Phase IIb Study of Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Stage II Surgical Repair of Right Ventricular Dependent Variants of Hypoplastic Left Heart Syndrome (AutoCell-S2)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 95
- 试验地点
- 8
- 主要终点
- Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)
研究概览
简要总结
Researchers want to better understand what happens to the heart when the stem cells are injected directly into the muscle of the right side of the heart during the Stage II palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS) or HLHS variant. Researchers want to see if there are changes in the heart's structure/function following this stem cell-based therapy and compared to children that have not had cell-based therapy.
详细描述
This is a Phase IIb trial to determine if the delivery of the autologous UCB-MNC product into the myocardium of the right ventricle of the heart at the time of Stage II surgical repair will provide an improvement in cardiac function, reaching growth and developmental milestones, and quality of life, while also providing a reduction in the cumulative days of hospitalization following Stage II surgical repair. Long-term improvement in cardiac function, reaching growth and developmental milestones, reaching Stage III surgical repair pre-op work-up, prolonging time to cardiac transplantation or death, and improving quality of life will also be determined.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 12 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of HLHS or HLHS variant with single right ventricular dependent CHD having undergone Stage I surgical repair and undergoing Stage II surgical repair.
- •Less than 13 months of age at time of Stage II surgical repair
- •Previous participation in the UCB collection protocol with autologous UCB-MNC product that is acceptable for use (treatment arm only)
排除标准
- •History of DMSO reaction (treatment arm only).
- •Parent(s) and/or legal guardian(s) unwilling to have their child participate or unwilling to follow the study procedures.
- •Severe chronic diseases at the discretion of the treating physician.
- •Extensive extra-cardiac syndromic features.
- •Known history of cancer.
- •Any of the following complications of his/her congenital heart disease:
- •Any condition requiring urgent, or unplanned interventional procedure within 15 days prior to Stage II surgical repair, unless complete and full cardiac recovery is documented by site investigator
- •Severe pulmonary hypertension (reported in the medical record as >70% systemic pressure)
- •Other clinical concerns as documented by a site investigator that would predict (more likely to happen than not to happen) a risk of severe complications or very poor outcome, not directly related to the stem cell product or its injection procedure, during or after Stage II surgical repair.
研究组 & 干预措施
Treatment
Autologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 1-3 million cells per kilogram body weight . This is a one time treatment at the time of Stage II Glenn surgery .
干预措施: Autologous (self) mononuclear cells derived from umbilical cord blood (Biological)
Treatment
Autologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 1-3 million cells per kilogram body weight . This is a one time treatment at the time of Stage II Glenn surgery .
干预措施: Stage II Surgical repair (Procedure)
Control
Clinical data will be collected before, during and after the Stage II surgical standard of care procedure. Requires additional imaging studies at 3 months that are not standard of care in addition to some blood tests that would be beyond standard of care. Information will be collected to document the clinical outcomes and will be compared with the information from the group receiving the investigational treatment used in this study.
干预措施: Stage II Surgical repair (Procedure)
结局指标
主要结局
Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)
时间窗: baseline, 12 months post-Stage II surgery
Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
Change in Right Ventricular Cardiac Function Measured by Circumferential Strain.
时间窗: baseline, 3 months post-Stage II surgery
Right ventricular cardiac function as measured by echocardiogram that includes studies for circumferential strain. Measured by percent.
Change in Right Ventricular Cardiac Function Measured by Longitudinal Strain.
时间窗: baseline, 3 months post-Stage II surgery
Right ventricular cardiac function as measured by echocardiogram that includes studies for longitudinal strain. Measured by percent.
次要结局
- Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)(baseline, Stage III surgery pre-op)
- Cumulative Days of Hospitalization(3-months post Stage II surgery)
- Change in Weight(baseline, every 6 months post-Stage II surgery)
- Change in Heart Rate(baseline, 3-months post Stage II surgery)
- Change in Oxygen Saturation(baseline, 3-months post Stage II surgery)
- Change in Arrhythmia and Heart Failure Medication(3-months post-surgery)
- Stage III Surgical Repair(Stage III surgery pre-op, approx. 4 years)
- Total Time Until Listed for Cardiac Transplantation(up to Stage III surgery pre-op, approx. 4 years)
- Total Time Until Death(approx 4 years)
研究者
Timothy J Nelson, MD, PhD
Program Director
ReGen Theranostics, Inc.