Safety Study of Autologous Cord Blood Stem Cell Treatment in Hypoplastic Left Heart Syndrome Patients Undergoing the Norwood Heart Operation
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Hypoplastic Left Heart Syndrome
- Sponsor
- Murdoch Childrens Research Institute
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Incidence of adverse cardiac events
- Status
- Completed
- Last Updated
- 3 years ago
Overview
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.
Investigators
Salvatore Pepe
Trial Study Coordinator
Murdoch Childrens Research Institute
Eligibility Criteria
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
- •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
- •is serum positive for HIV, hepatitis or other significant pathogen and has known allergies to penicillin, streptomycin or other antibiotic
Outcomes
Primary Outcomes
Incidence of adverse cardiac events
Time Frame: 1 month
Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke
Secondary Outcomes
- Change in right ventricular end-diastolic volume (% units)(baseline, 1 month, 3 months, 12 months)
- Change in right ventricular function -fractional shortening (% units)(baseline, 1 month, 3 months, 12 months)
- Change in right ventricular end-diastolic wall thickness (% units)(baseline, 1 month, 3 months, 12 months)
- Change in right ventricular end-systolic volume (% units)(baseline, 1 month, 3 months, 12 months)
- Increase in body weight(baseline, 1 month, 3 months, 12 months)
- Composite measure of height and head circumference(baseline, 1 month, 3 months, 12 months)