Safety of Autologous Cord Blood Cells in HLHS Patients During Norwood Heart Surgery

Study Overview
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Brief Summary
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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
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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.

Official Title
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Safety Study of Autologous Cord Blood Stem Cell Treatment in Hypoplastic Left Heart Syndrome Patients Undergoing the Norwood Heart Operation

Conditions
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Hypoplastic Left Heart Syndrome, Heart Defects, Congenital, Pediatric Disorder

Intervention / Treatment
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* Biological: Autologous Human Placental Cord Blood Mononuclear Cells


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Days
Maximum Age: 4 Days

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Safety
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: hCBMNC<br>Autologous human placental cord blood mononuclear cells (buffy coat fraction) | Biological: Autologous Human Placental Cord Blood Mononuclear Cells<br>* Autologous human placental cord blood mononuclear cells are infused into the coronary artery during the Norwood heart operation<br>* Other names: Cord blood buffycoat mononuclear cells, incl stem cells;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse cardiac events | Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke | 1 month | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in right ventricular function -fractional shortening (% units) | Measured by cardiac imaging with serial echocardiography and MRI scans | baseline, 1 month, 3 months, 12 months | 
| Change in right ventricular end-diastolic wall thickness (% units) | Measured by cardiac imaging with serial echocardiography and MRI scans | baseline, 1 month, 3 months, 12 months | 
| Change in right ventricular end-diastolic volume (% units) | Measured by cardiac imaging with serial echocardiography and MRI scans | baseline, 1 month, 3 months, 12 months | 
| Change in right ventricular end-systolic volume (% units) | Measured by cardiac imaging with serial echocardiography and MRI scans | baseline, 1 month, 3 months, 12 months | 
| Increase in body weight | Body weight measured in kilograms | baseline, 1 month, 3 months, 12 months | 
| Composite measure of height and head circumference | Body height and head circumference measured in meters | baseline, 1 month, 3 months, 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cord blood stem cell, Norwood heart surgery, congenital, heart