Lomecel-B Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase I/II Study (ELPIS)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
- 试验地点
- 10
- 主要终点
- Safety: To evaluate the safety and feasibility of intramyocardial injection of LMSCs during the Stage II (BDCPA) operation for HLHS via incidence of Treatment-Emergent Serious Adverse Events.
研究概览
简要总结
This study is designed to assess the safety, tolerability, and efficacy of Lomecel-B as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS. Lomecel-B will be delivered via intramyocardial injections
详细描述
This study is designed to assess the safety, tolerability, and efficacy of Lomecel-B (formerly LMSCs) as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS, which is typically performed at 4 - 6 months after birth. Lomecel-B will be delivered via intramyocardial injections.
A total of 30 patients will be enrolled in 2 stages with 3 Cohorts.
In the first stage, 10 consecutive HLHS patients will be enrolled and treated with Lomecel-B (Cohort A). The first 3 patients will be treated no less than 5 days apart, and will be evaluated for any treatment-emergent adverse events (TE-AEs) (e.g., induced myocardial infarction or perforation). These patients will undergo full evaluation for 5 days to demonstrate safety prior to proceeding with the remainder of the cohort. After 6 months post-treatment of the last patient of Cohort A, a formal safety review will be conducted prior to proceeding to the next phase.
The second stage is double-blinded, in which 20 HLHS patients will be randomized to either receive treatment with Lomecel-B (Cohort B, 10 patients), or will receive no cells and no injection (Cohort C, 10 patients).
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
- Double (Participant, Investigator)
盲法说明
Phase 1: no masking. Phase 2: HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio
入排标准
- 年龄范围
- 1 Day 至 1 Year（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •all patients must have HLHS (all types) requiring BDCPA surgery.
排除标准
- •all patients must not have any of the following.
- •Significant coronary artery sinusoids.
- •Requirement for mechanical circulatory support prior to BDCPA surgery.
- •Underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
- •Need for concomitant surgery for aortic coarctation or tricuspid valve repair.
- •HLHS and restrictive or intact atrial septum.
- •Undergoing the Stage I (Norwood) procedure that does not have HLHS.
- •Serum positivity for: HIV; hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV).
- •Parent/guardian that is unwilling or unable to comply with necessary follow-up.
- •Unsuitability for the study based on the Investigator's clinical opinion.
- •Documented chromosomal abnormalities
结局指标
主要结局
Safety: To evaluate the safety and feasibility of intramyocardial injection of LMSCs during the Stage II (BDCPA) operation for HLHS via incidence of Treatment-Emergent Serious Adverse Events.
时间窗: Evaluated through 1 year post-treatment.
The incidence of Treatment-Emergent Serious Adverse Events will be evaluated, including: sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support; aggravation of heart failure; myocardial infarction; unplanned cardiovascular operation for cardiac tamponade; infection during the first month post-treatment; and death.
次要结局
- Efficacy: Change from baseline in right ventricular ejection fraction (%).(Evaluated through 1 year post-treatment.)
- Efficacy: Change from baseline in right ventricular end-diastolic volume.(Evaluated through 1 year post-treatment.)
- Efficacy: Change in head circumference (in centimeters).(Evaluated through 1 year post-treatment.)
- Efficacy: Change from baseline in right ventricular end-diastolic diameter.(Evaluated through 1 year post-treatment.)
- Efficacy: Change from baseline tricuspid regurgitation.(Evaluated through 1 year post-treatment.)
- Efficacy: Change from baseline in right ventricular end-systolic volume.(Evaluated through 1 year post-treatment.)
- Efficacy: Change in weight (in kilograms).(Evaluated through 1 year post-treatment.)
- Efficacy: Change in height (in centimeters).(Evaluated through 1 year post-treatment.)
- Efficacy: Number of patients with Treatment-Emergent Adverse Events, and total number of occurrences of Treatment-Emergent Adverse Events, through-out participation in trial.(Evaluated through 1 year post-treatment.)