Pumps for Kids, Infants, and Neonates
试验速览
- 阶段
- 早期 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 7
- 试验地点
- 13
- 主要终点
- Adverse Events
研究概览
简要总结
PumpKIN is a multicenter, prospective, single-arm feasibility study; Evaluating the investigational Jarvik 2015 VAD in pediatric patients with heart failure. This feasibility trial will enroll 10 subjects at up to 7 sites in the US.
The primary objectives of this investigational device exemption (IDE) clinical investigation are to assess the feasibility of using the Jarvik 2015 in pediatric patients with severe heart failure who require mechanical circulatory support. Feasibility will be assessed by evaluating the safety profile of the Jarvik 2015 device in eligible subjects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Device Feasibility
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children must meet all of the following criteria:
- •Males and females within weight range 8 to 30 kg
- •Body surface area (BSA) 0.4 m2 to 1.0 m2
- •Cardiac Diagnosis:
- •a. Standard Cardiac Anatomy : Two-ventricle circulation, including cardiomyopathy, repaired structural heart disease (e.g. anomalous left coronary artery from the pulmonary artery [ALCAPA], aortic stenosis) or acquired heart disease (e.g., myocarditis, Kawasaki disease)
- •INTERMACS Profile 1 or 2 as evidenced by one or more of the following:
- •Inability to wean from extra-corporeal membrane oxygenation (ECMO) or other temporary circulatory support (TCS), OR
- •Inability to wean from mechanical ventilator support, OR
- •Inotrope-dependent, decompensated heart failure AND meet one or more of the following criteria within 48 hours prior to implant (unless otherwise noted) which is attributed to decompensated heart failure despite optimal medical therapy:
- •i. Urine output <0.5 cc/kg/hour for 12hr within 48 hours ii. Creatinine level >2 times the ULN for age iii. Alanine aminotransferase (ALT) or total bilirubin result >3 times the ULN for age (either qualifies the patient) iv. Mixed venous oxygen saturation (SvO2) <55% (or arteriovenous oxygen difference of >45%) in two repeated measurements v. Acidosis: Base excess >-5 in 2 or more measurements vi. Inability to tolerate appropriate enteral calories as prescribed by a registered dietician d. Inability to ambulate freely to participate fully in age-appropriate activities of daily living (ADLs) and/or cardiac rehabilitation/physical therapy
- •LVAD support is intended for bridge-to transplant. Subject is listed for transplant or eligible (i.e., no medical or surgical contraindications) to be listed for cardiac transplant, United Network for Organ Sharing (UNOS) status 1A, or equivalent
- •Written consent of parent(s) or legally authorized representative (LAR) where appropriate.
- •Children must not meet any of the following
排除标准
- •within 48 hours prior to device implant:
- •Known contraindication for systemic anticoagulation
- •Currently participating in an interventional trial whose protocol prevents effective application of Jarvik 2015, potentially has an independent effect on trial endpoints, or otherwise interferes with execution of the PumpKIN protocol
- •Stable inotrope dependence (INTERMACS profile 3)
- •Single ventricle anatomy
- •Presence of a mechanical heart valve
- •Unresolved malignancy
- •CPR with duration >30 consecutive minutes within 48 hours prior to device implant or CPR with uncertain neurological status prior to device implant
- •Renal dysfunction that is severe or, in the opinion of the investigator, irreversible
- •Hepatic dysfunction that is severe or, in the opinion of the investigator, irreversible
- •Severe or irreversible pulmonary dysfunction
- •ECMO use for >10 consecutive days
- •Unrepairable severe aortic insufficiency
- •Active, systemic infection unresponsive to antimicrobial therapy
- •Known cerebrovascular event within the past 30 days or uncertain neurological status
- •Severe right ventricular (RV) dysfunction or significant arrhythmia requiring treatment with an RV assist device (RVAD) (i.e., biventricular assist device)
- •Unmanageable bleeding per judgment of the investigator
- •Ventricular dysfunction that is likely to recover (e.g. myocarditis, metabolic myopathy where LV dysfunction is present solely with intermittent acidosis/crises).
- •Left ventricular end-diastolic dimension or left ventricular end-diastolic volume z score of < +2.5 end-diastolic dimension or ventricular end-diastolic volume z score of < +3.0
- •Left ventricular ejection fraction of >35%
结局指标
主要结局
Adverse Events
时间窗: 30 days or transplant/recovery (whichever comes first)
To evaluate the protocol defined (INTERMACS version 5.0) serious adverse events on Jarvik 2015 support up to transplant, recovery or 30 days of support
Clinical Feasibility
时间窗: 30 days or transplant/recovery (whichever comes first)
To assess the clinical feasibility of the investigational Jarvik 2015 VAD by evaluating survival in the absence of severe neurological impairment or death, or device failure up to the clinical endpoint defined as transplant, recovery or 30 days of support
Pivotal Trial continuation assessed by exploratory primary and secondary endpoints
时间窗: 12-months post-explant
To evaluate patients with respect to exploratory primary and secondary endpoints to inform the choice of appropriate primary and secondary endpoints for the pivotal trial.
Technical, surgical, and clinical milestones assessed by feasibility milestones
时间窗: up to 180 days or transplant/recovery
To describe the technical, surgical and clinical feasibility by achieving specific technical, surgical, and clinical milestones that typically accompany a successful VAD support run
次要结局
- Incidence of Adverse Events related to device(180 days post-implant)
- Neurological dysfunction(180 days post-implant)
- Incidence of Adverse Events per patient days of VAD support(180 days post-implant)
- King's Outcome Scale for Childhood Head Injury score (0 -5 scale, higher = worse outcome)(180 days and 12 months post-explant)
- TPN discontinuation(12 months post-explant)
- MRI(12 months post-explant)
- Overall survival in the absence of device failure(180 days or tranplant/recovery (whichever comes first))
- Survival at 12 months post-explant(12 months post-explant)
- Incidence rate of pump exchange(device explant)
- Pediatric Stroke Outcome Measure Neurological Exam (PSOM-NE) score (0 - 10 scale, higher = worse outcome)(180 days and 12 months post-explant)
- Vineland Adaptive Behavior Scales score (scoring and scale different by domain)(12 months post-explant)
- Ventricular Assist Device Quality of Life score (scoring and scale different per question)(30 days, 90 days, 12 months post-explant)
- Sedation(30 days post-implant)
- ALT level(180 days or immediately prior to explant)
- Survival in absence of severe neurological impairment(180 days or tranplant/recovery (whichever comes first))
- Survival at 30 days post-implant(30 days post-implant)
- Survival at 180 days post-implant(180 days post-implant)
- Time to pump failure(device explant)
- Pediatric Stroke Recurrence and Recovery Questionnaire score (0 -10 scale, higher = worse outcome)(180 days and 12 months post-explant)
- Pediatric Quality of Life score (0-4 scale per domain, higher = worse outcome)(12 months post-explant)
- Incidence rate of pump failure(device explant)
- Paralytics discontinuation(12 months post-explant)
- Extubation(30 days post-implant)
- Inotropic agents(12 months post-explant)
- Time to first pump exchange(device explant)
- Creatinine level(180 days or immediately prior to explant)