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

NCT ID: NCT06348043

Last Updated: 2024-04-04

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 22 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-08-31
• Study Completion Date: 2029-12-31

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.

Detailed Description

This is a NHLBI-funded study is a 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 where few LVAD options are available and require hospital confinement. Children weighing 8 to 30 kg will be eligible if they have end-stage heart failure refractory to medical therapy due to severe systolic dysfunction. Children with single ventricle heart disease, heart failure with preserved ejection fraction (HFpEF), those requiring BIVAD support, and those with irreversible end-organ dysfunction will be excluded. The primary probable benefit endpoint of the study is survival to heart transplant, recovery or 180 days of support in the absence of severe stroke. The primary safety endpoint of the study is freedom from any new symptomatic stroke. A total of 22 subjects will be enrolled in the trial at 14 sites in the US and Europe. Subjects will be enrolled over roughly 3 years (38 months) and followed for 1 year post explant. 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. Children

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

LVAD support with the Jarvik 2015

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

Group Type: EXPERIMENTAL

Jarvik 2015 LVAD

Intervention Type: DEVICE

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

Interventions

Jarvik 2015 LVAD

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

Intervention Type: DEVICE

Eligibility Criteria

Inclusion Criteria

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:

1. Males and females with body weight between 8 kg, and 30 kg

2. Body surface area (BSA) between 0.4 m2 and 1.0 m2

3. 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

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. Implant performed at a US or European site participating in the Jarvik 2015 IDE Pivotal Trial.

7. Written consent of parent(s) or legally authorized representative (LAR) where appropriate.

Exclusion Criteria

1. Single ventricle anatomy

2. Presence of a mechanical heart valve

3. 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).

11. 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

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Texas

OTHER

Sponsor Role: collaborator

Carelon Research

OTHER

Sponsor Role: collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Christopher Almond

Professor of Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

William T Mahle, MD

Role: STUDY_CHAIR

Children's Healthcare of Atlanta

Central Contacts

Christopher SD Almond, MD, MPH

Role: CONTACT

calmond@stanford.edu

6507237913

Ryan Davies, MD, MSC

Role: CONTACT

ryan.davies@UTsouthwestern.edu

Other Identifiers

1UG3HL163311-01A1

Identifier Type: NIH

Identifier Source: secondary_id

View Link

SU_PumpKIN

Identifier Type: -

Identifier Source: org_study_id