Safety of Autologous Cord Blood Cells in HLHS Patients During Norwood Heart Surgery
- Conditions
- Heart Defects, CongenitalHypoplastic Left Heart SyndromePediatric Disorder
- Interventions
- Biological: Autologous Human Placental Cord Blood Mononuclear Cells
- Registration Number
- NCT03431480
- Lead Sponsor
- Murdoch Childrens Research Institute
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 10
- 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
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description hCBMNC Autologous Human Placental Cord Blood Mononuclear Cells Autologous human placental cord blood mononuclear cells (buffy coat fraction)
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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
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-systolic volume (% 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
Trial Locations
- Locations (1)
Royal Children's Hospital
🇦🇺Melbourne, Victoria, Australia