A Phase I, Open Label, Safety Study of INXN-4001 Delivered Via Retrograde Coronary Sinus Infusion in Patients With an Outpatient Left Ventricular Assist Device (LVAD)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- To evaluate safety and feasibility of INXN-4001
研究概览
简要总结
To evaluate the safety of retrograde coronary sinus infusion (RCSI) of a novel triple-effector plasmid (INXN-4001) in outpatient LVAD recipients as assessed by incidence of all study intervention-related adverse events occurring up to 6 months post-RCSI (primary endpoints), and to evaluate general safety by assessing incidence of cardiac specific adverse events and the incidence of related serious adverse events at intervals up to 12 months post-infusion (or until cardiac transplantation or death).
详细描述
This is a first-in-human, phase I, open label, safety study of INXN-4001 delivered via RCSI in patients with outpatient LVAD. Twelve stable patients with implanted LVAD for mechanical support of end stage heart failure have been allocated into 2 cohorts (6 subjects each) to evaluate the safety of infusing the same amount of INXN-4001 (80mg) in 2 volumes (40mL and 80mL) at 20mL/min rate. Ongoing safety assessments include clinical labs, physical exams, ECG and medical history collected during clinic visits at: pre-treatment, day 3, then 1, 3, 6, 9, and 12 months after dosing via RCSI. During visits, subjects complete the KCCQ questionnaire and are evaluated via 6-min walk test (6MWT) prior to and during an LVAD wean interval. Daily activity data is collected throughout the study using a wearable biosensor (Actigraph).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female adult patients with a stable LVAD implanted for end-stage heart failure
- •Must be managed in an outpatient setting and on stable medication regimen
排除标准
- •Women who are pregnant or nursing
- •Patients who have been on another clinical trial for heart failure in the last 90 days, or have received any stem cell or gene therapy within the previous year.
- •Patient is not able to complete a Six Minute Walk Test or unable to tolerate an LVAD wean in the past 3 months
- •Patient has an active infection requiring systemic antibiotics or an autoimmune disease requiring systemic immunosuppressants
- •Patient has a history of arrhythmia, uncontrolled diabetes, diabetic retinopathy, systemic lupus erythematosus, macular degeneration, hyper-coagulation, or stroke
- •Patient has had a myocardial infarction related to ischemia within the past 30 days
- •Patient has had certain prior surgeries such as organ transplant, cardiac transplantation, left ventricle reduction surgery, or cardiomyoplasty
- •Patient has infectious disease, such as hepatitis B or C, or human immunodeficiency virus (HIV)
- •Patient has a history of cancer within the past 3 years
结局指标
主要结局
To evaluate safety and feasibility of INXN-4001
时间窗: 6 months
As assessed by incidence of all study intervention-related adverse events occurring up to 6 months post- treatment intervention-related adverse events
次要结局
- Feasibility of biosensor activity tracking(12 months)
- Quality of Life following treatment with INXN-4001(12 months)
- Overall safety of INXN-4001(12 months)
- Wean tolerability following treatment with INXN-4001(12 months)