Pumps for Kids, Infants and Neonates (PumpKIN): The Jarvik 2015 Pivotal Trial

NCT06348043 | Not Yet Recruiting DMC FDA Device

• 2 other identifiers: SU_PumpKIN1UG3HL163311-01A1
• Study Type: interventional
• Target: 22
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a 22-subject prospective, multicenter, single-arm clinical trial to evaluate the safety and effectiveness of the Jarvik 2015 left ventricular assist device (LVAD) as a bridge to heart transplant in children weighing 8 to 30 kilograms. Data generated from the study will be used to support FDA review and potential approval of the device under the Humanitarian Device Exemption (HDE) regulation, the FDA approval pathway for devices intended to treat rare and orphan diseases.

Conditions

Heart Failure With Reduced Ejection Fraction; Pediatric HD, Stage IV; LVAD

Keywords

LVAD; Pediatric heart failure; advanced cardiac therapies

Outcome Measures

Primary Outcomes (2)

• Survival to heart transplant, recovery or 180 days of support in the absence of severe stroke.

  Patient survival to heart transplant, recovery or 180 days of support in the absence of severe stroke as defined by the Pediatric Stroke Outcomes Measure (PSOM, (range 0 to 10 with 0 no deficit to 10 maximum deficit) performed at 12 months post-explant. Severe stroke is defined an abnormal PSOM score due to stroke that meets the following criteria: PSOM score of 3-4 in Part A (sensory and motor assessment) OR Score of 3-4 in Parts B and C combined (language comprehension and language production) OR Score of 2 in Part D (cognitive or behavioral deficit).

  180 days

• Freedom from symptomatic stroke

  Freedom from symptomatic stroke based on the 12-month Pediatric Stroke Outcome Measure (PSOM, symptomatic stroke defined as a PSOM \>0 due to stroke, PSOM score range 0-10 with higher scores corresponding to more severe stroke injury). Participants meet criteria for no stroke if they do not meet ACTION criteria for stroke at 12 months or stroke is present with a PSOM score of 0).

  12 months post-explant

Secondary Outcomes (4)

• Incidence rate of all protocol-defined SAE's through 180 days of support definitely or probably related to the device

  180 days

• Distribution of Pediatric Stroke Outcomes Measures

  12-months post explant for stroke occurring during the first 180 days of support

• The KOSCHI score

  12 months post-explant in children with strokes occurring during the first 180 days of support

• Overall survival on Jarvik 2015 support to 180 days

  180 days

Study Arms (1)

LVAD support with the Jarvik 2015

EXPERIMENTAL

Children implanted with the Jarvik 2015 LVAD as a bridge to heart transplant

Device: Jarvik 2015 LVAD

Interventions

Jarvik 2015 LVAD DEVICE

Medical device used to treat children awaiting heart transplant who fail optimal medical therapy.

LVAD support with the Jarvik 2015

Eligibility Criteria

• Age: 6 Months - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children with severe (Ross or New York Heart Association) class IV HF despite optimal medical therapy (Interagency Registry for Mechanically Assisted Circulatory Support \[INTERMACS\] profile 1 or 2 for pediatrics) who require mechanical circulatory support as a bridge to transplant and meet all the criteria below:
• Males and females with body weight between 8 kg, and 30 kg
• Body surface area (BSA) between 0.4 m2 and 1.0 m2
• 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., , Kawasaki disease) 4. INTERMACS Profile 1 or 2 as evidenced by: a. Inability to wean from extra-corporeal membrane oxygenation (ECMO) or other temporary circulatory support (TCS), OR b.Inability to wean from mechanical ventilator support, OR c. Inotrope-dependent,decompensatedHFANDmeetoneormoreofthe following severity criteria within 48 hours prior to implant (unless otherwise noted), attributed to decompensated HF despite optimal medical therapy: i. Urine output \<0.5 cc/kg/hour for 12hr ii. Creatinine level \>2 times the upper limit of normal (ULN) for age iii. Alanine aminotransferase (ALT) or total bilirubin level \>3 times the ULN for age (either qualifies the patient) iv. Mixed venous oxygen saturation (SvO2) \<55% (or arteriovenous oxygen difference \>45%) v. Acidosis: Base excess \>-5 vi. Inability to tolerate appropriate enteral calories vii. Inability to ambulate or participate 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 6. Implant performed at a US or European site participating in the Jarvik 2015 IDE Pivotal Trial.
• \. Written consent of parent(s) or legally authorized representative (LAR) where appropriate.

You may not qualify if:

• Single ventricle anatomy
• Presence of a mechanical heart valve
• Unrepairable severe aortic insufficiency 3. Stable inotrope dependence (INTERMACS profile 3) 4. Severe or irreversible renal dysfunction, liver dysfunction or pulmonary dysfunction. 5. Known contraindication to systemic anticoagulation (e.g., coagulopathy) 6. CPR with duration \>30 consecutive minutes within 48 hours prior to device implant or CPR with uncertain neurological status 7. ECMO use for \>10 consecutive days 8. Known cerebrovascular event within the past 30 days or uncertain neurological status 9. Unmanageable bleeding per judgment of the investigator 10. Ventricular dysfunction that is likely to recover (e.g., acute myocarditis, metabolic myopathy where LV dysfunction is present solely with intermittent acidosis/crises).
• \. A non-dilated LV as determined by a left ventricular end-diastolic dimension or left ventricular end-diastolic volume z score of \<+2.5 12. Systolic dysfunction that is not severe (LV ejection fraction of \>35%) (e.g., heart failure with preserved ejection fraction, aka primary diastolic dysfunction). 12. Unresolved malignancy 13. Active, systemic infection unresponsive to antimicrobials therapy 14. Currently participating in an interventional trial whose protocol prevents effective application of the Jarvik 2015 device, potentially has an independent effect on trial endpoints, or otherwise interferes with execution of the PumpKIN protocol

Sponsors & Collaborators

• Stanford University: lead
• University of Texas: collaborator
• Carelon Research: collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Officials

• William T Mahle, MD

  Children's Healthcare of Atlanta

  STUDY CHAIR

Central Study Contacts

Christopher SD Almond, MD, MPH

CONTACT

6507237913; calmond@stanford.edu

Ryan Davies, MD, MSC

CONTACT

ryan.davies@UTsouthwestern.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: March 29, 2024
• First Posted: April 4, 2024
• Study Start: August 1, 2024
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: April 4, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share