Clinical trial NCT02954497: Pumps for Kids, Infants, and Neonates
Official title: Pumps for Kids, Infants, and Neonates
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: EARLY_PHASE1
Conditions: Pediatric Heart Failure
Keywords: ventricular assist device
congenital heart disease
heart failure
Lead sponsor: Carelon Research
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
name: Jarvik Heart, Inc.
First posted: 2016-11-03
Enrollment: 7
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Jarvik 2015
Primary purpose: DEVICE_FEASIBILITY

Brief summary: 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.


Eligibility criteria: Children must meet all of the following criteria:

1. Males and females within weight range 8 to 30 kg
2. Body surface area (BSA) 0.4 m2 to 1.0 m2
3. 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)
4. INTERMACS Profile 1 or 2 as evidenced by one or more of the following:

   1. Inability to wean from extra-corporeal membrane oxygenation (ECMO) or other temporary circulatory support (TCS), OR
   2. Inability to wean from mechanical ventilator support, OR
   3. 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
5. 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
6. Written consent of parent(s) or legally authorized representative (LAR) where appropriate.

Children must not meet any of the following exclusion criteria within 48 hours prior to device implant:

1. Known contraindication for systemic anticoagulation
2. 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
3. Stable inotrope dependence (INTERMACS profile 3)
4. Single ventricle anatomy
5. Presence of a mechanical heart valve
6. Unresolved malignancy
7. CPR with duration \>30 consecutive minutes within 48 hours prior to device implant or CPR with uncertain neurological status prior to device implant
8. Renal dysfunction that is severe or, in the opinion of the investigator, irreversible
9. Hepatic dysfunction that is severe or, in the opinion of the investigator, irreversible
10. Severe or irreversible pulmonary dysfunction
11. ECMO use for \>10 consecutive days
12. Unrepairable severe aortic insufficiency
13. Active, systemic infection unresponsive to antimicrobial therapy
14. Known cerebrovascular event within the past 30 days or uncertain neurological status
15. Severe right ventricular (RV) dysfunction or significant arrhythmia requiring treatment with an RV assist device (RVAD) (i.e., biventricular assist device)
16. Unmanageable bleeding per judgment of the investigator
17. Ventricular dysfunction that is likely to recover (e.g. myocarditis, metabolic myopathy where LV dysfunction is present solely with intermittent acidosis/crises).
18. 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
19. Left ventricular ejection fraction of \>35%

Primary outcomes:
measure: Clinical Feasibility
timeFrame: 30 days or transplant/recovery (whichever comes first)
measure: Adverse Events
timeFrame: 30 days or transplant/recovery (whichever comes first)
measure: Technical, surgical, and clinical milestones assessed by feasibility milestones
timeFrame: up to 180 days or transplant/recovery
measure: Pivotal Trial continuation assessed by exploratory primary and secondary endpoints
timeFrame: 12-months post-explant
Secondary outcomes:
measure: Incidence of Adverse Events per patient days of VAD support
timeFrame: 180 days post-implant
measure: Incidence of Adverse Events related to device
timeFrame: 180 days post-implant
measure: Neurological dysfunction
timeFrame: 180 days post-implant
measure: Pediatric Stroke Outcome Measure Neurological Exam (PSOM-NE) score (0 - 10 scale, higher = worse outcome)
timeFrame: 180 days and 12 months post-explant
measure: King's Outcome Scale for Childhood Head Injury score (0 -5 scale, higher = worse outcome)
timeFrame: 180 days and 12 months post-explant
measure: Pediatric Stroke Recurrence and Recovery Questionnaire score (0 -10 scale, higher = worse outcome)
timeFrame: 180 days and 12 months post-explant
measure: Vineland Adaptive Behavior Scales score (scoring and scale different by domain)
timeFrame: 12 months post-explant
measure: Pediatric Quality of Life score (0-4 scale per domain, higher = worse outcome)
timeFrame: 12 months post-explant
measure: Ventricular Assist Device Quality of Life score (scoring and scale different per question)
timeFrame: 30 days, 90 days, 12 months post-explant
measure: Paralytics discontinuation
timeFrame: 12 months post-explant
measure: TPN discontinuation
timeFrame: 12 months post-explant
measure: Extubation
timeFrame: 30 days post-implant
measure: Inotropic agents
timeFrame: 12 months post-explant
measure: Sedation
timeFrame: 30 days post-implant
measure: MRI
timeFrame: 12 months post-explant
measure: Survival in absence of severe neurological impairment
timeFrame: 180 days or tranplant/recovery (whichever comes first)
measure: Overall survival in the absence of device failure
timeFrame: 180 days or tranplant/recovery (whichever comes first)
measure: Survival at 30 days post-implant
timeFrame: 30 days post-implant
measure: Survival at 180 days post-implant
timeFrame: 180 days post-implant
measure: Survival at 12 months post-explant
timeFrame: 12 months post-explant
measure: Time to pump failure
timeFrame: device explant
measure: Incidence rate of pump failure
timeFrame: device explant
measure: Time to first pump exchange
timeFrame: device explant
measure: Incidence rate of pump exchange
timeFrame: device explant
measure: Creatinine level
timeFrame: 180 days or immediately prior to explant
measure: ALT level
timeFrame: 180 days or immediately prior to explant
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital Stanford
state: California
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Dallas
country: United States
facility: Children's Medical Center - Dallas
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
Study officials:
affiliation: Children's Hospital of Atlanta, Emory University School of Medicine
name: William T Mahle, MD
role: STUDY_CHAIR
References: