Trial Outcomes & Findings for The CHILD Trial: Hypoplastic Left Heart Syndrome Study. (NCT NCT03406884)
NCT ID: NCT03406884
Last Updated: 2026-01-23
Results Overview
Safety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
25 participants
Primary outcome timeframe
30 days
Results posted on
2026-01-23
Participant Flow
Participant milestones
| Measure |
Open Label C-kit+ Cells Group A
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100 microliters per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Overall Study
STARTED
|
9
|
8
|
8
|
|
Overall Study
COMPLETED
|
9
|
8
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
The CHILD Trial: Hypoplastic Left Heart Syndrome Study.
Baseline characteristics by cohort
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
4.18 Months
STANDARD_DEVIATION 0.57 • n=270 Participants
|
4.66 Months
STANDARD_DEVIATION 0.85 • n=4 Participants
|
4.7 Months
STANDARD_DEVIATION 0.89 • n=9 Participants
|
4.51 Months
STANDARD_DEVIATION 0.77 • n=220 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=270 Participants
|
3 Participants
n=4 Participants
|
4 Participants
n=9 Participants
|
8 Participants
n=220 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=270 Participants
|
5 Participants
n=4 Participants
|
4 Participants
n=9 Participants
|
17 Participants
n=220 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=270 Participants
|
2 Participants
n=4 Participants
|
1 Participants
n=9 Participants
|
4 Participants
n=220 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=270 Participants
|
6 Participants
n=4 Participants
|
7 Participants
n=9 Participants
|
21 Participants
n=220 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=270 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=220 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=270 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=220 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=270 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=9 Participants
|
1 Participants
n=220 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=270 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=220 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=270 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=9 Participants
|
5 Participants
n=220 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=270 Participants
|
6 Participants
n=4 Participants
|
7 Participants
n=9 Participants
|
16 Participants
n=220 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=270 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=9 Participants
|
1 Participants
n=220 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=270 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=9 Participants
|
2 Participants
n=220 Participants
|
PRIMARY outcome
Timeframe: 30 daysSafety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Number of Incidence of Treatment Related Major Adverse Cardiac Events
|
1 Events
|
0 Events
|
6 Events
|
PRIMARY outcome
Timeframe: Day 1Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Number of C-kit+ Products
|
9 Products
|
7 Products
|
0 Products
|
PRIMARY outcome
Timeframe: Baseline, 6 Months, 12 monthsFeasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Number of Participants Completing Magnetic Resonance Imaging (MRI)
Month 12
|
6 Participants
|
6 Participants
|
2 Participants
|
|
Number of Participants Completing Magnetic Resonance Imaging (MRI)
Baseline
|
9 Participants
|
7 Participants
|
7 Participants
|
|
Number of Participants Completing Magnetic Resonance Imaging (MRI)
Month 6
|
6 Participants
|
6 Participants
|
5 Participants
|
PRIMARY outcome
Timeframe: Baseline, 6 Months, 12 monthsPopulation: sample size decreases due to attrition
Efficacy will be reported as the change in right ventricular function assessed as a percentage and will be measuring using serial echocardiograms and MRI scan.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Right Ventricular Function (RVEF)
Baseline
|
49.33 percentage
Standard Deviation 4.44
|
55 percentage
Standard Deviation 7.19
|
45.4 percentage
Standard Deviation 10.25
|
|
Change in Right Ventricular Function (RVEF)
Month 6
|
48 percentage
Standard Deviation 3.63
|
50.83 percentage
Standard Deviation 6.74
|
47 percentage
Standard Deviation 6.86
|
|
Change in Right Ventricular Function (RVEF)
Month 12
|
44.67 percentage
Standard Deviation 8.36
|
49.83 percentage
Standard Deviation 3.76
|
48.5 percentage
Standard Deviation 3.54
|
PRIMARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: sample size decreases due to attrition
Efficacy will be reported as the change in right ventricular end-diastolic assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Right Ventricular End-diastolic Volume (RVEDV)
Baseline
|
112.57 milliliters per square meter
Standard Deviation 20.75
|
119.1 milliliters per square meter
Standard Deviation 35.08
|
123.98 milliliters per square meter
Standard Deviation 18.92
|
|
Change in Right Ventricular End-diastolic Volume (RVEDV)
Month 6
|
129.5 milliliters per square meter
Standard Deviation 44.21
|
123.08 milliliters per square meter
Standard Deviation 11.83
|
130.27 milliliters per square meter
Standard Deviation 37.08
|
|
Change in Right Ventricular End-diastolic Volume (RVEDV)
Month 12
|
126.78 milliliters per square meter
Standard Deviation 23.55
|
113.8 milliliters per square meter
Standard Deviation 13.97
|
129 milliliters per square meter
Standard Deviation 18.1
|
PRIMARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: sample size decreases due to attrition
Efficacy will be reported as the change in right ventricular end-systolic volume assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Right Ventricular End-systolic Volume (RVESV)
Baseline
|
57.43 milliliters per square meter
Standard Deviation 13.22
|
55.53 milliliters per square meter
Standard Deviation 23.87
|
68.66 milliliters per square meter
Standard Deviation 22.04
|
|
Change in Right Ventricular End-systolic Volume (RVESV)
Month 6
|
68.38 milliliters per square meter
Standard Deviation 26.43
|
55.17 milliliters per square meter
Standard Deviation 21.36
|
70.66 milliliters per square meter
Standard Deviation 28.95
|
|
Change in Right Ventricular End-systolic Volume (RVESV)
Month 12
|
71.7 milliliters per square meter
Standard Deviation 21.64
|
56.66 milliliters per square meter
Standard Deviation 5.43
|
68.05 milliliters per square meter
Standard Deviation 12.8
|
PRIMARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: sample size decreases due to attrition
Efficacy will be reported as the change tricuspid regurgitation assessed as a percentage and will be measured using serial echocardiograms and MRI scan.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Tricuspid Regurgitation
Baseline
|
8.28 percentage
Standard Deviation 5.21
|
5 percentage
Standard Deviation 0
|
5 percentage
Standard Deviation 0
|
|
Change in Tricuspid Regurgitation
Month 6
|
35.07 percentage
Standard Deviation 27.81
|
9.23 percentage
Standard Deviation 6.03
|
15.35 percentage
Standard Deviation 16.95
|
|
Change in Tricuspid Regurgitation
Month 12
|
21.93 percentage
Standard Deviation 6.03
|
14.72 percentage
Standard Deviation 7.27
|
7.75 percentage
Standard Deviation 3.75
|
SECONDARY outcome
Timeframe: Up to 12 monthsIncidence of the following after the BDCPA / GLENN Procedure including: All-cause mortality; Cardiovascular mortality; Need for transplantation; Hospitalization for heart failure; Cardiovascular morbidity, including stroke or heart failure or sustained/symptomatic arrhythmias.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Number of Incidence of Serious Adverse Events
|
1 Events
|
0 Events
|
6 Events
|
SECONDARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: sample size decreases due to attrition
Changes in somatic growth velocity will be evaluated by length (cm) over 12 months post study product injection.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Somatic Growth Velocity - Length (cm)
Baseline
|
60.33 centimeters
Standard Deviation 3.82
|
59.5 centimeters
Standard Deviation 3.28
|
60.07 centimeters
Standard Deviation 4.94
|
|
Change in Somatic Growth Velocity - Length (cm)
Month 6
|
69.72 centimeters
Standard Deviation 5.57
|
69.79 centimeters
Standard Deviation 4.18
|
70.8 centimeters
Standard Deviation 5.39
|
|
Change in Somatic Growth Velocity - Length (cm)
Month 12
|
76.98 centimeters
Standard Deviation 5.87
|
77.03 centimeters
Standard Deviation 3.7
|
79.63 centimeters
Standard Deviation 4.02
|
SECONDARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: sample size decreases due to attrition
Changes in somatic growth velocity will be evaluated by weight (kg) over 12 months post study product injection.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Somatic Growth Velocity - Weight (kg)
Baseline
|
5.7 kilograms
Standard Deviation 0.66
|
5.6 kilograms
Standard Deviation 0.74
|
5.65 kilograms
Standard Deviation 0.69
|
|
Change in Somatic Growth Velocity - Weight (kg)
Month 6
|
7.67 kilograms
Standard Deviation 0.84
|
8.39 kilograms
Standard Deviation 1.21
|
8.36 kilograms
Standard Deviation 0.65
|
|
Change in Somatic Growth Velocity - Weight (kg)
Month 12
|
9.74 kilograms
Standard Deviation 1.65
|
10.45 kilograms
Standard Deviation 1.19
|
10.01 kilograms
Standard Deviation 0.68
|
SECONDARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: sample size decreases due to attrition
Changes in somatic growth velocity will be evaluated by head circumference (cm) over 12 months post study product injection.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Somatic Growth Velocity - Head Circumference (cm)
Baseline
|
38.51 centimeters
Standard Deviation 3.01
|
39.58 centimeters
Standard Deviation 2.69
|
38.33 centimeters
Standard Deviation 3.18
|
|
Change in Somatic Growth Velocity - Head Circumference (cm)
Month 6
|
45.04 centimeters
Standard Deviation 1.33
|
45.18 centimeters
Standard Deviation 1.62
|
45.42 centimeters
Standard Deviation 2.42
|
|
Change in Somatic Growth Velocity - Head Circumference (cm)
Month 12
|
47.88 centimeters
Standard Deviation 0.76
|
48.48 centimeters
Standard Deviation 1.57
|
46.92 centimeters
Standard Deviation 2.39
|
SECONDARY outcome
Timeframe: Baseline, 12 monthsPopulation: sample size decreases due to attrition
The Infant Toddler Quality of Life Survey (ITQOL) is a validated 47-item parent-reported questionnaire assessing multiple domains of child health, including overall health, physical abilities, growth and development, discomfort/pain, mood, and behavior. Domain scores are transformed to a 0-100 scale, where 0 reflects the poorest health-related quality of life and 100 reflects the best. Higher scores indicate better quality of life.
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Change in Infant Toddler Quality of Life Survey (ITQOL) - Overall Health
Baseline
|
86 score on a scale
Standard Deviation 16.36
|
62.14 score on a scale
Standard Deviation 24.64
|
77.86 score on a scale
Standard Deviation 12.2
|
|
Change in Infant Toddler Quality of Life Survey (ITQOL) - Overall Health
Month 12
|
88 score on a scale
Standard Deviation 6.71
|
85.71 score on a scale
Standard Deviation 13.36
|
72.5 score on a scale
Standard Deviation 13.69
|
SECONDARY outcome
Timeframe: Up to 12 monthsIncidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported
Outcome measures
| Measure |
Open Label C-kit+ Cells Group A
n=9 Participants
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 Participants
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 Participants
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Incidence of Mortality or Need for Transplantation
|
0 Participants
|
0 Participants
|
3 Participants
|
Adverse Events
Open Label C-kit+ Cells Group A
Serious events: 8 serious events
Other events: 5 other events
Deaths: 0 deaths
C-kit+ Cells Group B
Serious events: 4 serious events
Other events: 6 other events
Deaths: 0 deaths
No Intervention Group
Serious events: 4 serious events
Other events: 6 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Open Label C-kit+ Cells Group A
n=9 participants at risk
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 participants at risk
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 participants at risk
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Cardiac disorders
Acute right ventricular failure
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
Atrial flutter
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Cardiac disorders
Atrioventricular block complete
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
Bradycardia
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Cardiac disorders
Cardiac arrest
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
25.0%
2/8 • Number of events 3 • 1 year
|
|
Cardiac disorders
Cardiac failure
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
Cardiopulmonary failure
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
Right ventricular dysfunction
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
Supraventricular tachycardia
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Cardiac disorders
Tachyarrhythmia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
Tricuspid valve incompetence
|
22.2%
2/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Congenital, familial and genetic disorders
Coarctation of the aorta
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Dysphagia
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
Haematochezia
|
33.3%
3/9 • Number of events 3 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
Necrotising colitis
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
General disorders
Crying
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
General disorders
Vascular stent thrombosis
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
General disorders
Withdrawal syndrome
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Bacterial sepsis
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 4 • 1 year
|
|
Infections and infestations
Bronchiolitis
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
COVID-19
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
Infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
Mediastinitis
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Otitis media
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Parainfluenzae virus infection
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
Pneumonia
|
11.1%
1/9 • Number of events 3 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Sepsis
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
25.0%
2/8 • Number of events 3 • 1 year
|
|
Infections and infestations
Urinary tract infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
Viral infection
|
22.2%
2/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Viral myocarditis
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
upper respiratory infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Aortic restenosis
|
22.2%
2/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Cardiac function disturbance postoperative
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Injury, poisoning and procedural complications
Diaphragmatic injury
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Shunt stenosis
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Acidosis
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Failure to thrive
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Metabolism and nutrition disorders
Feeding intolerance
|
0.00%
0/9 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Nervous system disorders
Cerebrovascular accident
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Nervous system disorders
Infant irritability
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Nervous system disorders
Seizure
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Chylothorax
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Diaphragmatic paralysis
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Haemothorax
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
11.1%
1/9 • Number of events 1 • 1 year
|
25.0%
2/8 • Number of events 3 • 1 year
|
37.5%
3/8 • Number of events 4 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary vein stenosis
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Vascular disorders
Collateral circulation
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Vascular disorders
Deep vein thrombosis
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Vascular disorders
Hypotension
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Vascular disorders
Jugular vein thrombosis
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Vascular disorders
Peripheral artery occlusion
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Vascular disorders
Vena cava stenosis
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 2 • 1 year
|
|
Vascular disorders
Venous occlusion
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Open Label C-kit+ Cells Group A
n=9 participants at risk
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
C-kit+ Cells Group B
n=8 participants at risk
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
c-kit+ cells: The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
|
No Intervention Group
n=8 participants at risk
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
|
|---|---|---|---|
|
Cardiac disorders
Bradycardia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
Tricuspid valve incompetence
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
Haematochezia
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
Necrotising colitis
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
General disorders
Device embolisation
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
General disorders
Drug withdrawal syndrome
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
General disorders
Granuloma
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
General disorders
Pyrexia
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Bacteraemia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
COVID-19
|
0.00%
0/9 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Fungaemia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
Pneumonia
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Infections and infestations
Pseudomonas infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Urinary tract infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
Viral infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Accidental exposure to product by child
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Femur Fracture
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Injury, poisoning and procedural complications
Injury
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Medication Error
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Injury, poisoning and procedural complications
Spinal Fracture
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Injury, poisoning and procedural complications
Subdural Haematoma
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Nervous system disorders
Presyncope
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Nervous system disorders
Seizure
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Bronchomalacia
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Chylothorax
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary artery occlusion
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin Disorder
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Vascular disorders
Labile blood pressure
|
0.00%
0/9 • 1 year
|
0.00%
0/8 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place