Cardiomyopathy, Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne Muscular Dystrophy, Cardiomyopathy
Brief summary
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open-label fashion.
Detailed description
Approximately 24, and not more than 30, participants will be randomized into the study, in two sequential enrollment groups. Safety data from Group 1 will undergo a Data Safety Monitoring Board (DSMB) review prior to initiation of enrollment for Group 2. The first 6-8 randomized participants will comprise Group 1, and will include a minimum of 3 participants completing intracoronary infusion with CAP-1002. The DSMB will conduct a review of interim safety data through 72 hours post-Day 0 for at least 3 infused participants and for at least 6 participants overall. Enrollment of Group 2 will begin per DSMB recommendations following their review of the 72 hour safety data from Group 1. Group 2 will include approximately 18 participants. Screening and randomization will continue until at total of 12 participants are infused with CAP 1002 or 30 participants are randomized into the study, whichever comes first. All participants assigned to the active treatment arm will receive an intended total dose of up to 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior). Participants randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the participant on an ongoing basis, and will receive no infusion. Randomization will take place within 30 days of the first screening procedure. After completion of the screening procedures, eligible participants randomized to active treatment arm will receive CAP-1002 administered via intracoronary infusion on Day 0. Day 0 for eligible participants randomized to the usual care arm will occur 7 days after the date of randomization. All randomized participants will have a follow-up telephone call on Study Day 3, and study visits at Weeks 2 and 6, and at Months 3, 6 and 12 post Day 0.
Interventions
Intracoronary delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Sponsors
Capricor Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male participants 18 years of age or older must be able to provide informed consent and follow up with protocol procedures. Male participants at least 12 years of age but younger than 18 years of age must be able to provide assent with parent or guardian providing permission for study participation. Only male participants will be randomized into this study. 2. Documented diagnosis of Duchenne Muscular Dystrophy by genetic mutation analysis. 3. Cardiomyopathy with left ventricular scar by late gadolinium enhancement (LGE) in at least 4 segments as assessed by contrast-enhanced MRI and EF \>35% at the time of screening. 4. Use of evidence based medical-therapy in accordance with the DMD Care Considerations Working Group guidelines for the management of DMD, for at least three months prior to signing the consent form (or, providing assent) or documented contraindication or intolerance or patient preference. 5. Participants must be taking systemic glucocorticoids for at least six months prior to screening. 6. Participants must be 12 years of age or older at time of screening 7. Participants must be appropriate candidates for cardiac catheterization and intracoronary infusion of CAP-1002, in the judgement of the site's interventional cardiologist.
Exclusion criteria
1. Therapy with intravenous inotropic or vasoactive medications at the time of screening. 2. Inability to undergo cardiac catheterization and/or MRI without general anesthesia. 3. Immunologic incompatibility with all available Master Cell Banks (MCBs) by single-antigen bead (SAB) serum antibody profiling. 4. Planned or likely major surgery in the next 12 months after planned randomization. 5. Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of acquiring a LVAD. 6. Contraindication to cardiac MRI. 7. Known hypersensitivity to contrast agents. 8. Estimated glomerular filtration rate (GFR) \<60 mL/min, as calculated by the CKD-EPI cystatin C equation (Inker, Schmid et al. 2012). 9. Active infection not responsive to treatment. 10. Active systemic allergic reaction(s), connective tissue disease or autoimmune disorder(s). 11. History of cardiac tumor or cardiac tumor demonstrated on screening MRI. 12. History of previous stem cell therapy. 13. History of use of medications listed in Appendix 3 within 3 months prior to signing the Inform Consent Form / Assent through completion of the study infusion. 14. Known moderate-to-severe aortic stenosis/insufficiency or severe mitral stenosis/regurgitation. 15. Current active alcohol or drug abuse. 16. Known history of Human Immunodeficiency Virus (HIV) infection. 17. Known history of chronic viral hepatitis. 18. Abnormal liver function (alanine aminotransferase (ALT)/aspartate aminotransferase (AST) \>10 times the upper reference range) and/or abnormal hematology (hematocrit \<25%, White Blood Cells \<3000 μl, platelets \<100,000 μl) studies without a reversible, identifiable cause. 19. Known hypersensitivity to bovine products. 20. Known hypersensitivity to dimethyl sulfoxide (DMSO). 21. Uncontrolled diabetes (HbA1c \>9.0). 22. Inability to comply with protocol-related procedures, including required study visits. 23. Any condition or other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for the study. 24. Currently receiving investigational treatment on another clinical study or expanded access protocol, including any of the following: * Received investigational intervention within 30 days prior to randomization * Treatment and/or an incomplete follow-up to treatment with any investigational cell based therapy within 6 months prior to randomization * Active participation in other research therapy for cardiovascular repair/regeneration
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Experiencing Any of the Adjudicated Events | Within 72 hours post-infusion | Adjudicated Events reported included: New thrombolysis in myocardial infarction (TIMI) flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2noted immediately following infusion and persisting greater than (\>) 3 minutes, despite intracoronary vasodilator administration; sudden unexpected death within 72 hours of intracoronary infusion; and Major adverse cardiac event (MACE) within 72 hours of intracoronary infusion, including death, non-fatal myocardial infarction and hospitalization for cardiovascular event (including heart failure hospitalizations). |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Up to Month 12 post-infusion | An Adverse Event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily had to have causal relationship with treatment. TEAEs were defined as AEs that occurred or worsened in severity between the first dose of the investigational medicinal product (IMP) until the end of study. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. |
| Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Clinical chemistry parameters assessed were chloride, potassium and sodium. |
| Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Clinical chemistry parameter assessed was albumin. |
| Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Clinical chemistry parameter assessed was glucose. |
| Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Hematological parameters assessed were: platelets, white blood cells, basophils, eosinophils, lymphocytes, monocytes and neutrophils. |
| Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Hematological parameter assessed was hemoglobin. |
| Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Hematological parameter assessed was red blood cells. |
| Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Vital signs assessed were systolic and diastolic blood pressure. |
| Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Vital signs assessed was heart rate. |
| Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Vital signs assessed was respiratory rate. |
| Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Vital signs assessed was temperature. |
| Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | Cardiac physical examination parameters assessed were: jugular vein distension, edema, heart sounds, murmur, breath sounds. |
| Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | ECG parameters assessed were: PR Interval, QRS Duration, QT Interval, QTc interval and QT interval. |
| Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 | Baseline, Month 6 and Month 12 | ECG parameter assessed was ventricular rate; which depends on the degree of atrioventricular conduction. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Including Shoulder) at Month 6 and 12 | Baseline, Month 6 and 12 | The PUL Scale consists of an entry item to define the starting functional level and 21 items subdivided into shoulder level (4 items,), middle level (9 items), and distal level (8 items) dimensions. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for middle level, and 24 for distal level. The total score is calculated by the sum of all the scores of the three subscales (including the shoulder dimension) and ranged from 0 to 74. Higher scores over time indicate improvement and lower scores indicated severity. Change from Baseline in PUL overall score (including shoulder) at Month 6 and 12 is reported in this outcome measure. |
| Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12 | Baseline, Month 6 and 12 | The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter. |
| Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12 | Baseline, Month 6 and 12 | Percent predicted PEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort-dependent measure of lung function. |
| Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 6 and 12 | Baseline, Month 6 and 12 | FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer. |
| Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second at Month 6 and 12 | Baseline, Month 6 and 12 | FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer. |
| Change From Baseline in Percent Predicted Forced Vital Capacity Level at Month 6 and 12 | Baseline, Month 6 and 12 | FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer. |
| Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12 | Baseline, Month 6 and 12 | FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer. |
| Change From Baseline in Forced Expiratory Flow at 25-75% of FVC (FEF25-75%) at Month 6 and 12 | Baseline, Month 6 and 12 | Forced Expiratory Flow (FEF) is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF25-75% is defined as the mean forced expiratory flow between the 25% and 75% of the FVC (total amount of air exhaled from the lungs during the lung function test). |
| Change From Baseline in Percent Predicted Forced Expiratory Flow at 25-75% of FVC at Month 6 and 12 | Baseline, Month 6 and 12 | FEF is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF25-75% is defined as the mean forced expiratory flow between the 25% and 75% of the FVC (total amount of air exhaled from the lungs during the lung function test). |
| Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12 | Baseline, Month 6 and 12 | FET is defined as the time taken for an individual to complete a forceful exhalation after maximal inspiration. FET is measured by asking the subject to take a deep breath and blow it all out as fast as possible. |
| Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Baseline, 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. |
| Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Summary Score - Participant Responses at Month 6 and 12 | Baseline, Month 6 and 12 | The PedsQL-Duchenne muscular dystrophy (DMD) module consists of 18 items and comprised of 4 domains: Daily Activities, Treatment Barriers, Worry, Communication. Items are reverse scored and linearly transformed to a 0-100 scale. The overall range for total PedsQL score (18 items; mean of all items) was 0 (improvement) to 100 (severity). Decreasing scores over time indicate improvement. Change From Baseline in PedsQL total summary score- participant responses at Month 6 and 12 was reported in this outcome measure. |
| Change From Baseline in Pediatric Quality of Life Inventory Total Summary Score - Parent Responses at Month 6 and 12 | Baseline, Month 6 and 12 | The PedsQL-Duchenne muscular dystrophy (DMD) module consists of 18 items and comprised of 4 domains: Daily Activities, Treatment Barriers, Worry, Communication. Items are reverse scored and linearly transformed to a 0-100 scale. The overall range for total PedsQL score (18 items; mean of all items) was 0 (improvement) to 100 (severity). Decreasing scores over time indicate improvement. Change From Baseline in PedsQL total summary score- parent responses at Month 6 and 12 was reported in this outcome measure. |
| 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. |
| Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Baseline, Month 6 and 12 | The PODCI consists of 83 items and 5 core scales: Upper Extremity and Physical Function, Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort, and Happiness; a Global Functioning Scale. The Global Functioning Scale is the mean of the mean scores from 4 of the 5 core scales (all except the happiness core scale). The Global Functioning Scale and each of the core scales were standardized so that a score of 0 represents a poor outcome/worse health, while 100 is the best possible outcome/best health (i.e., complete range of each score is 0 to 100, with higher scores representing better functioning). |
| Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Baseline, Month 6 and Month 12 | Osteopontin, Cardiac stress biomarker (ST2) and Galectin-3 were DMD biomarkers assessed through serum samples. |
| Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Baseline, Month 6 and 12 | The PODCI consists of 83 items and 5 core scales: Upper Extremity and Physical Function, Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort, and Happiness; a Global Functioning Scale. The Global Functioning Scale is the mean of the mean scores from 4 of the 5 core scales (all except the happiness core scale). The Global Functioning Scale and each of the core scales were standardized so that a score of 0 represents a poor outcome/worse health, while 100 is the best possible outcome/best health (i.e., complete range of each score is 0 to 100, with higher scores representing better functioning). |
| 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%. |
| Absolute Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Baseline, Month 6 and Month 12 | Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. LV LGE scar tissue mass in grams was assessed by cardiac MRI. Absolute change was calculated as: post-baseline value-Baseline value. |
| Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Baseline, Month 6 and Month 12 | Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. Percent change in LV LGE scar tissue mass 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 (LV) Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Baseline, Month 6 and 12 | Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. Improvement in infarct size as a percent of LV mass was assessed by magnetic resonance imaging and reported in this outcome measure. |
| Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Baseline, Month 6 and 12 | Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. 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%. |
| Absolute Change From Baseline in Circumferential Strain at Month 6 and 12 | Baseline, Month 6 and Month 12 | Circumferential strain represents the change in length (shortening in systole, represented as a negative strain value) of the myocardium along the circumferential axis of the LV as viewed in the short axis. Improvement in Circumferential Strain was expressed in percentage and was assessed by cardiac MRI. |
| Percent Change From Baseline in Circumferential Strain at Month 6 and 12 | Baseline, Month 6 and Month 12 | Circumferential strain represents the change in length (shortening in systole, represented as a negative strain value) of the myocardium along the circumferential axis of the LV as viewed in the short axis; and was assessed by cardiac MRI. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value \*100%. |
| 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 change from baseline was assessed by magnetic resonance imaging. Change was calculated as: post-baseline value - baseline value. |
| 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 percent change from baseline 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 Performance of the Upper Limb (PUL) Overall Score (Excluding Shoulder) at Month 6 and 12 | Baseline, Month 6 and 12 | The PUL Scale consists of an entry item to define the starting functional level and 21 items subdivided into shoulder level (4 items), middle level (9 items), and distal level (8 items) dimensions. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for middle level, and 24 for distal level. The total score is calculated by the sum of all the scores of the three subscales (excluding the shoulder dimension) and ranged from 0 to 58. Higher scores indicated improvement and lower scores indicated severity. Change from Baseline in PUL overall score (excluding shoulder) at Month 6 and 12 is reported in this outcome measure. |
Countries
United States
Participant flow
Recruitment details
The study enrolled participants at 3 active sites between 07 January 2016 to 14 September 2017.
Pre-assignment details
Participants were randomized in 1:1 ratio to receive either Allogeneic Cardiosphere-Derived Cells (CAP-1002) or usual care. Usual care participants were considered as a reference comparator group and received no treatment.
Participants by arm
| Arm | Count |
|---|---|
| Usual Care Participants randomized to receive usual care continued to be cared for and treated in the manner the investigator deemed most appropriate for the participant on an ongoing basis and received no infusion. | 12 |
| CAP-1002 Participants received an intended total dose of 75 million (M) cardiosphere-derived cells (CDCs) infused as 25M cells into each of the three main coronary arteries supplying the anterior, lateral and inferior/posterior cardiac territories during a single, intracoronary procedure on Day 0 and were followed up for 12 months post-infusion. | 13 |
| Total | 25 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal of consent | 2 | 0 |
Baseline characteristics
| Characteristic | Usual Care | CAP-1002 | Total |
|---|---|---|---|
| Age, Continuous | 16.9 years STANDARD_DEVIATION 2.75 | 18.7 years STANDARD_DEVIATION 3.5 | 17.8 years STANDARD_DEVIATION 3.22 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 10 Participants | 11 Participants | 21 Participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 12 Participants | 13 Participants | 25 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 12 | 0 / 13 |
| other Total, other adverse events | 9 / 12 | 9 / 13 |
| serious Total, serious adverse events | 1 / 12 | 3 / 13 |
Outcome results
Primary
Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12
Clinical chemistry parameter assessed was albumin.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Baseline | 4.48 grams per deciliter | Standard Deviation 0.266 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Change at Month 6 | -0.09 grams per deciliter | Standard Deviation 0.181 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Change at Month 12 | -0.02 grams per deciliter | Standard Deviation 0.199 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Baseline | 3.87 grams per deciliter | Standard Deviation 0.417 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Change at Month 6 | 0.36 grams per deciliter | Standard Deviation 0.5 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Change at Month 12 | 0.32 grams per deciliter | Standard Deviation 0.402 |
Primary
Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12
Clinical chemistry parameter assessed was glucose.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Baseline | 87.1 milligrams per deciliter | Standard Deviation 8.2 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Change at Month 6 | 2.1 milligrams per deciliter | Standard Deviation 13.78 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Change at Month 12 | -4.1 milligrams per deciliter | Standard Deviation 12.44 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Baseline | 89.5 milligrams per deciliter | Standard Deviation 9.58 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Change at Month 6 | 12.3 milligrams per deciliter | Standard Deviation 20.56 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Change at Month 12 | 6.8 milligrams per deciliter | Standard Deviation 15 |
Primary
Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12
Clinical chemistry parameters assessed were chloride, potassium and sodium.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Chloride: Change at Month 6 | -0.4 millimoles per liter (mmol/L) | Standard Deviation 2.46 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Potassium: Change at Month 12 | -0.08 millimoles per liter (mmol/L) | Standard Deviation 0.531 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Potassium: Baseline | 4.28 millimoles per liter (mmol/L) | Standard Deviation 0.252 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Sodium: Baseline | 138.7 millimoles per liter (mmol/L) | Standard Deviation 2.23 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Chloride: Change at Month 12 | 0.2 millimoles per liter (mmol/L) | Standard Deviation 2.53 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Sodium: Change at Month 6 | -0.3 millimoles per liter (mmol/L) | Standard Deviation 2.9 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Potassium: Change at Month 6 | -0.15 millimoles per liter (mmol/L) | Standard Deviation 0.342 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Sodium: Change at Month 12 | 0.3 millimoles per liter (mmol/L) | Standard Deviation 1.83 |
| Usual Care | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Chloride: Baseline | 100.9 millimoles per liter (mmol/L) | Standard Deviation 2.54 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Sodium: Change at Month 12 | -1.2 millimoles per liter (mmol/L) | Standard Deviation 2.83 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Chloride: Baseline | 102.7 millimoles per liter (mmol/L) | Standard Deviation 3.52 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Chloride: Change at Month 6 | -0.8 millimoles per liter (mmol/L) | Standard Deviation 4.09 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Chloride: Change at Month 12 | -0.9 millimoles per liter (mmol/L) | Standard Deviation 3.95 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Potassium: Baseline | 4.00 millimoles per liter (mmol/L) | Standard Deviation 0.356 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Potassium: Change at Month 6 | 0.22 millimoles per liter (mmol/L) | Standard Deviation 0.286 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Potassium: Change at Month 12 | 0.31 millimoles per liter (mmol/L) | Standard Deviation 0.362 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Sodium: Baseline | 140.2 millimoles per liter (mmol/L) | Standard Deviation 2.51 |
| CAP-1002 | Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 | Sodium: Change at Month 6 | -1.5 millimoles per liter (mmol/L) | Standard Deviation 3.15 |
Primary
Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12
ECG parameters assessed were: PR Interval, QRS Duration, QT Interval, QTc interval and QT interval.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | PR Interval: Baseline | 116.2 millisecond | Standard Deviation 41.23 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | PR Interval: Change at Month 6 | 8.7 millisecond | Standard Deviation 37.02 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | PR Interval: Change at Month 12 | 8.3 millisecond | Standard Deviation 44.4 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QRS Duration: Baseline | 92.7 millisecond | Standard Deviation 8.11 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QRS Duration: Change at Month 6 | -0.8 millisecond | Standard Deviation 7.25 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QRS Duration: Change at Month 12 | 4.1 millisecond | Standard Deviation 9.53 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QT Interval: Baseline | 350.6 millisecond | Standard Deviation 18.76 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QT Interval: Change at Month 6 | 4.4 millisecond | Standard Deviation 16.07 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QT Interval: Change at Month 12 | 11.7 millisecond | Standard Deviation 23.9 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QTc Interval: Baseline | 419.6 millisecond | Standard Deviation 25.25 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QTc Interval: Change at Month 6 | 6.5 millisecond | Standard Deviation 11.84 |
| Usual Care | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QTc Interval: Change at Month 12 | 8.4 millisecond | Standard Deviation 6.42 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QTc Interval: Change at Month 6 | -5.8 millisecond | Standard Deviation 23.45 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | PR Interval: Baseline | 129.2 millisecond | Standard Deviation 21.31 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QT Interval: Baseline | 348.9 millisecond | Standard Deviation 21.55 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | PR Interval: Change at Month 6 | -2.8 millisecond | Standard Deviation 7.67 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QTc Interval: Baseline | 423.7 millisecond | Standard Deviation 23.6 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | PR Interval: Change at Month 12 | 1.0 millisecond | Standard Deviation 12.58 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QT Interval: Change at Month 6 | -3.2 millisecond | Standard Deviation 18.91 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QRS Duration: Baseline | 91.3 millisecond | Standard Deviation 10.03 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QTc Interval: Change at Month 12 | 1.2 millisecond | Standard Deviation 27.23 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QRS Duration: Change at Month 6 | 4.5 millisecond | Standard Deviation 7.58 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QT Interval: Change at Month 12 | -0.2 millisecond | Standard Deviation 18.21 |
| CAP-1002 | Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 | QRS Duration: Change at Month 12 | 4.5 millisecond | Standard Deviation 6.04 |
Primary
Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12
ECG parameter assessed was ventricular rate; which depends on the degree of atrioventricular conduction.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 | Baseline | 87.8 beats per minute | Standard Deviation 11.95 |
| Usual Care | Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 | Change at Month 6 | 0.8 beats per minute | Standard Deviation 7.11 |
| Usual Care | Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 | Change at Month 12 | -1.8 beats per minute | Standard Deviation 11.11 |
| CAP-1002 | Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 | Baseline | 89.8 beats per minute | Standard Deviation 14.43 |
| CAP-1002 | Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 | Change at Month 6 | 1.2 beats per minute | Standard Deviation 14.66 |
| CAP-1002 | Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 | Change at Month 12 | 2.2 beats per minute | Standard Deviation 14.62 |
Primary
Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12
Hematological parameter assessed was hemoglobin.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Baseline | 14.48 grams per deciliter | Standard Deviation 0.651 |
| Usual Care | Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Change at Month 6 | -0.05 grams per deciliter | Standard Deviation 1.054 |
| Usual Care | Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Change at Month 12 | -0.13 grams per deciliter | Standard Deviation 1.288 |
| CAP-1002 | Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Baseline | 13.76 grams per deciliter | Standard Deviation 1.254 |
| CAP-1002 | Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Change at Month 6 | 0.05 grams per deciliter | Standard Deviation 0.458 |
| CAP-1002 | Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Change at Month 12 | 0.29 grams per deciliter | Standard Deviation 0.808 |
Primary
Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12
Hematological parameter assessed was red blood cells.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Baseline | 5.01 10^6 cells per microliter | Standard Deviation 0.309 |
| Usual Care | Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Change at Month 6 | -0.05 10^6 cells per microliter | Standard Deviation 0.372 |
| Usual Care | Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Change at Month 12 | -0.07 10^6 cells per microliter | Standard Deviation 0.415 |
| CAP-1002 | Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Baseline | 4.68 10^6 cells per microliter | Standard Deviation 0.414 |
| CAP-1002 | Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Change at Month 6 | 0.20 10^6 cells per microliter | Standard Deviation 0.286 |
| CAP-1002 | Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Change at Month 12 | 0.23 10^6 cells per microliter | Standard Deviation 0.34 |
Primary
Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12
Hematological parameters assessed were: platelets, white blood cells, basophils, eosinophils, lymphocytes, monocytes and neutrophils.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Platelets: Baseline | 357.9 10^3 cells per microliter | Standard Deviation 68.36 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Platelets: Change at Month 6 | -24.5 10^3 cells per microliter | Standard Deviation 51.25 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Platelets: Change at Month 12 | -26.0 10^3 cells per microliter | Standard Deviation 45.72 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | White Blood Cells: Baseline | 9.83 10^3 cells per microliter | Standard Deviation 3.001 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | White Blood Cells: Change at Month 6 | 1.11 10^3 cells per microliter | Standard Deviation 4.01 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | White Blood Cells: Change at Month 12 | 0.42 10^3 cells per microliter | Standard Deviation 2.348 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Basophils: Baseline | 0.033 10^3 cells per microliter | Standard Deviation 0.0218 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Basophils: Change at Month 6 | -0.004 10^3 cells per microliter | Standard Deviation 0.0196 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Basophils: Change at Month 12 | 0.012 10^3 cells per microliter | Standard Deviation 0.0264 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Eosinophils: Baseline | 0.136 10^3 cells per microliter | Standard Deviation 0.1154 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Eosinophils: Change at Month 6 | -0.005 10^3 cells per microliter | Standard Deviation 0.1296 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Eosinophils: Change at Month 12 | 0.067 10^3 cells per microliter | Standard Deviation 0.1742 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Lymphocytes: Baseline | 2.793 10^3 cells per microliter | Standard Deviation 1.0429 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Lymphocytes: Change at Month 6 | -0.595 10^3 cells per microliter | Standard Deviation 1.1509 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Lymphocytes: Change at Month 12 | 0.771 10^3 cells per microliter | Standard Deviation 1.5711 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Monocytes: Baseline | 0.413 10^3 cells per microliter | Standard Deviation 0.1707 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Monocytes: Change at Month 6 | -0.082 10^3 cells per microliter | Standard Deviation 0.2248 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Monocytes: Change at Month 12 | 0.009 10^3 cells per microliter | Standard Deviation 0.2036 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Neutrophils: Baseline | 6.475 10^3 cells per microliter | Standard Deviation 2.7169 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Neutrophils: Change at Month 6 | 1.783 10^3 cells per microliter | Standard Deviation 4.2366 |
| Usual Care | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Neutrophils: Change at Month 12 | -0.451 10^3 cells per microliter | Standard Deviation 2.5038 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Eosinophils: Change at Month 6 | -0.073 10^3 cells per microliter | Standard Deviation 0.1515 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Platelets: Baseline | 318.6 10^3 cells per microliter | Standard Deviation 53.63 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Neutrophils: Baseline | 6.198 10^3 cells per microliter | Standard Deviation 2.9121 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Platelets: Change at Month 6 | 40.4 10^3 cells per microliter | Standard Deviation 45.39 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Eosinophils: Change at Month 12 | -0.078 10^3 cells per microliter | Standard Deviation 0.1366 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Platelets: Change at Month 12 | 24.0 10^3 cells per microliter | Standard Deviation 43.1 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Monocytes: Change at Month 6 | -0.273 10^3 cells per microliter | Standard Deviation 0.4337 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | White Blood Cells: Baseline | 10.57 10^3 cells per microliter | Standard Deviation 3.947 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Lymphocytes: Baseline | 3.381 10^3 cells per microliter | Standard Deviation 1.5423 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | White Blood Cells: Change at Month 6 | 0.81 10^3 cells per microliter | Standard Deviation 2.606 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Neutrophils: Change at Month 12 | 0.566 10^3 cells per microliter | Standard Deviation 1.9689 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | White Blood Cells: Change at Month 12 | -0.28 10^3 cells per microliter | Standard Deviation 1.556 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Lymphocytes: Change at Month 6 | -0.918 10^3 cells per microliter | Standard Deviation 1.9381 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Basophils: Baseline | 0.060 10^3 cells per microliter | Standard Deviation 0.1052 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Monocytes: Change at Month 12 | -0.284 10^3 cells per microliter | Standard Deviation 0.3238 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Basophils: Change at Month 6 | -0.033 10^3 cells per microliter | Standard Deviation 0.1064 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Lymphocytes: Change at Month 12 | -0.393 10^3 cells per microliter | Standard Deviation 1.5501 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Basophils: Change at Month 12 | -0.040 10^3 cells per microliter | Standard Deviation 0.1197 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Neutrophils: Change at Month 6 | 2.153 10^3 cells per microliter | Standard Deviation 3.4484 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Eosinophils: Baseline | 0.171 10^3 cells per microliter | Standard Deviation 0.1848 |
| CAP-1002 | Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 | Monocytes: Baseline | 0.705 10^3 cells per microliter | Standard Deviation 0.2608 |
Primary
Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12
Vital signs assessed were systolic and diastolic blood pressure.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Systolic Blood Pressure: Baseline | 111.3 millimeters of mercury | Standard Deviation 12.29 |
| Usual Care | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Systolic Blood Pressure: Change at Month 6 | 2.3 millimeters of mercury | Standard Deviation 18.7 |
| Usual Care | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Systolic Blood Pressure: Change at Month 12 | -0.3 millimeters of mercury | Standard Deviation 9.66 |
| Usual Care | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Diastolic Blood Pressure: Baseline | 65.9 millimeters of mercury | Standard Deviation 11.92 |
| Usual Care | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Diastolic Blood Pressure: Change at Month 6 | -0.3 millimeters of mercury | Standard Deviation 10.49 |
| Usual Care | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Diastolic Blood Pressure: Change at Month 12 | 3.5 millimeters of mercury | Standard Deviation 8.92 |
| CAP-1002 | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Diastolic Blood Pressure: Change at Month 6 | 0.1 millimeters of mercury | Standard Deviation 14.52 |
| CAP-1002 | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Systolic Blood Pressure: Baseline | 113.7 millimeters of mercury | Standard Deviation 12.72 |
| CAP-1002 | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Diastolic Blood Pressure: Baseline | 66.7 millimeters of mercury | Standard Deviation 14.12 |
| CAP-1002 | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Systolic Blood Pressure: Change at Month 6 | -2.1 millimeters of mercury | Standard Deviation 11.98 |
| CAP-1002 | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Diastolic Blood Pressure: Change at Month 12 | 1.5 millimeters of mercury | Standard Deviation 14.79 |
| CAP-1002 | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Systolic Blood Pressure: Change at Month 12 | 2.8 millimeters of mercury | Standard Deviation 17.67 |
Primary
Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12
Vital signs assessed was heart rate.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Baseline | 90.8 beats per minute | Standard Deviation 9.18 |
| Usual Care | Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Change at Month 6 | -3.0 beats per minute | Standard Deviation 13.42 |
| Usual Care | Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Change at Month 12 | -0.2 beats per minute | Standard Deviation 13.39 |
| CAP-1002 | Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Baseline | 87.1 beats per minute | Standard Deviation 14.71 |
| CAP-1002 | Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Change at Month 6 | 4.8 beats per minute | Standard Deviation 12.43 |
| CAP-1002 | Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Change at Month 12 | 4.4 beats per minute | Standard Deviation 11.24 |
Primary
Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12
Vital signs assessed was respiratory rate.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Baseline | 17.3 breaths per minute | Standard Deviation 2.27 |
| Usual Care | Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Change at Month 6 | -0.5 breaths per minute | Standard Deviation 1.75 |
| Usual Care | Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Change at Month 12 | 0.0 breaths per minute | Standard Deviation 2 |
| CAP-1002 | Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Baseline | 16.3 breaths per minute | Standard Deviation 2.81 |
| CAP-1002 | Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Change at Month 6 | 0.3 breaths per minute | Standard Deviation 3.71 |
| CAP-1002 | Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Change at Month 12 | 0.8 breaths per minute | Standard Deviation 4.21 |
Primary
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12
Vital signs assessed was temperature.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Baseline | 36.79 celsius | Standard Deviation 0.362 |
| Usual Care | Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Change at Month 6 | -0.13 celsius | Standard Deviation 0.49 |
| Usual Care | Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Change at Month 12 | -0.09 celsius | Standard Deviation 0.276 |
| CAP-1002 | Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Baseline | 36.68 celsius | Standard Deviation 0.36 |
| CAP-1002 | Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Change at Month 6 | 0.18 celsius | Standard Deviation 0.545 |
| CAP-1002 | Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Change at Month 12 | 0.05 celsius | Standard Deviation 0.609 |
Primary
Number of Participants Experiencing Any of the Adjudicated Events
Adjudicated Events reported included: New thrombolysis in myocardial infarction (TIMI) flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2noted immediately following infusion and persisting greater than (\>) 3 minutes, despite intracoronary vasodilator administration; sudden unexpected death within 72 hours of intracoronary infusion; and Major adverse cardiac event (MACE) within 72 hours of intracoronary infusion, including death, non-fatal myocardial infarction and hospitalization for cardiovascular event (including heart failure hospitalizations).
Time frame: Within 72 hours post-infusion
Population: Analysis was performed on safety population that included all participants who received CAP-1002 treatment on Day 0 and all participants in the usual care treatment group who remained in study as of the reference time point. Participants were summarized and analyzed per treatment actually received, regardless of their randomization assignment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Usual Care | Number of Participants Experiencing Any of the Adjudicated Events | TIMI 0-2 | 0 Participants |
| Usual Care | Number of Participants Experiencing Any of the Adjudicated Events | Sudden unexpected death | 0 Participants |
| Usual Care | Number of Participants Experiencing Any of the Adjudicated Events | MACE | 0 Participants |
| CAP-1002 | Number of Participants Experiencing Any of the Adjudicated Events | TIMI 0-2 | 0 Participants |
| CAP-1002 | Number of Participants Experiencing Any of the Adjudicated Events | Sudden unexpected death | 0 Participants |
| CAP-1002 | Number of Participants Experiencing Any of the Adjudicated Events | MACE | 0 Participants |
Primary
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12
Cardiac physical examination parameters assessed were: jugular vein distension, edema, heart sounds, murmur, breath sounds.
Time frame: Baseline, Month 6 and Month 12
Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Edema: Month 6 | 1 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Heart Sounds: Month 12 | 1 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Jugular Vein Distension: Month 12 | 0 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Murmur: Month 6 | 0 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Edema: Month 12 | 0 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Murmur: Month 12 | 0 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Breath Sounds: Month 6 | 1 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Jugular Vein Distension: Month 6 | 0 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Breath Sounds: Month 12 | 1 Participants |
| Usual Care | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Heart Sounds: Month 6 | 1 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Breath Sounds: Month 12 | 2 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Jugular Vein Distension: Month 6 | 0 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Jugular Vein Distension: Month 12 | 0 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Edema: Month 6 | 1 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Edema: Month 12 | 1 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Heart Sounds: Month 6 | 0 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Heart Sounds: Month 12 | 1 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Murmur: Month 6 | 0 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Breath Sounds: Month 6 | 0 Participants |
| CAP-1002 | Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 | Murmur: Month 12 | 0 Participants |
Primary
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
An Adverse Event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily had to have causal relationship with treatment. TEAEs were defined as AEs that occurred or worsened in severity between the first dose of the investigational medicinal product (IMP) until the end of study. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.
Time frame: Up to Month 12 post-infusion
Population: Analysis was performed on safety population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Usual Care | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | TEAEs | 10 Participants |
| Usual Care | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | SAEs | 1 Participants |
| CAP-1002 | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | TEAEs | 12 Participants |
| CAP-1002 | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | SAEs | 3 Participants |
Other Pre-specified
Absolute Change From Baseline in Circumferential Strain at Month 6 and 12
Circumferential strain represents the change in length (shortening in systole, represented as a negative strain value) of the myocardium along the circumferential axis of the LV as viewed in the short axis. Improvement in Circumferential Strain was expressed in percentage and was assessed by cardiac MRI.
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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Absolute Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 6 | -0.586 percentage circumferential strain | Standard Deviation 2.9542 |
| Usual Care | Absolute Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 12 | -0.173 percentage circumferential strain | Standard Deviation 2.6342 |
| CAP-1002 | Absolute Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 6 | -0.616 percentage circumferential strain | Standard Deviation 1.7732 |
| CAP-1002 | Absolute Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 12 | -0.707 percentage circumferential strain | Standard Deviation 2.4454 |
Other Pre-specified
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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | 0.74 percentage ejection fraction | Standard Deviation 5.497 |
| Usual Care | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | 0.624 percentage ejection fraction | Standard Deviation 4.396 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | 1.07 percentage ejection fraction | Standard Deviation 3.376 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | -0.35 percentage ejection fraction | Standard Deviation 2.703 |
Other Pre-specified
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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | 3.44 milliliters (mL) | Standard Deviation 14.311 |
| Usual Care | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 3.21 milliliters (mL) | Standard Deviation 12.309 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | 0.14 milliliters (mL) | Standard Deviation 8.538 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 6.16 milliliters (mL) | Standard Deviation 11.655 |
Other Pre-specified
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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | 0.60 mL | Standard Deviation 8.8 |
| Usual Care | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 1.29 mL | Standard Deviation 7.132 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | -0.79 mL | Standard Deviation 5.869 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 4.51 mL | Standard Deviation 8 |
Other Pre-specified
Absolute Change From Baseline in Left Ventricular (LV) Late Gadolinium Enhancement (LGE) at Month 6 and 12
Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. Improvement in infarct size as a percent of LV mass was assessed by magnetic resonance imaging and reported in this outcome measure.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on modified intent-to-treat (mITT) population that included all participants who received CAP-1002 treatment on Day 0 and all participants in the usual care treatment group who remained in study as of the reference time point and had at least one post-baseline observation. Participants were summarized and analyzed per treatment actually received, regardless of their randomization assignment. 'Number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Absolute Change From Baseline in Left Ventricular (LV) Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 6 | -0.53 percent of LV mass | Standard Deviation 2.865 |
| Usual Care | Absolute Change From Baseline in Left Ventricular (LV) Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 12 | -0.09 percent of LV mass | Standard Deviation 5.472 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular (LV) Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 6 | -1.11 percent of LV mass | Standard Deviation 1.63 |
| CAP-1002 | Absolute Change From Baseline in Left Ventricular (LV) Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 12 | -1.35 percent of LV mass | Standard Deviation 1.778 |
Other Pre-specified
Absolute Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12
Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. LV LGE scar tissue mass in grams was assessed by cardiac MRI. 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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Absolute Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 6 | 1.07 grams | Standard Deviation 2.564 |
| Usual Care | Absolute Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 12 | 0.99 grams | Standard Deviation 5.163 |
| CAP-1002 | Absolute Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 6 | 0.29 grams | Standard Deviation 3.038 |
| CAP-1002 | Absolute Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 12 | -0.15 grams | Standard Deviation 3.302 |
Other Pre-specified
Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12
Osteopontin, Cardiac stress biomarker (ST2) and Galectin-3 were DMD biomarkers assessed through serum samples.
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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Galectin-3: Month 6 | 0.368 nanograms per milliliter | Standard Deviation 1.8119 |
| Usual Care | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Galectin-3: Month 12 | 2.958 nanograms per milliliter | Standard Deviation 4.9781 |
| Usual Care | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Osteopontin: Month 6 | 9.238 nanograms per milliliter | Standard Deviation 24.0751 |
| Usual Care | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Osteopontin: Month 12 | 5.400 nanograms per milliliter | Standard Deviation 31.2415 |
| Usual Care | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | ST2: Month 6 | 3.99 nanograms per milliliter | Standard Deviation 15.574 |
| Usual Care | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | ST2: Month 12 | -7.61 nanograms per milliliter | Standard Deviation 14.928 |
| CAP-1002 | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | ST2: Month 6 | -9.43 nanograms per milliliter | Standard Deviation 16.294 |
| CAP-1002 | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Galectin-3: Month 6 | -0.175 nanograms per milliliter | Standard Deviation 2.9025 |
| CAP-1002 | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Osteopontin: Month 12 | 14.868 nanograms per milliliter | Standard Deviation 18.1615 |
| CAP-1002 | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Galectin-3: Month 12 | 0.829 nanograms per milliliter | Standard Deviation 1.3238 |
| CAP-1002 | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | ST2: Month 12 | -21.10 nanograms per milliliter | Standard Deviation 19.637 |
| CAP-1002 | Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12 | Osteopontin: Month 6 | 8.051 nanograms per milliliter | Standard Deviation 14.653 |
Other Pre-specified
Change From Baseline in Forced Expiratory Flow at 25-75% of FVC (FEF25-75%) at Month 6 and 12
Forced Expiratory Flow (FEF) is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF25-75% is defined as the mean forced expiratory flow between the 25% and 75% of the FVC (total amount of air exhaled from the lungs during the lung function test).
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Forced Expiratory Flow at 25-75% of FVC (FEF25-75%) at Month 6 and 12 | Month 12 | 0.437 liter per second | Standard Deviation 0.7612 |
| Usual Care | Change From Baseline in Forced Expiratory Flow at 25-75% of FVC (FEF25-75%) at Month 6 and 12 | Month 6 | 0.097 liter per second | Standard Deviation 1.0146 |
| CAP-1002 | Change From Baseline in Forced Expiratory Flow at 25-75% of FVC (FEF25-75%) at Month 6 and 12 | Month 6 | 0.139 liter per second | Standard Deviation 0.9876 |
| CAP-1002 | Change From Baseline in Forced Expiratory Flow at 25-75% of FVC (FEF25-75%) at Month 6 and 12 | Month 12 | 0.166 liter per second | Standard Deviation 1.0708 |
Other Pre-specified
Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12
FET is defined as the time taken for an individual to complete a forceful exhalation after maximal inspiration. FET is measured by asking the subject to take a deep breath and blow it all out as fast as possible.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12 | Month 6 | -0.433 seconds | Standard Deviation 1.5197 |
| Usual Care | Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12 | Month 12 | 0.086 seconds | Standard Deviation 0.6403 |
| CAP-1002 | Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12 | Month 6 | 0.547 seconds | Standard Deviation 2.6335 |
| CAP-1002 | Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12 | Month 12 | 0.181 seconds | Standard Deviation 2.5598 |
Other Pre-specified
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 6 and 12
FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 6 and 12 | Month 6 | 0.020 mL | Standard Deviation 0.3516 |
| Usual Care | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 6 and 12 | Month 12 | 0.121 mL | Standard Deviation 0.3145 |
| CAP-1002 | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 6 and 12 | Month 6 | -0.007 mL | Standard Deviation 0.2493 |
| CAP-1002 | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 6 and 12 | Month 12 | -0.015 mL | Standard Deviation 0.2297 |
Other Pre-specified
Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12
FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12 | Month 6 | -0.028 mL | Standard Deviation 0.2732 |
| Usual Care | Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12 | Month 12 | 0.000 mL | Standard Deviation 0.224 |
| CAP-1002 | Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12 | Month 6 | 0.007 mL | Standard Deviation 0.3101 |
| CAP-1002 | Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12 | Month 12 | -0.014 mL | Standard Deviation 0.2383 |
Other Pre-specified
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 change from baseline was assessed by magnetic resonance imaging. 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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | -8.20 percent | Standard Deviation 14.763 |
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | 2.28 percent | Standard Deviation 10.342 |
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | -2.73 percent | Standard Deviation 11.487 |
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | 3.94 percent | Standard Deviation 13.011 |
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | -4.52 percent | Standard Deviation 9.706 |
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | -2.74 percent | Standard Deviation 9.739 |
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | -0.59 percent | Standard Deviation 14.53 |
| Usual Care | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | 0.86 percent | Standard Deviation 12.177 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | 4.94 percent | Standard Deviation 11.981 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | 2.72 percent | Standard Deviation 14.256 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | 7.78 percent | Standard Deviation 11.47 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | 5.17 percent | Standard Deviation 11.757 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | 1.01 percent | Standard Deviation 13.728 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | 5.21 percent | Standard Deviation 12.704 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | 7.98 percent | Standard Deviation 12.494 |
| CAP-1002 | Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | 6.16 percent | Standard Deviation 8.353 |
Other Pre-specified
Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12
The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12 | Month 6 | -0.028 liter per second | Standard Deviation 0.7839 |
| Usual Care | Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12 | Month 12 | 0.095 liter per second | Standard Deviation 0.6134 |
| CAP-1002 | Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12 | Month 6 | -0.086 liter per second | Standard Deviation 1.2507 |
| CAP-1002 | Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12 | Month 12 | -0.101 liter per second | Standard Deviation 1.4427 |
Other Pre-specified
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12
The PODCI consists of 83 items and 5 core scales: Upper Extremity and Physical Function, Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort, and Happiness; a Global Functioning Scale. The Global Functioning Scale is the mean of the mean scores from 4 of the 5 core scales (all except the happiness core scale). The Global Functioning Scale and each of the core scales were standardized so that a score of 0 represents a poor outcome/worse health, while 100 is the best possible outcome/best health (i.e., complete range of each score is 0 to 100, with higher scores representing better functioning).
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 6 | 3.9 score on a scale | Standard Deviation 9.81 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 12 | -3.9 score on a scale | Standard Deviation 5.51 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 6 | -2.8 score on a scale | Standard Deviation 9.69 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 12 | -7.9 score on a scale | Standard Deviation 8.91 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Sports/Physical Functioning: Month 6 | 0.0 score on a scale | Standard Deviation 7.48 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Sports/Physical Functioning: Month 12 | -0.9 score on a scale | Standard Deviation 16.35 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Pain/Comfort: Month 6 | 8.6 score on a scale | Standard Deviation 22.38 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Pain/Comfort: Month 12 | 1.8 score on a scale | Standard Deviation 22.58 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Happiness: Month 6 | -2.5 score on a scale | Standard Deviation 16.2 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Happiness: Month 12 | -16.5 score on a scale | Standard Deviation 14.54 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Global Function: Month 6 | 2.6 score on a scale | Standard Deviation 9 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Global Function: Month 12 | -2.5 score on a scale | Standard Deviation 8.46 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Global Function: Month 6 | -0.8 score on a scale | Standard Deviation 8.01 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 6 | -2.5 score on a scale | Standard Deviation 13.17 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Pain/Comfort: Month 6 | 2.0 score on a scale | Standard Deviation 11.73 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 12 | -2.5 score on a scale | Standard Deviation 12.36 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Happiness: Month 12 | 8.1 score on a scale | Standard Deviation 10.71 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 6 | -1.2 score on a scale | Standard Deviation 6.19 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Pain/Comfort: Month 12 | -1.5 score on a scale | Standard Deviation 20.39 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 12 | -2.3 score on a scale | Standard Deviation 8.71 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Global Function: Month 12 | -2.1 score on a scale | Standard Deviation 8.15 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Sports/Physical Functioning: Month 6 | -1.6 score on a scale | Standard Deviation 10.21 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Happiness: Month 6 | 4.2 score on a scale | Standard Deviation 13.2 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12 | Sports/Physical Functioning: Month 12 | -2.9 score on a scale | Standard Deviation 9.6 |
Other Pre-specified
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12
The PODCI consists of 83 items and 5 core scales: Upper Extremity and Physical Function, Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort, and Happiness; a Global Functioning Scale. The Global Functioning Scale is the mean of the mean scores from 4 of the 5 core scales (all except the happiness core scale). The Global Functioning Scale and each of the core scales were standardized so that a score of 0 represents a poor outcome/worse health, while 100 is the best possible outcome/best health (i.e., complete range of each score is 0 to 100, with higher scores representing better functioning).
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 6 | -3.7 score on a scale | Standard Deviation 6.04 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 12 | -5.1 score on a scale | Standard Deviation 8.82 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 6 | -5.9 score on a scale | Standard Deviation 8.44 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 12 | -11.2 score on a scale | Standard Deviation 9.86 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Sports/Physical Functioning: Month 6 | -9.9 score on a scale | Standard Deviation 10.89 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Sports/Physical Functioning: Month 12 | -11.4 score on a scale | Standard Deviation 12.66 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Pain/Comfort: Month 6 | 6.4 score on a scale | Standard Deviation 16.75 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Pain/Comfort: Month 12 | 6.2 score on a scale | Standard Deviation 24.13 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Happiness: Month 6 | 2.0 score on a scale | Standard Deviation 14.94 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Happiness: Month 12 | -8.3 score on a scale | Standard Deviation 21.21 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Global Function: Month 6 | -3.3 score on a scale | Standard Deviation 4 |
| Usual Care | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Global Function: Month 12 | -5.4 score on a scale | Standard Deviation 4.59 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Global Function: Month 6 | -2.3 score on a scale | Standard Deviation 4.46 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 6 | -3.2 score on a scale | Standard Deviation 8.84 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Pain/Comfort: Month 6 | 1.0 score on a scale | Standard Deviation 13.34 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Upper Extremity and Physical Function: Month 12 | -1.8 score on a scale | Standard Deviation 14.25 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Happiness: Month 12 | 12.3 score on a scale | Standard Deviation 27.96 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 6 | -4.2 score on a scale | Standard Deviation 6.37 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Pain/Comfort: Month 12 | 2.2 score on a scale | Standard Deviation 13.03 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Transfer and Basic Mobility: Month 12 | -2.7 score on a scale | Standard Deviation 9.7 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Global Function: Month 12 | -2.2 score on a scale | Standard Deviation 5.73 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Sports/Physical Functioning: Month 6 | -2.5 score on a scale | Standard Deviation 7.34 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Happiness: Month 6 | 2.3 score on a scale | Standard Deviation 31.07 |
| CAP-1002 | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12 | Sports/Physical Functioning: Month 12 | -6.8 score on a scale | Standard Deviation 13.06 |
Other Pre-specified
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Summary Score - Participant Responses at Month 6 and 12
The PedsQL-Duchenne muscular dystrophy (DMD) module consists of 18 items and comprised of 4 domains: Daily Activities, Treatment Barriers, Worry, Communication. Items are reverse scored and linearly transformed to a 0-100 scale. The overall range for total PedsQL score (18 items; mean of all items) was 0 (improvement) to 100 (severity). Decreasing scores over time indicate improvement. Change From Baseline in PedsQL total summary score- participant responses at Month 6 and 12 was reported in this outcome measure.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Summary Score - Participant Responses at Month 6 and 12 | Month 6 | -9.83 score on a scale | Standard Deviation 10.507 |
| Usual Care | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Summary Score - Participant Responses at Month 6 and 12 | Month 12 | -11.24 score on a scale | Standard Deviation 11.293 |
| CAP-1002 | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Summary Score - Participant Responses at Month 6 and 12 | Month 6 | 3.64 score on a scale | Standard Deviation 16.225 |
| CAP-1002 | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Summary Score - Participant Responses at Month 6 and 12 | Month 12 | 4.28 score on a scale | Standard Deviation 16.455 |
Other Pre-specified
Change From Baseline in Pediatric Quality of Life Inventory Total Summary Score - Parent Responses at Month 6 and 12
The PedsQL-Duchenne muscular dystrophy (DMD) module consists of 18 items and comprised of 4 domains: Daily Activities, Treatment Barriers, Worry, Communication. Items are reverse scored and linearly transformed to a 0-100 scale. The overall range for total PedsQL score (18 items; mean of all items) was 0 (improvement) to 100 (severity). Decreasing scores over time indicate improvement. Change From Baseline in PedsQL total summary score- parent responses at Month 6 and 12 was reported in this outcome measure.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Pediatric Quality of Life Inventory Total Summary Score - Parent Responses at Month 6 and 12 | Month 6 | -1.40 score on a scale | Standard Deviation 13.495 |
| Usual Care | Change From Baseline in Pediatric Quality of Life Inventory Total Summary Score - Parent Responses at Month 6 and 12 | Month 12 | -6.69 score on a scale | Standard Deviation 7.627 |
| CAP-1002 | Change From Baseline in Pediatric Quality of Life Inventory Total Summary Score - Parent Responses at Month 6 and 12 | Month 6 | -0.21 score on a scale | Standard Deviation 17.334 |
| CAP-1002 | Change From Baseline in Pediatric Quality of Life Inventory Total Summary Score - Parent Responses at Month 6 and 12 | Month 12 | -0.43 score on a scale | Standard Deviation 19.429 |
Other Pre-specified
Change From Baseline in Percent Predicted Forced Expiratory Flow at 25-75% of FVC at Month 6 and 12
FEF is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF25-75% is defined as the mean forced expiratory flow between the 25% and 75% of the FVC (total amount of air exhaled from the lungs during the lung function test).
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Percent Predicted Forced Expiratory Flow at 25-75% of FVC at Month 6 and 12 | Month 6 | 0.2 percent predicted FEF | Standard Deviation 23.05 |
| Usual Care | Change From Baseline in Percent Predicted Forced Expiratory Flow at 25-75% of FVC at Month 6 and 12 | Month 12 | 5.6 percent predicted FEF | Standard Deviation 20.34 |
| CAP-1002 | Change From Baseline in Percent Predicted Forced Expiratory Flow at 25-75% of FVC at Month 6 and 12 | Month 6 | 1.1 percent predicted FEF | Standard Deviation 23.65 |
| CAP-1002 | Change From Baseline in Percent Predicted Forced Expiratory Flow at 25-75% of FVC at Month 6 and 12 | Month 12 | 0.9 percent predicted FEF | Standard Deviation 26.24 |
Other Pre-specified
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second at Month 6 and 12
FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second at Month 6 and 12 | Month 6 | -1.6 percent predicted FEV1 | Standard Deviation 13.88 |
| Usual Care | Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second at Month 6 and 12 | Month 12 | -2.1 percent predicted FEV1 | Standard Deviation 14.15 |
| CAP-1002 | Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second at Month 6 and 12 | Month 6 | -3.2 percent predicted FEV1 | Standard Deviation 7.82 |
| CAP-1002 | Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second at Month 6 and 12 | Month 12 | -4.3 percent predicted FEV1 | Standard Deviation 7.47 |
Other Pre-specified
Change From Baseline in Percent Predicted Forced Vital Capacity Level at Month 6 and 12
FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Percent Predicted Forced Vital Capacity Level at Month 6 and 12 | Month 6 | -3.6 percent predicted FVC | Standard Deviation 14.28 |
| Usual Care | Change From Baseline in Percent Predicted Forced Vital Capacity Level at Month 6 and 12 | Month 12 | -6.0 percent predicted FVC | Standard Deviation 12.13 |
| CAP-1002 | Change From Baseline in Percent Predicted Forced Vital Capacity Level at Month 6 and 12 | Month 6 | -3.1 percent predicted FVC | Standard Deviation 8.06 |
| CAP-1002 | Change From Baseline in Percent Predicted Forced Vital Capacity Level at Month 6 and 12 | Month 12 | -4.4 percent predicted FVC | Standard Deviation 7.17 |
Other Pre-specified
Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12
Percent predicted PEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort-dependent measure of lung function.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12 | Month 6 | -3.2 percent predicted PEF | Standard Deviation 10.45 |
| Usual Care | Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12 | Month 12 | -4.5 percent predicted PEF | Standard Deviation 8.9 |
| CAP-1002 | Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12 | Month 6 | -4.8 percent predicted PEF | Standard Deviation 17.53 |
| CAP-1002 | Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12 | Month 12 | -7.1 percent predicted PEF | Standard Deviation 20.46 |
Other Pre-specified
Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Excluding Shoulder) at Month 6 and 12
The PUL Scale consists of an entry item to define the starting functional level and 21 items subdivided into shoulder level (4 items), middle level (9 items), and distal level (8 items) dimensions. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for middle level, and 24 for distal level. The total score is calculated by the sum of all the scores of the three subscales (excluding the shoulder dimension) and ranged from 0 to 58. Higher scores indicated improvement and lower scores indicated severity. Change from Baseline in PUL overall score (excluding shoulder) at Month 6 and 12 is reported in this outcome measure.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, and 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Excluding Shoulder) at Month 6 and 12 | Month 6 | -1.8 score on a scale | Standard Deviation 4.38 |
| Usual Care | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Excluding Shoulder) at Month 6 and 12 | Month 12 | -1.2 score on a scale | Standard Deviation 2.64 |
| CAP-1002 | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Excluding Shoulder) at Month 6 and 12 | Month 6 | -0.3 score on a scale | Standard Deviation 2.9 |
| CAP-1002 | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Excluding Shoulder) at Month 6 and 12 | Month 12 | 1.3 score on a scale | Standard Deviation 6.12 |
Other Pre-specified
Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Including Shoulder) at Month 6 and 12
The PUL Scale consists of an entry item to define the starting functional level and 21 items subdivided into shoulder level (4 items,), middle level (9 items), and distal level (8 items) dimensions. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for middle level, and 24 for distal level. The total score is calculated by the sum of all the scores of the three subscales (including the shoulder dimension) and ranged from 0 to 74. Higher scores over time indicate improvement and lower scores indicated severity. Change from Baseline in PUL overall score (including shoulder) at Month 6 and 12 is reported in this outcome measure.
Time frame: Baseline, Month 6 and 12
Population: Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Including Shoulder) at Month 6 and 12 | Month 6 | 0.3 score on a scale | Standard Deviation 2.16 |
| Usual Care | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Including Shoulder) at Month 6 and 12 | Month 12 | -1.8 score on a scale | Standard Deviation 3.6 |
| CAP-1002 | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Including Shoulder) at Month 6 and 12 | Month 6 | -3.7 score on a scale | Standard Deviation 4.04 |
| CAP-1002 | Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Including Shoulder) at Month 6 and 12 | Month 12 | -4.0 score on a scale | Standard Deviation 0 |
Other Pre-specified
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 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 6 | -9.0 meters | Standard Deviation 2.83 |
| Usual Care | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 12 | -8.5 meters | Standard Deviation 0.71 |
| CAP-1002 | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 6 | -68.5 meters | Standard Deviation 21.92 |
| CAP-1002 | Change From Baseline in Six-Minute Walk Test at Month 6 and 12 | Month 12 | -117.0 meters | Standard Deviation 4.24 |
Other Pre-specified
Percent Change From Baseline in Circumferential Strain at Month 6 and 12
Circumferential strain represents the change in length (shortening in systole, represented as a negative strain value) of the myocardium along the circumferential axis of the LV as viewed in the short axis; and was assessed by cardiac MRI. 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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Percent Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 6 | -4.62 percent change | Standard Deviation 24.1 |
| Usual Care | Percent Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 12 | -1.50 percent change | Standard Deviation 21.755 |
| CAP-1002 | Percent Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 12 | -9.17 percent change | Standard Deviation 24.823 |
| CAP-1002 | Percent Change From Baseline in Circumferential Strain at Month 6 and 12 | Month 6 | -6.40 percent change | Standard Deviation 15.198 |
Other Pre-specified
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 percent change from baseline 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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | -14.12 percent change | Standard Deviation 24.899 |
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | -8.78 percent change | Standard Deviation 27.679 |
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | -4.54 percent change | Standard Deviation 35.009 |
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | 12.75 percent change | Standard Deviation 32.836 |
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | 6.40 percent change | Standard Deviation 33.55 |
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | 25.49 percent change | Standard Deviation 63.231 |
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | 8.87 percent change | Standard Deviation 32.843 |
| Usual Care | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | 1.56 percent change | Standard Deviation 37.898 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 12 | 17.17 percent change | Standard Deviation 39.263 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 6 | 16.25 percent change | Standard Deviation 46.546 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Anterior: Month 12 | 21.76 percent change | Standard Deviation 44.713 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 6 | 24.50 percent change | Standard Deviation 51.156 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Lateral: Month 12 | 28.22 percent change | Standard Deviation 40.267 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 6 | 31.19 percent change | Standard Deviation 46.995 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Inferior: Month 12 | 25.75 percent change | Standard Deviation 46.715 |
| CAP-1002 | Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12 | Septal: Month 6 | 9.68 percent change | Standard Deviation 38.06 |
Other Pre-specified
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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | 2.35 percent change | Standard Deviation 10.456 |
| Usual Care | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | 1.88 percent change | Standard Deviation 8.83 |
| CAP-1002 | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 6 | 2.44 percent change | Standard Deviation 7.301 |
| CAP-1002 | Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 | Month 12 | -0.88 percent change | Standard Deviation 5.58 |
Other Pre-specified
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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | 2.13 percent change | Standard Deviation 9.851 |
| Usual Care | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 1.86 percent change | Standard Deviation 9.567 |
| CAP-1002 | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 6 | -0.28 percent change | Standard Deviation 7.246 |
| CAP-1002 | Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12 | Month 12 | 3.91 percent change | Standard Deviation 8.479 |
Other Pre-specified
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, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | 0.96 percent change | Standard Deviation 10.987 |
| Usual Care | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 1.17 percent change | Standard Deviation 12.706 |
| CAP-1002 | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 6 | -2.12 percent change | Standard Deviation 10.558 |
| CAP-1002 | Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 | Month 12 | 4.43 percent change | Standard Deviation 9.881 |
Other Pre-specified
Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) at Month 6 and 12
Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. 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 6 and 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 6 | -0.23 percent change | Standard Deviation 11.482 |
| Usual Care | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 12 | 4.76 percent change | Standard Deviation 22.253 |
| CAP-1002 | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 6 | -5.09 percent change | Standard Deviation 8.48 |
| CAP-1002 | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) at Month 6 and 12 | Month 12 | -7.14 percent change | Standard Deviation 10.298 |
Other Pre-specified
Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12
Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. Percent change in LV LGE scar tissue 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 6 and Month 12
Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Usual Care | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 6 | 9.42 percent change | Standard Deviation 14.056 |
| Usual Care | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 12 | 10.86 percent change | Standard Deviation 23.17 |
| CAP-1002 | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 6 | -0.39 percent change | Standard Deviation 19.032 |
| CAP-1002 | Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12 | Month 12 | -2.81 percent change | Standard Deviation 19.557 |