Multicenter Trial of ECMO in Children with Severe Cardiac Failure using the Cardiohelp System

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PROJECT SUMMARY/ABSTRACT While ECMO is widely considered the standard-of-care for children with severe cardiac failure requiring emergent life support, no ECMO circuit has ever been FDA-approved or cleared for children. The primary reason is that pediatric clinicians have long had unfettered access to all the equipment needed to assemble an ECMO circuit without performing an FDA-approval trial by using equipment off-label that has been FDA-cleared for cardiopulmonary bypass. Nevertheless, studies suggest the Cardiohelp system can support children with cardiopulmonary failure for days or weeks with manageable complications. This resubmission responds to a favorable review of the “Multicenter Trial of ECMO for Children with Severe Cardiac Failure using the Cardiohelp System”, to evaluate safety and effectiveness of the System to provide up to 30d of circulatory support to children with severe cardiac failure where other options have failed, and clinical worsening is expected, in order to support FDA clearance of the system in children. The study will be conducted at 5 centers with extensive experience and favorable outcomes using the Cardiohelp system in children. The target N is 50 children aged 0 to 16 years (3 to 80 kg) receiving VA ECMO. The primary goal is to generate the data needed to secure FDA-clearance of the first complete ECMO circuit in children with cardiac failure, and to determine whether complications, i.e., hemolysis, circuit thrombosis/stroke, and renal failure are acceptable in children ≤ 10 kg compared to those > 10 kg. The trial IDE (G220250) has Approval from the FDA (Appendix B). Specific aims are: SPECIFIC AIM 1 (Effectiveness): To determine whether ≥30 of 50 children (≥60%) supported with the Cardiohelp system for severe cardiac failure survive to recovery, durable VAD implant, transplant, or 30 days of support in the absence of severe neurological injury due to stroke as defined by the PSOM score. SPECIFIC AIM 2 (Safety): To determine whether children supported with the Cardiohelp system for severe cardiac failure experience key severe adverse outcomes (symptomatic stroke; severe hemolysis; thromboembolism or major device malfunction leading to circuit change; renal failure) within pre- specified Performance Goals (PGs): SPECIFIC AIM 3 (Regulatory): To generate the data necessary to allow the Cardiohelp to serve as a predicate device (if worthy) to support FDA clearance of future ECMO systems under the 510(k) pathway. SPECIFIC AIM 4 (Regulatory): To harmonize SAE definitions across different forms of MCS to enable device comparisons irrespective of the presence of an oxygenator, as the line between VAD and ECMO is blurring. Trial infrastructure includes a CCC at Stanford University (IDE sponsor) and a DCC at Boston Children's Hospital. The trial will be led by Drs. Sleeper, Almond and Ozment, with complementary expertise (Statistical; Regulatory; ECMO & Critical Care) and a history of successful collaboration in trial execution.