere-Derived Cells (CAP-1002) is safe and effective in decreasing infarct size in patients with a myocardial infarction.
Interventions
Single dose, blinded, intracoronary infusion of 25 Million cardiosphere-derived cells
DRUGPlacebo
Single, blinded, intracoronary infusion of a placebo solution
Sponsors
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
California Institute for Regenerative Medicine (CIRM)
Capricor Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. History of MI (STEMI or NSTEMI) within the prior 12 months due to a coronary artery event and evidenced by at least two of the following: typical ischemic symptoms, serial ST-T changes (new ST elevation or new left bundle block) and/or elevated troponin or Creatine phosphokinase MB isoenzyme (CK-MB) \>5 times the upper limit of normal. Also at least one of the following: development of pathological Q wave ECG changes, imaging evidence of new loss of viable myocardium, or new regional wall motion abnormalities. 2. History of percutaneous coronary intervention (PCI), with stent placement resulting in Thrombolysis in Myocardial Infarction (TIMI) flow = 3, in the coronary artery supplying the infarcted, dysfunctional territory and through which the treatment will be infused. 3. At least one assessment of left ventricular ejection function (LVEF) \<=0.45 as determined by any one of the standard modalities (echocardiography, ventriculography, nuclear imaging, CT and/or MRI) prior to or during the screening period. * For participants that fulfill the criteria of Recent MI (i.e., within 90 days of MI) at time of screening visit: assessment must be post-reperfusion after index MI and the most recent test prior to or during the screening period. * For participants that fulfill the criteria of Chronic MI (i.e., greater than 90 days from MI) at the time of screening visit: assessment must be at least 21 days post-reperfusion after index MI and the most recent test prior to or during the screening period. Note: participants may screen as a Recent MI but be randomized into the Chronic MI strata if the infusion date is \> 90 days post-MI. 4. Left ventricular infarct size of \>= 15% of left ventricular mass in the qualifying infarct-related region to be infused as determined by centrally read screening MRI, with associated thinning and/or hypokinesis, akinesis, or dyskinesis, with no large aneurysmal area in the infarcted regions. 5. No further revascularization clinically indicated at the time the participants is assessed for participation in the clinical trial. 6. Ability to provide informed consent and follow-up with protocol procedures. 7. Age \>= 18 years.
Exclusion criteria
1. Participants with a history of coronary artery bypass surgery, and a patent graft (arterial or saphenous vein graft) attached to the coronary artery to be infused. 2. Diagnosed or suspected myocarditis. 3. History of cardiac tumor, or cardiac tumor demonstrated on screening MRI. 4. History of acute coronary syndrome in the 4 weeks prior to study infusion. 5. History of previous stem cell therapy. 6. History of radiation treatment to the central or left side of thorax. 7. Current or history (within the previous 5 years) of systematic auto-immune or connective tissue disease including, but not limited to, giant cell myocarditis, cardiac or systemic sarcoidosis, Dressler's syndrome, chronic recurrent or persistent pericarditis. 8. History of or current treatment with immunosuppressive, anti-inflammatory, or other agents to treat manifestations of systemic immunologic reactions, including chronic systemic corticosteroids, biologic agents targeting the immune system, anti-tumor and anti-neoplastic drugs, anti-vascular endothelial growth factor, or chemotherapeutic agents within 3 months prior to enrollment. 9. Prior implantable cardioverter defibrillator (ICD) and/or pacemaker placement where study imaging site has not been trained and certified specifically for this protocol to conduct cardiac MRI in participants with ICD and/or pacemaker placement. a. Presence of a pacemaker and/or ICD generator with any of the following limitations/conditions are excluded: i. Manufactured before the year 2000, ii. Leads implanted \< 6 weeks prior to signing informed consent, iii. Non-transvenous epicardial, abandoned, or no-fixation leads, iv. Subcutaneous ICDs, v. Leadless pacemakers, vi. Any other condition that, in the judgement of device-trained staff, would deem an MRI contraindicated. b. Pacemaker dependence with an ICD (Note: pacemaker-dependent candidates without an ICD are not excluded). c. A cardiac resynchronization therapy (CRT) device implanted \< 3 months prior to signing informed consent. 10. Estimated glomerular filtration rate \< 30 mL/min. 11. Participation in an on-going protocol studying an experimental drug or device, or participation in an interventional clinical trial within the last 30 days. 12. Diagnosis of arrhythmogenic right ventricular cardiomyopathy. 13. Current alcohol or drug abuse. 14. Pregnant/nursing women and women of child-bearing potential that do not agree to use at least two forms of active and highly reliable method(s) of contraception. Acceptable methods of contraception include contraceptive pills, depo-progesterone injections, a barrier contraceptive such as a condom with or without spermicide cream or gel, diaphragms or cervical cap with or without spermicide or gel, or an intrauterine device (IUD). 15. Human Immunodeficiency Virus (HIV) infection. 16. Viral hepatitis. 17. Uncontrolled diabetes (HbA1c\>9%). 18. Abnormal liver function (Serum Glutamic Pyruvic Transaminase/Alanine aminotransferase \> 3 times the upper reference range) and/or abnormal hematology (hematocrit \< 25%, White Blood Cell \< 3000 µl, platelets \< 100,000 µl) studies without a reversible, identifiable cause. 19. Sustained ventricular tachycardia (VT) or non-sustained ventricular tachycardia \> 30 beats, not associated with the acute phase of a previous MI (\> 48 hours after the MI onset) or a new acute ischemic episode. 20. Ventricular fibrillation not associated with a new acute ischemic episode. 21. New York Heart Association (NYHA) Class IV congestive heart failure. 22. Evidence of tumor on screening chest/abdominal/pelvic (body) CT scan. 23. Any prior transplant. 24. Known hypersensitivity to dimethyl sulfoxide (DMSO). 25. Known hypersensitivity to bovine products. 26. Any malignancy within 5 years (except for in-situ non-melanoma skin cancer and in-situ cervical cancer) of signing the informed consent form. 27. Any condition or other reason that, in the opinion of the Investigator or Medical Monitor, would render the participants unsuitable for the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Experiencing Any of the Adjudicated Events | Within 1-month post-infusion | Adjudicated Events reported included: Acute myocarditis; Death due to ventricular tachycardia (VT) or ventricular fibrillation (VF); Sudden unexpected death (defined as occurring within one hour of symptom onset, or un- witnessed death); and Major adverse cardiac event (MACE) (defined as the composite incidence of death, non- fatal recurrent MI, hospitalization for heart failure, emergency room treatment for heart failure, left ventricular assist device \[LVAD\] placement or heart transplant). |
| Percent Change From Baseline in Myocardium Mass Infarct Size at Month 12 | Baseline, Month 12 | Infarct size, expressed as a percentage, was calculated by dividing the sum of infarct areas from all sections by the sum of left ventricular (LV) areas from all sections (including those without infarct scar) and multiplying by 100. Percent improvement in infarct size defined by scar as a percent of LV mass was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Baseline, Month 6 and Month 12 | LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). Percent change in LVEF was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
| Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Baseline, Month 6 and Month 12 | LVEDV is the amount of blood in the heart's left ventricle just before the heart contracts. LVEDV was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value. |
| Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Baseline, Month 6 and Month 12 | LVEDV is the amount of blood in the heart's left ventricle just before the heart contracts. Percent change in LVEDV was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
| Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Baseline, Month 6 and Month 12 | LVESV is the amount of blood remaining in the ventricle at the end of systole, after the heart has contracted. LVESV was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value. |
| Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Baseline, Month 6 and Month 12 | LVESV is the amount of blood remaining in the ventricle at the end of systole, after the heart has contracted. Percent change in LVESV was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
| Percent Change From Baseline in Myocardium Mass Infarct Size at Month 6 and 12 | Baseline, Month 6 and Month 12 | Infarct size, expressed as a percentage, was calculated by dividing the sum of infarct areas from all sections by the sum of LV areas from all sections (including those without infarct scar) and multiplying by 100. Improvement in infarct size as a percent of LV mass was assessed by magnetic resonance imaging. |
| Absolute Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Baseline, Month 6 and Month 12 | Infarct size in grams was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value. |
| Percent Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Baseline, Month 6 and Month 12 | Percent change in infarct size was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
| Absolute Change From Baseline in Viable Mass at Month 6 and 12 | Baseline, Month 6 and Month 12 | Viable mass expressed in grams was assessed by magnetic resonance imaging. Myocardial viable mass refers to myocardial cells that are alive after myocardial injury, according to cellular, metabolic and contractile functions. Absolute change was calculated as: post-baseline value-Baseline value. |
| Percent Change From Baseline in Viable Mass at Month 6 and12 | Baseline, Month 6 and Month 12 | Percent change in viable mass was assessed by magnetic resonance imaging. Myocardial viable mass refers to myocardial cells that are alive after myocardial injury, according to cellular, metabolic and contractile functions. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
| Number of Participants Experiencing Any of the Adjudicated Events | Up to Month 12 post-infusion | Adjudicated Events reported included: Acute myocarditis; Death due to VT or VF; Sudden unexpected death (defined as occurring within one hour of symptom onset, or un- witnessed death); MACE (defined as the composite incidence of death, non- fatal recurrent MI, hospitalization for heart failure, emergency room treatment for heart failure, LVAD placement or heart transplant); New cardiac tumor formation on MRI imaging; Any hospitalization due to cardiovascular cause; Any inter-current cardiovascular illness or one related to CAP-1002 or placebo infusion, which prolongs hospitalization; New thrombolysis in myocardial infarction (TIMI) flow \<=1; Development of, or an increase in frequency of VT; Development of increased anti-human leukocyte antigen (anti-HLA) antibody levels (mean fluorescence intensity \[MFI\] \>= 1000; 5000) with development of sensitization to HLA antigens specific to CAP-1002 cardiosphere-derived cells donor. |
| Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Baseline, Month 6 and Month 12 | The regions assessed of the heart were: Anterior, Lateral, Inferior and Septal. Tissue mass recovery in the function of region receiving therapy expressed as percentage improvement was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
| Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Baseline, Month 6 and Month 12 | The six-minute walk test measures the distance a participant is able to walk over a total of six minutes on a hard, flat surface. The goal is for the participant to walk as far as possible in six minutes. The participant is allowed to self-pace and rest as needed as they traverse back and forth along a marked walkway. The total distance walked, in meters, was recorded for each participant. Longer distances indicate better outcomes. |
| Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Total Score at Month 6 and 12 | Baseline, Month 6 and Month 12 | Health related quality of life is measured using the Minnesota Living with Heart Failure questionnaire (MLHFQ). The MLHFQ is a patient-reported outcome to measure the patient's perceptions of the influence of heart failure on physical and emotional aspects of life. The questionnaire has 21 items to assess the impact of frequent physical symptoms of heart failure and the effects of heart failure on physical and emotional functions. Responses are recorded on six-point Likert scales, ranging from 0 (none) to 5 (very much). Total Scores are summed to a range of 0-105, in which with higher scores indicate worse health-related quality of life. |
| Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Baseline, Month 6 and Month 12 | The Short Form (36) Health Survey is a 36-item, patient-reported survey of participant health. The SF-36 consists of eight scaled scores (Physical Function, Physical Health, Emotional Problems, Energy/Fatigue, Emotional Well-Being, Social Functioning, Pain Scale and General Health) which are the weighted sums of the questions in their section. Each component on the SF-36 Item Health Survey is scored from 0-100 with higher scores reflecting better participant status. |
| Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Baseline, Month 6 and Month 12 | WPAI:SHP is a self-administered questionnaire that measures the effect of general health and symptom severity on work productivity and regular activities during the last 7 days. Four scores are derived as percent: Absenteeism, Presenteeism, Work Productivity Loss, Activity Impairment. Each of 4 scores expressed as impairment percentages with a total possible score range of 0 to 100, high percentage= more impairment, less productivity. |
| Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Baseline, Month 6 and Month 12 | PGA will ask participants to assess how their overall status has changed since prior to receiving the therapy. Possible PGA responses are 0=none, 1=mild, 2=moderate, and 3=severe. Change from Baseline was calculated as lowest PGA score on scheduled visits minus PGA score at Baseline which resulted in possible ranges from -3 to +3. Decreasing scores indicate improvement. |
| Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Baseline, Month 6 and Month 12 | New York Heart Association (NYHA) Classification: Class I Subject with cardiac disease but without resulting limitations of physical activity. Class II Subjects with cardiac disease resulting in slight limitation of physical activity. Class III Subjects with cardiac disease resulting in marked limitation of physical activity. Class IV Subjects with cardiac disease resulting in inability to carry on any physical activity without discomfort. Change from Baseline was calculated as lowest NYHA score on scheduled visits minus NYHA score at Baseline which resulted in possible ranges from -3 to +3. Decreasing scores indicate improvement. |
| Change From Baseline in N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Baseline, Month 6 and Month 12 | NT-proBNP was the cardiac biomarkers assessed through serum sample. |
| Change From Baseline in Log Transformed N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Baseline, Month 6 and Month 12 | NT-proBNP was the cardiac biomarkers assessed through serum sample. |
| Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Baseline, Month 6 and Month 12 | The regions assessed of the heart were: Anterior, Lateral, Inferior and Septal. Tissue mass recovery in the function of region receiving therapy expressed as percentage improvement was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value. |
| Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Baseline, Month 6 and Month 12 | LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). LVEF expressed as percentage ejection fraction was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value. |
Countries
United States
Participant flow
Recruitment details
The study enrolled participants at 30 active sites between 13 November 2012 to 03 July 2017. Study had 2 phases: Phase 1 (safety cohort) and Phase 2 (randomized double-blind cohort).
Pre-assignment details
After completion of screening procedures, and at least 4 weeks after myocardial infarction (MI), eligible Phase 1 participants received CAP-1002. In Phase 2, participants were randomized in 2:1 ratio to receive treatment with either CAP-1002 or placebo. Based on available clinical data at time of analysis, Sponsor decided not to pursue development of CAP-1002 in this indication; hence trial was stopped. Outcome measures data was planned to be collected and reported for Phase 2 part of study.
Participants by arm
| Arm | Count |
|---|---|
| Phase 1: Safety Cohort:12.5 M CDCs Participants with recent MI (defined as index MI more than 4 weeks but within 90 days prior to infusion), and chronic MI (defined as index MI more than 90 days but less than 12 months prior to infusion), randomized in safety cohort received a single infusion of CAP-1002 (12.5 M cells) suspended in cryopreservation solution on Day 0 and were followed up for 12 months post-infusion. | 4 |
| Phase 1: Safety Cohort: 25 M CDCs Participants with recent MI (defined as index MI more than 4 weeks but within 90 days prior to infusion), and chronic MI (defined as index MI more than 90 days but less than 12 months prior to infusion), randomized in safety cohort received a single infusion of CAP-1002 (25 M cells) suspended in cryopreservation solution on Day 0 and were followed up for 12 months post-infusion. | 10 |
| Phase 2: Randomized Treatment Cohort: Placebo Participants with recent MI (defined as index MI more than 4 weeks but within 90 days prior to infusion), and chronic MI (defined as index MI more than 90 days but less than 12 months prior to infusion), randomized to placebo received an infusion of 11.1 mL of cryopreservation solution on Day 0 and were followed up for 12 months post-infusion. | 44 |
| Phase 2: Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells Participants with recent MI (defined as index MI more than 4 weeks but within 90 days prior to infusion), and chronic MI (defined as index MI more than 90 days but less than 12 months prior to infusion), randomized to active treatment received a single infusion of CAP-1002 (25 M cells) suspended in cryopreservation solution on Day 0 and were followed up for 12 months post-infusion. | 77 |
| Total | 135 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Phase 1 (up to 12 Months) | Other | 0 | 1 | 0 | 0 |
| Phase 2 (up to 12 Months) | Lost to Follow-up | 0 | 0 | 0 | 2 |
| Phase 2 (up to 12 Months) | Study discontinued by Sponsor | 0 | 0 | 11 | 20 |
| Phase 2 (up to 12 Months) | Withdrawal by Subject | 0 | 0 | 0 | 1 |
Baseline characteristics
| Characteristic | Phase 1: Safety Cohort: 25 M CDCs | Phase 2: Randomized Treatment Cohort: Placebo | Phase 1: Safety Cohort:12.5 M CDCs | Phase 2: Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Total |
|---|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants | 44 Participants | 4 Participants | 77 Participants | 135 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 7 Participants | 0 Participants | 7 Participants | 15 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 0 Participants | 0 Participants | 2 Participants | 3 Participants |
| Race (NIH/OMB) White | 8 Participants | 36 Participants | 4 Participants | 67 Participants | 115 Participants |
| Sex: Female, Male Female | 1 Participants | 6 Participants | 0 Participants | 10 Participants | 17 Participants |
| Sex: Female, Male Male | 9 Participants | 38 Participants | 4 Participants | 67 Participants | 118 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 10 | 0 / 44 | 0 / 77 |
| other Total, other adverse events | 4 / 4 | 10 / 10 | 24 / 44 | 42 / 77 |
| serious Total, serious adverse events | 0 / 4 | 0 / 10 | 15 / 44 | 20 / 77 |
Outcome results
Primary
Number of Participants Experiencing Any of the Adjudicated Events
Adjudicated Events reported included: Acute myocarditis; Death due to ventricular tachycardia (VT) or ventricular fibrillation (VF); Sudden unexpected death (defined as occurring within one hour of symptom onset, or un- witnessed death); and Major adverse cardiac event (MACE) (defined as the composite incidence of death, non- fatal recurrent MI, hospitalization for heart failure, emergency room treatment for heart failure, left ventricular assist device \[LVAD\] placement or heart transplant).
Time frame: Within 1-month post-infusion
Population: Analysis was performed on safety population that included all participants who received investigational product. Data this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Acute myocarditis | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Death due to VT/VF | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Sudden unexpected death | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | MACE | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | MACE | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Acute myocarditis | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Sudden unexpected death | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Death due to VT/VF | 0 Participants |
Primary
Percent Change From Baseline in Myocardium Mass Infarct Size at Month 12
Infarct size, expressed as a percentage, was calculated by dividing the sum of infarct areas from all sections by the sum of left ventricular (LV) areas from all sections (including those without infarct scar) and multiplying by 100. Percent improvement in infarct size defined by scar as a percent of LV mass was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%.
Time frame: Baseline, Month 12
Population: Analysis was performed on Modified Intent-to-Treat (mITT) population that included all participants in the primary randomization cohort who received investigational product, had a baseline observation, and at least one post-baseline observation. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Myocardium Mass Infarct Size at Month 12 | -7.11 percent change | Standard Deviation 12.218 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Myocardium Mass Infarct Size at Month 12 | -8.80 percent change | Standard Deviation 10.645 |
p-value: 0.6453Regression, Linear
Secondary
Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12
The regions assessed of the heart were: Anterior, Lateral, Inferior and Septal. Tissue mass recovery in the function of region receiving therapy expressed as percentage improvement was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | -1.51 percent change | Standard Deviation 5.275 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | -0.98 percent change | Standard Deviation 4.51 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | 0.53 percent change | Standard Deviation 6.966 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | 0.39 percent change | Standard Deviation 5.707 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | 1.21 percent change | Standard Deviation 8.562 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | -0.78 percent change | Standard Deviation 6.255 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | -0.21 percent change | Standard Deviation 4.731 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | -2.48 percent change | Standard Deviation 4.948 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | -0.03 percent change | Standard Deviation 3.857 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | -1.97 percent change | Standard Deviation 6.445 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | -0.61 percent change | Standard Deviation 7.26 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | -1.90 percent change | Standard Deviation 8.681 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | -0.53 percent change | Standard Deviation 5.614 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | -1.15 percent change | Standard Deviation 3.727 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | -0.68 percent change | Standard Deviation 4.736 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | -1.52 percent change | Standard Deviation 4.851 |
Secondary
Absolute Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12
Infarct size in grams was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 6 | -1.72 grams | Standard Deviation 2.831 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 12 | -2.57 grams | Standard Deviation 4.858 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 6 | -1.66 grams | Standard Deviation 2.927 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 12 | -2.87 grams | Standard Deviation 4.256 |
Secondary
Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12
LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). LVEF expressed as percentage ejection fraction was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | 0.17 percent change ejection fraction | Standard Deviation 3.484 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | 0.01 percent change ejection fraction | Standard Deviation 4.089 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | -0.02 percent change ejection fraction | Standard Deviation 4.435 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | -0.62 percent change ejection fraction | Standard Deviation 5.239 |
Secondary
Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12
LVEDV is the amount of blood in the heart's left ventricle just before the heart contracts. LVEDV was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | 11.75 milliliters (mL) | Standard Deviation 23.129 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 7.59 milliliters (mL) | Standard Deviation 27.551 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | 3.92 milliliters (mL) | Standard Deviation 23.692 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 4.04 milliliters (mL) | Standard Deviation 30.511 |
Secondary
Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12
LVESV is the amount of blood remaining in the ventricle at the end of systole, after the heart has contracted. LVESV was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | 6.96 mL | Standard Deviation 17.39 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 4.51 mL | Standard Deviation 21.213 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | 2.63 mL | Standard Deviation 19 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 3.89 mL | Standard Deviation 25.476 |
Secondary
Absolute Change From Baseline in Viable Mass at Month 6 and 12
Viable mass expressed in grams was assessed by magnetic resonance imaging. Myocardial viable mass refers to myocardial cells that are alive after myocardial injury, according to cellular, metabolic and contractile functions. Absolute change was calculated as: post-baseline value-Baseline value.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Viable Mass at Month 6 and 12 | Month 6 | 2.40 grams | Standard Deviation 9.78 |
| Randomized Treatment Cohort: Placebo | Absolute Change From Baseline in Viable Mass at Month 6 and 12 | Month 12 | 1.48 grams | Standard Deviation 11.8 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Viable Mass at Month 6 and 12 | Month 6 | 0.95 grams | Standard Deviation 8.143 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Absolute Change From Baseline in Viable Mass at Month 6 and 12 | Month 12 | 0.96 grams | Standard Deviation 9.063 |
Secondary
Change From Baseline in Log Transformed N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12
NT-proBNP was the cardiac biomarkers assessed through serum sample.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Change From Baseline in Log Transformed N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 6 | -0.26 log pg/mL | Standard Deviation 0.732 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Log Transformed N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 12 | -0.46 log pg/mL | Standard Deviation 0.761 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Log Transformed N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 6 | -0.50 log pg/mL | Standard Deviation 0.557 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Log Transformed N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 12 | -0.63 log pg/mL | Standard Deviation 0.616 |
Secondary
Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Total Score at Month 6 and 12
Health related quality of life is measured using the Minnesota Living with Heart Failure questionnaire (MLHFQ). The MLHFQ is a patient-reported outcome to measure the patient's perceptions of the influence of heart failure on physical and emotional aspects of life. The questionnaire has 21 items to assess the impact of frequent physical symptoms of heart failure and the effects of heart failure on physical and emotional functions. Responses are recorded on six-point Likert scales, ranging from 0 (none) to 5 (very much). Total Scores are summed to a range of 0-105, in which with higher scores indicate worse health-related quality of life.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Total Score at Month 6 and 12 | Month 6 | -5.27 score on a scale | Standard Deviation 18.195 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Total Score at Month 6 and 12 | Month 12 | -8.47 score on a scale | Standard Deviation 16.58 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Total Score at Month 6 and 12 | Month 6 | -8.30 score on a scale | Standard Deviation 15.951 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Total Score at Month 6 and 12 | Month 12 | -8.89 score on a scale | Standard Deviation 20.636 |
Secondary
Change From Baseline in N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12
NT-proBNP was the cardiac biomarkers assessed through serum sample.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Change From Baseline in N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 12 | -283.2 pg/mL | Standard Deviation 571.86 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 6 | -126.4 pg/mL | Standard Deviation 621.45 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 6 | -361.6 pg/mL | Standard Deviation 626.56 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in N-terminal Pro-hormone Brain Natriuretic Peptide (NT-proBNP) Biomarker at Month 6 and 12 | Month 12 | -395.8 pg/mL | Standard Deviation 752.83 |
Secondary
Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12
The Short Form (36) Health Survey is a 36-item, patient-reported survey of participant health. The SF-36 consists of eight scaled scores (Physical Function, Physical Health, Emotional Problems, Energy/Fatigue, Emotional Well-Being, Social Functioning, Pain Scale and General Health) which are the weighted sums of the questions in their section. Each component on the SF-36 Item Health Survey is scored from 0-100 with higher scores reflecting better participant status.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Energy/Fatigue Scale: Month 12 | 8.59 score on a scale | Standard Deviation 23.044 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Well-Being Scale: Month 6 | 2.55 score on a scale | Standard Deviation 14.794 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Health Scale: Month 12 | 21.09 score on a scale | Standard Deviation 38.683 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Well-Being Scale: Month 12 | 0.50 score on a scale | Standard Deviation 17.996 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Function Scale: Month 12 | 3.91 score on a scale | Standard Deviation 13.182 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Social Functioning Scale: Month 6 | 8.24 score on a scale | Standard Deviation 20.163 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Problems Scale: Month 6 | 3.03 score on a scale | Standard Deviation 31.185 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Social Functioning Scale: Month 12 | 8.59 score on a scale | Standard Deviation 18.632 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Problems Scale: Month 12 | 11.46 score on a scale | Standard Deviation 41.139 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Pain Scale: Month 6 | 0.68 score on a scale | Standard Deviation 22.022 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Function Scale: Month 6 | 7.84 score on a scale | Standard Deviation 19.392 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Pain Scale: Month 12 | 2.50 score on a scale | Standard Deviation 21.166 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Energy/Fatigue Scale: Month 6 | 4.55 score on a scale | Standard Deviation 20.877 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | General Health Scale: Month 6 | 1.36 score on a scale | Standard Deviation 15.971 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | General Health Scale: Month 12 | -0.94 score on a scale | Standard Deviation 16.335 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Health Scale: Month 6 | 15.72 score on a scale | Standard Deviation 34.393 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | General Health Scale: Month 12 | 2.74 score on a scale | Standard Deviation 14.954 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | General Health Scale: Month 6 | 0.19 score on a scale | Standard Deviation 17.1 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Function Scale: Month 6 | 6.79 score on a scale | Standard Deviation 17.041 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Function Scale: Month 12 | 6.16 score on a scale | Standard Deviation 19.943 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Health Scale: Month 6 | 26.08 score on a scale | Standard Deviation 46.68 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Physical Health Scale: Month 12 | 37.42 score on a scale | Standard Deviation 41.171 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Problems Scale: Month 12 | 12.58 score on a scale | Standard Deviation 32.176 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Energy/Fatigue Scale: Month 6 | 7.29 score on a scale | Standard Deviation 18.984 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Energy/Fatigue Scale: Month 12 | 8.87 score on a scale | Standard Deviation 20.254 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Well-Being Scale: Month 6 | 2.18 score on a scale | Standard Deviation 14.833 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Well-Being Scale: Month 12 | 0.72 score on a scale | Standard Deviation 13.074 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Social Functioning Scale: Month 6 | 5.68 score on a scale | Standard Deviation 24.379 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Social Functioning Scale: Month 12 | 9.91 score on a scale | Standard Deviation 27.228 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Pain Scale: Month 6 | -1.01 score on a scale | Standard Deviation 21.773 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Pain Scale: Month 12 | 1.04 score on a scale | Standard Deviation 21.447 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Short Form (36) (SF-36) Scale Score at Month 6 and 12 | Emotional Problems Scale: Month 6 | 8.23 score on a scale | Standard Deviation 41.237 |
Secondary
Change From Baseline in Six-Minute Walk Test at Month 6 and 12
The six-minute walk test measures the distance a participant is able to walk over a total of six minutes on a hard, flat surface. The goal is for the participant to walk as far as possible in six minutes. The participant is allowed to self-pace and rest as needed as they traverse back and forth along a marked walkway. The total distance walked, in meters, was recorded for each participant. Longer distances indicate better outcomes.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 6 | 40.7 meters | Standard Deviation 92.49 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 12 | 10.5 meters | Standard Deviation 91.45 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 6 | 15.5 meters | Standard Deviation 108.43 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 12 | 25.4 meters | Standard Deviation 90.08 |
Secondary
Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12
WPAI:SHP is a self-administered questionnaire that measures the effect of general health and symptom severity on work productivity and regular activities during the last 7 days. Four scores are derived as percent: Absenteeism, Presenteeism, Work Productivity Loss, Activity Impairment. Each of 4 scores expressed as impairment percentages with a total possible score range of 0 to 100, high percentage= more impairment, less productivity.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Activity Impairment: Month 6 | -6.19 percentage impairment | Standard Deviation 27.67 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Absenteeism: Month 6 | -9.19 percentage impairment | Standard Deviation 22.91 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Absenteeism: Month 12 | -11.94 percentage impairment | Standard Deviation 30.569 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Presenteeism: Month 6 | -4.40 percentage impairment | Standard Deviation 22.376 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Presenteeism: Month 12 | -8.75 percentage impairment | Standard Deviation 22.472 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Work Productivity Loss: Month 6 | -9.67 percentage impairment | Standard Deviation 25.096 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Work Productivity Loss: Month 12 | -14.87 percentage impairment | Standard Deviation 30.538 |
| Randomized Treatment Cohort: Placebo | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Activity Impairment: Month 12 | -5.81 percentage impairment | Standard Deviation 24.87 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Activity Impairment: Month 12 | -12.64 percentage impairment | Standard Deviation 28.09 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Activity Impairment: Month 6 | -10.26 percentage impairment | Standard Deviation 29.843 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Presenteeism: Month 12 | -15.94 percentage impairment | Standard Deviation 33.201 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Absenteeism: Month 6 | -4.53 percentage impairment | Standard Deviation 20.707 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Work Productivity Loss: Month 12 | -18.77 percentage impairment | Standard Deviation 36.38 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Absenteeism: Month 12 | -8.00 percentage impairment | Standard Deviation 22.943 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Work Productivity Loss: Month 6 | -10.21 percentage impairment | Standard Deviation 33.012 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Change From Baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) Score at Month 6 and 12 | Presenteeism: Month 6 | -8.48 percentage impairment | Standard Deviation 28.903 |
Secondary
Number of Participants Experiencing Any of the Adjudicated Events
Adjudicated Events reported included: Acute myocarditis; Death due to VT or VF; Sudden unexpected death (defined as occurring within one hour of symptom onset, or un- witnessed death); MACE (defined as the composite incidence of death, non- fatal recurrent MI, hospitalization for heart failure, emergency room treatment for heart failure, LVAD placement or heart transplant); New cardiac tumor formation on MRI imaging; Any hospitalization due to cardiovascular cause; Any inter-current cardiovascular illness or one related to CAP-1002 or placebo infusion, which prolongs hospitalization; New thrombolysis in myocardial infarction (TIMI) flow \<=1; Development of, or an increase in frequency of VT; Development of increased anti-human leukocyte antigen (anti-HLA) antibody levels (mean fluorescence intensity \[MFI\] \>= 1000; 5000) with development of sensitization to HLA antigens specific to CAP-1002 cardiosphere-derived cells donor.
Time frame: Up to Month 12 post-infusion
Population: Analysis was performed on safety population that included all participants in the primary randomization cohort who received investigational product. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | TIMI <= 1 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Acute myocarditis | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Death due to VT/VF | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Sudden unexpected death | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | MACE | 5 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Cardiac tumor | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | CV hospitalization | 7 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Prolonged CV hospitalization | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | VT | 1 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Donor-specific antibody, MFI >= 1000 | 3 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants Experiencing Any of the Adjudicated Events | Donor-specific antibody, MFI >= 5000 | 1 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | VT | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | CV hospitalization | 10 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Acute myocarditis | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Donor-specific antibody, MFI >= 5000 | 1 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Death due to VT/VF | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Prolonged CV hospitalization | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Sudden unexpected death | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | TIMI <= 1 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | MACE | 5 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Donor-specific antibody, MFI >= 1000 | 5 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants Experiencing Any of the Adjudicated Events | Cardiac tumor | 0 Participants |
Secondary
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12
New York Heart Association (NYHA) Classification: Class I Subject with cardiac disease but without resulting limitations of physical activity. Class II Subjects with cardiac disease resulting in slight limitation of physical activity. Class III Subjects with cardiac disease resulting in marked limitation of physical activity. Class IV Subjects with cardiac disease resulting in inability to carry on any physical activity without discomfort. Change from Baseline was calculated as lowest NYHA score on scheduled visits minus NYHA score at Baseline which resulted in possible ranges from -3 to +3. Decreasing scores indicate improvement.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: - 2 | 3 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: + 2 | 1 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: + 2 | 1 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: + 3 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: - 1 | 15 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: - 3 | 1 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: - 3 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: - 2 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: 0 | 11 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: - 1 | 13 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: + 3 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: 0 | 26 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: + 1 | 2 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: + 1 | 3 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: + 1 | 4 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: + 2 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: + 3 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: - 3 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: - 2 | 4 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: - 1 | 17 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: 0 | 28 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: + 1 | 5 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: + 2 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 12: + 3 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: - 3 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: - 2 | 3 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: - 1 | 24 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Class at Month 6 and 12 | Month 6: 0 | 45 Participants |
Secondary
Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12
PGA will ask participants to assess how their overall status has changed since prior to receiving the therapy. Possible PGA responses are 0=none, 1=mild, 2=moderate, and 3=severe. Change from Baseline was calculated as lowest PGA score on scheduled visits minus PGA score at Baseline which resulted in possible ranges from -3 to +3. Decreasing scores indicate improvement.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: - 3 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: - 2 | 2 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: - 1 | 21 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 0 | 18 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 1 | 3 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 2 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 3 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: - 3 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: - 2 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: - 1 | 15 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 0 | 16 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 1 | 1 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 2 | 0 Participants |
| Randomized Treatment Cohort: Placebo | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 3 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 0 | 20 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: - 3 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: - 3 | 1 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: - 2 | 6 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 2 | 2 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: - 1 | 23 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: - 2 | 5 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 0 | 39 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 1 | 3 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 1 | 7 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: - 1 | 22 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 2 | 2 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 12: + 3 | 0 Participants |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Number of Participants With Change From Baseline in Patient Global Assessment (PGA) Score at Month 6 and 12 | Month 6: + 3 | 0 Participants |
Secondary
Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12
The regions assessed of the heart were: Anterior, Lateral, Inferior and Septal. Tissue mass recovery in the function of region receiving therapy expressed as percentage improvement was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | 6.50 percent change | Standard Deviation 33.009 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | 14.74 percent change | Standard Deviation 64.941 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | 15.18 percent change | Standard Deviation 79.223 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | 23.83 percent change | Standard Deviation 131.511 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | 2.33 percent change | Standard Deviation 18.196 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | -3.17 percent change | Standard Deviation 15.117 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | 178.68 percent change | Standard Deviation 940.725 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | -7.77 percent change | Standard Deviation 14.477 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | 15.29 percent change | Standard Deviation 91.948 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | 2.99 percent change | Standard Deviation 42.147 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | -0.39 percent change | Standard Deviation 42.897 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | 15.09 percent change | Standard Deviation 61.698 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | 6.22 percent change | Standard Deviation 75.838 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | 14.61 percent change | Standard Deviation 86.589 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | 78.79 percent change | Standard Deviation 382.48 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | 1.41 percent change | Standard Deviation 42.721 |
Secondary
Percent Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12
Percent change in infarct size was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 6 | -5.14 percent change | Standard Deviation 8.481 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 12 | -8.50 percent change | Standard Deviation 13.191 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 6 | -4.67 percent change | Standard Deviation 7.826 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Infarct Size (Scar Tissue Mass) at Month 6 and 12 | Month 12 | -8.86 percent change | Standard Deviation 11.249 |
Secondary
Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12
LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). Percent change in LVEF was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | 0.82 percent change | Standard Deviation 10.89 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | 0.01 percent change | Standard Deviation 10.228 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | 0.61 percent change | Standard Deviation 11.894 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | -1.04 percent change | Standard Deviation 13.956 |
Secondary
Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12
LVEDV is the amount of blood in the heart's left ventricle just before the heart contracts. Percent change in LVEDV was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | 5.64 percent change | Standard Deviation 10.742 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 4.68 percent change | Standard Deviation 12.91 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | 2.02 percent change | Standard Deviation 11.203 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 2.54 percent change | Standard Deviation 13.831 |
Secondary
Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12
LVESV is the amount of blood remaining in the ventricle at the end of systole, after the heart has contracted. Percent change in LVESV was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | 5.41 percent change | Standard Deviation 11.928 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 4.77 percent change | Standard Deviation 15.764 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | 2.34 percent change | Standard Deviation 14.257 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 4.13 percent change | Standard Deviation 18.572 |
Secondary
Percent Change From Baseline in Myocardium Mass Infarct Size at Month 6 and 12
Infarct size, expressed as a percentage, was calculated by dividing the sum of infarct areas from all sections by the sum of LV areas from all sections (including those without infarct scar) and multiplying by 100. Improvement in infarct size as a percent of LV mass was assessed by magnetic resonance imaging.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Myocardium Mass Infarct Size at Month 6 and 12 | Month 6 | -0.81 percent change | Standard Deviation 2.109 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Myocardium Mass Infarct Size at Month 6 and 12 | Month 12 | -1.31 percent change | Standard Deviation 2.684 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Myocardium Mass Infarct Size at Month 6 and 12 | Month 6 | -1.05 percent change | Standard Deviation 1.699 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Myocardium Mass Infarct Size at Month 6 and 12 | Month 12 | -1.83 percent change | Standard Deviation 2.432 |
Secondary
Percent Change From Baseline in Viable Mass at Month 6 and12
Percent change in viable mass was assessed by magnetic resonance imaging. Myocardial viable mass refers to myocardial cells that are alive after myocardial injury, according to cellular, metabolic and contractile functions. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. Data for this outcome measure was not planned to be collected and reported for Phase 1 part of the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Viable Mass at Month 6 and12 | Month 6 | 2.54 percent change | Standard Deviation 10.804 |
| Randomized Treatment Cohort: Placebo | Percent Change From Baseline in Viable Mass at Month 6 and12 | Month 12 | 2.79 percent change | Standard Deviation 10.635 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Viable Mass at Month 6 and12 | Month 6 | 1.07 percent change | Standard Deviation 7.698 |
| Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells | Percent Change From Baseline in Viable Mass at Month 6 and12 | Month 12 | 1.31 percent change | Standard Deviation 7.795 |