Trial Outcomes & Findings for HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) (NCT NCT02485938)

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

25 participants

Primary outcome timeframe

Within 72 hours post-infusion

Results posted on

2025-01-09

Participant Flow

The study enrolled participants at 3 active sites between 07 January 2016 to 14 September 2017.

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.

Participant milestones

Participant milestones
Measure	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.	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.
Overall Study STARTED	12	13
Overall Study COMPLETED	10	13
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Reasons for withdrawal
Measure	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.	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.
Overall Study Withdrawal of consent	2	0

Baseline Characteristics

HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.	Total n=25 Participants Total of all reporting groups
Age, Continuous	16.9 years STANDARD_DEVIATION 2.75 • n=5 Participants	18.7 years STANDARD_DEVIATION 3.50 • n=7 Participants	17.8 years STANDARD_DEVIATION 3.22 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	13 Participants n=7 Participants	25 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants	11 Participants n=7 Participants	21 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

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).

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Number of Participants Experiencing Any of the Adjudicated Events MACE	0 Participants	0 Participants
Number of Participants Experiencing Any of the Adjudicated Events TIMI 0-2	0 Participants	0 Participants
Number of Participants Experiencing Any of the Adjudicated Events Sudden unexpected death	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to Month 12 post-infusion

Population: Analysis was performed on safety population.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) TEAEs	10 Participants	12 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) SAEs	1 Participants	3 Participants

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Clinical chemistry parameters assessed were chloride, potassium and sodium.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
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	102.7 millimoles per liter (mmol/L) Standard Deviation 3.52
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	-0.8 millimoles per liter (mmol/L) Standard Deviation 4.09
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	-0.9 millimoles per liter (mmol/L) Standard Deviation 3.95
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	4.00 millimoles per liter (mmol/L) Standard Deviation 0.356
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	0.22 millimoles per liter (mmol/L) Standard Deviation 0.286
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	0.31 millimoles per liter (mmol/L) Standard Deviation 0.362
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	140.2 millimoles per liter (mmol/L) Standard Deviation 2.51
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 2.90	-1.5 millimoles per liter (mmol/L) Standard Deviation 3.15
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	-1.2 millimoles per liter (mmol/L) Standard Deviation 2.83

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Clinical chemistry parameter assessed was albumin.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 Baseline	4.48 grams per deciliter Standard Deviation 0.266	3.87 grams per deciliter Standard Deviation 0.417
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	0.36 grams per deciliter Standard Deviation 0.500
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	0.32 grams per deciliter Standard Deviation 0.402

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Clinical chemistry parameter assessed was glucose.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 Baseline	87.1 milligrams per deciliter Standard Deviation 8.20	89.5 milligrams per deciliter Standard Deviation 9.58
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	12.3 milligrams per deciliter Standard Deviation 20.56
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	6.8 milligrams per deciliter Standard Deviation 15.00

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Hematological parameters assessed were: platelets, white blood cells, basophils, eosinophils, lymphocytes, monocytes and neutrophils.

Outcome measures

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Hematological parameter assessed was hemoglobin.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 Baseline	14.48 grams per deciliter Standard Deviation 0.651	13.76 grams per deciliter Standard Deviation 1.254
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	0.05 grams per deciliter Standard Deviation 0.458
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	0.29 grams per deciliter Standard Deviation 0.808

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Hematological parameter assessed was red blood cells.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
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	4.68 10^6 cells per microliter Standard Deviation 0.414
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	0.20 10^6 cells per microliter Standard Deviation 0.286
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	0.23 10^6 cells per microliter Standard Deviation 0.340

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Vital signs assessed were systolic and diastolic blood pressure.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
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	113.7 millimeters of mercury Standard Deviation 12.72
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 18.70	-2.1 millimeters of mercury Standard Deviation 11.98
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	2.8 millimeters of mercury Standard Deviation 17.67
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	66.7 millimeters of mercury Standard Deviation 14.12
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	0.1 millimeters of mercury Standard Deviation 14.52
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	1.5 millimeters of mercury Standard Deviation 14.79

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Vital signs assessed was heart rate.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 Baseline	90.8 beats per minute Standard Deviation 9.18	87.1 beats per minute Standard Deviation 14.71
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	4.8 beats per minute Standard Deviation 12.43
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	4.4 beats per minute Standard Deviation 11.24

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Vital signs assessed was respiratory rate.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 Baseline	17.3 breaths per minute Standard Deviation 2.27	16.3 breaths per minute Standard Deviation 2.81
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	0.3 breaths per minute Standard Deviation 3.71
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 2.00	0.8 breaths per minute Standard Deviation 4.21

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Vital signs assessed was temperature.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 Baseline	36.79 celsius Standard Deviation 0.362	36.68 celsius Standard Deviation 0.360
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 Change at Month 6	-0.13 celsius Standard Deviation 0.490	0.18 celsius Standard Deviation 0.545
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 Change at Month 12	-0.09 celsius Standard Deviation 0.276	0.05 celsius Standard Deviation 0.609

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

Cardiac physical examination parameters assessed were: jugular vein distension, edema, heart sounds, murmur, breath sounds.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
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	0 Participants
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	0 Participants
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 Edema: Month 6	1 Participants	1 Participants
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 Edema: Month 12	0 Participants	1 Participants
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	0 Participants
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	1 Participants
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 Murmur: Month 6	0 Participants	0 Participants
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 Murmur: Month 12	0 Participants	0 Participants
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	0 Participants
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	2 Participants

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

ECG parameters assessed were: PR Interval, QRS Duration, QT Interval, QTc interval and QT interval.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
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	129.2 millisecond Standard Deviation 21.31
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	-2.8 millisecond Standard Deviation 7.67
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 44.40	1.0 millisecond Standard Deviation 12.58
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	91.3 millisecond Standard Deviation 10.03
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	4.5 millisecond Standard Deviation 7.58
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	4.5 millisecond Standard Deviation 6.04
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	348.9 millisecond Standard Deviation 21.55
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	-3.2 millisecond Standard Deviation 18.91
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 23.90	-0.2 millisecond Standard Deviation 18.21
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	423.7 millisecond Standard Deviation 23.60
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	-5.8 millisecond Standard Deviation 23.45
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	1.2 millisecond Standard Deviation 27.23

PRIMARY outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.

ECG parameter assessed was ventricular rate; which depends on the degree of atrioventricular conduction.

Outcome measures

Outcome measures
Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
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	2.2 beats per minute Standard Deviation 14.62
Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 Baseline	87.8 beats per minute Standard Deviation 11.95	89.8 beats per minute Standard Deviation 14.43
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	1.2 beats per minute Standard Deviation 14.66

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	1.07 percentage ejection fraction Standard Deviation 3.376
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	-0.35 percentage ejection fraction Standard Deviation 2.703

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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%.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	2.44 percent change Standard Deviation 7.301
Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12 Month 12	1.88 percent change Standard Deviation 8.830	-0.88 percent change Standard Deviation 5.580

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	0.14 milliliters (mL) Standard Deviation 8.538
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	6.16 milliliters (mL) Standard Deviation 11.655

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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%.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-0.28 percent change Standard Deviation 7.246
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	3.91 percent change Standard Deviation 8.479

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12 Month 6	0.60 mL Standard Deviation 8.800	-0.79 mL Standard Deviation 5.869
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	4.51 mL Standard Deviation 8.000

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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%.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-2.12 percent change Standard Deviation 10.558
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	4.43 percent change Standard Deviation 9.881

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	0.29 grams Standard Deviation 3.038
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	-0.15 grams Standard Deviation 3.302

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-0.39 percent change Standard Deviation 19.032
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	-2.81 percent change Standard Deviation 19.557

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-1.11 percent of LV mass Standard Deviation 1.630
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	-1.35 percent of LV mass Standard Deviation 1.778

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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%.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-7.14 percent change Standard Deviation 10.298
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	-5.09 percent change Standard Deviation 8.480

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
Absolute Change From Baseline in Circumferential Strain at Month 6 and 12 Month 6	-0.586 percentage circumferential strain Standard Deviation 2.9542	-0.616 percentage circumferential strain Standard Deviation 1.7732
Absolute Change From Baseline in Circumferential Strain at Month 6 and 12 Month 12	-0.173 percentage circumferential strain Standard Deviation 2.6342	-0.707 percentage circumferential strain Standard Deviation 2.4454

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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%.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
Percent Change From Baseline in Circumferential Strain at Month 6 and 12 Month 6	-4.62 percent change Standard Deviation 24.100	-6.40 percent change Standard Deviation 15.198
Percent Change From Baseline in Circumferential Strain at Month 6 and 12 Month 12	-1.50 percent change Standard Deviation 21.755	-9.17 percent change Standard Deviation 24.823

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	2.72 percent Standard Deviation 14.256
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	5.17 percent Standard Deviation 11.757
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	5.21 percent Standard Deviation 12.704
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	6.16 percent Standard Deviation 8.353
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	7.78 percent Standard Deviation 11.470
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	7.98 percent Standard Deviation 12.494
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 14.530	1.01 percent Standard Deviation 13.728
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	4.94 percent Standard Deviation 11.981

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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%.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	16.25 percent change Standard Deviation 46.546
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	21.76 percent change Standard Deviation 44.713
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	24.50 percent change Standard Deviation 51.156
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	28.22 percent change Standard Deviation 40.267
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	31.19 percent change Standard Deviation 46.995
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	25.75 percent change Standard Deviation 46.715
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 33.550	9.68 percent change Standard Deviation 38.060
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	17.17 percent change Standard Deviation 39.263

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, and 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-0.3 score on a scale Standard Deviation 2.90
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	1.3 score on a scale Standard Deviation 6.12

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=6 Participants 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.	CAP-1002 n=3 Participants 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.
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	-3.7 score on a scale Standard Deviation 4.04
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 3.60	-4.0 score on a scale Standard Deviation 0.00

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12 Month 12	0.095 liter per second Standard Deviation 0.6134	-0.101 liter per second Standard Deviation 1.4427
Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12 Month 6	-0.028 liter per second Standard Deviation 0.7839	-0.086 liter per second Standard Deviation 1.2507

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-4.8 percent predicted PEF Standard Deviation 17.53
Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12 Month 12	-4.5 percent predicted PEF Standard Deviation 8.90	-7.1 percent predicted PEF Standard Deviation 20.46

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-0.007 mL Standard Deviation 0.2493
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	-0.015 mL Standard Deviation 0.2297

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-3.2 percent predicted FEV1 Standard Deviation 7.82
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	-4.3 percent predicted FEV1 Standard Deviation 7.47

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-3.1 percent predicted FVC Standard Deviation 8.06
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	-4.4 percent predicted FVC Standard Deviation 7.17

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12 Month 6	-0.028 mL Standard Deviation 0.2732	0.007 mL Standard Deviation 0.3101
Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12 Month 12	0.000 mL Standard Deviation 0.2240	-0.014 mL Standard Deviation 0.2383

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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).

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=12 Participants 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.
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	0.139 liter per second Standard Deviation 0.9876
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	0.166 liter per second Standard Deviation 1.0708

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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).

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=12 Participants 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.
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	1.1 percent predicted FEF Standard Deviation 23.65
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	0.9 percent predicted FEF Standard Deviation 26.24

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12 Month 6	-0.433 seconds Standard Deviation 1.5197	0.547 seconds Standard Deviation 2.6335
Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12 Month 12	0.086 seconds Standard Deviation 0.6403	0.181 seconds Standard Deviation 2.5598

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=2 Participants 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.	CAP-1002 n=2 Participants 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.
Change From Baseline in Six-Minute Walk Test at Month 6 and 12 Month 12	-8.5 meters Standard Deviation 0.71	-117.0 meters Standard Deviation 4.24
Change From Baseline in Six-Minute Walk Test at Month 6 and 12 Month 6	-9.0 meters Standard Deviation 2.83	-68.5 meters Standard Deviation 21.92

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	3.64 score on a scale Standard Deviation 16.225
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	4.28 score on a scale Standard Deviation 16.455

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-0.21 score on a scale Standard Deviation 17.334
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	-0.43 score on a scale Standard Deviation 19.429

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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).

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	-1.5 score on a scale Standard Deviation 20.39
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	-2.5 score on a scale Standard Deviation 13.17
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	-2.5 score on a scale Standard Deviation 12.36
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	-1.2 score on a scale Standard Deviation 6.19
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	-2.3 score on a scale Standard Deviation 8.71
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	-1.6 score on a scale Standard Deviation 10.21
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	-2.9 score on a scale Standard Deviation 9.60
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	2.0 score on a scale Standard Deviation 11.73
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 16.20	4.2 score on a scale Standard Deviation 13.20
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	8.1 score on a scale Standard Deviation 10.71
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 9.00	-0.8 score on a scale Standard Deviation 8.01
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	-2.1 score on a scale Standard Deviation 8.15

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

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).

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	2.2 score on a scale Standard Deviation 13.03
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	-3.2 score on a scale Standard Deviation 8.84
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	-1.8 score on a scale Standard Deviation 14.25
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	-4.2 score on a scale Standard Deviation 6.37
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	-2.7 score on a scale Standard Deviation 9.70
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	-2.5 score on a scale Standard Deviation 7.34
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	-6.8 score on a scale Standard Deviation 13.06
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	1.0 score on a scale Standard Deviation 13.34
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	2.3 score on a scale Standard Deviation 31.07
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	12.3 score on a scale Standard Deviation 27.96
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 4.00	-2.3 score on a scale Standard Deviation 4.46
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	-2.2 score on a scale Standard Deviation 5.73

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6 and Month 12

Population: Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category.

Osteopontin, Cardiac stress biomarker (ST2) and Galectin-3 were DMD biomarkers assessed through serum samples.

Outcome measures

Outcome measures
Measure	Usual Care n=11 Participants 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.	CAP-1002 n=13 Participants 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.
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	14.868 nanograms per milliliter Standard Deviation 18.1615
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	-0.175 nanograms per milliliter Standard Deviation 2.9025
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	0.829 nanograms per milliliter Standard Deviation 1.3238
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	8.051 nanograms per milliliter Standard Deviation 14.6530
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	-9.43 nanograms per milliliter Standard Deviation 16.294
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	-21.10 nanograms per milliliter Standard Deviation 19.637

Adverse Events

Usual Care

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

CAP-1002

Serious events: 3 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Usual Care n=12 participants at risk 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.	CAP-1002 n=13 participants at risk 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.
Cardiac disorders Ventricular fibrillation	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
General disorders Pyrexia	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Infections and infestations Urinary tract infection	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Injury, poisoning and procedural complications Femur fracture	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Psychiatric disorders Confusional state	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.

Other adverse events

Additional Information

Vice President of Clinical Research and Development Operations

Capricor, Inc.

Phone: 858-727-1755

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Usual Care n=12 Participants 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.	CAP-1002 n=13 Participants 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.
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	24.0 10^3 cells per microliter Standard Deviation 43.10
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	6.198 10^3 cells per microliter Standard Deviation 2.9121
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	2.153 10^3 cells per microliter Standard Deviation 3.4484
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	0.566 10^3 cells per microliter Standard Deviation 1.9689
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	318.6 10^3 cells per microliter Standard Deviation 53.63
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	40.4 10^3 cells per microliter Standard Deviation 45.39
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	10.57 10^3 cells per microliter Standard Deviation 3.947
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 4.010	0.81 10^3 cells per microliter Standard Deviation 2.606
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	-0.28 10^3 cells per microliter Standard Deviation 1.556
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	0.060 10^3 cells per microliter Standard Deviation 0.1052
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	-0.033 10^3 cells per microliter Standard Deviation 0.1064
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	-0.040 10^3 cells per microliter Standard Deviation 0.1197
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	0.171 10^3 cells per microliter Standard Deviation 0.1848
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	-0.073 10^3 cells per microliter Standard Deviation 0.1515
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	-0.078 10^3 cells per microliter Standard Deviation 0.1366
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	3.381 10^3 cells per microliter Standard Deviation 1.5423
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	-0.918 10^3 cells per microliter Standard Deviation 1.9381
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	-0.393 10^3 cells per microliter Standard Deviation 1.5501
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	0.705 10^3 cells per microliter Standard Deviation 0.2608
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	-0.273 10^3 cells per microliter Standard Deviation 0.4337
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	-0.284 10^3 cells per microliter Standard Deviation 0.3238

Measure	Usual Care n=12 participants at risk 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.	CAP-1002 n=13 participants at risk 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.
Cardiac disorders Atrial Fibrillation	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	38.5% 5/13 • Number of events 5 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Cardiac disorders Atrioventricular Block Second Degree	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Cardiac disorders Bradycardia	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	15.4% 2/13 • Number of events 2 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Cardiac disorders Palpitations	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Cardiac disorders Supraventricular Tachycardia	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Cardiac disorders Ventricular Tachycardia	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	15.4% 2/13 • Number of events 2 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Gastrointestinal disorders Abdominal Discomfort	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Gastrointestinal disorders Haemorrhoids	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Gastrointestinal disorders Nausea	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
General disorders Catheter Site Pain	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Infections and infestations Body Tinea	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Infections and infestations Gastroenteritis Viral	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Infections and infestations Gastrointestinal infection	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Infections and infestations Sinusitis	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Infections and infestations Nasopharyngitis	16.7% 2/12 • Number of events 2 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	15.4% 2/13 • Number of events 5 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Injury, poisoning and procedural complications Fall	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Injury, poisoning and procedural complications Fibula Fracture	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Injury, poisoning and procedural complications Ligament Sprain	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Injury, poisoning and procedural complications Radius Fracture	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Injury, poisoning and procedural complications Tibia Fracture	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Investigations Heart Rate Irregular	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Investigations Monocyte Count Decreased	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Investigations Spirometry abnormal	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Investigations Troponin Increased	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Investigations Weight Increased	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Metabolism and nutrition disorders Insulin Resistance	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	15.4% 2/13 • Number of events 2 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Musculoskeletal and connective tissue disorders Back Pain	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Musculoskeletal and connective tissue disorders Muscle Spasms	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Musculoskeletal and connective tissue disorders Musculoskeletal Chest Pain	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Melanocytic Naevus	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Nervous system disorders Dizziness	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Nervous system disorders Headache	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	15.4% 2/13 • Number of events 2 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Renal and urinary disorders Haematuria	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	15.4% 2/13 • Number of events 2 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Renal and urinary disorders Nephrolithiasis	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Respiratory, thoracic and mediastinal disorders Sinus Congestion	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Skin and subcutaneous tissue disorders Dermatitis Contact	8.3% 1/12 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	0.00% 0/13 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Skin and subcutaneous tissue disorders Ecchymosis	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Vascular disorders Haematoma	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	15.4% 2/13 • Number of events 2 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.
Injury, poisoning and procedural complications Femur Fracture	0.00% 0/12 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.	7.7% 1/13 • Number of events 1 • Adverse Events data was collected from first infusion up to 12 Month post-infusion Analysis was performed on safety population.