Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS) : A Phase IIb Clinical Trial.
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
- 试验地点
- 10
- 主要终点
- Change in right ventricular ejection fraction (RVEF)
研究概览
简要总结
The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B. Lomecel-B contains human mesenchymal stem cells (MSCs) as the active ingredient. MSCs are special cells in the body that are able to change into other types of cells, such as heart, blood, and muscle cells. MSCs are found in various tissues of the body, such as the bone marrow, which is the spongy tissue inside of your bones. Lomecel-B uses MSCs from bone marrow of unrelated young healthy donors. These are called "allogeneic", and do not require donor matching to the patient.
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
- Triple (Participant, Investigator, Outcomes Assessor)
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Lomecel B Group
Participants randomized to receive Lomecel-B injections during their Stage II palliation.
干预措施: Lomecel-B medicinal signaling cells (Biological)
结局指标
主要结局
Change in right ventricular ejection fraction (RVEF)
时间窗: Baseline, 12 Months
Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.
次要结局
- Change in biomarkers/cytokines(Baseline, 12 Months)
- Change in right ventricular ejection fraction (RVEF)(Baseline, 6 Months)
- Change in right ventricular mass index at diastole(Baseline, 12 Months)
- Change in right ventricular end-diastolic volume index (RVEDVI)(Baseline, 12 Months)
- Change in right ventricular end-systolic volume index (RVESVI)(Baseline, 12 Months)
- Change in right ventricular global longitudinal strain and strain rate(Baseline, 12 Months)
- Change in right ventricular global circumferential strain and strain rate(Baseline, 12 Months)
- Change in right atrial volume index(Baseline, 12 Months)
- Change in tricuspid regurgitation severity(Baseline, 12 Months)
- Change in weight(Baseline, 12 Months)
- Change in length (height)(Baseline, 12 Months)
- Change in head circumference(Baseline, 12 Months)
- Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)(Baseline, 12 Months)
- Change in modified Ross Heart Failure Classification score(Baseline, 12 Months)
- Change in PedsQL™ Infant Scales(Baseline, 12 Months)
- Freedom from unplanned catheter intervention needed to address the pulmonary arteries or aorta.(Baseline, 12 Months)
- Participants experiencing treatment emergent serious adverse events (TE-SAEs)(30 days post Stage II palliation)
- Participants experiencing major adverse cardiac events (MACE)(Baseline, 12 Months)