Study Design

Study Design

The PumpKIN Clinical Trial consists of a prospective, multi-center, single-arm feasibility study that will evaluate the safety of the Jarvik 2015 as a bridge to transplant or recovery in children. The study is intended to determine if the safety and probable benefit of the Jarvik 2015 device is sufficient to merit the approval of a Humanitarian Device Exemption (HDE) application by the Food and Drug Administration (FDA).

Each year in the US, nearly 1,800 infants die from complications of congenital heart disease (CHD) and another 350 develop cardiomyopathy which, for many, will lead to severe heart failure.1 Since 1982, more than 6,000 pediatric heart transplants have taken place and, due to improvements in perioperative care worldwide, these numbers continue to increase.Although the median wait-time of 61 days for a pediatric heart is significantly shorter2 than the 172 days for an adult,3 the number of pediatric donor hearts is insufficient and the swelling ranks of children in need of a transplant is resulting in a disproportionate number of pediatric wait-list deaths occurring in children weighing less than 25 kg.4

There are multiple, complex reasons for the lack of an ideal pediatric ventricular assist device (VAD): the heterogeneity of pediatric cardiac failure diagnoses, multiple congenital malformations, and acquired cardiomyopathy in addition to the heterogeneity in patient size, age and therapeutic goals (bridge to transplant, recovery, or decision). VADs currently used in the pediatric population are restricted to larger patients in whom VADs designed for adults or modified for use in larger children will fit appropriately. These are children five years of age or older, >25 kg or who meet the body surface area (>0.9 m2 BSA) criterion. With all of these issues, the availability of a low-risk long-term device for >65% of the pediatric transplant wait list is still not realized.

Study Device

The Jarvik 2015 VAD is a miniaturized continuous flow left ventricular assistance device developed through the NHLBI’s pediatric Circulatory Support Program. The Jarvik has emerged as a promising alternative to the Berlin Heart EXCOR® Pediatric VAD for infants and smaller children who require mechanical support as a bridge to transplant. Data from pre-clinical studies suggest this fully implantable axial flow device may have a more favorable adverse event profile; in particular, it may be less thrombogenic because of its valve-less continuous flow technology and more resistant to infection because of its internalized pump head and cannulae. Because the Jarvik 2015 is fully implantable and powered by a portable driver and battery pack, is more suitable for home discharge, similar to currently available adult continuous flow devices.

References

1Baldwin JT, Borovetz HS, Duncan BW, et al. The National Heart, Lung, and Blood Institute Pediatric Circulatory Support Program. Circulation 2006;113:147-55.
2Morales DL, Dreyer WJ, Denfield SW, et al. Over two decades of pediatric heart transplantation: how has survival changed? The Journal of thoracic and cardiovascular surgery 2007;133:632-9.
3Bove AA, Kashem A, Cross RC, et al. Factors affecting survival after heart transplantation: comparison of pre- and post-1999 listing protocols. The Journal of Heart and Lung Transplantation 2006;25:42-7.
4Almond CS, Thiagarajan RR, Piercey GE, et al. Waiting list mortality among children listed for heart transplantation in the United States. Circulation 2009;119:717-27.