Safety Study of Autologous Cord Blood Stem Cell Treatment in Hypoplastic Left Heart Syndrome Patients Undergoing the Norwood Heart Operation
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Incidence of adverse cardiac events
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Days 至 4 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Incidence of adverse cardiac events
时间窗: 1 month
Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke
次要结局
- 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)
研究者
Salvatore Pepe
Trial Study Coordinator
Murdoch Childrens Research Institute