Autologous Cardiac Stem Cell Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 4
- 主要终点
- Number of Incidence of Treatment Related Major Adverse Cardiac Events
研究概览
简要总结
The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, re-hospitalizations, changes in health status, the need for transplantation, or mortality.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Day 至 21 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For inclusion in the study, subjects must meet all of the inclusion criteria:
- •Subjects with hypoplastic left heart syndrome (HLHS) (all types) requiring Stage I Norwood operation.
排除标准
- •Candidates will be excluded from the study if any of the following conditions are met:
- •Subjects undergoing the Stage I Norwood operation who do not have HLHS.
- •Subjects requiring mechanical circulatory support immediately prior to Stage II BDCPA operation (within 5 days).
- •Parent or guardian unwilling or unable to comply with necessary follow-up(s).
- •Mother is serum positive for HIV 1/2, hepatitis B surface antigen or viremic hepatitis C and Treponema pallidum.
- •Subjects who are unsuitable for inclusion in the study in the opinion of the investigator(s).
研究组 & 干预措施
Open label C-kit+ cells Group A
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
干预措施: c-kit+ cells (Biological)
C-kit+ cells Group B
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
干预措施: c-kit+ cells (Biological)
No Intervention Group
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
结局指标
主要结局
Number of Incidence of Treatment Related Major Adverse Cardiac Events
时间窗: 30 days
Safety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.
Number of C-kit+ Products
时间窗: Day 1
Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
Number of Participants Completing Magnetic Resonance Imaging (MRI)
时间窗: Baseline, 6 Months, 12 months
Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
Change in Right Ventricular Function (RVEF)
时间窗: Baseline, 6 Months, 12 months
Efficacy will be reported as the change in right ventricular function assessed as a percentage and will be measuring using serial echocardiograms and MRI scan.
Change in Right Ventricular End-diastolic Volume (RVEDV)
时间窗: Baseline, 6 months, 12 months
Efficacy will be reported as the change in right ventricular end-diastolic assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Change in Right Ventricular End-systolic Volume (RVESV)
时间窗: Baseline, 6 months, 12 months
Efficacy will be reported as the change in right ventricular end-systolic volume assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Change in Tricuspid Regurgitation
时间窗: Baseline, 6 months, 12 months
Efficacy will be reported as the change tricuspid regurgitation assessed as a percentage and will be measured using serial echocardiograms and MRI scan.
次要结局
- Number of Incidence of Serious Adverse Events(Up to 12 months)
- Change in Somatic Growth Velocity - Length (cm)(Baseline, 6 months, 12 months)
- Change in Somatic Growth Velocity - Weight (kg)(Baseline, 6 months, 12 months)
- Change in Somatic Growth Velocity - Head Circumference (cm)(Baseline, 6 months, 12 months)
- Change in Infant Toddler Quality of Life Survey (ITQOL) - Overall Health(Baseline, 12 months)
- Incidence of Mortality or Need for Transplantation(Up to 12 months)