Researchers want to better understand what happens to the heart when the stem cells are injected directly into the muscle of the right side of the heart during the Stage II palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS) or HLHS variant. Researchers want to see if there are changes in the heart's structure/function following this stem cell-based therapy and compared to children that have not had cell-based therapy.
This is a Phase IIb trial to determine if the delivery of the autologous UCB-MNC product into the myocardium of the right ventricle of the heart at the time of Stage II surgical repair will provide an improvement in cardiac function, reaching growth and developmental milestones, and quality of life, while also providing a reduction in the cumulative days of hospitalization following Stage II surgical repair. Long-term improvement in cardiac function, reaching growth and developmental milestones, reaching Stage III surgical repair pre-op work-up, prolonging time to cardiac transplantation or death, and improving quality of life will also be determined.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
95
The investigational product will be delivered into the right myocardium via sub-epicardial injections of 0.1 mL per kg body weight to achieve the target dose of 1-3 million TNC per kg body weight.at the time of Stage II surgical repair.
This operation usually is performed about six months after Stage I surgery to divert half of the blood to the lungs when circulation through the lungs no longer needs as much pressure from the ventricle. The shunt to the pulmonary arteries is disconnected and the right pulmonary artery is connected directly to the superior vena cava, the vein that brings deoxygenated blood from the upper part of the body to the heart. This sends half of the deoxygenated blood directly to the lungs without going through the ventricle.
Children's of Alabama
Birmingham, Alabama, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Ocshner Medical Center
New Orleans, Louisiana, United States
Children's Hospitals of Minnesota
Minneapolis, Minnesota, United States
Cincinnati Children's Hospital and Medical Center
Cincinnati, Ohio, United States
Oklahoma University Medical Center
Oklahoma City, Oklahoma, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)
Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
Time frame: baseline, 3 months post-Stage II surgery
Change in Right Ventricular Cardiac Function Measured by Circumferential Strain.
Right ventricular cardiac function as measured by echocardiogram that includes studies for circumferential strain. Measured by percent.
Time frame: baseline, 3 months post-Stage II surgery
Change in Right Ventricular Cardiac Function Measured by Longitudinal Strain.
Right ventricular cardiac function as measured by echocardiogram that includes studies for longitudinal strain. Measured by percent.
Time frame: baseline, 3 months post-Stage II surgery
Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)
Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
Time frame: baseline, 12 months post-Stage II surgery
Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)
Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
Time frame: baseline pre-op, hospital discharge from Stage II surgery (an average of 1-6 weeks)
Cumulative Days of Hospitalization
Cumulative days of hospitalization per patient following discharge for the Stage II surgical repair.
Time frame: 1-month post Stage II surgery
Change in Weight
Change in weight between baseline and 3-months post Stage II surgery. Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.
Time frame: baseline, 3-months post Stage II surgery
Change in Heart Rate
Change in heart rate between baseline and 3-months post Stage II surgery. Heart rate as measured at home by subject's caregiver as standard of care per subject's provider. Caregivers will be instructed by the site staff on the best way to perform.
Time frame: baseline, 3-months post Stage II surgery
Change in Oxygen Saturation
Change in oxygen saturation between baseline and 3-months post Stage II surgery. Oxygen saturation as measured at home by subject's caregiver using the pulse oximetry device provided as standard of care by subject's provider. Caregivers will be instructed by the site to use the same pulse oximetry device for every home measurement of oxygen saturation.
Time frame: baseline, 3-months post Stage II surgery
Change in Weight
Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.
Time frame: baseline, every 6 months post-Stage II surgery
Change in Arrhythmia and Heart Failure Medication
Changes in arrhythmia and heart failure medications will be recorded by caregiver on the concomitant medication diary throughout the subject's participation in the study starting at the time of consent.
Time frame: 3-months post-surgery
Stage III Surgical Repair
The number of research subjects eligible for Stage III surgical repair
Time frame: Stage III surgery pre-op, approx. 4 years
Total Time Until Listed for Cardiac Transplantation
The number of days between Stage II surgery and listed on cardiac transplantation list.
Time frame: up to Stage III surgery pre-op, approx. 4 years
Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)
Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
Time frame: baseline, Stage III surgery pre-op
Total Time Until Death
The number of days between Stage II surgery and death occurrence
Time frame: approx 4 years
Cumulative Days of Hospitalization
Cumulative days of hospitalization per patient following discharge for the Stage II surgical repair.
Time frame: 3-months post Stage II surgery
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