Heart Defects, Congenital, Hypoplastic Left Heart Syndrome, Pediatric Disorder
Conditions
Keywords
cord blood stem cell, Norwood heart surgery, congenital, heart
Brief summary
This study aims to evaluate the safety and feasibility of coronary infusion of autologous placental cord blood mononuclear cells during the Norwood heart operation in newborn hypoplastic left heart syndrome (HLHS) patients.
Detailed description
This is a Phase I, open label safety study. Autologous cord blood mononuclear cells (stem cell containing, cord blood buffy coat fraction) was delivered by coronary infusion during the Norwood cardiopulmonary bypass operation in 10 antenatally diagnosed HLHS patients within 2-3 days of birth. Safety and clinical status monitoring was performed to Stage II surgical intervention for HLHS (at approximately 3 months of age). Treated patients will continue to be assessed beyond the trial during follow up between Stage II and III (Fontan) surgical interventions.
Interventions
BIOLOGICALAutologous Human Placental Cord Blood Mononuclear Cells
Autologous human placental cord blood mononuclear cells are infused into the coronary artery during the Norwood heart operation
Sponsors
Murdoch Childrens Research Institute
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Safety
Eligibility
Sex/Gender
ALL
Age
2 Days to 4 Days
Healthy volunteers
No
Inclusion criteria
* Male and females with antenatally diagnosed Hypoplastic Left Heart Syndrome (all types requiring Norwood operation) * Written informed consent by parents/legal guardian * Successful aseptic collection of autologous cord blood unit and transfer of buffy coat mononucleocyte fraction to cold storage pending cardiac surgery within 2-4 days
Exclusion criteria
Patient: * does not have autologous cord blood cells available at the time of cardiopulmonary bypass surgery * has evidence of arrhythmia requiring anti-arrhythmia therapy * has an additional congenital diagnosis that contributes to conditions such as an immune system disorder, immune deficiency, complex metabolic disorder, brain dysplasia or progressive neurological degenerative disorder * has other clinical complexity deemed by the cardiac surgeon to make the patient unsuitable for inclusion in the trial Mother: • is serum positive for HIV, hepatitis or other significant pathogen and has known allergies to penicillin, streptomycin or other antibiotic
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of adverse cardiac events | 1 month | Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in right ventricular end-diastolic wall thickness (% units) | baseline, 1 month, 3 months, 12 months | Measured by cardiac imaging with serial echocardiography and MRI scans |
| Change in right ventricular end-diastolic volume (% units) | baseline, 1 month, 3 months, 12 months | Measured by cardiac imaging with serial echocardiography and MRI scans |
| Change in right ventricular function -fractional shortening (% units) | baseline, 1 month, 3 months, 12 months | Measured by cardiac imaging with serial echocardiography and MRI scans |
| Increase in body weight | baseline, 1 month, 3 months, 12 months | Body weight measured in kilograms |
| Composite measure of height and head circumference | baseline, 1 month, 3 months, 12 months | Body height and head circumference measured in meters |
| Change in right ventricular end-systolic volume (% units) | baseline, 1 month, 3 months, 12 months | Measured by cardiac imaging with serial echocardiography and MRI scans |
Countries
Australia
Outcome results
None listed