Pumps for Kids, Infants, and Neonates

Pediatric Heart Failure Clinical Trial

— PumpKIN  

Official title:

Pumps for Kids, Infants, and Neonates

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02954497
• Other study ID #: C01367
• Status: Active, not recruiting
• Phase: Early Phase 1
• Start date: October 22, 2018
• Est. completion date: September 15, 2023

Study information

• Verified date: November 2022
• Source: Carelon Research
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 7
• Est. completion date: September 15, 2023
• Est. primary completion date: June 9, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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%

Related Conditions & MeSH terms

• Heart Failure
• Pediatric Heart Failure

Intervention

Device:
• Surgical placement of the Jarvik 2015 VAD
The Jarvik 2015 VAD is a miniaturized, fully implantable, continuous flow left ventricular assist device.

Procedure:
• Surgical Placement of Jarvik 2015 VAD
Surgical Implant of the Jarvik 2015 VAD is a miniaturized, fully implantable, continuous flow left ventricular assist device.

Locations

Country	Name	City	State
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Children's Medical Center - Dallas	Dallas	Texas
United States	Texas Children's Hospital	Houston	Texas
United States	Columbia University Medical Center	New York	New York
United States	Lucile Packard Children's Hospital Stanford	Palo Alto	California

Sponsors (3)

Lead Sponsor	Collaborator
Carelon Research	Jarvik Heart, Inc., National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Clinical Feasibility	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	30 days or transplant/recovery (whichever comes first)
Primary	Adverse Events	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	30 days or transplant/recovery (whichever comes first)
Primary	Technical, surgical, and clinical milestones assessed by feasibility milestones	To describe the technical, surgical and clinical feasibility by achieving specific technical, surgical, and clinical milestones that typically accompany a successful VAD support run	up to 180 days or transplant/recovery
Primary	Pivotal Trial continuation assessed by exploratory primary and secondary endpoints	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.	12-months post-explant
Secondary	Incidence of Adverse Events per patient days of VAD support	The incidence rate of protocol-defined AEs (INTERMACS, version 5) per patient-day of VAD support up to the first 180 days post-implant.	180 days post-implant
Secondary	Incidence of Adverse Events related to device	Incidence rate of protocol-defined AEs possibly, probably, or definitely related to the device per day of mechanical circulatory support, up to the first 180 days post-implant.	180 days post-implant
Secondary	Neurological dysfunction	The incidence of new neurological dysfunction up to 180 days post-implant.	180 days post-implant
Secondary	Pediatric Stroke Outcome Measure Neurological Exam (PSOM-NE) score (0 - 10 scale, higher = worse outcome)	Pediatric Stroke Outcome Measure-Neurological (PSOM-NE) score measured at 180 days post-implant or immediately prior to explant, and at 12 months post-explant.	180 days and 12 months post-explant
Secondary	King's Outcome Scale for Childhood Head Injury score (0 -5 scale, higher = worse outcome)	King's Outcome Scale for Childhood Head Injury (KOSCHI) score measured at 180 days post-implant or immediately prior to explant, and at 12 months post-explant.	180 days and 12 months post-explant
Secondary	Pediatric Stroke Recurrence and Recovery Questionnaire score (0 -10 scale, higher = worse outcome)	Pediatric Stroke Recurrence and Recovery Questionnaire (RRQ) measured at 180 days post-implant or immediately prior to explant, and at 12 months post-explant.	180 days and 12 months post-explant
Secondary	Vineland Adaptive Behavior Scales score (scoring and scale different by domain)	Vineland Adaptive Behavior Scales (VABS II) measured at 12 months post-explant.	12 months post-explant
Secondary	Pediatric Quality of Life score (0-4 scale per domain, higher = worse outcome)	PedsQL20 measured at 180 days post-implant or immediately prior to explant and 12 months post-explant.	12 months post-explant
Secondary	Ventricular Assist Device Quality of Life score (scoring and scale different per question)	VAD QL measured at 30 and 90 days post-implant, and every 90 days thereafter, while on Jarvik 2015 VAD support.	30 days, 90 days, 12 months post-explant
Secondary	Paralytics discontinuation	Time to discontinuation of paralytics	12 months post-explant
Secondary	TPN discontinuation	Time to discontinuation of total parenteral nutrition (TPN)	12 months post-explant
Secondary	Extubation	Time to extubation	30 days post-implant
Secondary	Inotropic agents	Time to discontinuation of inotropic agents	12 months post-explant
Secondary	Sedation	Time to discontinuation of sedation such that subject is alert and interactive (e.g., engaging in age-appropriate play).	30 days post-implant
Secondary	MRI	Brain Magnetic Resonance Imaging (MRI) performed 12 months post-explant to assess for clinically silent neurologic injuries.	12 months post-explant
Secondary	Survival in absence of severe neurological impairment	Overall survival in the absence of a severe neurologic impairment and in the absence of device failure at 180 days following VAD implant and meeting any of the following outcomes: Survival to induction of anesthesia for cardiac transplant surgery, Recovery (removal of VAD and alive at 30 days post-explant), Survival at 180 consecutive days of VAD support	180 days or tranplant/recovery (whichever comes first)
Secondary	Overall survival in the absence of device failure	Overall survival in the absence of device failure but without regard for neurologic status, meeting any of the following outcomes: Survival to induction of anesthesia for cardiac transplant surgery, Survival to recovery (removal of Jarvik 2015 VAD and alive at 30 days post-explant), Survival at 180 consecutive days of Jarvik 2015 VAD support	180 days or tranplant/recovery (whichever comes first)
Secondary	Survival at 30 days post-implant	Survival at 30 days post-implant	30 days post-implant
Secondary	Survival at 180 days post-implant	Survival at 180 days post-implant	180 days post-implant
Secondary	Survival at 12 months post-explant	Survival at 12 months post-explant	12 months post-explant
Secondary	Time to pump failure	Time to first pump failure	device explant
Secondary	Incidence rate of pump failure	Incidence rate of pump failures	device explant
Secondary	Time to first pump exchange	Time to first pump exchanges	device explant
Secondary	Incidence rate of pump exchange	Incidence rate of pump exchanges	device explant
Secondary	Creatinine level	Proportion of subjects with a creatinine level below the ULNULN	180 days or immediately prior to explant
Secondary	ALT level	Proportion of subjects with an alanine aminotransferase (ALT) level below the ULNULN	180 days or immediately prior to explant