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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Experimental | CAP-1002 is an investigational product consisting of allogeneic cardiosphere-derived cells (CDCs). All subjects assigned to the active treatment arm will receive an intended total dose of 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior). If any of the three coronary arteries are deemed by the infusing Investigator to supply less than 30% of the left ventricular myocardium, the infusing Investigator may choose to infuse only 12.5M cells into that coronary artery or arteries. Therefore the full dose of CAP-1002 delivered may range from 50M cells to 75M cells provided that all three arteries are infused. |
|
| Usual Care | No Intervention | Subjects randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the subject on an ongoing basis, and will receive no infusion. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Drug | Intracoronary delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | Within 72 hours post-infusion |
| 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. | Up to Month 12 post-infusion |
| 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. | Baseline, Month 6 and Month 12 |
| Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Clinical chemistry parameter assessed was albumin. | Baseline, Month 6 and Month 12 |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. |
Inclusion Criteria:
Exclusion Criteria:
Therapy with intravenous inotropic or vasoactive medications at the time of screening.
Inability to undergo cardiac catheterization and/or MRI without general anesthesia.
Immunologic incompatibility with all available Master Cell Banks (MCBs) by single-antigen bead (SAB) serum antibody profiling.
Planned or likely major surgery in the next 12 months after planned randomization.
Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of acquiring a LVAD.
Contraindication to cardiac MRI.
Known hypersensitivity to contrast agents.
Estimated glomerular filtration rate (GFR) <60 mL/min, as calculated by the CKD-EPI cystatin C equation (Inker, Schmid et al. 2012).
Active infection not responsive to treatment.
Active systemic allergic reaction(s), connective tissue disease or autoimmune disorder(s).
History of cardiac tumor or cardiac tumor demonstrated on screening MRI.
History of previous stem cell therapy.
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.
Known moderate-to-severe aortic stenosis/insufficiency or severe mitral stenosis/regurgitation.
Current active alcohol or drug abuse.
Known history of Human Immunodeficiency Virus (HIV) infection.
Known history of chronic viral hepatitis.
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.
Known hypersensitivity to bovine products.
Known hypersensitivity to dimethyl sulfoxide (DMSO).
Uncontrolled diabetes (HbA1c >9.0).
Inability to comply with protocol-related procedures, including required study visits.
Any condition or other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for the study.
Currently receiving investigational treatment on another clinical study or expanded access protocol, including any of the following:
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| Name | Affiliation | Role |
|---|---|---|
| Mark Awadalla | Capricor Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cedars-Sinai Medical Center | Los Angeles | California | 90048 | United States | ||
| University of Florida |
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.
The study enrolled participants at 3 active sites between 07 January 2016 to 14 September 2017.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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|
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.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | CAP-1002 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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). | 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. | Posted | Count of Participants | Participants | Within 72 hours post-infusion |
|
Adverse Events data was collected from first infusion up to 12 Month post-infusion
Analysis was performed on safety population.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ventricular fibrillation | Cardiac disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial Fibrillation | Cardiac disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Vice President of Clinical Research and Development Operations | Capricor, Inc. | 858-727-1755 | clinicaltrialsgov@capricor.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 18, 2016 | Feb 29, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 6, 2017 | Feb 29, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D009202 | Cardiomyopathies |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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|
| Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 |
Clinical chemistry parameter assessed was glucose. |
| Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Hematological parameter assessed was hemoglobin. | Baseline, Month 6 and Month 12 |
| Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Hematological parameter assessed was red blood cells. | Baseline, Month 6 and Month 12 |
| Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Vital signs assessed were systolic and diastolic blood pressure. | Baseline, Month 6 and Month 12 |
| Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Vital signs assessed was heart rate. | Baseline, Month 6 and Month 12 |
| Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Vital signs assessed was respiratory rate. | Baseline, Month 6 and Month 12 |
| Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Vital signs assessed was temperature. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| Baseline, Month 6 and Month 12 |
| 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%. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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%. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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%. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and 12 |
| 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%. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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%. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and Month 12 |
| 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%. | Baseline, Month 6 and Month 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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). | Baseline, Month 6 and 12 |
| 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). | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and 12 |
| 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). | Baseline, Month 6 and 12 |
| 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). | Baseline, Month 6 and 12 |
| 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. | Baseline, Month 6 and Month 12 |
| Gainesville |
| Florida |
| 32611 |
| United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
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. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| OG000 |
| 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. |
| OG001 | 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. |
|
|
| 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. | Analysis was performed on safety population. | Posted | Count of Participants | Participants | Up to Month 12 post-infusion |
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| 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. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | millimoles per liter (mmol/L) | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 | Clinical chemistry parameter assessed was albumin. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | grams per deciliter | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 | Clinical chemistry parameter assessed was glucose. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | milligrams per deciliter | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | 10^3 cells per microliter | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 | Hematological parameter assessed was hemoglobin. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | grams per deciliter | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 | Hematological parameter assessed was red blood cells. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | 10^6 cells per microliter | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 | Vital signs assessed were systolic and diastolic blood pressure. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | millimeters of mercury | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 | Vital signs assessed was heart rate. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | beats per minute | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 | Vital signs assessed was respiratory rate. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | breaths per minute | Baseline, Month 6 and Month 12 |
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| Primary | Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 | Vital signs assessed was temperature. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | celsius | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Count of Participants | Participants | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | millisecond | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | beats per minute | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percentage ejection fraction | Baseline, Month 6 and Month 12 |
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| 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%. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | milliliters (mL) | Baseline, Month 6 and Month 12 |
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| 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%. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | mL | Baseline, Month 6 and Month 12 |
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| 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%. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | grams | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 and Month 12 |
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| 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. | 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. | Posted | Mean | Standard Deviation | percent of LV mass | Baseline, Month 6 and 12 |
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| 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%. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percentage circumferential strain | Baseline, Month 6 and Month 12 |
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| 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%. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent | Baseline, Month 6 and Month 12 |
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| 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%. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 and Month 12 |
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| 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. | Analysis was performed on mITT population. Here, and 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | liter per second | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent predicted PEF | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | mL | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent predicted FEV1 | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent predicted FVC | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | mL | Baseline, Month 6 and 12 |
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| 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). | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | liter per second | Baseline, Month 6 and 12 |
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| 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). | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | percent predicted FEF | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | seconds | Baseline, Month 6 and 12 |
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| 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. | 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. | Posted | Mean | Standard Deviation | meters | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 6 and 12 |
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| 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). | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 6 and 12 |
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| 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). | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 6 and 12 |
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| 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. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | Mean | Standard Deviation | nanograms per milliliter | Baseline, Month 6 and Month 12 |
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| 0 |
| 12 |
| 1 |
| 12 |
| 9 |
| 12 |
| EG001 | 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. | 0 | 13 | 3 | 13 | 9 | 13 |
| Pyrexia | General disorders | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | Systematic Assessment |
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| Femur fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Confusional state | Psychiatric disorders | Systematic Assessment |
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| Atrioventricular Block Second Degree | Cardiac disorders | Systematic Assessment |
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| Bradycardia | Cardiac disorders | Systematic Assessment |
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| Palpitations | Cardiac disorders | Systematic Assessment |
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| Supraventricular Tachycardia | Cardiac disorders | Systematic Assessment |
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| Ventricular Tachycardia | Cardiac disorders | Systematic Assessment |
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| Abdominal Discomfort | Gastrointestinal disorders | Systematic Assessment |
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| Haemorrhoids | Gastrointestinal disorders | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | Systematic Assessment |
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| Catheter Site Pain | General disorders | Systematic Assessment |
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| Body Tinea | Infections and infestations | Systematic Assessment |
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| Gastroenteritis Viral | Infections and infestations | Systematic Assessment |
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| Gastrointestinal infection | Infections and infestations | Systematic Assessment |
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| Sinusitis | Infections and infestations | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
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| Fibula Fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Ligament Sprain | Injury, poisoning and procedural complications | Systematic Assessment |
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| Radius Fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Tibia Fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Heart Rate Irregular | Investigations | Systematic Assessment |
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| Monocyte Count Decreased | Investigations | Systematic Assessment |
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| Spirometry abnormal | Investigations | Systematic Assessment |
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| Troponin Increased | Investigations | Systematic Assessment |
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| Weight Increased | Investigations | Systematic Assessment |
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| Insulin Resistance | Metabolism and nutrition disorders | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Back Pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Muscle Spasms | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Musculoskeletal Chest Pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Melanocytic Naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
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| Dizziness | Nervous system disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Haematuria | Renal and urinary disorders | Systematic Assessment |
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| Nephrolithiasis | Renal and urinary disorders | Systematic Assessment |
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| Sinus Congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Dermatitis Contact | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Ecchymosis | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Haematoma | Vascular disorders | Systematic Assessment |
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| Femur Fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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Not provided
Not provided
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| Chloride: Change at Month 6 |
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| Chloride: Change at Month 12 |
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| Potassium: Baseline |
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| Potassium: Change at Month 6 |
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| Potassium: Change at Month 12 |
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| Sodium: Baseline |
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| Sodium: Change at Month 6 |
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| Sodium: Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Platelets: Change at Month 6 |
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| Platelets: Change at Month 12 |
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| White Blood Cells: Baseline |
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| White Blood Cells: Change at Month 6 |
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| White Blood Cells: Change at Month 12 |
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| Basophils: Baseline |
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| Basophils: Change at Month 6 |
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| Basophils: Change at Month 12 |
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| Eosinophils: Baseline |
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| Eosinophils: Change at Month 6 |
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| Eosinophils: Change at Month 12 |
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| Lymphocytes: Baseline |
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| Lymphocytes: Change at Month 6 |
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| Lymphocytes: Change at Month 12 |
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| Monocytes: Baseline |
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| Monocytes: Change at Month 6 |
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| Monocytes: Change at Month 12 |
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| Neutrophils: Baseline |
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| Neutrophils: Change at Month 6 |
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| Neutrophils: Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Systolic Blood Pressure: Change at Month 6 |
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| Systolic Blood Pressure: Change at Month 12 |
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| Diastolic Blood Pressure: Baseline |
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| Diastolic Blood Pressure: Change at Month 6 |
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| Diastolic Blood Pressure: Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Jugular Vein Distension: Month 12 |
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| Edema: Month 6 |
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| Edema: Month 12 |
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| Heart Sounds: Month 6 |
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| Heart Sounds: Month 12 |
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| Murmur: Month 6 |
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| Murmur: Month 12 |
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| Breath Sounds: Month 6 |
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| Breath Sounds: Month 12 |
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| PR Interval: Change at Month 6 |
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| PR Interval: Change at Month 12 |
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| QRS Duration: Baseline |
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| QRS Duration: Change at Month 6 |
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| QRS Duration: Change at Month 12 |
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| QT Interval: Baseline |
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| QT Interval: Change at Month 6 |
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| QT Interval: Change at Month 12 |
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| QTc Interval: Baseline |
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| QTc Interval: Change at Month 6 |
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| QTc Interval: Change at Month 12 |
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| Change at Month 6 |
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| Change at Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Anterior: Month 12 |
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| Lateral: Month 6 |
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| Lateral: Month 12 |
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| Inferior: Month 6 |
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| Inferior: Month 12 |
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| Septal: Month 6 |
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| Septal: Month 12 |
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| Anterior: Month 12 |
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| Lateral: Month 6 |
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| Lateral: Month 12 |
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| Inferior: Month 6 |
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| Inferior: Month 12 |
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| Septal: Month 6 |
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| Septal: Month 12 |
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| Month 12 |
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| Month 12 |
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| Month 12 |
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| Upper Extremity and Physical Function: Month 12 |
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| Transfer and Basic Mobility: Month 6 |
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| Transfer and Basic Mobility: Month 12 |
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| Sports/Physical Functioning: Month 6 |
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| Sports/Physical Functioning: Month 12 |
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| Pain/Comfort: Month 6 |
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| Pain/Comfort: Month 12 |
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| Happiness: Month 6 |
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| Happiness: Month 12 |
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| Global Function: Month 6 |
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| Global Function: Month 12 |
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| Upper Extremity and Physical Function: Month 12 |
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| Transfer and Basic Mobility: Month 6 |
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| Transfer and Basic Mobility: Month 12 |
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| Sports/Physical Functioning: Month 6 |
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| Sports/Physical Functioning: Month 12 |
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|
| Pain/Comfort: Month 6 |
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|
| Pain/Comfort: Month 12 |
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|
| Happiness: Month 6 |
|
|
| Happiness: Month 12 |
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|
| Global Function: Month 6 |
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|
| Global Function: Month 12 |
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| Galectin-3: Month 12 |
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| Osteopontin: Month 6 |
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| Osteopontin: Month 12 |
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| ST2: Month 6 |
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| ST2: Month 12 |
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